An extension study to find out if multiple treatment cycles of rozanolixizumab are safe and work in children with myasthenia gravis

Phase 1

Conditions: Generalized Myasthenia Gravis
MedDRA version: 21.1Level: PTClassification code: 10028417Term: Myasthenia gravis Class: 100000004852
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: CTIS2022-502075-34-00

Lead Sponsor: CB Biopharma

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

Study participant must meet one of the following: --Study participant completed MG0006 according to the protocol --Study participant completed the MG0006 Treatment Period and has a worsening of gMG symptoms in the Observation Period of MG0006

Exclusion Criteria

-Study participant met any mandatory withdrawal or mandatory permanent IMP discontinuation criteria in MG0006 or permanently discontinued IMP -Study participant has a known hypersensitivity to any components of the IMP or other FcRn drugs -Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant’s ability to participate in this study

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged =2 at the time of the Screening Visit of MG0006;Secondary Objective: Evaluate the activity of rozanolixizumab;Primary end point(s): -Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit -Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit -Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):-Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP) -Absolute change in total IgG from Baseline to the end of Week 6 of each TP -Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP -Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP