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N-MOmentum: A Clinical Research Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorders

Sponsor
MedImmune LLC (Industry)
Overall Status
Completed
CT.gov ID
NCT02200770
Collaborator
(none)
231
Enrollment
98
Locations
2
Arms
67.2
Actual Duration (Months)
2.4
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

To compare the efficacy of inebilizumab (MEDI-551) versus placebo in reducing the risk of an neuromyelitis optica/neuromyelitis optica- spectrum disorders (NMO/NMOSD) attack in participants with NMO/NMOSD.

Condition or Disease Intervention/Treatment Phase
Phase 2/Phase 3

Detailed Description

Inebilizumab is a genetically engineered humanized monoclonal antibody that binds to the B cell specific surface antigen cluster of differentiation (CD19) resulting in the depletion of B cells. Inebilizumab depletes antibody-secreting plasmablasts and some plasma cells, which are generally CD19 positive and CD20 negative.

The main objective of this study is to determine whether inebilizumab compare to placebo decreases the risk of an attack in participants with NMO/NMOSD.

This is a multicenter, multinational, randomized, double-masked, placebo controlled study with an open-label extension period to evaluate the efficacy and safety of intravenous (IV) inebilizumab in adult participants with NMO/NMOSD. After a screening period, eligible participants will enter a randomized-controlled period (RCP) of maximum 197 days where they will be randomized in a 3:1 ratio to receive either IV inebilizumab or placebo. NMO/NMOSD attacks will be evaluated by the investigator and confirmed against the attack criteria by an independent Adjudication Committee (AC). Participants for whom the attack was confirmed by the AC will be given the option to enroll into an open label period (OLP) with inebilizumab treatment. Participants who complete the RCP without experiencing an attack will be given the option to enroll into an OLP with inebilizumab treatment. The OLP will continue for a minimum of 1 year and a maximum of 3 years after the last participant enter the OLP. All participants who discontinue from the RCP or the OLP will continue in a Safety Follow-up for a total of 12 months from last dose to evaluate the long-term safety of the investigational product.

Study Design

Study Type:
Interventional
Actual Enrollment :
231 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Double-masked, Placebo-controlled Study With Open-label Period to Evaluate the Efficacy and Safety of MEDI-551 in Adult Subjects With Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders.
Actual Study Start Date :
Apr 1, 2015
Actual Primary Completion Date :
Oct 26, 2018
Actual Study Completion Date :
Nov 6, 2020

Arms and Interventions

Arm Intervention/Treatment
Placebo Comparator: Placebo/Inebilizumab

Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants will receive IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who enter OLP will receive IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants will have choice to enter in the SFP at any point during RCP or OLP and will be free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants will continue in the SFP for 12 months from last dose of study drug.

Drug: Inebilizumab
Participants will receive IV inebilizumab 300 mg.
Other Names:
  • MEDI-551

    • Other: Placebo
    Participants will receive IV placebo matched to inebilizumab.
    Experimental: Inebilizumab/Inebilizumab

    AQP4-IgG sero positive and sero negative participants will IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who enter OLP will receive IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants will have choice to enter in the SFP at any point during RCP or OLP and will be free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants will continue in the SFP for 12 months from last dose of study drug.

    Drug: Inebilizumab
    Participants will receive IV inebilizumab 300 mg.
    Other Names:
  • MEDI-551

    • Other: Placebo
    Participants will receive IV placebo matched to inebilizumab.

    Outcome Measures

    Primary Outcome Measures

    1. Time to Adjudication Committee (AC)-Determined Neuromyelitis Optica Spectrum Disorder (NMOSD) Attack During RCP [Day 1 (Baseline) through Day 197]

      The NMOSD attack is defined as the presence of new or worsening symptom(s) related to NMOSD that meet at least one of the 18 protocol-defined attack criteria. These criteria were developed in conjunction with a panel of disease experts and with Food and Drug Administration input, and were intended to be clinically meaningful, objective, quantifiable, and able to be used worldwide. Only attacks positively adjudicated by the AC were used for the primary analysis.

    Secondary Outcome Measures

    1. Percentage of Participants With Worsening in Expanded Disability Severity Scale (EDSS) Score From Baseline to the Last Visit of RCP [Day 1 (Baseline) through Day 197]

      EDSS and its associated functional system (FS) score provide a system for quantifying disability and monitoring changes in the level of disability over time. EDSS is a scale for assessing neurologic impairment in multiple sclerosis (MS). It consists of 7 FS (visual FS, brainstem FS, pyramidal FS, cerebellar FS, sensory FS, bowel and bladder FS, and cerebral FS) which are used to derive EDSS score ranging from 0 (normal neurological exam) to 10 (death from MS). A negative change from baseline indicates improvement. A participant was considered to have a worsening in overall EDSS score of at least 2 if baseline EDSS score was 0, or at least 1 point if baseline EDSS score is 1 to 5, or at least 0.5 point if baseline EDSS score is 5.5 or more.

    2. Change From Baseline in Low-Contrast Visual Acuity Binocular Score to the Last Visit of RCP [Day 1 (Baseline) through Day 197]

      Low-contrast visual acuity test is used to determine the number of letters that can be read on a standardized low-contrast Landolt C Broken Rings Chart held at a distance of 3 meters. Binocular score is the number of letters read correctly on an eye chart using both eyes simultaneously. The total score ranges from 0-70. Higher score indicates better vision.

    3. Cumulative Number of Active Magnetic Resonance Imaging (MRI) Lesions During RCP [From Screening (Day -28) to Day 197]

      The number of new gadolinium-enhancing lesions and new or enlarging T2 lesions were measured by MRI of the brain, optic nerve, and spinal cord.

    4. Number of NMOSD-related In-patient Hospitalizations During RCP [Day 1 (Baseline) through Day 197]

      Participants with relapsing NMOSD have recurrent attacks that can be severe and result in blindness, paralysis, and even death and consequently, such attacks frequently result in in-patient hospitalizations. In-patient hospitalization is defined as a stay in hospital that goes beyond midnight of the first day of admission.

    5. Annualized AC-determined NMOSD Attack Rate During Any Exposure to Inebilizumab [For participants randomized to inebilizumab: Day 1 of RCP through end of OLP (approximately 3.5 years); and for participants randomized to placebo: Day 1 of OLP through the end of OLP (approximately 3 years)]

      Annualized attack rate is defined as total number of AC-determined attacks divided by total person years. Total person-years is calculated as the sum of the person-years for individual participant. Person-year for individual participant = (Date of last day before safety follow-up period - first inebilizumab dose date +1)/365.25. Annualized AC-determined NMOSD attack rate during any exposure to inebilizumab (in RCP and OLP) is reported.

