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

Not yet recruiting

• Conditions: Myasthenia Gravis
• Interventions: Drug: Zilbrysq

• Registration Number: NCT06815133
• Lead Sponsor: Centre Hospitalier Universitaire de Nice

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-02
• Study First Submitted: 2025-02-04
• Study First Submitted QC: 2025-02-04
• Last Update Submitted: 2025-02-04
• Study First Posted: 2025-02-07
• Last Update Posted: 2025-02-07
• Study Start: 2025-03-01
• Primary Completion: 2025-11-01
• Study Completion: 2026-03-03

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 55

Inclusion Criteria

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

Exclusion Criteria

• None

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan	Zilbrysq	-

Primary Outcome Measures

Name	Time	Method
MG-ADL scores	At 1 month and at 3 months	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 Outcome Measures

Name	Time	Method
Evaluation of tolerance (M3) through the description of the frequency of Adverse event	At 3 months	number of adverse event per month
Adverse events possible association with the treatment (causality)	At 3 months	causality (Certain / probable / possible / Unlikely / conditional / Unassessable)
Garches scale scores	At the Inclusion, at 1 Month and at 3 months	The Garches scale is a 9-item based on clinical examination which measures the severity of myasthenia symptoms and muscle function.
Evaluation of tolerance (M3) through the description of type of Adverse events	At 3 months	Collect data on adverse events: total number of events, classification by type (mild, moderate, severe based on the " Common Terminology Criteria for Adverse Events (CTCAE) " scale)
Quality of life evaluation (MG-QoL15r)	At the inclusion, at 1 Month and at 3 months	Myasthenia gravis quality of life-15 revised (MG-QoL51r) is a 15-item quality of life scale designed to assess important aspects of the patient's experience related to myasthenia gravis

Trial Locations

Locations (1)

CHU NICE; 🇫🇷 Nice, Alpes Maritimes, France