GC012F in Patients With Autoimmune Diseases

NCT07072884 | Not Yet Recruiting Early Phase 1 DMC

• 1 other identifier: 24105/D8318N00001
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is an open-label, early exploratory main clinical study to evaluate the safety and efficacy of GC012F Injection in subjects with autoimmune diseases (AID), as well as to assess its pharmacokinetic (PK) and pharmacodynamic (PD) profiles.

Conditions

Systemic Sclerosis (SSc); Idiopathic Inflammatory Myopathy (IIM)

Outcome Measures

Primary Outcomes (2)

• Proportion of subjects with dose-limiting toxicity (DLT) within 28 days after infusion.

  28 days after infusion.

• Post-infusion adverse events and their proportion.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

Secondary Outcomes (6)

• Chimeric antigen receptor T cell (CAR-T) levels in peripheral blood at each time point.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

• Chimeric antigen receptor transgene levels in peripheral blood at each time point.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

• Quantification of cytokines in peripheral blood at each time point.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

• Quantification of immunoglobulins in peripheral blood at each time point.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

• Changes in patient reported outcomes (PROs) from baseline at each time point: health assessment questionnaire disability index (HAQ-DI) .

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

Other Outcomes (3)

• Detection rate of CAR-T cell antibody after GC012F infusion.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

• Changes in antibody concentrations resulting from previous vaccination.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

• Detection rate of RCL after GC012F infusion.

  From the signing of informed consent through 672 days (96 weeks) after cell infusion or initiation of a new therapy, whichever occurs first.

Study Arms (1)

3.0×10^5 CAR-T cells/kg

EXPERIMENTAL

Drug: GC012F injection

Interventions

GC012F injection DRUG

Subjects who meet the CAR-T cell infusion criteria will receive GC012F Injection infusion within 48-72 hours after completion of lymphodepleting preconditioning.

3.0×10^5 CAR-T cells/kg

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The subject or legal representative voluntarily signs the written informed consent form (ICF) personally, and is willing and able to comply with study procedures;
• Aged 18 to 70 years (inclusive) at the time of signing the informed consent form, male or female;
• 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);
• Screening laboratory test results must meet the following criteria (except for indicators related to the diseases under study):
• Neutrophil count ≥ 1.0 × 10\^9/L; hemoglobin ≥ 80 g/L; platelet count ≥ 50 × 10\^9/L;
• 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);
• Creatinine clearance ≥ 60 mL/min, or estimated glomerular filtration rate (eGFR) ≥ 45 mL/min/1.73 m\^2;
• Activated partial thromboplastin time (APTT) ≤ 1.5 × ULN, and prothrombin time (PT) ≤ 1.5 × ULN;
• Left ventricular ejection fraction (LVEF) ≥ 50% as determined by echocardiogram, with no evidence of pericardial effusion.
• Women of childbearing potential must:
• Have a negative serum β-human chorionic gonadotropin (β-hCG) pregnancy test result at screening as confirmed by the investigator;
• 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).
• 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.
• The required venous access for collection can be set up, with no contraindications for leukapheresis.

You may not qualify if:

• History of severe hypersensitivity or allergy;
• Contraindications or hypersensitivity to fludarabine, cyclophosphamide, or any component of the investigational product;
• Presence of the following cardiac disorders:
• New York Heart Association (NYHA) Class III or IV congestive heart failure;
• Myocardial infarction or coronary artery bypass grafting within 6 months prior to screening;
• History of clinically significant ventricular arrhythmia or unexplained syncope not caused by vasovagal reactions or dehydration, or corrected QT interval \> 480 ms at screening;
• History of severe non-ischemic cardiomyopathy.
• 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;
• 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;
• Any of the following test results is positive:
• Human immunodeficiency virus (HIV) antibody positive;
• 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;
• Hepatitis C virus (HCV) antibody positive with HCV ribonucleic acid (RNA) above the lower limit of detection of the assay;
• 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).
• Active tuberculosis or latent tuberculosis that has not received appropriate treatment prior to screening;

Sponsors & Collaborators

• Qiong Fu: lead
• Gracell Biotechnologies (Shanghai) Co., Ltd.: collaborator

MeSH Terms

Conditions

Scleroderma, Systemic; Myositis

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Central Study Contacts

Qiong Fu

CONTACT

13585603288; Fuqiong5@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief Physician of Rheumatology

Study Record Dates

• First Submitted: June 18, 2025
• First Posted: July 18, 2025
• Study Start: August 18, 2025
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): March 31, 2029
• Last Updated: July 18, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share