An Exploratory Clinical Study of GC012F Injection for Refractory gMG (NCT06419166) | Clinical Trial Compass
WithdrawnEarly Phase 1
An Exploratory Clinical Study of GC012F Injection for Refractory gMG
Stopped: The study stopped early, before enrolling its first participant.
China0Started 2024-12-01
Plain-language summary
This study is a single-arm, open-label early exploratory clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of GC012F injection in subjects with refractory GMG. Additionally, the study aims to assess the pharmacokinetic (PK), pharmacodynamic (PD) characteristics, and immunogenicity of GC012F injection in subjects.
Who can participate
Age range18 Years ā 75 Years
SexALL
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Inclusion criteria
ā. Subjects or his/her legal proxy/guardian voluntary signing the ICF, and willing and able to follow the procedure in this study.
ā. Aged ā„18 years old, no gender limitation;
ā. Patients with confirmed refractory GMG, and the clinical classification is IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa and IVb) in screening;
ā. Patients whose MG-ADL score is 5 or more, and the proportion of ocular symptoms is less than 50% in the total score;
ā. Patients with poor efficacy of conventional treatment and/or no effective treatment means relapse or exacerbation despite conventional hormone, immunosuppressant (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporin A, cyclophosphamide, etc.), or rituximab treatment;
ā. Patients who are on corticosteroids, the dose of prednisone should not exceed 20 mg/d (or no more than an equivalent dose of another corticosteroid) during the 3 weeks prior to apheresis, and the dose isn't escalated during 3weeksk prior to apheresis, the dose isn't changed within 4 weeks prior to infusion;
ā. Patients with positive MG-specific autoantibodies in the screening period: acetylcholine receptor autoantibody (anti-AChR) titer or muscle-specific tyrosine kinase autoantibody (anti-MuSK) or low-density lipoprotein receptor-associated protein 4 autoantibody (anti-LRP4) or anti-acetylcholine receptor cluster antibody must be higher than the upper limit of the laboratory reference normal value;
ā. Life expectancy ā„3 months;
Exclusion criteria
ā. Subjects have a history of severe hypersensitivity or allergy;
What they're measuring
1
Incidence of DLT
Timeframe: Within 28 days after GC012F injection infusion
2
Frequency and severity of abnormal findings in electrocardiograms
Timeframe: Within 96 weeks after GC012F injection infusion
3
Frequency and severity of abnormal findings of adverse events.
Timeframe: Within 96 weeks after GC012F injection infusion
4
Frequency and severity of abnormal findings in physical examinations
Timeframe: Within 96 weeks after GC012F injection infusion
5
Frequency and severity of abnormal findings in laboratory tests
Timeframe: Within 96 weeks after GC012F injection infusion
6
Frequency and severity of abnormal findings in vital signs
Timeframe: Within 96 weeks after GC012F injection infusion
7
RP1D
Timeframe: 2 years after GC012F injection infusion
8
MTD
Timeframe: 2 years after GC012F injection infusion
ā. Any contraindication for fludarabine, cyclophosphamide and any component of the investigational product;
ā. Subjects with any of the following heart diseases:
ā. Congestive heart failure (New York Heart Association (NYHA) Class III or IV);
ā. Experienced myocardial infarction or underwent coronary artery bypass grafting (CABG) within 6 months prior to screening period;
ā. Clinically significant ventricular arrhythmias or a history of unexplained syncope not due to vasovagal reaction or dehydration; or a QTc interval \>480 ms during screening;
ā. History of severe non-ischemic cardiomyopathy.
ā. Accompanied by other uncontrolled malignancies. Subjects with the following conditions should be excluded: early-stage tumors that have received radical treatment (carcinoma in situ or grade 1 tumors, or non-ulcerated primary melanoma with a depth \<1 mm and no involvement of lymph nodes), basal cell skin cancer, skin squamous cell carcinoma, cervical carcinoma in situ, or breast cancer in situ that has received potential radical treatment;