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

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.

Study Overview

• Status: Recruiting
• Conditions: Myasthenia Gravis
• Intervention / Treatment: Drug: Zilbrysq

• Study Type: Observational
• Enrollment (Estimated): 55

Contacts and Locations

• Study Contact: Name: Sabrina Sacconi; Phone Number: +33 04 92 03 57 57; Email: sacconi.s@chu-nice.fr
• Study Contact Backup: Name: Abderhmane Slioui; Phone Number: +33 04 92 03 89 53; Email: slioui.a@chu-nice.fr

Study Locations

• France
  • Alpes Maritimes
    • Nice, Alpes Maritimes, France, 06000
      • Recruiting
      • CHU Nice
      • Contact: Sabrina Sacconi; Phone Number: +33 04 92 03 57 57; Email: sacconi.s@chu-nice.fr
      • Contact: Abderhmane Slioui; Phone Number: +33 04 92 03 89 53; Email: slioui.a@chu-nice.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population will be selected from all the public hospitals in France where patients with confirmed myasthenia gravis with anti-AChR antibodies are given Zilucoplan as treatment under restricted access

Description

Inclusion Criteria:

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

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan

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); 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),

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
MG-ADL scores	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	At the inclusion, at 1 month, at 3 months and at 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Garches scale scores	The Garches scale is a 9-item based on clinical examination which measures the severity of myasthenia symptoms and muscle function.	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	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)	At 6 months
Evaluation of tolerance (M6) through the description of the frequency of Adverse event	number of adverse event per month	At 6 months
Adverse events possible association with the treatment (causality)	causality (Certain / probable / possible / Unlikely / conditional / Unassessable)	At 6 months
Quality of life evaluation (MG-QoL15r)	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	At the inclusion, at 1 month, at 3 months and at 6 months

Collaborators and Investigators

• Sponsor: Centre Hospitalier Universitaire de Nice

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2025
• Primary Completion (Estimated): February 1, 2026
• Study Completion (Estimated): March 3, 2026

Study Registration Dates

• First Submitted: February 4, 2025
• First Submitted That Met QC Criteria: February 4, 2025
• First Posted (Actual): February 7, 2025

Study Record Updates

• Last Update Posted (Estimated): December 3, 2025
• Last Update Submitted That Met QC Criteria: November 25, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis
• Other Study ID Numbers: 25Neuro01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No