Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months

NCT ID: NCT06815133

Last Updated: 2025-12-03

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 55 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2025-03-01
• Study Completion Date: 2026-03-03

Brief 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.

Conditions

Myasthenia Gravis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan

Zilbrysq

Intervention Type: DRUG

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),

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),

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Confirmed diagnosis of myasthenia gravis with anti-AChR antibodies in patients treated with Zilucoplan

Exclusion Criteria

• None

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Centre Hospitalier Universitaire de Nice

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

CHU NICE

Nice, Alpes Maritimes, France

Site Status: RECRUITING

Countries

France

Central Contacts

Sabrina Sacconi

Role: CONTACT

sacconi.s@chu-nice.fr

04 92 03 57 57 ext. +33

Abderhmane Slioui

Role: CONTACT

slioui.a@chu-nice.fr

04 92 03 89 53 ext. +33

Facility Contacts

Sabrina Sacconi

Role: primary

sacconi.s@chu-nice.fr

04 92 03 57 57 ext. +33

Abderhmane Slioui

Role: backup

slioui.a@chu-nice.fr

04 92 03 89 53 ext. +33

Other Identifiers

25Neuro01

Identifier Type: -

Identifier Source: org_study_id