Status:
ACTIVE_NOT_RECRUITING
Multi-omics Profiling of Patients With Aplastic Anemia Before and After CD7-CAR-T Therapy
Lead Sponsor:
Xuzhou Medical University
Conditions:
Aplastic Anaemia
CAR-T
Eligibility:
All Genders
18-70 years
Brief Summary
Aplastic anemia (AA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and hypocellular bone marrow, with immune-mediated destruction of hematopoietic stem and progenito...
Detailed Description
This is a single-center, single-arm, prospective clinical study evaluating the safety and efficacy of CD7-directed CAR-T cell therapy in adult patients with refractory or relapsed severe aplastic anem...
Eligibility Criteria
Inclusion
- Age ≥18 years at the time of informed consent, of Chinese nationality. ECOG performance status 0-1, with an expected survival time ≥3 months.
- Diagnosis of severe aplastic anemia (SAA) and meeting at least one of the following:
- Relapsed or refractory after standard therapy without achieving complete remission.
- Ineligible for, without access to, or refusing allogeneic hematopoietic stem cell transplantation.
- Laboratory values at screening meeting all of the following:
- Serum creatinine ≤2.5 × upper limit of normal (ULN). Oxygen saturation ≥90% at baseline. Total bilirubin ≤3 × ULN. ALT and AST ≤3 × ULN. Adequate venous access for mononuclear cell collection and no contraindications to leukapheresis.
- Female participants of childbearing potential must have a negative high-sensitivity serum pregnancy test (β-hCG) at screening and before the first dose of fludarabine/cyclophosphamide. Male and female participants of childbearing potential must agree to use effective contraception from informed consent through at least 36 months after CD7-CAR-T infusion.
- After review by the investigator team, the overall benefit of trial participation is judged to outweigh potential risks.
- Ability to understand and sign informed consent, and willingness to comply with study procedures and restrictions.
Exclusion
- History of or treatment for other malignancies within 5 years prior to screening, except adequately treated cervical carcinoma in situ, basal or squamous cell skin cancer, curatively treated localized prostate cancer, or ductal carcinoma in situ.
- Severe systemic diseases, including:
- NYHA class III or IV congestive heart failure. Stroke, myocardial infarction, or hemodynamically unstable arrhythmia within 6 months prior to consent.
- Left ventricular ejection fraction (LVEF) \<50% by echocardiography. Severe or uncontrolled concomitant illness within 14 days prior to consent, including active infection.
- Pregnant or breastfeeding women. Positive serology for HIV. Positive for hepatitis B surface antigen or detectable HBV DNA. Positive for hepatitis C antibody with detectable HCV RNA. Positive syphilis serology (TP-Ab and RPR). Positive CMV DNA.
- History of life-threatening hypersensitivity to CD7-CAR-T cells or any excipients (including DMSO), or known hypersensitivity to biologics such as antibodies or cytokines.
- Contraindications to fludarabine or cyclophosphamide therapy. History of alcohol dependence, substance abuse, or psychiatric disorders that may interfere with study compliance.
- Any other condition that, in the opinion of the investigator, makes the participant unsuitable for the study.
Key Trial Info
Start Date :
August 1 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
July 31 2027
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT07139600
Start Date
August 1 2024
End Date
July 31 2027
Last Update
August 24 2025
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Xuzhou Medical University
Xuzhou, Jiangsu, China, 221006