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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 September 2022
• Main ID: NCT04124965
• Date of registration: 11/10/2019
• Prospective Registration: Yes
• Primary sponsor: UCB Biopharma SRL
• Public title: A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
• Scientific 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
• Date of first enrolment: October 29, 2019
• Target sample size: 71
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04124965
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

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

Contacts

• Name: UCB Cares
• Affiliation: 001 844 599 22733 (UCB)

Key inclusion & exclusion criteria

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)



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Generalized Myasthenia Gravis

Intervention(s)

Drug: Rozanolixizumab

Primary Outcome(s)

Percentage of participants with treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of study medication [Time Frame: From Baseline until End of Study (up to Week 60)]

Percentage of participants with treatment-emergent adverse events (TEAEs) [Time Frame: From Baseline until End of Study (up to Week 60)]

Secondary Outcome(s)

Percentage of participants using rescue medication (intravenous infusion of immunoglobulin G (IVIg) or plasma exchange (PEX)) during the study [Time Frame: From Baseline during Treatment and Observation Periods (up to Week 60)]

Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at each scheduled assessment during Treatment and Observation Periods [Time Frame: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60)]

Change from Baseline in Myasthenia Gravis-Composite (MG-C) score at each scheduled assessment during Treatment and Observation Periods [Time Frame: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60)]

Change from Baseline in Quantitative Myasthenia Gravis (QMG) score at each scheduled assessment during Treatment and Observation Periods [Time Frame: From Baseline at each scheduled assessment during Treatment and Observation Periods (up to Week 60)]

Secondary ID(s)

MG0004

2019-000969-21

Source(s) of Monetary Support

Please refer to primary and secondary sponsors