A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

Sponsor

UCB Biopharma SRL (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04650854

Collaborator

(none)

165

Enrollment

Locations

Arms

32.9

Anticipated Duration (Months)

2.4

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).

Condition or Disease	Intervention/Treatment	Phase
Generalized Myasthenia Gravis	Drug: Rozanolixizumab	Phase 3

Study Design

Study Type:

Interventional

Actual Enrollment :

165 participants

Allocation:

Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

Actual Study Start Date :

Feb 3, 2021

Anticipated Primary Completion Date :

Oct 31, 2023

Anticipated Study Completion Date :

Oct 31, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Rozanolixizumab dosage regimen 1 Study participants randomized/assigned to dosage regimen 1 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.	Drug: Rozanolixizumab Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2. Other Names: UCB7665
Experimental: Rozanolixizumab dosage regimen 2 Study participants randomized/assigned to dosage regimen 2 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.	Drug: Rozanolixizumab Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2. Other Names: UCB7665

Arm

Intervention/Treatment

Experimental: Rozanolixizumab dosage regimen 1

Study participants randomized/assigned to dosage regimen 1 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.

Drug: Rozanolixizumab

Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.

Other Names:

UCB7665

Experimental: Rozanolixizumab dosage regimen 2

Study participants randomized/assigned to dosage regimen 2 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.

Drug: Rozanolixizumab

Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.

Other Names:

UCB7665

Outcome Measures

Primary Outcome Measures

Percentage of participants with treatment-emergent adverse events (TEAEs) [From Baseline (Day 1) to End of Study (average of 20 months)]
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP) [From Baseline (Day 1) to End of Study (average of 20 months)]
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.

Secondary Outcome Measures

Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement.
Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. The total QMG score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.
Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. The total MG-C score is obtained by summing the responses to each individual item (10 items; Grade:0-9 depending on item). The score ranges from 0 to 50, with lower scores indicating lower disease activity.
Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar symptoms' score within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
MG-ADL responder (≥2.0-point improvement from Baseline [Day 1] to end of Day 43) within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. A MG-ADL responder is defined as achieving at least 2.0-point improvement in the MG-ADL score from Baseline.
Time to MG-ADL response (≥2.0-point improvement from Baseline [Day 1]) within one treatment cycle [From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)]
The Outcome Measure is applicable for the first 3 treatment cycles. Time to achieve MG-ADL response, defined as at least 2.0-point improvement from Baseline.
Time between consecutive treatment cycles [From end of the 6-week treatment cycle (Day 43) to the next 6-week treatment cycle (Day 1), assessed up to 2.5 years]
Time between consecutive treatment cycles: Study participants will be assessed for MG worsening prior to repeated cycles. In case of symptom worsening (eg, an increase of 2.0 points on the MG-ADL or 3.0 points on the QMG scale) between assessments, resulting in a need for additional treatment, study participants will undergo another 6-week treatment cycle followed by an Observation Period, based on the Investigator's discretion.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Study participant must meet one of the following:

completed MG0003 [NCT03971422]
required rescue therapy during the Observation Period in MG0003 or
completed at least 6 visits in MG0004 [NCT04124965]

Body weight ≥35 kg at Baseline (Day 1)
Study participants may be male or female

Exclusion Criteria:

Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal Fc receptor (FcRn) medications
Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, or MG0004, or permanently discontinued study drug in either study
Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab
Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mg0007 50092	Orange	California	United States	92868
2	Mg0007 50099	San Francisco	California	United States	94117
3	Mg0007 50122	Miami	Florida	United States	33136
4	Mg0007 50120	Miami	Florida	United States	33144
5	Mg0007 50073	Tampa	Florida	United States	33612
6	Mg0007 50075	Augusta	Georgia	United States	30912-0004
7	Mg0007 50323	Honolulu	Hawaii	United States	96817
8	Mg0007 50114	Indianapolis	Indiana	United States	46202
9	Mg0007 50121	Lexington	Kentucky	United States	40536-0284
10	Mg0007 50077	New York	New York	United States	10021-4823
11	Mg0007 50090	Winston-Salem	North Carolina	United States	27157
12	Mg0007 50096	Philadelphia	Pennsylvania	United States	19104
13	Mg0007 50113	Houston	Texas	United States	77030
14	Mg0007 50071	Edmonton		Canada
15	Mg0007 50066	Montreal		Canada
16	Mg0007 50124	Montreal		Canada
17	Mg0007 50070	Quebec		Canada
18	Mg0007 50069	Toronto		Canada
19	Mg0007 40125	Ostrava - Poruba		Czechia
20	Mg0007 40124	Praha 2		Czechia
21	Mg0007 40128	Aalborg		Denmark
22	Mg0007 40127	Aarhus N		Denmark
23	Mg0007 40126	Copenhagen		Denmark
24	Mg0007 40129	Bordeaux		France
25	Mg0007 40360	Limoges		France
26	Mg0007 40132	Nice Cedex 1		France
27	Mg0007 40133	Paris		France
28	Mg0007 40131	Strasbourg		France
29	Mg0007 20160	Tbilisi		Georgia
30	Mg0007 20161	Tbilisi		Georgia
31	Mg0007 20163	Tbilisi		Georgia
32	Mg0007 20164	Tbilisi		Georgia
33	Mg0007 20165	Tbilisi		Georgia
34	Mg0007 40134	Essen		Germany
35	Mg0007 40140	Göttingen		Germany
36	Mg0007 40139	Jena		Germany
37	Mg0007 40078	Leipzig		Germany
38	Mg0007 40177	Münster		Germany
39	Mg0007 40283	Bologna		Italy
40	Mg0007 40144	Milano		Italy
41	Mg0007 40307	Napoli		Italy
42	Mg0007 40146	Pavia		Italy
43	Mg0007 40148	Roma		Italy
44	Mg0007 40150	Roma		Italy
45	Mg0007 20035	Bunkyo-ku		Japan
46	Mg0007 20068	Chiba-shi		Japan
47	Mg0007 20078	Hanamaki-shi		Japan
48	Mg0007 20079	Hiroshima		Japan
49	Mg0007 20075	Kobe		Japan
50	Mg0007 20071	Nagasaki-shi		Japan
51	Mg0007 20077	Sendai		Japan
52	Mg0007 20070	Shinjuku-ku		Japan
53	Mg0007 20076	Shinjuku-ku		Japan
54	Mg0007 20032	Suita		Japan
55	Mg0007 40155	Gdansk		Poland
56	Mg0007 40154	Lodz		Poland
57	Mg0007 40151	Lublin		Poland
58	Mg0007 40153	Poznan		Poland
59	Mg0007 20169	Novosibirsk		Russian Federation
60	Mg0007 20001	Saint-petersburg		Russian Federation
61	Mg0007 20028	Saint-petersburg		Russian Federation
62	Mg0007 20055	Saint-petersburg		Russian Federation
63	Mg0007 40467	NIS		Serbia
64	Mg0007 40160	Barcelona		Spain
65	Mg0007 40157	Hospitalet de Llobregat		Spain
66	Mg0007 40350	Murcia		Spain
67	Mg0007 40308	San Sebastián de Los Reyes		Spain
68	Mg0007 20081	Taipei		Taiwan
69	Mg0007 20086	Taipei		Taiwan