A Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults With Generalized Myasthenia Gravis
Sponsor
Momenta Pharmaceuticals, Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT03772587
Collaborator
(none)
68
61
5
14.5
1.1
0.1
Study Details
Study Description
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, and efficacy of M281 administered to participants with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing standard of care therapy.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Study Type:
Interventional
Actual Enrollment
:
68 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults With Generalized Myasthenia Gravis
Actual Study Start Date
:
Apr 10, 2019
Actual Primary Completion Date
:
Jun 25, 2020
Actual Study Completion Date
:
Jun 25, 2020
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Placebo Comparator: Group 1
|
Other: Placebo
Placebo administered as intravenous (IV) infusion
|
| Experimental: Group 2
|
Drug: M281
M281 administered as IV infusion
|
| Experimental: Group 3
|
Drug: M281
M281 administered as IV infusion
|
| Experimental: Group 4
|
Drug: M281
M281 administered as IV infusion
|
| Experimental: Group 5
|
Drug: M281
M281 administered as IV infusion
|
Outcome Measures
Primary Outcome Measures
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) as a Measure of Safety and Tolerability [Up to Day 113]
An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAE are defined as any AE occurring during or after the initiation of the first infusion of study drug.
- Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) [Up to Day 113]
An AE is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug. An SAE is defined as any AE occurring at any dose that results in any of the following outcomes: death, life-threatening AE, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect.
- Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESI) [Up to Day 113]
An AE is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug. For this study, any common terminology criteria for adverse events (CTCAE) Grade 3 or higher event of severe infection or hypoalbuminemia was considered as AESI.
- Change From Baseline to Day 57 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Total Score [Baseline to Day 57]
The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.
Secondary Outcome Measures
- Change From Baseline in Total MG-ADL Score as a Function of Total Serum Immunoglobulin G (IgG) at Day 57 [Baseline and Day 57]
Estimate of additional change from baseline in MG-ADL total score for every 10 percent (%) additional reduction in IgG based on a linear regression of change from baseline in MG-ADL total score on percent reduction in IgG at Day 57. The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.
- Change From Baseline in Total MG-ADL Score as a Response to Percent Change in Total Serum IgG, for Participants Positive for Anti-acetylcholine Receptor (Anti-AChR) Antibodies, at Day 57 [Baseline and Day 57]
Estimate of additional change from baseline in MG-ADL total score for every 10% additional reduction in IgG based on a linear regression of change from baseline in MG-ADL total score on percent reduction in IgG at Day 57 in anti-AChR positive participants. The MG-ADL was used to assess participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). Total score is sum of eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.
- Change From Baseline in Total Quantitative Myasthenia Gravis (QMG) Score as a Function of Total Serum IgG at Day 57 [Baseline and Day 57]
Estimate of additional change from baseline in QMG total score for every 10% additional reduction in IgG based on a linear regression of change from baseline in QMG total score on percent reduction in IgG at Day 57. The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment.
- Change From Baseline in Total QMG Score as a Response to Percent Change in Total Serum IgG, for Participants Positive for Anti-acetylcholine Receptor (Anti-AChR) Antibodies, at Day 57 [Baseline and Day 57]
Estimate of additional change from baseline in QMG total score for every 10% additional reduction in IgG based on a linear regression of change from baseline in QMG total score on percent reduction in IgG at Day 57 in anti-AChR positive participants. The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment.
- Number of Participants With a 2-, 3-, 4-, 5-, 6-, 7-, or Greater Than or Equal to (>=) 8-point Improvement in Total MG-ADL Score at Day 57 [Day 57]
The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.
- Change From Baseline in Total QMG Score at Day 57 [Baseline and Day 57]
The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment.
- Number of Participants With a 3-, 4-, 5-, 6-, 7-, or >= 8-point Improvement in Total QMG Score at Day 57 [Day 57]
The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment.
- Change From Baseline in Total Revised Myasthenia Gravis Quality of Life - 15 (MG-QoL-15r) Scale Score at Day 57 [Baseline and Day 57]
The MG-QoL15r was used to assess the participant's limitations related to living with MG. Each of the 15 questions were rated by the participant on a 3-point scale (0= Not at all, 1= somewhat, 2=very much) based on a recall period of "over the past few weeks". The total score is the sum of the 15 question scores and ranges from 0 to 30. Higher scores indicated more limitation.
- Change From Baseline in Total Serum IgG at Day 57 [Baseline and Day 57]
Change from baseline in total serum IgG was reported. Blood samples were collected for analysis of total serum IgG.
- Change From Baseline in Total MG-ADL Score at Day 85 and Day 113 [Baseline, Day 85 and Day 113]
The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities.
- Change From Baseline in Total QMG Score at Day 85 and Day 113 [Baseline, Day 85 and Day 113]
The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment.
- Change From Baseline in Total MG-QoL15r Score at Day 85 and Day 113 [Baseline, Day 85 and Day 113]
The MG-QoL15r was used to assess the participant's limitations related to living with MG. Each of the 15 questions were rated by the participant on a 3-point scale (0= Not at all, 1= somewhat, 2=very much) based on a recall period of "over the past few weeks". The total score is the sum of the 15 question scores and ranges from 0 to 30. Higher scores indicated more limitation.
- Number of Participants With Shift From Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification at Day 57 [Baseline and Day 57]
The MGFA was used to assess the participant's MG severity. MGFA classification identifies the subgroup participants with MG who share distinct clinical features or severity of disease: Class I (ocular MG), classes II, III and IV generalized MG with mild, moderate and severe disease, respectively; Class V MG crisis. Separate subclasses under classes II, III and IV are designed: "a" if the predominant weakness is affecting limb/axial weakness or both; subclass "b" if the predominant weakness is affecting oropharyngeal or respiratory muscles or both. In the MGFA classification, lower roman numerals mean less severity. Changes in MGFA classification (regardless of subclass) are categorized as "Improved" (example, III to II), "Same" (example, II to II), or "Worsened" (example, II to III).
