Clinical trial · Observational
Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months
Multicenter Retrospective Study on the Short- and Medium-Term Efficacy and Tolerance of Zilucoplan Therapy in a Cohort of French Patients With Anti-AChR Myasthenia Gravis
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This study called "Multicenter Retrospective Study on the Short- and Medium-Term Efficacy and Tolerance of Zilucoplan Therapy in a Cohort of French Patients with Anti-AChR Myasthenia Gravis", is investigating the effects of a new treatment called Zilucoplan (generic name: ZILBRYSQ) on patients in France with a condition known as myasthenia gravis. Myasthenia gravis is an autoimmune disease that causes muscle weakness that worsens throughout the day with activity and impacts the ability to work and perform daily activities. The study aims to see if Zilucoplan can improve muscle strength and quality of life over three months, while also assessing its tolerance and safety by monitoring side effects. Researchers hope this treatment will offer significant benefits for people with this challenging condition.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myasthenia Gravis | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Zilbrysq | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- label
- Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan
- interventionNames
- Drug: Zilbrysq
Primary outcomes (1)
- measure
- MG-ADL scores
- timeFrame
- At the inclusion, at 1 month, at 3 months and at 6 months
- description
- The MG-ADL is an 8-item patient-reported scale that measures myasthenia gravis symptoms and functional status. It assesses the impact of Myasthenia Gravis on the daily functions of the patients
Secondary outcomes (5)
- measure
- Garches scale scores
- timeFrame
- At the inclusion, at 1 month, at 3 months and at 6 months
- description
- The Garches scale is a 9-item based on clinical examination which measures the severity of myasthenia symptoms and muscle function.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: \- Confirmed diagnosis of myasthenia gravis with anti-AChR antibodies in patients treated with Zilucoplan Exclusion Criteria: * None
References
Publications (1)
- DERIVEDTammam G, Slioui A, Friedman D, Acket B, Cavalli M, Bresch S, Lagrange E, Sole G, Noury JB, Barbat Du Closel L, Marois C, Pakleza AN, Tard C, Salort-Campana E, Merle PE, Annane D, Attarian S, Duval F, Frachet S, Svahn J, Pegat A, Bouhour F, Taieb G, Praline J, Puma A, Fabry V, Villa L, Beltran S, Behin A, Cintas P, Sacconi S. Real-world effectiveness and safety of zilucoplan in patients with anti-AChR myasthenia gravis: A retrospective cohort study in France. J Neuromuscul Dis. 2026 Mar 29:22143602261437792. doi: 10.1177/22143602261437792. Online ahead of print. PMID 41904994