Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Zilbrysq.
- Who it may be relevant to
- Registry conditions: Myasthenia Gravis. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
Overview
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.
Interventions
- Drug Zilbrysq
Zilbrysq is a medicine used to treat generalized myasthenia gravis anti-AChR (a disease that leads to muscle weakness and tiredness) in adults whose immune system produces antibodies against a protein called the acetylcholine receptor. It is given as a subcutaneous injection (under the skin) once daily in a prefilled syringe that can be self-injected, and the dose depends on the patient's body weight. There are 3 available doses: 16.6 mg/0.416 mL for patients weighing less than 56 kg (123 lbs);
Primary outcome measures
- MG-ADL scores [Time frame: At the inclusion, at 1 month, at 3 months and at 6 months]
Secondary outcome measures (5)
- Garches scale scores [Time frame: At the inclusion, at 1 month, at 3 months and at 6 months]
- Evaluation of tolerance (M6) through the description of type of Adverse events [Time frame: At 6 months]
- Evaluation of tolerance (M6) through the description of the frequency of Adverse event [Time frame: At 6 months]
- Adverse events possible association with the treatment (causality) [Time frame: At 6 months]
- Quality of life evaluation (MG-QoL15r) [Time frame: At the inclusion, at 1 month, at 3 months and at 6 months]
Eligibility criteria
Inclusion criteria
\- Confirmed diagnosis of myasthenia gravis with anti-AChR antibodies in patients treated with Zilucoplan
Exclusion criteria
- None
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
France · 1 center
- CHU NICE — Nice
Publications
- Tammam 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- PMID 41904994
Identifiers
NCT: NCT06815133 · 25Neuro01