- Number of Participants With Shift From Baseline in MGFA Classification to Day 113 [Baseline to Day 113]
The MGFA was used to assess the participant's MG severity. MGFA classification identifies the subgroup participants with MG who share distinct clinical features or severity of disease: Class I (ocular MG), classes II, III and IV generalized MG with mild, moderate and severe disease, respectively; Class V MG crisis. Separate subclasses under classes II, III and IV are designed: "a" if the predominant weakness is affecting limb/axial weakness or both; subclass "b" if the predominant weakness is affecting oropharyngeal or respiratory muscles or both. In the MGFA classification, lower roman numerals mean less severity. Changes in MGFA classification (regardless of subclass) are categorized as "Improved" (example, III to II), "Same" (example, II to II), or "Worsened" (example, II to III).
- Change From Baseline in Total Serum IgG at Day 85 and Day 113 [Baseline, Day 85 and Day 113]
Change from baseline in total serum IgG at Day 85 and Day 113 was analyzed. Blood samples were collected for analysis of total serum IgG.
- Serum Concentrations of Nipocalimab [Baseline (Pre Infusion and Post Infusion), Day 15 (Pre Infusion), Day 29 (Pre Infusion), Day 43 (Pre Infusion), Day 57 (Pre Infusion and Post Infusion) and Day 85]
Serum concentrations of nipocalimab were reported. Concentrations below the lowest quantifiable concentration (< LLOQ) that is <0.15 microgram/milliliter (mcg/mL) was treated as zero in calculating the summary statistics.
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Participants must be ≥18 years of age with a documented history of Generalized Myasthenia Gravis (gMG) and clinical signs/symptoms of gMG, not pregnant or breastfeeding, and no history of any neurologic disorder other than MG that might interfere with the accuracy of study assessments.
Additional, more specific criteria are defined in the protocol.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Momenta Investigational Site | Phoenix | Arizona | United States | 85013 |
| 2 | Momenta Investigational Site | Tucson | Arizona | United States | 85724 |
| 3 | Momenta Investigational Site | Los Angeles | California | United States | 90048 |
| 4 | Momenta Investigational Site | Orange | California | United States | 92868 |
| 5 | Momenta Investigational Site | Stanford | California | United States | 94305 |
| 6 | Momenta Investigational Site | Aurora | Colorado | United States | 80045 |
| 7 | Momenta Investigational Site | New Haven | Connecticut | United States | 06511 |
| 8 | Momenta Investigational Site | Boca Raton | Florida | United States | 33487 |
| 9 | Momenta Investigational Site | Maitland | Florida | United States | 32751 |
| 10 | Momenta Investigational Site | Port Charlotte | Florida | United States | 33952 |
| 11 | Momenta Investigational Site | Saint Petersburg | Florida | United States | 33713 |
| 12 | Momenta Investigational Site | Augusta | Georgia | United States | 30912 |
| 13 | Momenta Investigational Site | Meridian | Idaho | United States | 83642 |
| 14 | Momenta Investigational Site | Lake Barrington | Illinois | United States | 60010 |
| 15 | Momenta Investigational Site | Fairway | Kansas | United States | 66205 |
| 16 | Momenta Investigational Site | Boston | Massachusetts | United States | 02114 |
| 17 | Momenta Investigational Site | Boston | Massachusetts | United States | 02115 |
| 18 | Momenta Investigational Site | Boston | Massachusetts | United States | 02215 |
| 19 | Momenta Investigational Site | Detroit | Michigan | United States | 48202 |
| 20 | Momenta Investigational Site | East Lansing | Michigan | United States | 48824 |
| 21 | Momenta Investigational Site | Columbia | Missouri | United States | 65212 |
| 22 | Momenta Investigational Site | New Brunswick | New Jersey | United States | 08550 |
| 23 | Momenta Investigational Site | New York | New York | United States | 10021 |
| 24 | Momenta Investigational Site | Charlotte | North Carolina | United States | 28207 |
| 25 | Momenta Investigational Site | Durham | North Carolina | United States | 27710 |
| 26 | Momenta Investigational Site | Raleigh | North Carolina | United States | 27607 |
| 27 | Momenta Investigational Site | Cincinnati | Ohio | United States | 45267 |
| 28 | Momenta Investigational Site | Columbus | Ohio | United States | 43210 |
| 29 | Momenta Investigational Site | Cordova | Tennessee | United States | 38106 |
| 30 | Momenta Investigational Site | Austin | Texas | United States | 78756 |
| 31 | Momenta Investigational Site | Round Rock | Texas | United States | 78681 |
| 32 | Momenta Investigational Site | Leuven | Vlaams Brabant | Belgium | 3000 |
| 33 | Momenta Investigational Site | Antwerp | Belgium | 2650 | |
| 34 | Momenta Investigational Site | Bruxelles | Belgium | 1070 | |
| 35 | Momenta Investigational Site | Edmonton | Alberta | Canada | T6G 2G3 |
| 36 | Momenta Investigational Site | London | Ontario | Canada | N6A 5A5 |
| 37 | Momenta Investigational Site | Toronto | Ontario | Canada | M5G 2C4 |
| 38 | Momenta Investigational Site | Quebec City | Quebec | Canada | G1J 1Z4 |
| 39 | Momenta Investigational Site | Gottingen | Niedersachsen | Germany | 37075 |
| 40 | Momenta Investigational Site | Düsseldorf | Germany | 40225 | |
| 41 | Momenta Investigational Site | Gummersbach | Germany | 51643 | |
| 42 | Momenta Investigational Site | Munster | Germany | 48149 | |
| 43 | Momenta Investigational Site | Cefalu | Italy | 90015 | |
| 44 | Momenta Investigational Site | Messina | Italy | 98125 | |
| 45 | Momenta Investigational Site | Milano | Italy | 20133 | |
| 46 | Momenta Investigational Site | Krakow | Poland | 31-505 | |
| 47 | Momenta Investigational Site | Lodz | Poland | 90-324 | |
| 48 | Momenta Investigational Site | Warsaw | Poland | 01-684 | |
| 49 | Momenta Investigational Site | Warsaw | Poland | 01-868 | |
| 50 | Momenta Investigational Site | Barcelona | Catalan | Spain | 08035 |
