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

ConditionGeneralized Myasthenia Gravis (gMG)

Trial statusRecruiting

Trial phaseEarly Phase 1

Trial typeInterventional

Biological sexAll

Age18-75

SponsorDaishi Tian

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Subjects or their legal representatives voluntarily sign a written informed consent and are willing and able to comply with the procedures of the study;

Subjects aged 18-75 years old (both inclusive), male or female;

Subjects with confirmed refractory gMG of classes IIa - IVb by MGFA clinical classification (including classes IIa, IIb, IIIa, IIIb, IVa and IVb) at screening;

Subjects with the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score of ≥6, the proportion of ocular symptoms of <50% of the total score, and the Quantitative Myasthenia Gravis (QMG) score of ≥11;

Disqualifiers

Have a history of severe hypersensitivity or allergy;

Contraindications or hypersensitivity to fludarabine, cyclophosphamide and any component of the test drug;

Subjects who have received intravenous immunoglobulin or plasma exchange therapy or immunoadsorption therapy within 4 weeks prior to infusion;

Received CD20-targeted drugs within 6 months prior to apheresis;

Trial design

Design model

Single group

Treatments tested in this trial

GC012F injection
Drug
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.

Treatment groups

6 Participants

are divided into 1 treatment group

Group A: 3×10^5/kgExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

The frequency and severity of adverse events.

Time frame

Since signing the ICF until 24-week post-infusion or withdrawal from the study, whichever comes first.

Secondary outcomes

Analysis of cell dynamics (CK) parameters of the copy number of the GC012F CAR gene in peripheral blood- Cmax(based on data availability).

Time frame

Since signing the ICF until 24-week post-infusion or withdrawal from the study, whichever comes first.

Analysis of cell dynamics (CK) parameters of the copy number of the GC012F CAR gene in peripheral blood- Tmax(based on data availability).

Time frame

Since signing the ICF until 24-week post-infusion or withdrawal from the study, whichever comes first.

Analysis of cell dynamics (CK) parameters of the copy number of the GC012F CAR gene in peripheral blood- AUC(based on data availability).

Time frame

Since signing the ICF until 24-week post-infusion or withdrawal from the study, whichever comes first.

T/B lymphocyte subsets in peripheral blood after GC012F infusion.

Time frame

Since signing the ICF until 24-week post-infusion or withdrawal from the study, whichever comes first.

Other outcomes

To evaluate the immunogenicity of GC012F injection

The changes in serum immunoglobulin concentrations (including IgG, IgM, IgA, and IgE) within 24 weeks after the infusion of GC012F.

Time frame

Since signing the ICF until 24-week post-infusion or withdrawal from the study, whichever comes first.

To evaluate immune reconstitution after infusion of GC012F injection

Incidence and titer of anti-CAR AZD0120 antibody response during the study period.

Time frame

Since signing the ICF until 24-week post-infusion or premature withdrawal from the study, whichever comes first.

To determine the presence of replication competent lentivirus (RCL) and lentiviral integration site in subjects after infusion of GC012F lnjection.

Detection rate of RCL

Time frame

Since signing the ICF until 24-week post-infusion or withdrawal from the study, whichever comes first.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Daishi Tian

Lead sponsor

Tongji Hospital

Sponsor institution

Gracell Biotechnologies (Shanghai) Co., Ltd.

Collaborator

Source ClinicalTrials.gov

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