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

Generalized Myasthenia Gravis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04124965
• Other study ID #: MG0004
• Secondary ID: 2019-000969-21
• Status: Completed
• Phase: Phase 3
• Start date: October 29, 2019
• Est. completion date: September 1, 2021

Study information

• Verified date: August 2023
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 71
• Est. completion date: September 1, 2021
• Est. primary completion date: September 1, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

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

Locations

Country	Name	City	State
Canada	Mg0004 50066	Montréal
Canada	Mg0004 50070	Québec
Canada	Mg0004 50069	Toronto
Czechia	Mg0004 40125	Ostrava-Poruba
Czechia	Mg0004 40124	Praha 2
Denmark	Mg0004 40128	Aalborg
France	Mg0004 40129	Bordeaux
France	Mg0004 40132	Nice
France	Mg0004 40133	Paris
France	Mg0004 40131	Strasbourg
Germany	Mg0004 40140	Göttingen
Germany	Mg0004 40078	Leipzig
Germany	Mg0004 40177	Münster
Italy	Mg0004 40144	Milano
Italy	Mg0004 40146	Pavia
Italy	Mg0004 40148	Roma
Italy	Mg0004 40150	Roma
Japan	Mg0004 20078	Hanamaki shi
Japan	Mg0004 20079	Hiroshima
Japan	Mg0004 20077	Miyagi
Japan	Mg0004 20076	Shinjuku-Ku
Japan	Mg0004 20032	Suita
Poland	Mg0004 40155	Gdansk
Poland	Mg0004 40154	Lódz
Poland	Mg0004 40151	Lublin
Russian Federation	Mg0004 20027	Moscow
Russian Federation	Mg0004 20001	Saint Petersburg
Russian Federation	Mg0004 20028	Saint Petersburg
Russian Federation	Mg0004 20055	Saint Petersburg
Spain	Mg0004 40159	Barcelona
Spain	Mg0004 40157	Hospitalet de Llobregat
Taiwan	Mg0004 20080	Taichung
Taiwan	Mg0004 20081	Taipei
United States	Mg0004 50114	Indianapolis	Indiana
United States	Mg0004 50121	Lexington	Kentucky
United States	Mg0004 50072	Los Angeles	California
United States	Mg0004 50120	Miami	Florida
United States	Mg0004 50077	New York	New York
United States	Mg0004 50096	Philadelphia	Pennsylvania
United States	Mg0004 50081	Phoenix	Arizona
United States	Mg0004 50073	Tampa	Florida
United States	Mg0004 50088	Washington	District of Columbia
United States	Mg0004 50090	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
UCB Biopharma SRL

Countries where clinical trial is conducted

United States,  Canada,  Czechia,  Denmark,  France,  Germany,  Italy,  Japan,  Poland,  Russian Federation,  Spain,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	A TEAE is defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.	From Baseline until End of Study (up to Week 60)
Primary	Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication	A TEAE is defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.	From Baseline until End of Study (up to Week 60)
Secondary	Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods	The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported outcome (PRO) instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement.	Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Secondary	Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods	MG-C scale is a validated assessment and scale tests 10 items with individual items being weighted differently. The items included ptosis/upward gaze (range: 0 [>45 second] - 3 [Immediate]), double vision on lateral gaze (range: 0 [>45 second] - 4 [Immediate]), eye closure (range: 0 [Normal] - 2 [severe weakness]), talking (range: 0 [Normal] - 6 [difficult to understand speech]), chewing (range: 0 [Normal] - 6 [gastric tube]), swallowing (range: 0 [Normal] - 6 [gastric tube]), breathing (range: 0 [Normal] - 9 [ventilator dependence]), neck flexion (range: 0 [Normal] - 4 [severe weakness]), shoulder abduction (range: 0 [Normal] - 5 [severe weakness]) and hip flexion (range: 0 [Normal] - 5 [severe weakness]), lower scores= lower disease activity. Total MG-C score was obtained by summing responses to each individual item and score ranges from 0 to 50, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.	Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Secondary	Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods	The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.	Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Secondary	Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX))	Rescue therapy consisted of IVIg or PEX. Study participants who experienced 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.	From Baseline until End of Study (up to Week 60)