| 51 | Momenta Investigational Site | Barcelona | Catalan | Spain | 08041 |
| 52 | Momenta Investigational Site | Badalona | Cataluna | Spain | 08036 |
| 53 | Momenta Investigational Site | L'hospitalet De Llobregat | Cataluna | Spain | 08907 |
| 54 | Momenta Investigational Site | San Sebastian | Gipuzkoa | Spain | 20014 |
| 55 | Momenta Investigational Site | Madrid | Spain | 28007 | |
| 56 | Momenta Investigational Site | Madrid | Spain | 28040 | |
| 57 | Momenta Investigational Site | Sevilla | Spain | 41013 | |
| 58 | Momenta Investigational Site | Valencia | Spain | 46026 | |
| 59 | Momenta Investigational Site | Birmingham | United Kingdom | B15 2TH | |
| 60 | Momenta Investigational Site | Sheffield | United Kingdom | S11 9NE | |
| 61 | Momenta Investigational Site | Southampton | United Kingdom | SO16 6YD |
Sponsors and Collaborators
- Momenta Pharmaceuticals, Inc.
Investigators
- Study Director: Momenta General Queries, Momenta Pharmaceuticals, Inc.
Study Documents (Full-Text)
More Information
Publications
None provided.Responsible Party:
Momenta Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT03772587
Other Study ID Numbers:
- MOM-M281-004
- 2018-002247-28
First Posted:
Dec 11, 2018
Last Update Posted:
Oct 27, 2021
Last Verified:
Sep 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Momenta Pharmaceuticals, Inc.
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Placebo | Nipocalimab (M281) 5 Milligrams/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received intravenous (IV) infusion of placebo matching to nipocalimab once every 2 weeks (Q2W) starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once every 4 weeks (Q4W) starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Period Title: Overall Study | |||||
| STARTED | 14 | 14 | 13 | 13 | 14 |
| COMPLETED | 13 | 14 | 12 | 12 | 14 |
| NOT COMPLETED | 1 | 0 | 1 | 1 | 0 |
Baseline Characteristics
| Arm/Group Title | Placebo | Nipocalimab 5 Milligrams/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) | Total |
|---|---|---|---|---|---|---|
| Arm/Group Description | Participants received intravenous (IV) infusion of placebo matching to nipocalimab once every 2 weeks (Q2W) starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once every 4 weeks (Q4W) starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. | Total of all reporting groups |
| Overall Participants | 14 | 14 | 13 | 13 | 14 | 68 |
| Age (Count of Participants) | ||||||
| <=18 years |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Between 18 and 65 years |
8
57.1%
|
9
64.3%
|
9
69.2%
|
9
69.2%
|
8
57.1%
|
43
63.2%
|
| >=65 years |
6
42.9%
|
5
35.7%
|
4
30.8%
|
4
30.8%
|
6
42.9%
|
25
36.8%
|
| Age (years) [Mean (Standard Deviation) ] | ||||||
| Mean (Standard Deviation) [years] |
57.7
(17.85)
|
54.8
(17.64)
|
49
(19.54)
|
53.1
(15.4)
|
59.9
(15.03)
|
55
(17.07)
|
| Sex: Female, Male (Count of Participants) | ||||||
| Female |
8
57.1%
|
6
42.9%
|
9
69.2%
|
9
69.2%
|
5
35.7%
|
37
54.4%
|
| Male |
6
42.9%
|
8
57.1%
|
4
30.8%
|
4
30.8%
|
9
64.3%
|
31
45.6%
|
| Race/Ethnicity, Customized (Count of Participants) | ||||||
| American Indian or Alaska Native |
0
0%
|
0
0%
|
1
7.7%
|
0
0%
|
0
0%
|
1
1.5%
|
| Asian |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Black or African American |
0
0%
|
2
14.3%
|
0
0%
|
0
0%
|
0
0%
|
2
2.9%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| White |
12
85.7%
|
12
85.7%
|
12
92.3%
|
13
100%
|
14
100%
|
63
92.6%
|
| More than one race |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Other |
2
14.3%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
2
2.9%
|
| Race/Ethnicity, Customized (Count of Participants) | ||||||
| Hispanic or Latino |
4
28.6%
|
1
7.1%
|
3
23.1%
|
1
7.7%
|
0
0%
|
9
13.2%
|
| Not Hispanic or Latino |
10
71.4%
|
13
92.9%
|
10
76.9%
|
11
84.6%
|
14
100%
|
58
85.3%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
1
7.7%
|
0
0%
|
1
1.5%
|
| Region of Enrollment (Count of Participants) | ||||||
| BELGIUM |
1
7.1%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
1
1.5%
|
| CANADA |
2
14.3%
|
2
14.3%
|
0
0%
|
0
0%
|
1
7.1%
|
5
7.4%
|
| GERMANY |
1
7.1%
|
3
21.4%
|
1
7.7%
|
0
0%
|
0
0%
|
5
7.4%
|
| ITALY |
2
14.3%
|
2
14.3%
|
1
7.7%
|
0
0%
|
0
0%
|
5
7.4%
|
| POLAND |
1
7.1%
|
2
14.3%
|
1
7.7%
|
3
23.1%
|
1
7.1%
|
8
11.8%
|
| SPAIN |
4
28.6%
|
0
0%
|
6
46.2%
|
2
15.4%
|
3
21.4%
|
15
22.1%
|
| UNITED KINGDOM |
0
0%
|
0
0%
|
0
0%
|
3
23.1%
|
1
7.1%
|
4
5.9%
|
| UNITED STATES |
3
21.4%
|
5
35.7%
|
4
30.8%
|
5
38.5%
|
8
57.1%
|
25
36.8%
|
Outcome Measures
| Title | Number of Participants With Treatment-emergent Adverse Events (TEAEs) as a Measure of Safety and Tolerability |
|---|---|
| Description | An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAE are defined as any AE occurring during or after the initiation of the first infusion of study drug. |
| Time Frame | Up to Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The safety population included all participants who received any amount of nipocalimab or placebo. |
| Arm/Group Title | Placebo | Nipocalimab 5 Milligrams/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received intravenous (IV) infusion of placebo matching to nipocalimab once every 2 weeks (Q2W) starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once every 4 weeks (Q4W) starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 14 | 14 | 13 | 13 | 14 |
| Count of Participants [Participants] |
11
78.6%
|
12
85.7%
|
9
69.2%
|
12
92.3%
|
12
85.7%
|
| Title | Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) |
|---|---|
| Description | An AE is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug. An SAE is defined as any AE occurring at any dose that results in any of the following outcomes: death, life-threatening AE, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect. |
