Evaluating Rozanolixizumab for Children with Moderate to Severe Myasthenia Gravis
An Open-label, Single-arm Study Evaluating the Activity, Safety, and Pharmacokinetics of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
This study is testing a new treatment called rozanolixizumab to see if it can help children with moderate to severe Myasthenia Gravis who haven't had enough success with other treatments.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 2 Years to 17 Years |
| Sex | All |
| Sponsor | UCB Pharma Industry-sponsored |
| Drugs / interventions | rozanolixizumab |
| Locations | 14 sites (Denton, Texas and 13 other locations) |
| Trial ID | NCT06149559 on ClinicalTrials.gov |
What this trial studies
This study assesses the safety and tolerability of rozanolixizumab, administered subcutaneously, in pediatric participants aged 2 to under 18 years diagnosed with moderate to severe generalized Myasthenia Gravis (gMG). Participants must have a documented presence of specific autoantibodies and have previously received conventional treatments for gMG. The study aims to provide additional therapeutic options for children who have not responded adequately to existing treatments.
Who should consider this trial
Good fit: Ideal candidates are children aged 2 to under 18 years with a confirmed diagnosis of generalized Myasthenia Gravis and inadequate response to current treatments.
Not a fit: Patients with severe weakness affecting their ability to participate in the study may not benefit from this treatment.
Why it matters
Potential benefit: If successful, this treatment could offer a new therapeutic option for children suffering from moderate to severe Myasthenia Gravis.
How similar studies have performed: Other studies have shown promise with similar approaches in treating Myasthenia Gravis, but this specific treatment is being evaluated in a pediatric population, making it a novel application.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Study participant must be ≥2 to \<18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation * Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening * Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening * Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening * Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin (IVIg)) Exclusion Criteria: * Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline * Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-neonatal-Fc receptor (FcRn) medications * Study participant with any active or untreated thymoma * Study participant has a history of thymectomy within 6 months prior to Screening * Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP * Study participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study
Where this trial is running
Denton, Texas and 13 other locations
- Mg0006 50574 — Denton, Texas, United States (Withdrawn)
- Mg0006 40290 — Bologna, Italy (Recruiting)
- Mg0006 40144 — Milan, Italy (Recruiting)
- Mg0006 40733 — Naples, Italy (Recruiting)
- Mg0006 20340 — Fuchu-shi, Japan (Recruiting)
- Mg0006 20339 — Ōbu, Japan (Recruiting)
- Mg0006 20343 — Sagamihara, Japan (Recruiting)
- Mg0006 40155 — Warsaw, Poland (Recruiting)
- Mg0006 40734 — Warsaw, Poland (Recruiting)
- Mg0006 20081 — Taipei, Taiwan (Recruiting)
- Mg0006 20095 — Taipei, Taiwan (Recruiting)
- Mg0006 40841 — Altindağ/ankara, Turkey (Türkiye) (Recruiting)
- Mg0006 40843 — Ankara, Turkey (Türkiye) (Recruiting)
- Mg0006 40836 — Kocaeli, Turkey (Türkiye) (Recruiting)
Study contacts
- Study coordinator: UCB Cares
- Email: ucbcares@ucb.com
- Phone: 1-844-599-2273
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.