    6. Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) During RCP [Day 1 (Baseline) through Day 197]

      An adverse event (AE) is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the RCP.

    7. Number of Participants With TEAEs and TESAEs During OLP [Day 198 through end of OLP period (maximum of 3 years after the last participant entered, until regulatory approval or study discontinuation, whichever occurs first) (approximately 3 years)]

      An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the OLP.

    8. Number of Participants With TEAEs and TESAEs During SFP (Open-label Population) [Every 3 months for a total of 1 year after the last dose of study drug (approximately 3 years)]

      An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the OLP. Participant who prematurely discontinued from the RCP or OLP entered in the SFP.

    9. Number of Participants With TEAEs and TESAEs During SFP (Non-OLP Population) [Every 3 months for a total of 1 year after the last dose of study drug (approximately 3 years)]

      An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the OLP. The SFP started when a participant prematurely discontinues from the RCP or OLP.

    10. Number of Participants With at Least a 2-Grade Shift From Baseline to Worst Toxicity Grade in Hematology and Chemistry During RCP [Day 1 (Baseline) through Day 197]

      Number of participants with at least a 2-grade shift from baseline to worst toxicity grade in hematology and chemistry during RCP is reported.

    11. Number of Participants With at Least a 2-Grade Shift From Baseline to Worst Toxicity Grade in Hematology and Chemistry During OLP [Day 198 through end of OLP (maximum of 3 years after the last participant enters, until regulatory approval or study discontinuation, whichever occurs first) (approximately 3 years)]

      Number of participants with at least a 2-grade shift from baseline to worst toxicity grade in hematology and chemistry during OLP is reported.

    12. Time to Maximum Serum Concentration (Tmax) of Inebilizumab (During RCP) [Dose 1 (Pre and post dose on Day 1 and Day 8); and Dose 2 (pre and post dose on Day 15; and Days 29, 57, 85, 113, 155, and 197)]

      Time to maximum serum concentration of inebilizumab during RCP is reported.

    13. Maximum Observed Serum Concentration (Cmax) of Inebilizumab (During RCP) [Dose 1 (Pre and post dose on Day 1 and Day 8); and Dose 2 (pre and post dose on Day 15; and Days 29, 57, 85, 113, 155, and 197)]

      Maximum observed serum concentration of inebilizumab during RCP is reported.

    14. Area Under the Serum Concentration Time Curve of the Dosing Interval (AUC0-14d) of Inebilizumab (During RCP) [Dose 1 (Pre and post dose on Day 1 and Day 8); and Dose 2 (pre and post dose on Day 15; and Days 29, 57, 85, 113, 155, and 197)]

      Area under the serum concentration time curve of the dosing interval (AUC0-14d) of inebilizumab during RCP is reported.

    15. Number of Participants With Positive Anti-Drug Antibodies (ADA) Titer to Inebilizumab (During RCP) [Pre and post dose on Day 1; and on Days 29, 85, and 197]

      Number of participants with positive ADA titer to inebilizumab during RCP is reported.

    16. Number of Participants With Positive Anti-Drug Antibodies (ADA) Titer to Inebilizumab (During OLP) [Pre and post dose on Day 1; and on Days 92, 183, 274, and then every 6 months (maximum of 3 years after the last participant enters, until regulatory approval or study discontinuation, whichever occurs first) (approximately 3 years)]

      Number of participants with positive ADA titer to inebilizumab in OLP is reported.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Men and women 18 years or older with diagnosis of NMO/NMOSD

    2. Confirmation of NMO/NMOSD status:

    3. AQP4-IgG sero-positive NMO/NMOSD with at least one attack requiring rescue therapy in the last year or two attacks requiring rescue therapy in the last 2 years

    4. AQP4-IgG sero-negative NMO with at least one attack requiring rescue therapy in the last year or two attacks requiring rescue therapy in the last 2 years

    5. Able and willing to give written informed consent and comply with the requirements of the study protocol.

    6. EDSS <= 7.5 (8 in special circumstances)

    7. Men and women of reproductive potential must agree to use a highly effective method of birth control from screening to 6 months after final dose of the investigational product.

    Exclusion Criteria:

    1. Lactating and pregnant females

    2. Treatment with any investigational agent within 4 weeks of screening

    3. Known history of a severe allergy or reaction to any component of the investigational product formulation or history of anaphylaxis following any biologic therapy.

    4. Known active severe bacterial, viral, or other infection or any major episode of infection requiring hospitalization.

    5. History of alcohol, drug, or chemical abuse, or a recent history of such abuse < 1 year prior to randomization

    6. Receipt of the following at any time prior to randomization:

    7. Alemtuzumab

    8. Total lymphoid irradiation

    9. Bone marrow transplant

    10. T-cell vaccination therapy

    11. Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior screening and B-cells below the lower limit of normal.

    12. Receipt of intravenous immunoglobulin (IVIG) within 1 month prior to randomization.

    13. Receipt of any of the following within 3 months prior to randomization:

    14. Natalizumab (Tysabri®).

    15. Cyclosporin

    16. Methotrexate

    17. Mitoxantrone

    18. Cyclophosphamide

    19. Tocilizumab

    20. Eculizumab

    21. History of Hepatitis B and/or Hepatitis C (Hep B/C at screening)

    22. Known history of a primary immunodeficiency (congenital or acquired) or an underlying condition such as human immunodeficiency virus (HIV) infection

    23. History of malignancies, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy > 3 months prior to randomization

    24. Any concomitant disease other than NMO/NMOSD that required treatment with oral or intravenous steroids at doses over 20 mg a day for over 21 days