| Time Frame | Up to Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The safety population included all participants who received any amount of nipocalimab or placebo. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 14 | 14 | 13 | 13 | 14 |
| Count of Participants [Participants] |
2
14.3%
|
0
0%
|
1
7.7%
|
0
0%
|
0
0%
|
| Title | Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESI) |
|---|---|
| Description | An AE is any untoward medical event that occurs in a participant administered an investigational product and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as any AE occurring during or after the initiation of the first infusion of study drug. For this study, any common terminology criteria for adverse events (CTCAE) Grade 3 or higher event of severe infection or hypoalbuminemia was considered as AESI. |
| Time Frame | Up to Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The safety population included all participants who received any amount of nipocalimab or placebo. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 14 | 14 | 13 | 13 | 14 |
| Count of Participants [Participants] |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Title | Change From Baseline to Day 57 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Total Score |
|---|---|
| Description | The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities. |
| Time Frame | Baseline to Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent-to-Treat (ITT) population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this outcome measure (OM). |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 11 | 14 | 12 | 13 | 14 |
| Mean (Standard Deviation) [score on a scale] |
-1.8
(3.22)
|
-2.5
(2.41)
|
-3.9
(3.00)
|
-1.5
(2.82)
|
-3.9
(3.66)
|
| Title | Change From Baseline in Total MG-ADL Score as a Function of Total Serum Immunoglobulin G (IgG) at Day 57 |
|---|---|
| Description | Estimate of additional change from baseline in MG-ADL total score for every 10 percent (%) additional reduction in IgG based on a linear regression of change from baseline in MG-ADL total score on percent reduction in IgG at Day 57. The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities. |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants over all arms with both an MG-ADL score and an IgG result at both baseline and Day 57 are included in the analysis. Participants are combined over all groups because the analysis correlates MG-ADL change with IgG change and arm was not included as a factor in the pre-specified analysis. Therefore, results by arm are not available. |
| Arm/Group Title | Placebo/Nipocalimab (All Participants) |
|---|---|
| Arm/Group Description | All participants combined over all arms received IV infusion either of placebo or nipocalimab (5 mg/kg [once Q4W]/30 mg/kg[once Q4W]/60 mg/kg[once Q4W]/60 mg/kg [once Q2W]) starting Day 1 up to Day 57. |
| Measure Participants | 58 |
| Least Squares Mean (Standard Error) [score on a scale per 10% IgG reduction] |
-0.30
(0.136)
|
| Title | Change From Baseline in Total MG-ADL Score as a Response to Percent Change in Total Serum IgG, for Participants Positive for Anti-acetylcholine Receptor (Anti-AChR) Antibodies, at Day 57 |
|---|---|
| Description | Estimate of additional change from baseline in MG-ADL total score for every 10% additional reduction in IgG based on a linear regression of change from baseline in MG-ADL total score on percent reduction in IgG at Day 57 in anti-AChR positive participants. The MG-ADL was used to assess participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). Total score is sum of eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities. |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All anti-AChR positive participants over all arms with both an MG-ADL score and an IgG result at both baseline and Day 57 are included in the analysis. Participants are combined over all groups because the analysis correlates MG-ADL change with IgG change and arm was not included as a factor in the pre-specified analysis. Therefore, results by arm are not available. |
| Arm/Group Title | Placebo/Nipocalimab (All Anti-AChR Positive Participants) |
|---|---|
| Arm/Group Description | All anti-AChR positive participants combined over all arms received IV infusion of either placebo or nipocalimab (5 mg/kg [once Q4W]/30 mg/kg[once Q4W]/60 mg/kg[once Q4W]/60 mg/kg [once Q2W]) starting Day 1 up to Day 57. |
| Measure Participants | 55 |
| Least Squares Mean (Standard Error) [score on a scale per 10% IgG reduction] |
-0.33
(0.144)
|
| Title | Change From Baseline in Total Quantitative Myasthenia Gravis (QMG) Score as a Function of Total Serum IgG at Day 57 |
|---|---|
| Description | Estimate of additional change from baseline in QMG total score for every 10% additional reduction in IgG based on a linear regression of change from baseline in QMG total score on percent reduction in IgG at Day 57. The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment. |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants over all arms with both an QMG score and an IgG result at both baseline and Day 57 are included in the analysis. Participants are combined over all groups because the analysis correlates QMG change with IgG change and arm was not included as a factor in the pre-specified analysis. Therefore, results by arm are not available. |
| Arm/Group Title | Placebo/Nipocalimab (All Participants) |
|---|---|
| Arm/Group Description | All participants combined over all arms received IV infusion of either placebo or nipocalimab (5 mg/kg [once Q4W]/30 mg/kg[once Q4W]/60 mg/kg[once Q4W]/60 mg/kg [once Q2W]) starting Day 1 up to Day 57. |
| Measure Participants | 58 |
| Least Squares Mean (Standard Error) [score on a scale per 10% IgG reduction] |
-0.38
(0.174)
|
| Title | Change From Baseline in Total QMG Score as a Response to Percent Change in Total Serum IgG, for Participants Positive for Anti-acetylcholine Receptor (Anti-AChR) Antibodies, at Day 57 |
|---|---|
