A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

Sponsor

UCB Biopharma SRL (Industry)

Overall Status

Completed

CT.gov ID

NCT04124965

Collaborator

(none)

Enrollment

Locations

Arms

22.1

Actual Duration (Months)

1.9

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of the MycarinGstudy is to evaluate the long-term safety, tolerability and long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG).

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

Study Design

Study Type:

Interventional

Actual Enrollment :

71 participants

Allocation:

Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Randomized, Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

Actual Study Start Date :

Oct 29, 2019

Actual Primary Completion Date :

Sep 1, 2021

Actual Study Completion Date :

Sep 1, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Rozanolixizumab dosage regimen 1 Study participants randomized to dosage regimen 1 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched 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 to dosage regimen 2 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched 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 to dosage regimen 1 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched 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 to dosage regimen 2 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched 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 until End of Study (up to Week 60)]
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 treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of study medication [From Baseline until End of Study (up to Week 60)]
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 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at each scheduled assessment during Treatment and Observation Periods [From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60)]
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 in Myasthenia Gravis-Composite (MG-C) score at each scheduled assessment during Treatment and Observation Periods [From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60)]
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 in Quantitative Myasthenia Gravis (QMG) score at each scheduled assessment during Treatment and Observation Periods [From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60)]
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.
Percentage of participants using rescue medication (intravenous infusion of immunoglobulin G (IVIg) or plasma exchange (PEX)) during the study [From Baseline during Treatment and Observation Periods (up to Week 60)]
Rescue therapy for the study will consist of IVIg or PEX. Study participants who experience disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Participant was eligible for MG0003 [NCT03971422] or MGC003 at the time of enrollment into either study and the participant either completed the observation Period of MG0003 or MGC003 or required rescue therapy during the Observation Period of the lead-in studies
Body weight ≥35 kg at Visit 1
Study participants may be male or female

Exclusion Criteria:

Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest X-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold Plus
Participant has received a live vaccination within 8 weeks prior to the Baseline visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of study medication
Study participant has experienced hypersensitivity reaction after exposure to other anti-neonatal Fc receptor (FcRn) drugs - Study participant with severe (defined as Grade 3 on the myasthenia gravis-activates of daily living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis
Participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the study participant's ability to participate in this study
Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria MG0003 [NCT03971422] or MGC003, or discontinued study medication in either study, with the exception of discontinuation due to a need for rescue treatment
Study participant is not considered capable of adhering to the protocol visit schedule, or medication intake according to the judgment of the Investigator
Study participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation since the last visit in MG0003 as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mg0004 50081	Phoenix	Arizona	United States	85013
2	Mg0004 50072	Los Angeles	California	United States	90033
3	Mg0004 50120	Miami	Florida	United States	33144
4	Mg0004 50073	Tampa	Florida	United States	33612
5	Mg0004 50114	Indianapolis	Indiana	United States	46202
6	Mg0004 50121	Lexington	Kentucky	United States	40536
7	Mg0004 50077	New York	New York	United States	10021
8	Mg0004 50090	Winston-Salem	North Carolina	United States	27157
9	Mg0004 50096	Philadelphia	Pennsylvania	United States	19104
10	Mg0004 50066	Montréal		Canada
11	Mg0004 50070	Québec		Canada
12	Mg0004 50069	Toronto		Canada
13	Mg0004 40125	Ostrava-Poruba		Czechia
14	Mg0004 40124	Praha 2		Czechia
15	Mg0004 40128	Aalborg		Denmark
16	Mg0004 40129	Bordeaux		France
17	Mg0004 40132	Nice		France
18	Mg0004 40133	Paris		France
19	Mg0004 40131	Strasbourg		France
20	Mg0004 40140	Göttingen		Germany
21	Mg0004 40078	Leipzig		Germany
22	Mg0004 40177	Münster		Germany
23	Mg0004 40146	Pavia		Italy
24	Mg0004 40150	Roma		Italy
25	Mg0004 20078	Hanamaki shi		Japan
26	Mg0004 20079	Hiroshima		Japan
27	Mg0004 20076	Shinjuku-Ku		Japan
28	Mg0004 20032	Suita		Japan
29	Mg0004 40155	Gdańsk		Poland
30	Mg0004 40151	Lublin		Poland
31	Mg0004 40154	Łódź		Poland
32	Mg0004 20027	Moscow		Russian Federation
33	Mg0004 20001	Saint Petersburg		Russian Federation
34	Mg0004 20028	Saint Petersburg		Russian Federation
35	Mg0004 40159	Barcelona		Spain
36	Mg0004 40157	Hospitalet de Llobregat		Spain
37	Mg0004 20081	Taipei		Taiwan