Actively Recruiting
An Open-label, Single-arm Study Evaluating the Activity, Safety, and Pharmacokinetics of Rozanolixizumab in Pediatric Participants With Moderate to Severe Generalized Myasthenia Gravis
Led by UCB Biopharma SRL · Updated on 2026-05-08
12
Participants Needed
14
Research Sites
10 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are assessing the safety and tolerability of subcutaneous administration of rozanolixizumab in children aged 2 to under 18 years who have moderate to severe generalized Myasthenia Gravis (gMG). This open-label, single-arm study aims to evaluate the activity, safety, and pharmacokinetics of rozanolixizumab in this pediatric population. Participants must have a documented diagnosis of gMG with specific autoantibodies and have an unsatisfactory response to existing treatments. Participants will receive predefined doses of rozanolixizumab for a 6-week treatment period. The study does not include a placebo or comparison group. Rozanolixizumab is given as a solution for injection under the skin. The study includes monitoring for safety and drug activity, including measurement of drug levels and antibody responses. During the study, participants will be regularly assessed for treatment-emergent adverse events, antibody levels, and changes in their Myasthenia Gravis symptoms using daily living and quantitative scores. Assessments will occur up to 18 weeks from baseline, covering treatment and observation periods. Safety monitoring includes evaluation of adverse events and local tolerability at scheduled visits. The total participation duration is up to 18 weeks, including treatment and follow-up.
CONDITIONS
Brief Title
A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be between 2 and under 18 years old at consent
- Must have a confirmed diagnosis of generalized Myasthenia Gravis with specific autoantibodies to acetylcholine receptor or muscle-specific kinase
- Must have Myasthenia Gravis Foundation of America Clinical Classification II to IVa
- Must have received conventional treatments for generalized Myasthenia Gravis before the study
- Must have had an unsatisfactory response or worsening of symptoms needing additional therapy such as plasma exchange or intravenous immunoglobulin
You will not qualify if you...
- Severe weakness affecting throat or breathing muscles, or myasthenic crisis at screening or baseline
- Known allergy to any components of rozanolixizumab or similar anti-neonatal-Fc receptor medications
- Active or untreated thymoma
- Thymectomy within 6 months before screening
- Active infection requiring hospitalization or serious infection within 6 weeks before first dose
- Received a live vaccine within 4 weeks before baseline or plans to receive one during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 weeks
Participants receive pre-defined doses of rozanolixizumab for 6 weeks to evaluate safety and activity.
Visits at baseline and weekly up to Week 6
Duration - Up to 12 weeks after treatment
Participants are monitored for safety and treatment effects after the treatment period.
Regular visits during follow-up period up to Week 18
Trial Site Locations
Total: 14 locations
1
Mg0006 50574
Denton, Texas, United States, 76208
Withdrawn
2
Mg0006 40290
Bologna, Italy
Actively Recruiting
3
Mg0006 40144
Milan, Italy
Actively Recruiting
4
Mg0006 40733
Naples, Italy
Actively Recruiting
5
Mg0006 20340
Fuchu-shi, Japan
Actively Recruiting
6
Mg0006 20339
Ōbu, Japan
Actively Recruiting
7
Mg0006 20343
Sagamihara, Japan
Actively Recruiting
8
Mg0006 40155
Warsaw, Poland
Actively Recruiting
9
Mg0006 40734
Warsaw, Poland
Actively Recruiting
10
Mg0006 20081
Taipei, Taiwan
Actively Recruiting
11
Mg0006 20095
Taipei, Taiwan
Actively Recruiting
12
Mg0006 40841
Altindağ/ankara, Turkey (Türkiye)
Actively Recruiting
13
Mg0006 40843
Ankara, Turkey (Türkiye)
Actively Recruiting
14
Mg0006 40836
Kocaeli, Turkey (Türkiye)
Actively Recruiting
Research Team
U
UCB Cares
U
UCB Cares
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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