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NCT Number: NCT06815133

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

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • None

Treatment and study plan

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),

Primary outcomes

  1. MG-ADL scores

    Time frame: At the inclusion, at 1 month, at 3 months and at 6 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 outcomes

  1. Garches scale scores

    Time frame: At the inclusion, at 1 month, at 3 months and at 6 months

    The Garches scale is a 9-item based on clinical examination which measures the severity of myasthenia symptoms and muscle function.

  2. Evaluation of tolerance (M6) through the description of type of Adverse events

    Time frame: At 6 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)

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

    Time frame: At 6 months

    number of adverse event per month

  4. Adverse events possible association with the treatment (causality)

    Time frame: At 6 months

    causality (Certain / probable / possible / Unlikely / conditional / Unassessable)

  5. Quality of life evaluation (MG-QoL15r)

    Time frame: At the inclusion, at 1 month, at 3 months and at 6 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

Study contacts

Contact information is provided by the study sponsor or research team.

Abderhmane Slioui

CONTACT

[email protected]

04 92 03 89 53 ext. +33

Sabrina Sacconi

CONTACT

[email protected]

04 92 03 57 57 ext. +33

Sponsors and collaborators

Lead sponsor

Centre Hospitalier Universitaire de Nice

Other

Registry information

Official study 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

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Feb 7, 2025
Registry last updated
Dec 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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