| Description | Estimate of additional change from baseline in QMG total score for every 10% additional reduction in IgG based on a linear regression of change from baseline in QMG total score on percent reduction in IgG at Day 57 in anti-AChR positive participants. The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment. |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All anti-AChR positive participants over all arms with both an QMG score and an IgG result at both baseline and Day 57 are included in the analysis. Participants are combined over all groups because the analysis correlates QMG change with IgG change and arm was not included as a factor in the pre-specified analysis. Therefore, results by arm are not available. |
| Arm/Group Title | Placebo/Nipocalimab (All Anti-AChR Positive Participants) |
|---|---|
| Arm/Group Description | All anti-AChR positive participants combined over all arms received IV infusion of either placebo or nipocalimab (5 mg/kg [once Q4W]/30 mg/kg[once Q4W]/60 mg/kg[once Q4W]/60 mg/kg [once Q2W]) starting Day 1 up to Day 57. |
| Measure Participants | 55 |
| Least Squares Mean (Standard Error) [score on a scale per 10% IgG reduction] |
-0.45
(0.181)
|
| Title | Number of Participants With a 2-, 3-, 4-, 5-, 6-, 7-, or Greater Than or Equal to (>=) 8-point Improvement in Total MG-ADL Score at Day 57 |
|---|---|
| Description | The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities. |
| Time Frame | Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Population analyzed included ITT participants for whom data was available at Day 57. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 11 | 14 | 12 | 13 | 14 |
| 2-point Improved |
7
50%
|
9
64.3%
|
10
76.9%
|
7
53.8%
|
12
85.7%
|
| 3-point Improved |
5
35.7%
|
9
64.3%
|
8
61.5%
|
5
38.5%
|
11
78.6%
|
| 4-point Improved |
1
7.1%
|
5
35.7%
|
5
38.5%
|
3
23.1%
|
7
50%
|
| 5-point Improved |
1
7.1%
|
2
14.3%
|
5
38.5%
|
2
15.4%
|
6
42.9%
|
| 6-point Improved |
1
7.1%
|
1
7.1%
|
3
23.1%
|
1
7.7%
|
3
21.4%
|
| 7-point Improved |
1
7.1%
|
1
7.1%
|
3
23.1%
|
0
0%
|
2
14.3%
|
| >=8-point Improved |
1
7.1%
|
0
0%
|
1
7.7%
|
0
0%
|
2
14.3%
|
| Title | Change From Baseline in Total QMG Score at Day 57 |
|---|---|
| Description | The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment. |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 11 | 13 | 10 | 11 | 13 |
| Mean (Standard Deviation) [score on a scale] |
-3.7
(2.94)
|
-3.5
(4.10)
|
-4.1
(3.45)
|
-1.5
(2.54)
|
-5.9
(5.30)
|
| Title | Number of Participants With a 3-, 4-, 5-, 6-, 7-, or >= 8-point Improvement in Total QMG Score at Day 57 |
|---|---|
| Description | The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment. |
| Time Frame | Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Population analyzed included ITT participants for whom data was available at Day 57. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 11 | 13 | 10 | 11 | 13 |
| 3-point Improved |
8
57.1%
|
6
42.9%
|
6
46.2%
|
4
30.8%
|
10
71.4%
|
| 4-point Improved |
5
35.7%
|
5
35.7%
|
6
46.2%
|
3
23.1%
|
10
71.4%
|
| 5-point Improved |
5
35.7%
|
5
35.7%
|
5
38.5%
|
1
7.7%
|
8
57.1%
|
| 6-point Improved |
2
14.3%
|
5
35.7%
|
4
30.8%
|
1
7.7%
|
5
35.7%
|
| 7-point Improved |
2
14.3%
|
5
35.7%
|
1
7.7%
|
0
0%
|
3
21.4%
|
| >= 8-point Improved |
2
14.3%
|
3
21.4%
|
1
7.7%
|
0
0%
|
3
21.4%
|
| Title | Change From Baseline in Total Revised Myasthenia Gravis Quality of Life - 15 (MG-QoL-15r) Scale Score at Day 57 |
|---|---|
| Description | The MG-QoL15r was used to assess the participant's limitations related to living with MG. Each of the 15 questions were rated by the participant on a 3-point scale (0= Not at all, 1= somewhat, 2=very much) based on a recall period of "over the past few weeks". The total score is the sum of the 15 question scores and ranges from 0 to 30. Higher scores indicated more limitation. |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 11 | 14 | 12 | 13 | 14 |
| Mean (Standard Deviation) [score on a scale] |
-2.0
(4.58)
|
-1.7
(4.16)
|
-6.8
(5.73)
|
-1.2
(1.91)
|
-3.7
(5.37)
|
| Title | Change From Baseline in Total Serum IgG at Day 57 |
|---|---|
| Description | Change from baseline in total serum IgG was reported. Blood samples were collected for analysis of total serum IgG. |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 12 | 13 | 10 | 11 | 13 |
| Mean (Standard Deviation) [gram/liter (g/L)] |
-0.3
(1.82)
|
-1.5
(1.01)
|
-3.4
(1.01)
|
-1.7
(1.23)
|
-7.6
(2.27)
|
| Title | Change From Baseline in Total MG-ADL Score at Day 85 and Day 113 |
|---|---|
| Description | The MG-ADL was used to assess the participant's MG symptom severity. It assesses eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, and eyelid droop) which were rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score is the sum of the eight function scores and ranges from 0 to 24. Higher scores indicated greater symptom severity/difficulty in performing daily living activities. |
| Time Frame | Baseline, Day 85 and Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM and 'n' (number analyzed) included the number of participants evaluated for specific timepoints. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 12 | 14 | 12 | 13 | 14 |
| Day 85 |
-2.2
(2.64)
|
-2.1
(2.40)
|
-3.7
(2.69)
|
-1.9
(2.29)
|
-3.6
(2.79)
|
| Day 113 |
-2.6
(3.09)
|
-1.0
(2.25)
|
-2.8
(2.33)
|
-2.4
(2.78)
|
-2.6
(3.30)
|
| Title | Change From Baseline in Total QMG Score at Day 85 and Day 113 |
|---|---|
| Description | The QMG test was used to assess the participant's strength. The quantitative results of each of the 13 strength components were mapped to a 4-point scale where 0 equals to (=) none, 1= mild, 2= moderate and 3= severe. The total score is the sum of the 13 scale scores and ranges from 0 to 39. Higher scores indicated more severe impairment. |
| Time Frame | Baseline, Day 85 and Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM and 'n' (number analyzed) included the number of participants evaluated for specific timepoints. |
