Actively Recruiting
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
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-07-24
15
Participants Needed
1
Research Sites
12 weeks
Total Duration
On this page
Sponsors
I
Institute of Hematology & Blood Diseases Hospital, China
Lead Sponsor
N
Nanjing Bioheng Biotech Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating RD13-02, a universal CD7 CAR T-cell therapy, in adults with relapsed or refractory severe aplastic anemia (SAA). This early-phase study aims to explore the safety, tolerability, how the drug moves and acts in the body, and its effectiveness in this condition. The trial is sponsored by the Institute of Hematology & Blood Diseases Hospital in China and focuses on patients who have not responded to standard treatments. The trial uses a single-arm, open-label design with a dose escalation phase followed by an expansion phase. Each phase includes a screening period of up to 2 weeks, a 7-day conditioning treatment with chemotherapy, then a 28-day treatment period where participants receive a single intravenous infusion of the RD13-02 CAR T cells. The CAR T cells are genetically modified from healthy donors to target CD7 on affected cells while minimizing risks like graft-versus-host disease. Continuous heart monitoring occurs during and after the infusion. Participants will be closely monitored for up to 6 months after the infusion. Regular assessments include safety checks for side effects or toxicities, and measurements of blood cell response at 3 and 6 months. The study involves physical exams, laboratory tests, and ongoing observation to evaluate how well the treatment is tolerated and how it affects the disease. The total study duration includes screening, treatment, and follow-up phases.
CONDITIONS
Brief Title
CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willing and able to provide written informed consent
- Age 18 years and older up to 75 years
- Diagnosed with severe aplastic anemia based on UK Hematology Guidelines
- Had at least one course of standard therapy with ATG or ALG combined with TPO-RA that was ineffective or relapsed within 6 months
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Willing and able to comply with study procedures
You will not qualify if you...
- Blood cell reduction and bone marrow diseases from other causes (e.g., hemolytic PNH, hypoplastic MDS/AML)
- Received ATG/ALG treatment less than 6 months ago or TPO-RA less than 4 months ago unless stable
- Significant neurological disease history posing safety risks
- History or plans for allogeneic bone marrow, stem cell, or solid organ transplantation
- History of autologous or allogeneic CAR T therapy
- Primary immunodeficiency diseases
- Significant cardiovascular dysfunction or recent congestive heart failure
- Liver or lung dysfunction beyond specified thresholds
- Abnormal QTcF intervals on ECG
- History of malignancy except certain treated cancers
- Pregnant or breastfeeding females or planning pregnancy during or 12 months after the study
- Significant chronic or uncontrolled infections
- Positive tests for hepatitis B, C, or HIV
- Recent live attenuated vaccination
- Abnormal blood clotting tests beyond limits
- Severe kidney impairment (eGFR <30 mL/min/1.73 m2)
- History of drug or alcohol abuse within past year
- Participation in other experimental studies recently
- Hypersensitivity to study drug components
- Any condition that may affect participation or safety as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 2 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 7 days
Participants undergo lymphodepleting chemotherapy with Fludarabine and Cyclophosphamide before receiving the CAR T cell infusion.
Daily visits for 7 days
Duration - 28 days
Participants receive a single intravenous infusion of RD13-02 CAR T cells with continuous cardiac monitoring during and up to 2 hours after infusion.
1 infusion visit with monitoring plus follow-up visits during treatment period
Duration - Up to 6 months
Participants are monitored for safety, response, and other outcomes up to 6 months after the first infusion.
Regular follow-up visits during 6-month period
Trial Site Locations
Total: 1 location
1
Regenerative Medicine Clinic Center
Tianjin, Tianjin Municipality, China
Actively Recruiting
Research Team
J
Jun Shi, PhD
L
Lele Zhang, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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