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Research Site Birmingham Alabama United States 35294
    2 Research Site Sacramento California United States 95817
    3 Research Site San Francisco California United States 94158
    4 Research Site Aurora Colorado United States 80010
    5 Research Site New Haven Connecticut United States 06511
    6 Research Site Maitland Florida United States 32751
    7 Research Site Chicago Illinois United States 60637
    8 Research Site Kansas City Kansas United States 66160
    9 Research Site Baltimore Maryland United States 21287
    10 Research Site Detroit Michigan United States 48201
    11 Research Site Rochester Minnesota United States 55905
    12 Research Site Saint Louis Missouri United States 63131-2374
    13 Research Site Raleigh North Carolina United States 27607
    14 Research Site Cincinnati Ohio United States 45219
    15 Research Site Cleveland Ohio United States 44195
    16 Research Site Mansfield Ohio United States 44906
    17 Research Site Dallas Texas United States 75390
    18 Research Site Houston Texas United States 77030
    19 Research Site Richmond Virginia United States 23298
    20 Research Site Melbourne Australia 3065
    21 Research Site Sofia Bulgaria 1113
    22 Research Site Sofia Bulgaria 1309
    23 Research Site Sofia Bulgaria 1431
    24 Research Site Varna Bulgaria 9010
    25 Research Site Vancouver British Columbia Canada V6T 2B5
    26 Research Site Barranquilla Colombia 080020
    27 Research Site Bogota Colombia 110131
    28 Research Site Bogota Colombia 110231
    29 Research Site Cali Colombia 760032
    30 Research Site Olomouc Czechia 775 20
    31 Research Site Praha 2 Czechia 121 11
    32 Research Site Teplice Czechia 415 29
    33 Research Site Tallinn Estonia 10617
    34 Research Site Tartu Estonia 51014
    35 Research Site Berlin Germany 10117
    36 Research Site Dresden Germany 01307
    37 Research Site Düsseldorf Germany 40225
    38 Research Site Leipzig Germany 04103
    39 Research Site Muenster Germany 48149
    40 Research Site Rostock Germany 18147
    41 Research Site HongKong Hong Kong
    42 Research Site Esztergom Hungary 2500
    43 Research Site Nyíregyháza Hungary 4400
    44 Research Site Szeged Hungary 6725
    45 Research Site Jerusalem Israel 91120
    46 Research Site Ramat Gan Israel 52621
    47 Research Site Tel Aviv Israel 6423906
    48 Research Site Aomori-shi Japan 030-8553
    49 Research Site Bunkyo-ku Japan 113-8431
    50 Research Site Kyoto-shi Japan 604-8453
    51 Research Site Ota-ku Japan 145-0065
    52 Research Site Sendai-shi Japan 980-8574
    53 Research Site Tsukuba Japan 305-8577
    54 Research Site Goyang Korea, Republic of 410-769
    55 Research Site Jongno-gu Korea, Republic of 110-744
    56 Research Site Seoul Korea, Republic of 135-710
    57 Research Site Seoul Korea, Republic of 143729
    58 Research Site Ciudad De Mexico Mexico 14269
    59 Research Site Mexico City Mexico 03310
    60 Research Site Monterrey Mexico 64460
    61 Research Site San Luis Potosi Mexico 78090
    62 Research Site Chisinau Moldova, Republic of 2028
    63 Research Site Auckland New Zealand 1023
    64 Research Site Bellavista Peru CALLAO 2
    65 Research Site Lima Peru LIMA 01
    66 Research Site Katowice Poland 40-595
    67 Research Site Krakow Poland 31-637
    68 Research Site Lublin Poland 20-954
    69 Research Site Lódz Poland 90-324
    70 Research Site Olsztyn Poland 10-560
    71 Research Site Warszawa Poland 02-097
    72 Research Site Warszawa Poland 02-957
    73 Research Site Belgorod Russian Federation 308007
    74 Research Site Kazan Russian Federation 420021
    75 Research Site Khabarovsk Russian Federation 680009
    76 Research Site Krasnoyarsk Russian Federation 660037
    77 Research Site Moscow Russian Federation 123367
    78 Research Site Moscow Russian Federation 127018
    79 Research Site Nizhniy Novgorod Russian Federation 603155
    80 Research Site Novosibirsk Russian Federation 63007
    81 Research Site Omsk Russian Federation 644033
    82 Research Site Saint-Petersburg Russian Federation 197110
    83 Research Site Ufa Russian Federation 450005
    84 Research Site Belgrade Serbia 11129
    85 Research Site Cape Town South Africa 7505
    86 Research Site Cape Town South Africa 7925
    87 Research Site Madrid Spain 28040
    88 Research Site Changhua City Taiwan 50006
    89 Research Site Hualien City Taiwan 97002
    90 Research Site Tainan City Taiwan 70403
    91 Research Site Bangkok Thailand 10700
    92 Research Site Muang Thailand 40002
    93 Research Site Muang Thailand 50200
    94 Research Site Istanbul Turkey 34098
    95 Research Site Istanbul Turkey 34147
    96 Research Site Istanbul Turkey 34890
    97 Research Site Izmir Turkey 35170
    98 Research Site Samsun Turkey 55139

    Sponsors and Collaborators

    • MedImmune LLC

    Investigators

    • Study Director: MedImmune, LLC MedImmune, LLC, MedImmune LLC

    Study Documents (Full-Text)

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    MedImmune LLC
    ClinicalTrials.gov Identifier:
    NCT02200770
    Other Study ID Numbers:
    • CD-IA-MEDI-551-1155
    • 2014-000253-36
    First Posted:
    Jul 25, 2014
    Last Update Posted:
    Dec 3, 2021
    Last Verified:
    Nov 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by MedImmune LLC
    NMO, NMOSD, Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorders, autoimmune, demyelination, MEDI-551, monoclonal antibody, Devic's syndrome, B-cell
    Additional relevant MeSH terms:
    Neuromyelitis Optica
    Disease

    Study Results

    Participant Flow

    Recruitment Details
    Pre-assignment Detail
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Period Title: Randomized-controlled Period (RCP)
    STARTED 56 175
    Treated 56 174
    COMPLETED 54 169
    NOT COMPLETED 2 6
    Period Title: Randomized-controlled Period (RCP)
    STARTED 51 165
    COMPLETED 43 131
    NOT COMPLETED 8 34

    Baseline Characteristics

    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab Total
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. Total of all reporting groups
    Overall Participants 56 174 230
    Age (Years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [Years]
    42.6
    (13.9)
    43.0
    (11.6)
    42.9
    (12.2)
    Sex: Female, Male (Count of Participants)
    Female
    50
    89.3%
    159
    91.4%
    209
    90.9%
    Male
    6
    10.7%
    15
    8.6%
    21
    9.1%
    Ethnicity (NIH/OMB) (Count of Participants)
    Hispanic or Latino
    15
    26.8%
    28
    16.1%
    43
    18.7%
    Not Hispanic or Latino
    41
    73.2%
    146
    83.9%
    187
    81.3%
    Unknown or Not Reported
    0
    0%
    0
    0%
    0
    0%
    Race (NIH/OMB) (Count of Participants)
    American Indian or Alaska Native
    5
    8.9%
    14
    8%
    19
    8.3%
    Asian
    8
    14.3%
    39
    22.4%
    47
    20.4%
    Native Hawaiian or Other Pacific Islander
    0
    0%
    0
    0%
    0
    0%
    Black or African American
    5
    8.9%
    15
    8.6%
    20
    8.7%
    White
    28
    50%
    92
    52.9%
    120
    52.2%
    More than one race
    0
    0%
    1
    0.6%
    1
    0.4%
    Unknown or Not Reported
    10
    17.9%
    13
    7.5%
    23
    10%

