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

NCT06815133 | Recruiting

• 1 other identifier: 25Neuro01
• Study Type: observational
• Target: 55
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• Garches scale scores

  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

  At 6 months

• Evaluation of tolerance (M6) through the description of the frequency of Adverse event

  At 6 months

• Adverse events possible association with the treatment (causality)

  At 6 months

• Quality of life evaluation (MG-QoL15r)

  At the inclusion, at 1 month, at 3 months and at 6 months

Study Arms (1)

Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan

Drug: Zilbrysq

Interventions

Zilbrysq 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),

Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

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

You may not qualify if:

• None

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Nice: lead

Study Sites (1)

CHU NICE

Nice, Alpes Maritimes, 06000, France

RECRUITING

Related Publications (1)

• 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-AChR myasthenia gravis: A retrospective cohort study in France. J Neuromuscul Dis. 2026 Mar 29:22143602261437792. doi: 10.1177/22143602261437792. Online ahead of print.

  PMID: 41904994 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Sabrina Sacconi

CONTACT

04 92 03 57 57; sacconi.s@chu-nice.fr

Abderhmane Slioui

CONTACT

04 92 03 89 53; slioui.a@chu-nice.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2025
• First Posted: February 7, 2025
• Study Start: March 1, 2025
• Primary Completion: February 1, 2026
• Study Completion: March 3, 2026
• Last Updated: December 3, 2025

Record last verified: 2025-11

Locations

FR (1)