Actively Recruiting
An Early Exploratory Clinical Study of GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis
Led by Daishi Tian · Updated on 2025-09-08
6
Participants Needed
1
Research Sites
29 weeks
Total Duration
On this page
Sponsors
D
Daishi Tian
Lead Sponsor
G
Gracell Biotechnologies (Shanghai) Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting an early exploratory clinical study to examine the safety, tolerability, and initial effectiveness of GC012F Injection in adults with refractory generalized myasthenia gravis (GMG). This study also evaluates how the drug moves through and affects the body, along with its immune response. The trial aims to provide early data on this treatment for patients who have not responded well to conventional therapies. The study involves a single group of participants receiving one dose level of CAR-T cells at 3 × 10^5 cells per kilogram of body weight. The process includes screening, apheresis collection to obtain cells, lymphodepletion pretreatment, infusion of the GC012F Injection, followed by safety and efficacy monitoring. Dose adjustments may be made based on safety observations. About six participants will be enrolled, and the study allows for backup lower doses if certain safety concerns arise. Participants will be closely monitored for side effects and treatment response for 24 weeks after infusion or until withdrawal. Researchers will assess adverse events, cell behavior in the blood, immune cell changes, muscle weakness scores, quality of life, and antibody levels. Follow-up continues until disease progression, withdrawal, or other endpoints. This comprehensive monitoring helps understand the treatment’s safety and potential benefits in refractory GMG over time.
CONDITIONS
Brief Title
GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis(24103)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subjects or their legal representatives voluntarily sign informed consent and can follow study procedures
- Age between 18 and 75 years, male or female
- Confirmed refractory generalized myasthenia gravis classified as MGFA classes IIa to IVb at screening
- MG-ADL score of 6 or higher with less than 50% ocular symptoms, and QMG score of 11 or higher
- Poor response or relapse risk after conventional therapies including hormones, immunosuppressants, or biological agents
- Stable prednisone dose not exceeding 30 mg/day for at least 4 weeks before infusion
- Laboratory tests meeting specified blood counts, liver, kidney, coagulation, and heart function criteria
- Female participants of childbearing potential with negative pregnancy test and agreement to avoid breastfeeding during study and follow-up
- Male participants with partners of childbearing potential and female participants agree to effective contraception during study and follow-up
- Adequate venous access for blood collection and no contraindications to leukocyte collection
You will not qualify if you...
- History of severe hypersensitivity or allergy
- Contraindications or allergies to fludarabine, cyclophosphamide, or study drug components
- Received intravenous immunoglobulin, plasma exchange, or immunoadsorption within 4 weeks prior to infusion
- Received CD20-targeted drugs within 6 months prior to apheresis
- Received Tacrolimus, Cyclosporine, Azathioprine, or Mycophenolate Mofetil within 1 week before apheresis
- Treatment with neonatal Fc receptor antagonists or complement inhibitors within 1 week before apheresis
- Heart diseases including class III/IV heart failure, recent unstable angina or heart attack, significant arrhythmias, severe cardiomyopathy, or severe cardiovascular abnormalities
- Uncontrolled malignancies except certain early-stage treated tumors
- Serious infections requiring intravenous therapy or active tuberculosis
- Dementia or significant mental status changes
- History of central nervous system or neurodegenerative diseases such as epilepsy, stroke, severe brain injury, Parkinson's disease, or mental illness
- Positive tests for HIV, hepatitis B or C with active infection, or syphilis
- Prior CAR-T cell therapy
- Previous organ or allogeneic bone marrow transplantation
- Recent surgery within 2 weeks before lymphodepletion or planned surgery during study (except minor local anesthesia surgeries)
- Received live attenuated vaccines within 4 weeks before lymphodepletion
- Participation in other clinical trials or recent study drug use within specified time frames
- Pregnant or lactating females not agreeing to stop breastfeeding, or men planning families within 1 year after treatment
- Current suicidal intentions or history of suicidal behavior
- Any condition preventing full participation or likely to confuse study results per investigator judgment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 week
Participants undergo apheresis collection and baseline assessments prior to lymphodepletion pretreatment.
1 to 2 visits including apheresis and baseline assessments
Duration - Several days
Participants receive lymphodepletion conditioning regimen before the CAR-T cell infusion.
Daily visits during conditioning period
Duration - 1 day
Participants receive a single infusion of GC012F injection (CAR-T cells) after lymphodepletion.
1 infusion visit (in-person)
Duration - 24 weeks
Participants are monitored for dose-limiting toxicities and preliminary efficacy for 24 weeks post infusion.
Weekly visits for 4 weeks, then periodic visits up to 24 weeks
Duration - Up to several years
Participants continue to be followed for safety, cell persistence, and disease progression until withdrawal or study end.
Periodic visits as scheduled by the study team
Trial Site Locations
Total: 1 location
1
Tongji Hospital of Tongji Medical Colledge, Huazhong University of Science and Technology
Wuhan, Hubei, China, 430000
Actively Recruiting
Research Team
C
Chuan Qin Sub-I
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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