Severe Aplastic AnemiaPhase 1NCT06622694
CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia
A Phase 1 study testing RD13-02 in people with Severe Aplastic Anemia and Relapse, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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) Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Severe Aplastic Anemia, Relapse, Refractory
- What is being tested?
- RD13-02
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 19 Jun 2025to 31 Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
- History of malignancy, excluding adequately treated or surgically removed non-melanoma skin cancer or in situ cancers (e.g., cervical cancer, bladder cancer, breast cancer) without residual disease.
- Pregnant or breastfeeding females, or females planning to become pregnant during the study or within 12 months after RD13-02 infusion.
- History or signs of significant chronic active or recurrent infections, or significant chronic active or recurrent infections requiring antibiotics, antivirals, or antifungal treatments based on screening laboratory results.
- Uncontrolled infections at screening. Patients with uncomplicated urinary tract infections (UTIs) and uncomplicated bacterial pharyngitis responding to active treatment may be included with investigator and medical monitor approval.
- Positive screening for HBsAg, HCV RNA, or HIV. If a subject is HBsAg negative but HBcAb positive, hepatitis B virus DNA testing should be performed; subjects with positive hepatitis B virus DNA should be excluded from the study.
- Vaccination with live attenuated vaccines within 4 weeks prior to enrollment, or plans to receive live attenuated vaccines during the study.
- PT, APTT, or INR >1.2×ULN, except for subjects on stable doses of anticoagulants who can discontinue treatment prior to using intravenous access procedures.
- eGFR <30 mL/min/1.73 m² calculated using the CKD-EPI formula; if eGFR ≥30 mL/min/1.73 m² but <45 mL/min/1.73 m², adjust fludarabine dosing in the lymphodepleting regimen based on renal function.
- History of drug or alcohol abuse within the past year.
- Currently enrolled in other experimental device or drug studies, or less than 30 days since ending other experimental device or drug interventions, or less than 5 half-lives (whichever is longer).
- History of hypersensitivity or life-threatening reactions to any component or formulation of the study drug or treatment (including lymphodepleting regimen). For detailed information about study drug components, refer to the investigator brochure (IB).
- Any condition the investigator believes may affect participation, pose safety risks to the subjects, or potentially confound the interpretation of study results.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Regenerative Medicine Clinic Center
Tianjin, Tianjin Municipality
Lele Zhang, PhD, 15811139278, zhanglele@ihcams.ac.cn
About this study
Official title: 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
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)
Read the detailed description
This trial used a single-arm, open-label, and "3+3" dose-escalation design. It is divided into two phases, the dose escalation phase and the expansion phase. The dose escalation and expansion phases each consisted of a screening period of up to 2 weeks, a 7-day conditioning period, a 28-day treatment period, and a follow-up period (to 6 months after the first infusion).
- Conditions
- Severe Aplastic Anemia, Relapse, Refractory, CAR T-cell Therapy
- Being tested
- RD13-02
- Medical subject headings
- Anemia, Aplastic, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RD13-02Biological
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: RD13-02 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: After cell infusion
Secondary outcomes (1)
Overall hematologic response rates (ORR)
A response in any one or more cell lineages, including platelet response, erythroid response and neutrophil response
Measured: At 3 and 6 months post-cell infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2024
- Study start19 Jun 2025
- Primary completion7 Oct 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Collaborators
- Nanjing Bioheng Biotech Co., Ltd.
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06622694
- Sponsor's protocol ID
- IIT2024081
- Status last verified
- Jul 2025
- First submitted
- 30 Sep 2024
- Last updated
- 24 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06622694
- Acronym / sponsor ID
- IIT2024081
- Conditions
- Severe Aplastic Anemia, Relapse, Refractory, CAR T-cell Therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Nanjing Bioheng Biotech Co., Ltd.
- Study start
- 19 Jun 2025 (actual)
- Primary completion
- 7 Oct 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 2 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
This trial used a single-arm, open-label, and "3+3" dose-escalation design. It is divided into two phases, the dose escalation phase and the expansion phase. The dose escalation and expansion phases each consisted of a screening period of up to 2 weeks, a 7-day conditioning period, a 28-day treatment period, and a follow-up period (to 6 months after the first infusion).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RD13-02 | Experimental | Biological: RD13-02RD13-02 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (27 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RD13-02Before 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. | Biological | RD13-02 |
Primary outcome
The incidence of dose-limiting toxicities (DLT), serious adverse events (SAE), and other treatment-emergent adverse events (TEAE)
Time frame: After cell infusion
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.
1 secondary outcome
Overall hematologic response rates (ORR)
Time frame: At 3 and 6 months post-cell infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RD13-02 in people with Severe Aplastic Anemia and Relapse, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jun Shi, PhD (13752253515, shijun@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of dose-limiting toxicities (DLT), serious adverse events (SAE), and other treatment-emergent adverse events (TEAE) (time frame: After cell infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2025.