Myasthenia GravisNCT06815133
Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months
An observational study of Myasthenia Gravis, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- Zilbrysq
- Type of treatment
- Injection or infusion
- How many people
- 55 participants
- Where
- 1 siteFrance
- Study dates
- 1 Mar 2025to 3 Mar 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Who may be able to join
- Confirmed diagnosis of myasthenia gravis with anti-AChR antibodies in patients treated with Zilucoplan
Who cannot join
- None
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
CHU NICE
Nice, Alpes Maritimes, 06000
Sabrina Sacconi, 04 92 03 57 57 ext. +33, sacconi.s@chu-nice.fr
Abderhmane Slioui, 04 92 03 89 53 ext. +33, slioui.a@chu-nice.fr
About this study
Official 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
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
- Being tested
- Zilbrysq
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZilbrysqDrug
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),
| Study group | Role | What they receive |
|---|---|---|
| Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan | Not described Receives: Drug: Zilbrysq |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: At the inclusion, at 1 month, at 3 months and at 6 months
Secondary outcomes (5)
Garches scale scores
The Garches scale is a 9-item based on clinical examination which measures the severity of myasthenia symptoms and muscle function.
Measured: 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)
Measured: At 6 months
Evaluation of tolerance (M6) through the description of the frequency of Adverse event
number of adverse event per month
Measured: At 6 months
Adverse events possible association with the treatment (causality)
causality (Certain / probable / possible / Unlikely / conditional / Unassessable)
Measured: 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
Measured: At the inclusion, at 1 month, at 3 months and at 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2025
- Study start1 Mar 2025
- Primary completion1 Feb 2026 (estimated)
- Study completion3 Mar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06815133
- Sponsor's protocol ID
- 25Neuro01
- Status last verified
- Nov 2025
- First submitted
- 4 Feb 2025
- Last updated
- 3 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 41904994
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06815133
- Acronym / sponsor ID
- 25Neuro01
- Conditions
- Myasthenia Gravis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- 1 Mar 2025 (actual)
- Primary completion
- 1 Feb 2026 (estimated)
- Study completion
- 3 Mar 2026 (estimated)
- First posted
- 7 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan | – | Drug: Zilbrysq |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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
Key inclusion criteria (1 listed)
- Confirmed diagnosis of myasthenia gravis with anti-AChR antibodies in patients treated with Zilucoplan
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZilbrysqZilbrysq 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), | Drug | Patients with Anti-AChR Myasthenia Gravis treated with Zilucoplan |
Primary outcome
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
5 secondary outcomes
Garches scale scores
Time frame: 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
Time frame: At 6 months
Evaluation of tolerance (M6) through the description of the frequency of Adverse event
Time frame: At 6 months
Adverse events possible association with the treatment (causality)
Time frame: At 6 months
Quality of life evaluation (MG-QoL15r)
Time frame: At the inclusion, at 1 month, at 3 months and at 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2025.
- Derived from this study: PubMed 41904994Tammam 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myasthenia Gravis, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sabrina Sacconi (04 92 03 57 57 ext. +33, sacconi.s@chu-nice.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
MG-ADL scores (time frame: At the inclusion, at 1 month, at 3 months and at 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06815133, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Dec 2025.