    Outcome Measures

    1. Primary Outcome
    Title Time to Adjudication Committee (AC)-Determined Neuromyelitis Optica Spectrum Disorder (NMOSD) Attack During RCP
    Description The NMOSD attack is defined as the presence of new or worsening symptom(s) related to NMOSD that meet at least one of the 18 protocol-defined attack criteria. These criteria were developed in conjunction with a panel of disease experts and with Food and Drug Administration input, and were intended to be clinically meaningful, objective, quantifiable, and able to be used worldwide. Only attacks positively adjudicated by the AC were used for the primary analysis.
    Time Frame Day 1 (Baseline) through Day 197

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 56 174
    Median (95% Confidence Interval) [Days]
    NA
    NA
    Statistical Analysis 1
    Statistical Analysis Overview Comparison Group Selection Placebo/Inebilizumab, Inebilizumab/Inebilizumab
    Comments
    Type of Statistical Test Superiority
    Comments
    Statistical Test of Hypothesis p-Value <0.0001
    Comments
    Method Regression, Cox
    Comments
    Method of Estimation Estimation Parameter Hazard Ratio (HR)
    Estimated Value 0.272
    Confidence Interval (2-Sided) 95%
    0.1496 to 0.4961
    Parameter Dispersion Type:
    Value:
    Estimation Comments
    2. Secondary Outcome
    Title Percentage of Participants With Worsening in Expanded Disability Severity Scale (EDSS) Score From Baseline to the Last Visit of RCP
    Description EDSS and its associated functional system (FS) score provide a system for quantifying disability and monitoring changes in the level of disability over time. EDSS is a scale for assessing neurologic impairment in multiple sclerosis (MS). It consists of 7 FS (visual FS, brainstem FS, pyramidal FS, cerebellar FS, sensory FS, bowel and bladder FS, and cerebral FS) which are used to derive EDSS score ranging from 0 (normal neurological exam) to 10 (death from MS). A negative change from baseline indicates improvement. A participant was considered to have a worsening in overall EDSS score of at least 2 if baseline EDSS score was 0, or at least 1 point if baseline EDSS score is 1 to 5, or at least 0.5 point if baseline EDSS score is 5.5 or more.
    Time Frame Day 1 (Baseline) through Day 197

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 56 174
    Number [Percentage of Participants]
    33.9
    60.5%
    14.9
    8.6%
    Statistical Analysis 1
    Statistical Analysis Overview Comparison Group Selection Placebo/Inebilizumab, Inebilizumab/Inebilizumab
    Comments
    Type of Statistical Test Superiority
    Comments
    Statistical Test of Hypothesis p-Value 0.0033
    Comments
    Method Regression, Logistic
    Comments
    Method of Estimation Estimation Parameter Odds Ratio (OR)
    Estimated Value 0.352
    Confidence Interval (2-Sided) 95%
    0.1755 to 0.7059
    Parameter Dispersion Type:
    Value:
    Estimation Comments
    3. Secondary Outcome
    Title Change From Baseline in Low-Contrast Visual Acuity Binocular Score to the Last Visit of RCP
    Description Low-contrast visual acuity test is used to determine the number of letters that can be read on a standardized low-contrast Landolt C Broken Rings Chart held at a distance of 3 meters. Binocular score is the number of letters read correctly on an eye chart using both eyes simultaneously. The total score ranges from 0-70. Higher score indicates better vision.
    Time Frame Day 1 (Baseline) through Day 197

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment. Participants with low contrast visual acuity binocular score were analyzed for this outcome measure.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 56 171
    Least Squares Mean (Standard Error) [Score on scale]
    1.442
    (1.217)
    1.576
    (0.935)
    Statistical Analysis 1
    Statistical Analysis Overview Comparison Group Selection Placebo/Inebilizumab, Inebilizumab/Inebilizumab
    Comments
    Type of Statistical Test Superiority
    Comments
    Statistical Test of Hypothesis p-Value 0.9026
    Comments
    Method ANCOVA
    Comments
    Method of Estimation Estimation Parameter Mean Difference (Net)
    Estimated Value 0.134
    Confidence Interval (2-Sided) 95%
    -2.0254 to 2.2941
    Parameter Dispersion Type: Standard Error of the Mean
    Value: 1.096
    Estimation Comments
    4. Secondary Outcome
    Title Cumulative Number of Active Magnetic Resonance Imaging (MRI) Lesions During RCP
    Description The number of new gadolinium-enhancing lesions and new or enlarging T2 lesions were measured by MRI of the brain, optic nerve, and spinal cord.
    Time Frame From Screening (Day -28) to Day 197

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment. Participants with observed MRI lesions were analyzed for this outcome measure.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 32 79
    Mean (Standard Deviation) [Number of lesions]
    2.3
    (1.3)
    1.6
    (1.0)
    Statistical Analysis 1
    Statistical Analysis Overview Comparison Group Selection Placebo/Inebilizumab, Inebilizumab/Inebilizumab
    Comments
    Type of Statistical Test Superiority
    Comments
    Statistical Test of Hypothesis p-Value 0.0034
    Comments
    Method Negative Binomial Regression
    Comments
    Method of Estimation Estimation Parameter Rate Ratio
    Estimated Value 0.566
    Confidence Interval (2-Sided) 95%
    0.3866 to 0.8279
    Parameter Dispersion Type:
    Value:
    Estimation Comments
    5. Secondary Outcome
    Title Number of NMOSD-related In-patient Hospitalizations During RCP
    Description Participants with relapsing NMOSD have recurrent attacks that can be severe and result in blindness, paralysis, and even death and consequently, such attacks frequently result in in-patient hospitalizations. In-patient hospitalization is defined as a stay in hospital that goes beyond midnight of the first day of admission.
    Time Frame Day 1 (Baseline) through Day 197

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment. Participants with in-patient hospitalization were analyzed for this outcome measure.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 8 11
    Mean (Standard Deviation) [Number of In-patient Hospitalizations]
    1.4
    (0.7)
    1.0
    (0)
    Statistical Analysis 1
    Statistical Analysis Overview Comparison Group Selection Placebo/Inebilizumab, Inebilizumab/Inebilizumab
    Comments
    Type of Statistical Test Superiority
    Comments
    Statistical Test of Hypothesis p-Value 0.0146
    Comments
    Method Negative Binomial Regression
    Comments
    Method of Estimation Estimation Parameter Rate Ratio
    Estimated Value 0.317
    Confidence Interval (2-Sided) 95%
    0.1257 to 0.7972
    Parameter Dispersion Type:
    Value:
    Estimation Comments
    6. Secondary Outcome
    Title Annualized AC-determined NMOSD Attack Rate During Any Exposure to Inebilizumab
    Description Annualized attack rate is defined as total number of AC-determined attacks divided by total person years. Total person-years is calculated as the sum of the person-years for individual participant. Person-year for individual participant = (Date of last day before safety follow-up period - first inebilizumab dose date +1)/365.25. Annualized AC-determined NMOSD attack rate during any exposure to inebilizumab (in RCP and OLP) is reported.
    Time Frame For participants randomized to inebilizumab: Day 1 of RCP through end of OLP (approximately 3.5 years); and for participants randomized to placebo: Day 1 of OLP through the end of OLP (approximately 3 years)

