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New treatment option for Generalized Myasthenia Gravis (gMG)
Official title GC012F Injection in the Treatment of Refractory Generalized Myasthenia Gravis(24103)
ClinicalTrials.gov ID: NCT07058298
What this study is testing
What is GC012F injection?
GC012F injection is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for generalized myasthenia gravis (gmg).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join if
- To be enrolled, people must meet all of the following criteria:
- people or their legal representatives voluntarily sign a written informed consent and are willing and able to comply with the procedures of the study;
- people aged 18-75 years old (both inclusive), male or female;
- people with confirmed refractory gMG of classes IIa - IVb by MGFA clinical classification (including classes IIa, IIb, IIIa, IIIb, IVa and IVb) at...
- people with the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score of ≥6, the proportion of ocular symptoms of \<50% of the total score...
You likely can't join if
- Participants who meet any of the following criteria are not included in the study:
- Have a history of severe hypersensitivity or allergy;
- Contraindications or hypersensitivity to fludarabine, cyclophosphamide and any component of the test drug;
- people 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;
- Received Tacrolimus, Cyclosporine, Azathioprine, Mycophenol Mofetil within 1 week before apheresis;
See the full eligibility criteria
Who can join
- To be enrolled, people must meet all of the following criteria:
- people or their legal representatives voluntarily sign a written informed consent and are willing and able to comply with the procedures of the study;
- people aged 18-75 years old (both inclusive), male or female;
- people with confirmed refractory gMG of classes IIa - IVb by MGFA clinical classification (including classes IIa, IIb, IIIa, IIIb, IVa and IVb) at screening;
- people 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;
- people with poor response and/or who do not respond to the conventional therapies, that means people who are still at risk of relapse or exacerbation after conventional therapies with hormones, immunosuppressants (e.g...
- For patients who are taking corticosteroids, the dose of prednisone should not exceed 30 mg/day (or an equivalent dose of other corticosteroids), and the dose must be stable for at least 4 weeks before infusion;
- The laboratory test results during the screening period meet the following criteria:
- Neutrophil count ≥ 1.0×10\^9/L; Hemoglobin ≥ 8.0 g/dL; Platelet count≥50×10\^9/L;
- Alanine aminotransferase ≤ 3× upper limit of normal (ULN); Aspartate aminotransferase ≤ 3×ULN; total bilirubin (TBIL) \< 2× ULN (for people with Gilbert's syndrome), direct bilirubin (DBIL)) ≤ 1.5×ULN
- Creatinine clearance (19.3 Appendix 3) ≥ 30 mL/min;
- Activated partial thromboplastin time (APTT) ≤ 1.5×ULN, prothrombin time (PT)≤ 1.5×ULN;
- Subject's left ventricular ejection fraction (LVEF) is ≥ 50% by echocardiography, with no evidence of pericardial effusion as determined;
- Female people of child-bearing age must:
- At screening, a negative serum β human chorionic gonadotropin β-hCG pregnancy test result confirmed by the investigator;
- Who agree to avoid breastfeeding during the study period until at least 1 year after the infusion of GC012F Injection or until two consecutive flow cytometry tests show the absence of CAR-T cells (whichever occurs...
- Male people with sexual partners and female people of potential child-bearing age shall agree to take effective contraceptive measures (e.g., oral contraceptive pills, intrauterine device or condom) from the screening...
- people for whom venous access available for blood collection can be established, and with no contraindications to leukocyte collection.
What rules you out
- Participants who meet any of the following criteria are not included in the study:
- Have a history of severe hypersensitivity or allergy;
- Contraindications or hypersensitivity to fludarabine, cyclophosphamide and any component of the test drug;
- people 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;
- Received Tacrolimus, Cyclosporine, Azathioprine, Mycophenol Mofetil within 1 week before apheresis;
- Treatment with neonatal Fc receptor (FcRn) antagonists within 1 week prior to apheresis;
- Patients who have received complement inhibitors (e.g., eculizumab, etc.) within 1 weeks before apheresis ;
- people with any of the following heart diseases:
- Class III or Class IV congestive heart failure based on New York Heart Association (NYHA) Functional Classification;
- Unstable angina, myocardial infarction or coronary artery bypass grafting (CABG) within 6 months prior to screening;
- Clinically significant ventricular arrhythmia or a history of unexplained syncope not due to vasovagal reaction or dehydration; or a QTc interval \>480 ms at screening;
- Has a history of severe non-ischemic cardiomyopathy;
- Severe cardiovascular abnormalities (such as brain natriuretic peptide (BNP)/troponin and other indices) and the investigator judges that such people are not eligible for enrollment.
- people with other uncontrolled malignancies. The following conditions will be excluded: early-stage tumors that have been treated by radical surgery (carcinoma in situ or grade 1 tumors, or non-ulcerative primary...
- Serious underlying medical conditions, such as:
- Viral, bacterial, fungal, or other infections that are uncontrollable or requiring systemic intravenous therapy (including tuberculosis infection with clear evidence of disease activity) as demonstrated by evidence;
- Dementia or mental status changes as demonstrated by significant clinical evidence;
- History of any central nervous system (CNS) or neurodegenerative diseases, (e.g., Epilepsy, Convulsion, Paralysis, Aphasia, Stroke, Severe brain injury, Dementia, Parkinson's disease, Mental Illness).
- Positive result for any of the following tests:
- Positive test result of human immunodeficiency virus (HIV) antibody;
- Positive test result of hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) (HBV DNA copy number will be detected for HBcAb- or HBsAb-positive patients. If the copy number is lower than the lower...
- Positive test result of hepatitis C virus (HCV) antibody with the copy number of HCV RNA higher than the lower limit of detection; or with known history of hepatitis C without completion of antiviral therapy for ≥24...
- Positive test result of Syphilis antibody.
- Prior treatment with a CAR-T product for any target;
- Previous organ or allogeneic bone marrow transplantation;
- people who have undergone surgery within 2 weeks prior to lymphodepletion pretreatment or plan to undergo surgery during the study (except people who schedule for local anesthesia surgery, but the surgery cannot be...
- Received live attenuated vaccine within 4 weeks prior to lymphodepletion pretreatment;
- people who have received study drugs in other clinical trials within 4 weeks prior to signing the informed consent form, or whose ICF signing date is within 5 half-lives of the last dose they had taken in other clinical...
- Pregnant females or lactating females who do not agree to give up breastfeeding, during the period of participation in this study or men with a family plan within 1 year of receiving study treatment and females;
- people with suicidal intentions at present based on the Columbia-Suicide Severity Rating Scale (C-SSRS), i.e., the answer to Question 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) or...
- According to the researcher's judgment, there are circumstances that may prevent the subject from participating in the full trial, confuse the trial results, or participation in this study is not in the best interests...
The study team makes the final eligibility decision.
Where it's taking place
- Wuhan, Hubei, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Wuhan, Hubei, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.