A study to evaluate rozanolixizumab in study participants with generalized myasthenia gravis

JRCT ID: jRCT2021200038

Registered date:09/02/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Generalized Myasthenia Gravis
• Date of first enrollment: 09/02/2021
• Target sample size: 18
• Countries of recruitment: Belgium,Japan,Canada,Japan,Czech Republic,,Japan,Denmark,Japan,France,Japan,Georgia,Japan,Germany,Japan,Hungary,Japan,Italy,Japan,Poland,Japan,Russian Federation,Japan,Serbia,Japan,Spain,Japan,Taiwan,Japan,UK,Japan,US,Japan
• Study type: Interventional
• Intervention(s): Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.

Outcome(s)

Primary Outcome

1. Percentage of participants with treatment-emergent adverse events (TEAEs)
2. Percentage of participants with (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP)

Secondary Outcome

1. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MGADL) score within one treatment cycle
2. Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle
3. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle
4. 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
5. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO ' Physical Fatigue' score within one treatment cycle
6. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO ' Bulbar symptoms' score within one treatment cycle
7. MG-ADL responder (>=2.0-point improvement from Baseline [Day 1] to end of Day 43) within one treatment cycle
8. Time to MG-ADL response (>=2.0-point improvement from Baseline [Day 1]) within one treatment cycle
9. Time between consecutive treatment cycles

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Study participant must meet one of the following:
a) completed MG0003 [NCT03971422]
b) required rescue therapy during the Observation Period in MG0003
or
c) completed at least 6 visits in MG0004 [NCT04124965]
- Body weight >=35 kg at Baseline (Day 1)
- Study participants may be male or female

Exclude 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

Related Information

• Primary Sponsor: SUGIHARA Kazuhiro
• Secondary ID(s): 2020-003230-20,NCT04650854