    Outcome Measure Data

    Analysis Population Description
    Any inebilizumab population included all participants who received at least one dose of inebilizumab either in the RCP or OLP.
    Arm/Group Title Any Inebilizumab
    Arm/Group Description AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 in RCP or IV inebilizumab 300 mg on both Day 1 and Day 15 in OLP ; followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP.
    Measure Participants 225
    Number [Annualized attack rate]
    0.086
    7. Secondary Outcome
    Title Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) During RCP
    Description An adverse event (AE) is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the RCP.
    Time Frame Day 1 (Baseline) through Day 197

    Outcome Measure Data

    Analysis Population Description
    As-treated population included all participants who received any dose of study drug and analyzed according to the treatment received.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 56 174
    TEAEs
    41
    73.2%
    127
    73%
    TESAEs
    6
    10.7%
    9
    5.2%
    8. Secondary Outcome
    Title Number of Participants With TEAEs and TESAEs During OLP
    Description An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the OLP.
    Time Frame Day 198 through end of OLP period (maximum of 3 years after the last participant entered, until regulatory approval or study discontinuation, whichever occurs first) (approximately 3 years)

    Outcome Measure Data

    Analysis Population Description
    Open-label population included all participants who received at least one dose of inebilizumab during OLP.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 51 165
    TEAEs
    45
    80.4%
    144
    82.8%
    TESAEs
    19
    33.9%
    22
    12.6%
    9. Secondary Outcome
    Title Number of Participants With TEAEs and TESAEs During SFP (Open-label Population)
    Description An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the OLP. Participant who prematurely discontinued from the RCP or OLP entered in the SFP.
    Time Frame Every 3 months for a total of 1 year after the last dose of study drug (approximately 3 years)

    Outcome Measure Data

    Analysis Population Description
    Open-label population included all participants who received at least one dose of inebilizumab during OLP.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 51 165
    TEAEs
    3
    5.4%
    5
    2.9%
    TESAEs
    2
    3.6%
    1
    0.6%
    10. Secondary Outcome
    Title Number of Participants With TEAEs and TESAEs During SFP (Non-OLP Population)
    Description An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event is any AE that resulted in death, life threatening, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, is a congenital anomaly/birth defect in offspring of a study participant, is an important medical event that may jeopardize the participant or may require medical intervention. TEAEs are defined as events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug during the OLP. The SFP started when a participant prematurely discontinues from the RCP or OLP.
    Time Frame Every 3 months for a total of 1 year after the last dose of study drug (approximately 3 years)

    Outcome Measure Data

    Analysis Population Description
    Non-OLP population included all participants who received any dose of study drug, analyzed according to the treatment received in RCP, but did not roll over to OLP.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 5 9
    TEAEs
    2
    3.6%
    3
    1.7%
    TESAEs
    1
    1.8%
    1
    0.6%
    11. Secondary Outcome
    Title Number of Participants With at Least a 2-Grade Shift From Baseline to Worst Toxicity Grade in Hematology and Chemistry During RCP
    Description Number of participants with at least a 2-grade shift from baseline to worst toxicity grade in hematology and chemistry during RCP is reported.
    Time Frame Day 1 (Baseline) through Day 197

    Outcome Measure Data

    Analysis Population Description
    As-treated population included all participants who received any dose of study drug and analyzed according to the treatment received. 'Number of Participants analyzed' signifies participants who were analyzed for this outcome measure.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 56 173
    Hemoglobin (decreased)
    0
    0%
    2
    1.1%
    Leukocytes (decreased)
    1
    1.8%
    11
    6.3%
    Lymphocytes (decreased)
    5
    8.9%
    35
    20.1%
    Lymphocytes (increased)
    4
    7.1%
    1
    0.6%
    Neutrophils (decreased)
    0
    0%
    10
    5.7%
    Alanine Aminotransferase (increased)
    1
    1.8%
    4
    2.3%
    Aspartate Aminotransferase (increased)
    2
    3.6%
    1
    0.6%
    Bilirubin (increased)
    2
    3.6%
    1
    0.6%
    Cholesterol (increased)
    2
    3.6%
    5
    2.9%
    Creatinine (increased)
    1
    1.8%
    5
    2.9%
    Gamma glutamyl transferase (increased)
    1
    1.8%
    6
    3.4%
    Glucose (decreased)
    1
    1.8%
    1
    0.6%
    Glucose (increased)
    5
    8.9%
    1
    0.6%
    Potassium (decreased)
    0
    0%
    1
    0.6%
    Sodium (decreased)
    1
    1.8%
    2
    1.1%
    Triglycerides (increased)
    2
    3.6%
    7
    4%
    12. Secondary Outcome
    Title Number of Participants With at Least a 2-Grade Shift From Baseline to Worst Toxicity Grade in Hematology and Chemistry During OLP
    Description Number of participants with at least a 2-grade shift from baseline to worst toxicity grade in hematology and chemistry during OLP is reported.
    Time Frame Day 198 through end of OLP (maximum of 3 years after the last participant enters, until regulatory approval or study discontinuation, whichever occurs first) (approximately 3 years)

