GC012F in Patients With Autoimmune Diseases

Launched by QIONG FU · Jul 9, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called GC012F Injection for people with certain autoimmune diseases, specifically Systemic Sclerosis and Idiopathic Inflammatory Myopathy. These are conditions where the body’s immune system mistakenly attacks healthy tissues. The main goal of the trial is to find out if this treatment is safe and if it helps improve symptoms. Researchers will also look at how the medicine behaves in the body and how it affects the immune system.

To take part in the study, participants need to be between 18 and 70 years old and meet specific health requirements, such as having certain blood counts and heart function within safe limits. They must agree to follow certain safety measures, including using effective birth control during and after the study because the treatment may affect fertility or pregnancy. Participants will go through tests and procedures, including blood draws and possibly a process called leukapheresis, which collects certain blood cells for treatment. The study is still not open for recruiting, and people with serious heart problems, infections, recent cancer, or certain other health issues won’t be eligible. If you or your loved one are considering this trial, it’s important to discuss all details with the study team to understand the risks and benefits.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Only subjects who meet all of the following inclusion criteria prior to enrollment are eligible for this study. In addition, subjects should meet the relevant inclusion criteria for each indication as specified in the corresponding section of each sub-study protocol.
• • 1. The subject or legal representative voluntarily signs the written informed consent form (ICF) personally, and is willing and able to comply with study procedures;
• • 2. Aged 18 to 70 years (inclusive) at the time of signing the informed consent form, male or female;
• • 3. Willing to adhere to study-specific contraception requirements until 2 years after infusion or until two consecutive flow cytometry tests indicate that CAR-T cells are no longer present in females of childbearing potential and male subjects (whichever occurs later);
• 4. Screening laboratory test results must meet the following criteria (except for indicators related to the diseases under study):
• • 1. Neutrophil count ≥ 1.0 × 10\^9/L; hemoglobin ≥ 80 g/L; platelet count ≥ 50 × 10\^9/L;
• • 2. Alanine aminotransferase (ALT) ≤ 3 × upper limit of normal (ULN); aspartate aminotransferase (AST) ≤ 3 × ULN (unless ALT and/or AST increase is assessed by the investigator as related to IIM); total bilirubin \< 2 × ULN (for subjects with Gilbert's syndrome, direct bilirubin ≤ 1.5 × ULN);
• • 3. Creatinine clearance ≥ 60 mL/min, or estimated glomerular filtration rate (eGFR) ≥ 45 mL/min/1.73 m\^2;
• • 4. Activated partial thromboplastin time (APTT) ≤ 1.5 × ULN, and prothrombin time (PT) ≤ 1.5 × ULN;
• • 5. Left ventricular ejection fraction (LVEF) ≥ 50% as determined by echocardiogram, with no evidence of pericardial effusion.
• 5. Women of childbearing potential must:
• • 1. Have a negative serum β-human chorionic gonadotropin (β-hCG) pregnancy test result at screening as confirmed by the investigator;
• • 2. Agree to avoid breastfeeding during the study period and until at least 2 years after infusion of GC012F Injection, or until two consecutive flow cytometry tests indicate that CAR-T cells are no longer present (whichever occurs later).
• • 6. Male subjects with sexual partners and female subjects of childbearing potential must agree to use highly effective contraception methods (e.g., oral contraceptives, intrauterine devices, or condoms) starting from screening and continuing for at least 2 years after GC012F Injection infusion or until two consecutive flow cytometry tests indicate that CAR-T cells are no longer present (whichever occurs later). Male subjects must agree to use condoms during any sexual contact with pregnant women or women of childbearing potential for at least 2 years after GC012F Injection infusion, even if they have undergone successful vasoligation.
• • 7. The required venous access for collection can be set up, with no contraindications for leukapheresis.
• Exclusion Criteria:
• • Subjects who meet any of the following criteria are not eligible for the study. In addition, subjects who meet the relevant exclusion criteria for each indication as specified in the corresponding section of each sub-study protocol must not be enrolled.