| Arm/Group Title | Placebo | Nipocalimab 5 Milligram/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 10 | 14 | 10 | 9 | 13 |
| Day 85 |
-4.0
(2.62)
|
-3.6
(3.23)
|
-4.8
(2.49)
|
-2.0
(2.60)
|
-5.1
(3.52)
|
| Day 113 |
-4.7
(3.04)
|
-2.1
(2.40)
|
-4.2
(3.08)
|
-3.2
(2.28)
|
-3.3
(5.69)
|
| Title | Change From Baseline in Total MG-QoL15r Score at Day 85 and Day 113 |
|---|---|
| Description | The MG-QoL15r was used to assess the participant's limitations related to living with MG. Each of the 15 questions were rated by the participant on a 3-point scale (0= Not at all, 1= somewhat, 2=very much) based on a recall period of "over the past few weeks". The total score is the sum of the 15 question scores and ranges from 0 to 30. Higher scores indicated more limitation. |
| Time Frame | Baseline, Day 85 and Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM and 'n' (number analyzed) included the number of participants evaluated for specific timepoints. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 12 | 14 | 12 | 13 | 13 |
| Day 85 |
-2.5
(2.84)
|
-2.9
(3.74)
|
-6.5
(5.66)
|
-0.7
(4.25)
|
-3.5
(5.61)
|
| Day 113 |
-3.2
(3.90)
|
-1.6
(4.20)
|
-4.2
(4.32)
|
-1.0
(2.92)
|
-2.5
(6.09)
|
| Title | Number of Participants With Shift From Baseline in Myasthenia Gravis Foundation of America (MGFA) Classification at Day 57 |
|---|---|
| Description | The MGFA was used to assess the participant's MG severity. MGFA classification identifies the subgroup participants with MG who share distinct clinical features or severity of disease: Class I (ocular MG), classes II, III and IV generalized MG with mild, moderate and severe disease, respectively; Class V MG crisis. Separate subclasses under classes II, III and IV are designed: "a" if the predominant weakness is affecting limb/axial weakness or both; subclass "b" if the predominant weakness is affecting oropharyngeal or respiratory muscles or both. In the MGFA classification, lower roman numerals mean less severity. Changes in MGFA classification (regardless of subclass) are categorized as "Improved" (example, III to II), "Same" (example, II to II), or "Worsened" (example, II to III). |
| Time Frame | Baseline and Day 57 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 12 | 12 | 11 | 12 | 12 |
| Improved |
6
42.9%
|
3
21.4%
|
7
53.8%
|
4
30.8%
|
7
50%
|
| Same |
6
42.9%
|
9
64.3%
|
3
23.1%
|
8
61.5%
|
4
28.6%
|
| Worsened |
0
0%
|
0
0%
|
1
7.7%
|
0
0%
|
1
7.1%
|
| Title | Number of Participants With Shift From Baseline in MGFA Classification to Day 113 |
|---|---|
| Description | The MGFA was used to assess the participant's MG severity. MGFA classification identifies the subgroup participants with MG who share distinct clinical features or severity of disease: Class I (ocular MG), classes II, III and IV generalized MG with mild, moderate and severe disease, respectively; Class V MG crisis. Separate subclasses under classes II, III and IV are designed: "a" if the predominant weakness is affecting limb/axial weakness or both; subclass "b" if the predominant weakness is affecting oropharyngeal or respiratory muscles or both. In the MGFA classification, lower roman numerals mean less severity. Changes in MGFA classification (regardless of subclass) are categorized as "Improved" (example, III to II), "Same" (example, II to II), or "Worsened" (example, II to III). |
| Time Frame | Baseline to Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 9 | 9 | 8 | 7 | 11 |
| Improved |
3
21.4%
|
2
14.3%
|
3
23.1%
|
2
15.4%
|
3
21.4%
|
| Same |
6
42.9%
|
5
35.7%
|
4
30.8%
|
5
38.5%
|
5
35.7%
|
| Worsened |
0
0%
|
2
14.3%
|
1
7.7%
|
0
0%
|
3
21.4%
|
| Title | Change From Baseline in Total Serum IgG at Day 85 and Day 113 |
|---|---|
| Description | Change from baseline in total serum IgG at Day 85 and Day 113 was analyzed. Blood samples were collected for analysis of total serum IgG. |
| Time Frame | Baseline, Day 85 and Day 113 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population included all randomized participants. Here 'N' (number of participants analyzed) included all participants who were evaluated for this OM and 'n' (number analyzed) included the number of participants evaluated for specific timepoints. |
| Arm/Group Title | Placebo | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of placebo matching to nipocalimab once Q2W starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 11 | 14 | 11 | 9 | 13 |
| Day 85 |
-0.5
(1.02)
|
-1.4
(1.93)
|
-3.8
(1.28)
|
-1.2
(1.02)
|
-5.7
(2.30)
|
| Day 113 |
-0.6
(1.19)
|
-0.7
(1.48)
|
-1.2
(0.78)
|
-0.7
(1.09)
|
-2.2
(1.73)
|
| Title | Serum Concentrations of Nipocalimab |
|---|---|
| Description | Serum concentrations of nipocalimab were reported. Concentrations below the lowest quantifiable concentration (< LLOQ) that is <0.15 microgram/milliliter (mcg/mL) was treated as zero in calculating the summary statistics. |
| Time Frame | Baseline (Pre Infusion and Post Infusion), Day 15 (Pre Infusion), Day 29 (Pre Infusion), Day 43 (Pre Infusion), Day 57 (Pre Infusion and Post Infusion) and Day 85 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The safety population included all participants who received any amount of nipocalimab or placebo. Here 'n' (number analyzed) included the number of participants evaluated for specific timepoints. |
| Arm/Group Title | Nipocalimab 5 mg/kg | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) |
|---|---|---|---|---|
| Arm/Group Description | Participants received IV infusion of 5 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. |
| Measure Participants | 14 | 13 | 13 | 14 |
| Baseline (Pre Infusion) |
0.0
(0.0)
|
0.02
(0.066)
|
0.0
(0.0)
|
0.0
(0.0)
|
| Baseline (Post Infusion) |
107.35
(39.826)
|
708.07
(95.185)
|
1739.93
(287.127)
|
1794.93
(1308.695)
|
| Day 15 (Pre Infusion) |
0.0
(0.0)
|
0.0
(0.0)
|
22.56
(36.198)
|
25.38
(33.402)
|
| Day 29 (Pre Infusion) |
0.0
(0.0)
|
0.02
(0.052)
|
0.0
(0.0)