    Outcome Measure Data

    Analysis Population Description
    Open-label population included all participants who received at least one dose of study drug during OLP.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 51 165
    Hemoglobin (decreased)
    2
    3.6%
    6
    3.4%
    Leukocytes (decreased)
    1
    1.8%
    12
    6.9%
    Lymphocytes (decreased)
    9
    16.1%
    21
    12.1%
    Lymphocytes (increased)
    1
    1.8%
    4
    2.3%
    Neutrophils (decreased)
    1
    1.8%
    16
    9.2%
    Alanine aminotransferase (increased)
    4
    7.1%
    2
    1.1%
    Albumin (decreased)
    0
    0%
    3
    1.7%
    Alkaline phosphatase (increased)
    1
    1.8%
    1
    0.6%
    Aspartate aminotransferase (increased)
    2
    3.6%
    3
    1.7%
    Cholesterol (increased)
    1
    1.8%
    5
    2.9%
    Creatinine (increased)
    3
    5.4%
    7
    4%
    Gamma glutamyl transferase (increased)
    1
    1.8%
    10
    5.7%
    Glucose (decreased)
    0
    0%
    3
    1.7%
    Glucose (increased)
    5
    8.9%
    5
    2.9%
    Potassium (decreased)
    0
    0%
    1
    0.6%
    Potassium (increased)
    1
    1.8%
    2
    1.1%
    Sodium (decreased)
    2
    3.6%
    1
    0.6%
    Triglycerides (increased)
    4
    7.1%
    6
    3.4%
    Urate (increased)
    1
    1.8%
    1
    0.6%
    Hemoglobin (increased)
    0
    0%
    1
    0.6%
    Bilirubin
    2
    3.6%
    0
    0%
    13. Secondary Outcome
    Title Time to Maximum Serum Concentration (Tmax) of Inebilizumab (During RCP)
    Description Time to maximum serum concentration of inebilizumab during RCP is reported.
    Time Frame Dose 1 (Pre and post dose on Day 1 and Day 8); and Dose 2 (pre and post dose on Day 15; and Days 29, 57, 85, 113, 155, and 197)

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment. 'Number analyzed' signifies participants who had adequate pharmacokinetic sample of inebilizumab per the specified dose levels (Dose 1 and Dose 2).
    Arm/Group Title Inebilizumab/Inebilizumab
    Arm/Group Description AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 173
    Dose 1
    0.07
    Dose 2
    0.07
    14. Secondary Outcome
    Title Maximum Observed Serum Concentration (Cmax) of Inebilizumab (During RCP)
    Description Maximum observed serum concentration of inebilizumab during RCP is reported.
    Time Frame Dose 1 (Pre and post dose on Day 1 and Day 8); and Dose 2 (pre and post dose on Day 15; and Days 29, 57, 85, 113, 155, and 197)

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment. 'Number analyzed' signifies participants who had adequate pharmacokinetic sample of inebilizumab per the specified dose levels (Dose 1 and Dose 2).
    Arm/Group Title Inebilizumab/Inebilizumab
    Arm/Group Description AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 173
    Dose 1
    97.7
    (37.4)
    Dose 2
    108
    (45.4)
    15. Secondary Outcome
    Title Area Under the Serum Concentration Time Curve of the Dosing Interval (AUC0-14d) of Inebilizumab (During RCP)
    Description Area under the serum concentration time curve of the dosing interval (AUC0-14d) of inebilizumab during RCP is reported.
    Time Frame Dose 1 (Pre and post dose on Day 1 and Day 8); and Dose 2 (pre and post dose on Day 15; and Days 29, 57, 85, 113, 155, and 197)

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment. 'Number analyzed' signifies participants who had adequate pharmacokinetic sample of inebilizumab per the specified dose levels (Dose 1 and Dose 2).
    Arm/Group Title Inebilizumab/Inebilizumab
    Arm/Group Description AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 167
    Dose 1
    667
    (31.3)
    Dose 2
    967
    (39.6)
    16. Secondary Outcome
    Title Number of Participants With Positive Anti-Drug Antibodies (ADA) Titer to Inebilizumab (During RCP)
    Description Number of participants with positive ADA titer to inebilizumab during RCP is reported.
    Time Frame Pre and post dose on Day 1; and on Days 29, 85, and 197

    Outcome Measure Data

    Analysis Population Description
    The ITT population included all participants who were randomized, received any study drug, and grouped according to their randomized treatment. 'Number analyzed' signifies the number of participants who had adequate ADA samples.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 56 174
    ADA positive at anytime including baseline (BL)
    8
    14.3%
    17
    9.8%
    ADA positive at BL; not detected post-BL
    0
    0%
    5
    2.9%
    ADA positive post-BL; positive at BL
    4
    7.1%
    7
    4%
    ADA positive post-BL; not detected at BL
    4
    7.1%
    5
    2.9%
    17. Secondary Outcome
    Title Number of Participants With Positive Anti-Drug Antibodies (ADA) Titer to Inebilizumab (During OLP)
    Description Number of participants with positive ADA titer to inebilizumab in OLP is reported.
    Time Frame Pre and post dose on Day 1; and on Days 92, 183, 274, and then every 6 months (maximum of 3 years after the last participant enters, until regulatory approval or study discontinuation, whichever occurs first) (approximately 3 years)

    Outcome Measure Data

    Analysis Population Description
    Open-label population included all participants who received at least one dose of inebilizumab during OLP. 'Number analyzed' signifies the number of participants who had adequate ADA samples.
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    Measure Participants 51 165
    ADA positive at any time including baseline (BL)
    9
    16.1%
    22
    12.6%
    ADA positive at BL; not detected post-BL
    0
    0%
    5
    2.9%
    ADA positive post-BL and positive at BL
    4
    7.1%
    7
    4%
    ADA positive post-BL; not detected at BL
    5
    8.9%
    10
    5.7%