• • 1. History of severe hypersensitivity or allergy;
• • 2. Contraindications or hypersensitivity to fludarabine, cyclophosphamide, or any component of the investigational product;
• 3. Presence of the following cardiac disorders:
• • 1. New York Heart Association (NYHA) Class III or IV congestive heart failure;
• • 2. Myocardial infarction or coronary artery bypass grafting within 6 months prior to screening;
• • 3. History of clinically significant ventricular arrhythmia or unexplained syncope not caused by vasovagal reactions or dehydration, or corrected QT interval \> 480 ms at screening;
• • 4. History of severe non-ischemic cardiomyopathy.
• • 4. Any active malignancy or history of malignancy within 5 years prior to screening. The following are exceptions: early-stage tumors that have received curative treatment (carcinoma in situ or stage I tumors, non-ulcerative primary melanoma with a depth \< 1 mm and no lymph node involvement), basal cell carcinoma of the skin, squamous cell carcinoma of the skin, thyroid carcinoma in situ or early-stage thyroid cancer that has received curative treatment, cervical carcinoma in situ, or breast cancer in situ that has received potentially curative treatment;
• • 5. Clinically significant hemorrhage symptoms or a definite tendency to hemorrhage within 6 months prior to screening, such as hemorrhage of digestive tract and hemorrhagic gastric ulcer; hereditary or acquired hemorrhage or thrombotic tendencies (e.g., hemophilia, coagulation disorders, and hypersplenism); arterial or venous thrombotic events within 6 months prior to screening, such as cerebrovascular diseases (including cerebral hemorrhage and cerebral infarction), deep vein thrombosis and/or pulmonary embolism;
• 6. Any of the following test results is positive:
• • 1. Human immunodeficiency virus (HIV) antibody positive;
• • 2. Hepatitis B surface antigen (HBsAg) positive; or hepatitis B core antibody (HBcAb) positive with hepatitis B virus deoxyribonucleic acid (DNA) above the lower limit of detection of the assay;
• • 3. Hepatitis C virus (HCV) antibody positive with HCV ribonucleic acid (RNA) above the lower limit of detection of the assay;
• • 4. Syphilis antibody positive (confirmation by a confirmatory test for syphilis is required if necessary, such as fluorescent treponemal antibody-absorption test and Treponema pallidum particle agglutination assay).
• • 7. Active tuberculosis or latent tuberculosis that has not received appropriate treatment prior to screening;
• 8. Severe underlying medical conditions, such as:
• • 1. Evidence of viral, bacterial, fungal, or other infections that are uncontrolled or need systemic intravenous treatment;
• • 2. Clear clinical evidence of dementia or altered mental status;
• • 3. Any other central nervous system disease or history of neurodegenerative diseases, such as epilepsy, seizure, paralysis, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, and psychosis.
• • 9. Prior therapy targeting cluster of differentiation (CD)19 and/or B-cell maturation antigen, or CAR-T product therapy targeting any antigen;
• • 10. Thymectomy within 12 months prior to signing the ICF;
• • 11. Treatment with CD20-targeted agents within 6 months prior to signing the ICF;
• • 12. Receipt of other investigational products within 4 weeks prior to signing the ICF, or if the time from the last dose of a previous investigational product to the ICF signing date is still within 5 half-lives of that drug (whichever is longer);
• • 13. Receipt of other monoclonal antibody drugs (e.g., infliximab, certolizumab pegol, golimumab, etanercept, abatacept, adalimumab, or tocilizumab) within 4 weeks prior to apheresis;
• • 14. Receipt of cyclophosphamide within 4 weeks prior to apheresis;
• • 15. Major surgery within 8 weeks prior to signing the ICF, or planned surgery during the study;
• • 16. History of organ transplantation;
• • 17. Pregnant women, breastfeeding women who do not agree to discontinue breastfeeding, or males and females who plan to conceive during the study or within 2 years after receiving study treatment;
• • 18. Any condition, as determined by the investigator, that would interfere with the subject's full participation in the study, confound the study results, or render participation in the study not in the best interest of the subject.