|
58.34
(113.158)
|
| Day 43 (Pre Infusion) |
0.0
(0.0)
|
0.0
(0.0)
|
0.0
(0.0)
|
61.28
(80.513)
|
| Day 57 (Pre Infusion) |
0.0
(0.0)
|
0.02
(0.71)
|
0.0
(0.0)
|
35.95
(54.440)
|
| Day 57 (Post Infusion) |
105.65
(43.331)
|
752.47
(154.608)
|
0.0
(0.0)
|
1568.92
(288.500)
|
| Day 85 |
0.0
(0.0)
|
0.03
(0.08)
|
0.0
(0.0)
|
0.0
(0.0)
|
Adverse Events
| Time Frame | Up to Day 113 | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | The safety population included all participants who received any amount of nipocalimab or placebo. | |||||||||
| Arm/Group Title | Placebo | Nipocalimab 5 Milligrams/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) | |||||
| Arm/Group Description | Participants received intravenous (IV) infusion of placebo matching to nipocalimab once every 2 weeks (Q2W) starting Day 1 up to Day 57. | Participants received IV infusion of 5 mg/kg nipocalimab once every 4 weeks (Q4W) starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 30 mg/kg nipocalimab once Q4W starting Day 1 up to Day 57. To maintain blinding, participants received matching placebo on Days 15 and 43. | Participants received IV infusion of 60 mg/kg nipocalimab single dose on Day 1. To maintain blinding, participants received matching placebo on Days 15, 29, 43 and 57. | Participants received IV infusion of 60 mg/kg nipocalimab once Q2W starting Day 1 up to Day 57. | |||||
All Cause Mortality |
||||||||||
| Placebo | Nipocalimab 5 Milligrams/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
Serious Adverse Events |
||||||||||
| Placebo | Nipocalimab 5 Milligrams/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 2/14 (14.3%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Musculoskeletal and connective tissue disorders | ||||||||||
| Musculoskeletal Pain | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Nervous system disorders | ||||||||||
| Ischaemic Stroke | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Myasthenia Gravis | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
Other (Not Including Serious) Adverse Events |
||||||||||
| Placebo | Nipocalimab 5 Milligrams/Kilogram (mg/kg) | Nipocalimab 30 mg/kg | Nipocalimab 60 mg/kg | Nipocalimab 60 mg/kg (Q2W) | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 10/14 (71.4%) | 12/14 (85.7%) | 8/13 (61.5%) | 12/13 (92.3%) | 12/14 (85.7%) | |||||
| Blood and lymphatic system disorders | ||||||||||
| Iron Deficiency Anaemia | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Thrombocytopenia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Cardiac disorders | ||||||||||
| Palpitations | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Ear and labyrinth disorders | ||||||||||
| Tinnitus | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Endocrine disorders | ||||||||||
| Hypothyroidism | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Eye disorders | ||||||||||
| Blepharitis | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Eye Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Eyelid Ptosis | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Vision Blurred | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Gastrointestinal disorders | ||||||||||
| Abdominal Pain Upper | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 1/14 (7.1%) | |||||
| Constipation | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Diarrhoea | 1/14 (7.1%) | 1/14 (7.1%) | 1/13 (7.7%) | 2/13 (15.4%) | 2/14 (14.3%) | |||||
| Dysphagia | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Gastric Ulcer | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Gastrooesophageal Reflux Disease | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Nausea | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Salivary Hypersecretion | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Toothache | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Vomiting | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| General disorders | ||||||||||
| Chest Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Feeling Cold | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Feeling Hot | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Hernia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Infusion Site Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Malaise | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Oedema Peripheral | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 2/14 (14.3%) | |||||
| Peripheral Swelling | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Vessel Puncture Site Pruritus | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Vessel Puncture Site Swelling | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Infections and infestations | ||||||||||
| Asymptomatic Bacteriuria | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Bronchitis | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Cellulitis | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Conjunctivitis | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Herpes Zoster | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Hordeolum | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Influenza | 0/14 (0%) | 1/14 (7.1%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Lower Respiratory Tract Infection | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Nasopharyngitis | 0/14 (0%) | 1/14 (7.1%) | 1/13 (7.7%) | 2/13 (15.4%) | 2/14 (14.3%) | |||||
| Pharyngitis | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Pneumonia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Sinusitis | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Upper Respiratory Tract Infection | 1/14 (7.1%) | 1/14 (7.1%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Urinary Tract Infection | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 2/14 (14.3%) | |||||
| Viral Upper Respiratory Tract Infection | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Injury, poisoning and procedural complications | ||||||||||