    Adverse Events

    Time Frame From Day 1 through the end of study (approximately 5 years 10 months)
    Adverse Event Reporting Description Adverse events are reported for all 3 study periods combined (RCP, OLP, and SFP).
    Arm/Group Title Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Arm/Group Description Aquaporin-4-antibody (AQP4-IgG) sero positive and sero negative participants received IV dose of placebo matched to inebilizumab on Day 1 and Day 15 of the RCP. The participants who entered OLP received IV inebilizumab 300 mg on both Day 1 and Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug. AQP4-IgG sero positive and sero negative participants received IV dose of inebilizumab 300 mg on Day 1 and Day 15 of RCP. The participants who entered OLP received IV inebilizumab 300 mg on Day 1 and matching placebo on Day 15, followed by a single IV dose of inebilizumab 300 mg every 6 months until maximum of 3 years after the last participant enters the OLP. Participants had choice to enter in the SFP at any point during RCP or OLP and were free to pursue other treatment options otherwise prohibited during the RCP and OLP. Participants continued in the SFP for 12 months from last dose of study drug.
    All Cause Mortality
    Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 1/56 (1.8%) 2/174 (1.1%)
    Serious Adverse Events
    Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 24/56 (42.9%) 28/174 (16.1%)
    Blood and lymphatic system disorders
    Neutropenia 0/56 (0%) 0 1/174 (0.6%) 1
    Ear and labyrinth disorders
    Deafness 0/56 (0%) 0 1/174 (0.6%) 1
    Endocrine disorders
    Adrenal insufficiency 0/56 (0%) 0 1/174 (0.6%) 1
    Steroid withdrawal syndrome 0/56 (0%) 0 1/174 (0.6%) 1
    Eye disorders
    Blindness unilateral 0/56 (0%) 0 1/174 (0.6%) 1
    Cataract 0/56 (0%) 0 1/174 (0.6%) 2
    Vision blurred 0/56 (0%) 0 1/174 (0.6%) 1
    Visual acuity reduced 1/56 (1.8%) 1 0/174 (0%) 0
    Gastrointestinal disorders
    Abdominal pain upper 0/56 (0%) 0 1/174 (0.6%) 1
    Constipation 1/56 (1.8%) 1 0/174 (0%) 0
    Diarrhoea 0/56 (0%) 0 1/174 (0.6%) 1
    Gastrooesophageal reflux disease 1/56 (1.8%) 1 0/174 (0%) 0
    Ileus 0/56 (0%) 0 1/174 (0.6%) 1
    Umbilical hernia 0/56 (0%) 0 1/174 (0.6%) 1
    General disorders
    Chest pain 1/56 (1.8%) 1 0/174 (0%) 0
    Pyrexia 1/56 (1.8%) 1 1/174 (0.6%) 1
    Hepatobiliary disorders
    Hepatic failure 1/56 (1.8%) 1 0/174 (0%) 0
    Hepatic function abnormal 0/56 (0%) 0 1/174 (0.6%) 1
    Cholangitis acute 0/56 (0%) 0 1/174 (0.6%) 1
    Cholecystitis acute 1/56 (1.8%) 1 1/174 (0.6%) 1
    Infections and infestations
    Covid-19 pneumonia 0/56 (0%) 0 1/174 (0.6%) 1
    Renal abscess 0/56 (0%) 0 1/174 (0.6%) 1
    Appendicitis 0/56 (0%) 0 1/174 (0.6%) 1
    Appendicitis perforated 1/56 (1.8%) 1 0/174 (0%) 0
    Atypical pneumonia 0/56 (0%) 0 1/174 (0.6%) 1
    Bacteraemia 0/56 (0%) 0 1/174 (0.6%) 1
    Bronchiolitis 1/56 (1.8%) 1 0/174 (0%) 0
    Covid-19 0/56 (0%) 0 1/174 (0.6%) 1
    Cellulitis 0/56 (0%) 0 1/174 (0.6%) 1
    Central nervous system infection 0/56 (0%) 0 1/174 (0.6%) 1
    Chorioretinitis 0/56 (0%) 0 1/174 (0.6%) 1
    Hepatitis a 1/56 (1.8%) 1 0/174 (0%) 0
    Herpes zoster 0/56 (0%) 0 1/174 (0.6%) 1
    Influenza 0/56 (0%) 0 1/174 (0.6%) 1
    Intervertebral discitis 1/56 (1.8%) 1 0/174 (0%) 0
    Meningitis viral 1/56 (1.8%) 1 0/174 (0%) 0
    Neuroborreliosis 1/56 (1.8%) 1 0/174 (0%) 0
    Osteomyelitis 1/56 (1.8%) 1 0/174 (0%) 0
    Pneumonia 3/56 (5.4%) 3 3/174 (1.7%) 5
    Pneumonia bacterial 0/56 (0%) 0 1/174 (0.6%) 1
    Progressive multifocal leukoencephalopat 0/56 (0%) 0 1/174 (0.6%) 1
    Pyelonephritis chronic 1/56 (1.8%) 1 0/174 (0%) 0
    Sepsis 1/56 (1.8%) 1 0/174 (0%) 0
    Septic shock 1/56 (1.8%) 1 0/174 (0%) 0
    Sinusitis 1/56 (1.8%) 1 0/174 (0%) 0
    Subcutaneous abscess 1/56 (1.8%) 1 0/174 (0%) 0
    Urinary tract infection 7/56 (12.5%) 11 3/174 (1.7%) 3
    Injury, poisoning and procedural complications
    Burns third degree 0/56 (0%) 0 1/174 (0.6%) 1
    Foot fracture 0/56 (0%) 0 1/174 (0.6%) 1
    Infusion related reaction 0/56 (0%) 0 1/174 (0.6%) 1
    Overdose 0/56 (0%) 0 1/174 (0.6%) 2
    Wrist fracture 0/56 (0%) 0 1/174 (0.6%) 1
    Investigations
    International normalised ratio increased 0/56 (0%) 0 1/174 (0.6%) 1
    Weight decreased 0/56 (0%) 0 1/174 (0.6%) 1
    Metabolism and nutrition disorders
    Hypoglycaemia 1/56 (1.8%) 1 0/174 (0%) 0
    Hyponatraemia 2/56 (3.6%) 2 0/174 (0%) 0
    Musculoskeletal and connective tissue disorders
    Back pain 1/56 (1.8%) 1 1/174 (0.6%) 1
    Arthralgia 0/56 (0%) 0 2/174 (1.1%) 2
    Arthritis 0/56 (0%) 0 1/174 (0.6%) 1
    Connective tissue disorder 0/56 (0%) 0 1/174 (0.6%) 1
    Muscular weakness 2/56 (3.6%) 2 0/174 (0%) 0
    Pain in extremity 0/56 (0%) 0 1/174 (0.6%) 1
    Rhabdomyolysis 1/56 (1.8%) 1 0/174 (0%) 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer female 1/56 (1.8%) 1 0/174 (0%) 0
    Colon cancer 0/56 (0%) 0 1/174 (0.6%) 1
    Nervous system disorders
    Optic neuritis 0/56 (0%) 0 1/174 (0.6%) 1
    Peripheral nerve palsy 0/56 (0%) 0 1/174 (0.6%) 1
    Cerebrovascular accident 1/56 (1.8%) 1 0/174 (0%) 0
    Lumbosacral radiculopathy 1/56 (1.8%) 1 0/174 (0%) 0
    Migraine 1/56 (1.8%) 1 0/174 (0%) 0
    Myelitis transverse 0/56 (0%) 0 1/174 (0.6%) 1
    Neuromyelitis optica spectrum disorder 2/56 (3.6%) 2 3/174 (1.7%) 3
    Post cardiac arrest syndrome 0/56 (0%) 0 1/174 (0.6%) 1
    Seizure 1/56 (1.8%) 1 0/174 (0%) 0
    Unresponsive to stimuli 1/56 (1.8%) 2 0/174 (0%) 0
    Uraemic encephalopathy 1/56 (1.8%) 1 0/174 (0%) 0
    Psychiatric disorders
    Delirium 1/56 (1.8%) 1 0/174 (0%) 0
    Depression 1/56 (1.8%) 1 0/174 (0%) 0
    Renal and urinary disorders
    Calculus bladder 0/56 (0%) 0 1/174 (0.6%) 1
    Acute kidney injury 2/56 (3.6%) 2 0/174 (0%) 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure 1/56 (1.8%) 1 0/174 (0%) 0
    Dyspnoea 1/56 (1.8%) 3 0/174 (0%) 0
    Pickwickian syndrome 1/56 (1.8%) 1 0/174 (0%) 0
    Pulmonary embolism 1/56 (1.8%) 1 0/174 (0%) 0
    Respiratory failure 1/56 (1.8%) 2 1/174 (0.6%) 1
    Sleep apnoea syndrome 1/56 (1.8%) 1 0/174 (0%) 0
    Skin and subcutaneous tissue disorders
    Drug reaction with eosinophilia and syst 1/56 (1.8%) 1 0/174 (0%) 0
    Vascular disorders
    Shock 0/56 (0%) 0 1/174 (0.6%) 1
    Other (Not Including Serious) Adverse Events
    Placebo/Inebilizumab Inebilizumab/Inebilizumab
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 48/56 (85.7%) 138/174 (79.3%)
    Blood and lymphatic system disorders
    Anaemia 4/56 (7.1%) 4 11/174 (6.3%) 12
    Eye disorders
    Dry eye 6/56 (10.7%) 6 4/174 (2.3%) 4
    Eye pain 5/56 (8.9%) 5 8/174 (4.6%) 27
    Gastrointestinal disorders
    Abdominal pain 4/56 (7.1%) 4 3/174 (1.7%) 3
    Constipation 7/56 (12.5%) 7 9/174 (5.2%) 13
    Diarrhoea 8/56 (14.3%) 10 15/174 (8.6%) 19
    Gastritis 3/56 (5.4%) 3 3/174 (1.7%) 3
    Nausea 9/56 (16.1%) 9 15/174 (8.6%) 31
    Vomiting 6/56 (10.7%) 8 9/174 (5.2%) 11
    General disorders
    Fatigue 3/56 (5.4%) 3 12/174 (6.9%) 16
    Influenza like illness 4/56 (7.1%) 6 3/174 (1.7%) 6
    Pyrexia 5/56 (8.9%) 12 9/174 (5.2%) 12
    Infections and infestations
    Bronchitis 5/56 (8.9%) 6 12/174 (6.9%) 13
    Conjunctivitis 3/56 (5.4%) 5 5/174 (2.9%) 6
    Influenza 4/56 (7.1%) 10 17/174 (9.8%) 20
    Nasopharyngitis 11/56 (19.6%) 17 38/174 (21.8%) 69
    Oral herpes 4/56 (7.1%) 8 5/174 (2.9%) 6
    Rhinitis 2/56 (3.6%) 3 9/174 (5.2%) 10
    Upper respiratory tract infection 10/56 (17.9%) 15 28/174 (16.1%) 51
    Urinary tract infection 20/56 (35.7%) 54 40/174 (23%) 77
    Injury, poisoning and procedural complications
    Fall 3/56 (5.4%) 3 11/174 (6.3%) 18
    Infusion related reaction 9/56 (16.1%) 16 22/174 (12.6%) 47
    Metabolism and nutrition disorders
    Hypokalaemia 4/56 (7.1%) 4 1/174 (0.6%) 1
    Musculoskeletal and connective tissue disorders
    Arthralgia 12/56 (21.4%) 14 28/174 (16.1%) 37
    Muscle spasms 4/56 (7.1%) 6 5/174 (2.9%) 8
    Muscular weakness 4/56 (7.1%) 4 4/174 (2.3%) 9
    Myalgia 3/56 (5.4%) 6 5/174 (2.9%) 5
    Pain in extremity 9/56 (16.1%) 14 11/174 (6.3%) 20
    Back pain 8/56 (14.3%) 8 24/174 (13.8%) 38
    Nervous system disorders
    Dizziness 3/56 (5.4%) 3 7/174 (4%) 7
    Headache 13/56 (23.2%) 16 31/174 (17.8%) 82
    Hypoaesthesia 2/56 (3.6%) 3 12/174 (6.9%) 24
    Paraesthesia 2/56 (3.6%) 2 12/174 (6.9%) 15
    Psychiatric disorders
    Depression 6/56 (10.7%) 6 8/174 (4.6%) 8
    Insomnia 6/56 (10.7%) 6 10/174 (5.7%) 10
    Renal and urinary disorders
    Micturition urgency 3/56 (5.4%) 3 2/174 (1.1%) 2
    Respiratory, thoracic and mediastinal disorders
    Cough 6/56 (10.7%) 15 17/174 (9.8%) 20
    Rhinorrhoea 3/56 (5.4%) 6 3/174 (1.7%) 5
    Skin and subcutaneous tissue disorders
    Alopecia 3/56 (5.4%) 3 5/174 (2.9%) 5
    Eczema 4/56 (7.1%) 4 4/174 (2.3%) 5
    Pruritus 6/56 (10.7%) 6 7/174 (4%) 7
    Rash 4/56 (7.1%) 6 7/174 (4%) 11
    Vascular disorders
    Hypertension 3/56 (5.4%) 4 5/174 (2.9%) 11
    Hypotension 4/56 (7.1%) 4 2/174 (1.1%) 2

