An Early Exploratory Clinical Study of GC012F Injection in the Treatment of Refractory Generalize… (NCT06759948) | Clinical Trial Compass
Not Yet RecruitingPhase 1
An Early Exploratory Clinical Study of GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis
China18 participantsStarted 2025-02-15
Plain-language summary
This is a single-arm, open-label and early exploratory clinical study, with the purpose to study the safety, tolerability and initial clinical efficacy of GC012F Injection in the treatment of refractory GMG and to evaluate the PK, PD characteristics and immunogenicity in subjects with refractory GMG infused with GC012F Injection.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Subjects or their legal representative voluntary signing the ICF, and willing and able to follow the procedure in this study.
. Aged between 18 and 75 years old (including 75), no gender limitation;
. Patients with confirmed refractory GMG, and the clinical classification according to MGFA is IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa and IVb) at screening;
. At screening, the MG-ADL score must be ≥6, with ocular symptoms accounting for less than 50% of the total score, and QMG must be ≥11;
. Ineffectiveness of conventional treatment and/or lack of effective therapeutic options, referring to relapse or exacerbation after standard treatments with hormones, immunosuppressants (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A, cyclophosphamide, methotrexate, etc.), or biological agents (e.g., rituximab);
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Primary endpoints
Timeframe: Since signing the ICF until 24-week post-infusion or premature withdrawal from the study, whichever comes first.
2
Primary endpoints
Timeframe: Since signing the ICF until 24-week post-infusion or premature withdrawal from the study, whichever comes first.
. If the subject is currently using corticosteroids, the dose of prednisone must not exceed 30 mg/day (or the equivalent dose of other corticosteroids) and must remain stable for at least 4 weeks prior to infusion;
. Only subjects with positive MG-specific autoantibodies: the titer/level of anti-acetylcholine receptor (anti-AChR) antibodies or muscle-specific kinase (anti-MuSK) antibodies must be above the reference laboratory's upper normal limit (UNL);
. The results of laboratory test during screening period shall meet all following criteria:
Exclusion criteria
. Subjects have a history of severe hypersensitivity or allergy;
. Any contraindication for fludarabine, cyclophosphamide and any component of the investigational product;
. Administration of intravenous immunoglobulin or plasmapheresis or immunoadsorption treatment within 4 weeks prior to infusion;
. Use of B-cell targeting drugs, including but not limited to rituximab, Belimumab, and Telitacicept, within 6 months prior to apheresis;
. Used tacrolimus, cyclosporine, azathioprine, mycophenolate, mycophenolate, methotrexate, etc., within 3 weeks prior to apheresis;
. Treatment with neonatal Fc receptor (FcRn) antagonists (such as Efgartigimod, etc.) within 3 weeks prior to apheresis
. Use of complement inhibition therapy (such as eculizumab, etc.) within 3 weeks prior to apheresis;
. Subjects with any of the following heart diseases: