A Clinical Study of Safety, Efficacy, and Pharmacokinetics of Universal CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia
Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
This trial is exploratory research aimed at evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD13-02, a universal CD7 CAR T therapy, in subjects with relapsed/refractory severe aplastic anemia (SAA)
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
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• Willing and able to provide written informed consent.
• Age ≥18 years and ≤75 years.
• Diagnosed with severe aplastic anemia: Diagnostic criteria refer to the UK Hematology Guidelines (Br J Haematol, 2024, 204(3): 784-804): 1) Bone marrow cellularity \<25% (or 25-50% but \<30% residual hematopoietic cell area); 2) At least two of the following conditions must be met: ANC \<0.5×10\^9/L, PLT \<20×10\^9/L, absolute reticulocyte count \<60×10\^9/L; 3) Exclusion of other congenital or acquired hematopoietic failure disorders.
• Had at least one course of standard-dose therapy with anti-thymocyte globulin (ATG) or anti-lymphocyte globulin (ALG) combined with TPO-RA that was ineffective or relapsed after remission within the past 6 months.
• Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
• Willing and able to comply with the study procedures.
Locations
Other Locations
China
Regenerative Medicine Clinic Center
RECRUITING
Tianjin
Contact Information
Primary
Jun Shi, PhD
shijun@ihcams.ac.cn
13752253515
Backup
Lele Zhang, PhD
zhanglele@ihcams.ac.cn
15811139278
Time Frame
Start Date:2025-06-19
Estimated Completion Date:2026-12-31
Participants
Target number of participants:15
Treatments
Experimental: RD13-02
RD13-02 is a genetically engineered T cell product derived from healthy donors, designed to express a chimeric antigen receptor (CAR) targeting CD7 on its cell membrane. This modification enables the CAR T cells to specifically kill CD7-positive tumor cells. CD7 is expressed on the surface of both normal and malignant T cells. The extracellular domain of RD13-02 consists of a humanized single-chain antibody targeting CD7, while the co-stimulatory domain is derived from TNFRSF9 (CD137 or 4-1BB). The CAR is cloned into a clinical-grade retroviral vector and introduced into human T cells via retroviral transduction. To prevent fratricide, RD13-02 has had surface CD7 expression knocked out, and TCR knockout is implemented to prevent graft-versus-host disease (GvHD). HLA knockout and NK cell introduction further reduce the risk of rejection.