| Contusion | 0/14 (0%) | 1/14 (7.1%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Fall | 0/14 (0%) | 1/14 (7.1%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Limb Injury | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Muscle Rupture | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Muscle Strain | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Palate Injury | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Skin Laceration | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Spinal Compression Fracture | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Investigations | ||||||||||
| Alanine Aminotransferase Increased | 1/14 (7.1%) | 1/14 (7.1%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Aspartate Aminotransferase Increased | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Bacterial Test | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Blood Creatine Phosphokinase Increased | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Blood Pressure Increased | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Carbohydrate Antigen 19-9 Increased | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Helicobacter Test Positive | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Liver Function Test Increased | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Lymphocyte Count Decreased | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Neutrophil Count Increased | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Neutrophil Percentage Increased | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Urine Analysis Abnormal | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Weight Decreased | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Metabolism and nutrition disorders | ||||||||||
| Glucose Tolerance Impaired | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Hyperglycaemia | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Hypophosphataemia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Musculoskeletal and connective tissue disorders | ||||||||||
| Arthralgia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 2/13 (15.4%) | 0/14 (0%) | |||||
| Arthritis | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Back Pain | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 1/13 (7.7%) | 1/14 (7.1%) | |||||
| Muscle Spasms | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Muscle Twitching | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 1/14 (7.1%) | |||||
| Musculoskeletal Chest Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Musculoskeletal Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 2/13 (15.4%) | 0/14 (0%) | |||||
| Neck Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Rotator Cuff Syndrome | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Spinal Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Nervous system disorders | ||||||||||
| Dizziness | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 2/13 (15.4%) | 0/14 (0%) | |||||
| Dysgeusia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Headache | 1/14 (7.1%) | 2/14 (14.3%) | 1/13 (7.7%) | 2/13 (15.4%) | 1/14 (7.1%) | |||||
| Hypoaesthesia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 2/13 (15.4%) | 0/14 (0%) | |||||
| Mononeuropathy | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Myasthenia Gravis | 1/14 (7.1%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Presyncope | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Tension Headache | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Reproductive system and breast disorders | ||||||||||
| Dysmenorrhoea | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||
| Catarrh | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Cough | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Dysphonia | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Dyspnoea | 0/14 (0%) | 0/14 (0%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Oropharyngeal Pain | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Skin and subcutaneous tissue disorders | ||||||||||
| Dermatitis Allergic | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 1/13 (7.7%) | 0/14 (0%) | |||||
| Erythema | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Pruritus | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Rash | 0/14 (0%) | 1/14 (7.1%) | 0/13 (0%) | 0/13 (0%) | 3/14 (21.4%) | |||||
| Rash Erythematous | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Skin Swelling | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Swelling Face | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
| Vascular disorders | ||||||||||
| Brachiocephalic Vein Thrombosis | 0/14 (0%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 1/14 (7.1%) | |||||
| Hypertension | 0/14 (0%) | 2/14 (14.3%) | 1/13 (7.7%) | 0/13 (0%) | 0/14 (0%) | |||||
| Hypotension | 1/14 (7.1%) | 0/14 (0%) | 0/13 (0%) | 0/13 (0%) | 0/14 (0%) | |||||
Limitations/Caveats
Limitations to this study include the small sample sizes of each treatment arm and the study activity disruption due to the COVID-19 pandemic, especially the missed Quantitative Myasthenia Gravis (QMG) assessments which hampered the analysis of the endpoint.
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
If an investigator wishes to publish information from the study, a copy of the manuscript must be provided to the sponsor for review at least 60 days before submission for publication or presentation. If requested by the sponsor in writing, the investigator will withhold such publication for up to an additional 60 days.
Results Point of Contact
| Name/Title | DIRECTOR CLINICAL RESEARCH |
|---|---|
| Organization | Momenta Pharmaceuticals, Inc. |
| Phone | 844-434-4210 |
| ClinicalTrialDisclosure@its.jnj.com |
Responsible Party:
Momenta Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT03772587
Other Study ID Numbers:
- MOM-M281-004
- 2018-002247-28
First Posted:
Dec 11, 2018
Last Update Posted:
Oct 27, 2021
Last Verified:
Sep 1, 2021