CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia

NCT06622694 | Recruiting Phase 1

• 1 other identifier: IIT2024081
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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)

Conditions

Severe Aplastic Anemia; Relapse; Refractory; CAR T-cell Therapy

Outcome Measures

Primary Outcomes (1)

• The incidence of dose-limiting toxicities (DLT), serious adverse events (SAE), and other treatment-emergent adverse events (TEAE)

  The severity of adverse events will be graded according to NCI CTCAE version 5. Based on System Organ Class and Preferred Terms, adverse events will be summarized by incidence.

  After cell infusion

Secondary Outcomes (1)

• Overall hematologic response rates (ORR)

  At 3 and 6 months post-cell infusion

Study Arms (1)

RD13-02

EXPERIMENTAL

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.

Biological: RD13-02

Interventions

RD13-02 BIOLOGICAL

Before cell infusion (Day 0), subjects will undergo a lymphodepleting chemotherapy based on the "Fludarabine + Cyclophosphamide" (FC regimen). Prior to the lymphodepleting chemotherapy, subjects must undergo an assessment, and only those meeting the criteria will be eligible for the lymphodepleting chemotherapy. The CAR T infusion should be performed at least 48 hours after the completion of the lymphodepleting chemotherapy. The infusion will be administered intravenously as a single dose. It should be completed within 30 minutes, with continuous cardiac monitoring during the infusion to observe heart rate (HR), blood pressure (BP), respiratory rate (R), and peripheral oxygen saturation (SpO2) for up to 2 hours post-infusion.

RD13-02

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Willing and able to provide written informed consent.
• Age ≥18 years and ≤75 years.
• 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.

You may not qualify if:

• Blood cell reduction and hypoplastic bone marrow diseases due to other causes (e.g., hemolytic PNH, hypoplastic MDS/AML, antibody-mediated pancytopenia).
• Received ATG/ALG treatment less than 6 months ago; received TPO-RA treatment less than 4 months ago. Note: Patients on stable doses of cyclosporine or hematopoietic agents at screening can be enrolled if laboratory values are stable.
• Significant neurological disease history, including dementia, stroke without paralysis, aphasia, seizures, or any neurological history that may pose safety risks regarding cell therapy as judged by the investigator.
• History of allogeneic bone marrow or stem cell transplantation, or solid organ transplantation (e.g., kidney, lung, heart), or plans for such transplantation in the future.
• History of autologous or allogeneic CAR T therapy.
• Any primary immunodeficiency disease.
• Significant cardiovascular dysfunction history or current significant cardiovascular dysfunction, particularly:
• Signs or symptoms of congestive heart failure classified as NYHA class ≥III within 12 months prior to enrollment.
• Echocardiogram showing left ventricular ejection fraction (LVEF) \<45% (assessed during screening).
• History of any serious arrhythmia, currently on treatment for arrhythmias, significant history of myocardial infarction, clinically significant valvular heart disease (including mild or greater aortic regurgitation or moderate or greater mitral regurgitation), recurrent syncope, or significant hypercoagulable vascular events (e.g., transient ischemic attack or stroke); history of pulmonary embolism.
• Liver or lung dysfunction, defined as:
• Serum alanine aminotransferase (ALT)/aspartate aminotransferase (AST) ≥2.5× the upper limit of normal (ULN);
• Total bilirubin ≥1.5×ULN, except in subjects with Gilbert's syndrome.
• Pulmonary hypertension, including secondary pulmonary hypertension, classified as WHO functional class \>2.
• Males with QTcF \>450 msec; females with QTcF \>470 msec, based on a single ECG or the average of three repeat ECGs taken more than 3 minutes apart.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• Nanjing Bioheng Biotech Co., Ltd.: collaborator

Study Sites (1)

Regenerative Medicine Clinic Center

Tianjin, Tianjin Municipality, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic; Recurrence

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Jun Shi, PhD

CONTACT

13752253515; shijun@ihcams.ac.cn

Lele Zhang, PhD

CONTACT

‭15811139278‬; zhanglele@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Regenerative Medical Center

Study Record Dates

• First Submitted: September 30, 2024
• First Posted: October 2, 2024
• Study Start: June 19, 2025
• Primary Completion (Estimated): October 7, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: July 24, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)