NCT07058298

GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis(24103)

An Early Exploratory Clinical Study of GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Daishi Tian · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This study is a single-arm, open-label, early-stage exploratory clinical study designed to evaluate safety, tolerability and preliminary efficacy of GC012F injection in the treatment of subjects with refractory GMG,the PK, PD characteristics and immunogenicity of GC012F injection in subjects were evaluated. This trial is divided into:screening period, apheresis day, baseline period, lymphodepletion pretreatment period, infusion day, safety and efficacy follow-up period, long-term follow-up period and withdrawal visits.Eligible subjects will undergo apheresis collection and then receive the CAR-T infusion after the manufacturing of the products. Subjects in the lymphodepletion cohort will receive the conditioning regimen and then receive CAR-T cell infusion (per the schedule of assessment). Subjects who meet the cell infusion criteria will receive CAR-T cell infusion at the doses levels which were specified in the protocol, and the dose levels could be adjusted according to safety and clinical efficacy. In this study, the infusion dose of CAR-T cells was set up in one dose group: 3×10\^5/kg. About 6 participants are planned to be included. The subjects will be monitored for DLT for 28 days after the infusion of GC012F injection. For the first 3 patients to receive GC012F infusion, if ≤1/3 of the patients develop a DLT at a given dose level, the additional 3 patients will be enrolled in this cohort. If 2/3 or more DLTs occur at this dose level, subsequent subjects may be given a backup dose group of 2.0×10\^5/kg or 1.0×10\^5/kg after discussion between the investigator and the collaborator. In the completion dose group of all subjects ,after the DLT observation period, DLTs will be observed for this dose group , all clinical study data collected during the period, especially safety data, were evaluated by the investigator discuss with the collaborator whether to add new subjects to the dose group or explore higher doses group. After CAR-T cell infusion, subjects will be followed up for safety, cell proliferation, survival, and efficacy until the subject progresses on the disease, or withdraws from the study and refuses to accept follow-up , or dies, or withdraws informed consent, or is lost to follow-up (whichever occurs first).

Conditions

• Generalized Myasthenia Gravis (gMG)

Interventions

Type	Name	Description
DRUG	GC012F injection	Subjects will be infused with GC012F Injection within 48-72 hours after lymphodepletion pretreatment,the infusion doses will be (CAR-T cell count) 3 × 10\^5/kg. Note: for subjects weighing ≤70 kg: number of infused CAR-T cells = 3 (±20%) × 10\^5 × body weight(kg); for subjects weighing \>70 kg: number of infused CART cells (fixed doses of GC012F Injection) = 3 (±20%) × 10\^5 × 70 kg.

Timeline

• Start date: 2025-07-15
• Primary completion: 2027-02-13
• Completion: 2027-09-10
• First posted: 2025-07-10
• Last updated: 2025-09-08

Locations

1 site across 1 country: China