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Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months
Sponsor: Centre Hospitalier Universitaire de Nice
Summary
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.
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
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
55
Start Date
2025-03-01
Completion Date
2026-03-03
Last Updated
2025-12-03
Healthy Volunteers
No
Conditions
Interventions
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); 23 mg/0.574 mL for patients between 56 kg and less than 77 kg (123-170 lbs); and 32.4 mg/0.81 mL for patients weighing 77 kg and above (\>170 lbs). This can be injected into the abdomen, front of the thighs, and back of the upper arms (only if administered by a caregiver),
Locations (1)
CHU NICE
Nice, Alpes Maritimes, France