CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-75
SponsorInstitute of Hematology & Blood Diseases Hospital, China
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
RD13-02
Biological/VaccineBefore 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.
Treatment groups
15 Participants
are divided into 1 treatment groupGroup A: RD13-02Experimental treatment 1 intervention
Trial outcomes
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.
Time frame
After cell infusionSecondary outcomes
1
Overall hematologic response rates (ORR)
A response in any one or more cell lineages, including platelet response, erythroid response and neutrophil response
Time frame
At 3 and 6 months post-cell infusionSponsors and contacts
Click on the lead sponsor to view all of their trials.
Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Nanjing Bioheng Biotech Co., Ltd.
Collaborator
Source ClinicalTrials.gov