    Limitations/Caveats

    In the safety follow-up period (SFP), only 1 participant from 'Placebo/Inebilizumab' arm rolled over to SFP and no participant from 'Inebilizumab /Inebilizumab' arm rolled over to SFP. For EudraCT result posting, a study period with any one of the arm with zero participants started is not acceptable (EudraCT limitation). Therefore, not included SFP in 'Participant Flow' section to keep the data consistent across registry portals.

    More Information

    Certain Agreements

    Principal Investigators are NOT employed by the organization sponsoring the study.

    Viela Bio has 60 days to review results communications prior to public release and may delete information that compromises ongoing studies or is considered proprietary. This restriction is not intended to compromise the objective scientific integrity of the manuscript, it is understood that results shall be published regardless of outcome. The PIs also agree for data to be presented first as a joint, multicentre publication.

    Results Point of Contact

    Name/Title Global Clinical Lead
    Organization AstraZeneca Clinical study Information Center
    Phone +1-877-240-9479
    Email information.center@astrazeneca.com
    Responsible Party:
    MedImmune LLC
    ClinicalTrials.gov Identifier:
    NCT02200770
    Other Study ID Numbers:
    • CD-IA-MEDI-551-1155
    • 2014-000253-36
    First Posted:
    Jul 25, 2014
    Last Update Posted:
    Dec 3, 2021
    Last Verified:
    Nov 1, 2021