Aplastic AnaemiaNCT07139600
Multi-omics Profiling of Patients With Aplastic Anemia Before and After CD7-CAR-T Therapy
An observational study of Aplastic Anaemia and CAR-T, run by Xuzhou Medical University at 1 site in China.
Trial summary
Aplastic anemia (AA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and hypocellular bone marrow, with immune-mediated destruction of hematopoietic stem and progenitor cells (HSPCs) playing a central role in its pathogenesis. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Aplastic Anaemia, CAR-T, CD7 Positive
- What is being tested?
- CD7 CAR-T cells infusion
- Type of treatment
- Injection or infusion
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2024to 31 Jul 2027
- How long will I take part?
- 3 Years
- Phase
- Observational
- 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 70 years
- All sexes
- Adult, Older adult
Study population: Adult patients (≥18 years) with refractory or relapsed severe aplastic anemia (SAA) after standard immunosuppressive therapy, or ineligible for/allogeneic hematopoietic stem cell transplantation. Participants must have ECOG 0-1, life expectancy ≥3 months, suitable venous access for leukapheresis, and meet basic lab criteria. Key exclusions include active infections, severe cardiovascular disease, recent malignancy, pregnancy/breastfeeding, or known HIV, HBV, or HCV infection.
Who may be able to join
- Age ≥18 years at the time of informed consent, of Chinese nationality. ECOG performance status 0-1, with an expected survival time ≥3 months.
- Diagnosis of severe aplastic anemia (SAA) and meeting at least one of the following:
- Relapsed or refractory after standard therapy without achieving complete remission.
- Ineligible for, without access to, or refusing allogeneic hematopoietic stem cell transplantation.
- Laboratory values at screening meeting all of the following:
- Serum creatinine ≤2.5 × upper limit of normal (ULN). Oxygen saturation ≥90% at baseline. Total bilirubin ≤3 × ULN. ALT and AST ≤3 × ULN. Adequate venous access for mononuclear cell collection and no contraindications to leukapheresis.
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- Female participants of childbearing potential must have a negative high-sensitivity serum pregnancy test (β-hCG) at screening and before the first dose of fludarabine/cyclophosphamide. Male and female participants of childbearing potential must agree to use effective contraception from informed consent through at least 36 months after CD7-CAR-T infusion.
- After review by the investigator team, the overall benefit of trial participation is judged to outweigh potential risks.
- Ability to understand and sign informed consent, and willingness to comply with study procedures and restrictions.
Who cannot join
- History of or treatment for other malignancies within 5 years prior to screening, except adequately treated cervical carcinoma in situ, basal or squamous cell skin cancer, curatively treated localized prostate cancer, or ductal carcinoma in situ.
- Severe systemic diseases, including:
- NYHA class III or IV congestive heart failure. Stroke, myocardial infarction, or hemodynamically unstable arrhythmia within 6 months prior to consent.
- Left ventricular ejection fraction (LVEF) <50% by echocardiography. Severe or uncontrolled concomitant illness within 14 days prior to consent, including active infection.
- Pregnant or breastfeeding women. Positive serology for HIV. Positive for hepatitis B surface antigen or detectable HBV DNA. Positive for hepatitis C antibody with detectable HCV RNA. Positive syphilis serology (TP-Ab and RPR). Positive CMV DNA.
- History of life-threatening hypersensitivity to CD7-CAR-T cells or any excipients (including DMSO), or known hypersensitivity to biologics such as antibodies or cytokines.
- Contraindications to fludarabine or cyclophosphamide therapy. History of alcohol dependence, substance abuse, or psychiatric disorders that may interfere with study compliance.
- Any other condition that, in the opinion of the investigator, makes the participant unsuitable for the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study of Multi-omics Changes in Aplastic Anemia Patients Receiving CD7-CAR-T Therapy
Aplastic anemia (AA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and hypocellular bone marrow, with immune-mediated destruction of hematopoietic stem and progenitor cells (HSPCs) playing a central role in its pathogenesis. Although immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT) have improved survival, a significant proportion of patients remain refractory, relapse after treatment, or lack suitable donors for transplantation. Therefore, novel therapeutic strategies are urgently needed.
Chimeric antigen receptor T (CAR-T) cell therapy has demonstrated remarkable efficacy in hematologic malignancies. CD7 is an early surface marker of T-lineage cells and is dispensable for T cell development and function, making it a promising therapeutic target. This exploratory study aims to investigate the molecular and cellular mechanisms of CD7-CAR-T therapy in AA patients by analyzing multi-omics changes before and after treatment.
Read more
This is a prospective, single-center, single-arm, open-label study enrolling patients with relapsed or refractory severe aplastic anemia. Participants will receive autologous CD7-CAR-T cells following lymphodepletion. Multi-omics profiling, including genomics, transcriptomics, proteomics, and immunophenotyping, will be performed on patient samples before and after infusion. The primary objective is to explore dynamic molecular changes associated with treatment response and disease progression. Secondary objectives include safety evaluation and preliminary assessment of efficacy.
Findings from this study may provide mechanistic insights into CD7-CAR-T therapy in AA and inform the development of innovative immunotherapies for bone marrow failure syndromes.
Read the detailed description
This is a single-center, single-arm, prospective clinical study evaluating the safety and efficacy of CD7-directed CAR-T cell therapy in adult patients with refractory or relapsed severe aplastic anemia (SAA). SAA is an acquired bone marrow failure disorder characterized by pancytopenia and hypocellular marrow without malignant infiltration. Standard immunosuppressive therapy (IST) has improved survival, but approximately 30% of patients fail initial treatment, and relapse or progression to myelodysplastic syndrome or acute myeloid leukemia remains a clinical challenge. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) improves outcomes for younger patients but is limited by donor availability and graft-versus-host disease risks.
Eligible participants are adults (≥18 years) with ECOG performance status 0-1, life expectancy ≥3 months, and refractory or relapsed SAA after IST, or ineligible for, lacking access to, or refusing allo-HSCT. Key laboratory criteria include creatinine ≤2.5× upper limit of normal, total bilirubin ≤3× ULN, ALT and AST ≤3× ULN, and baseline oxygen saturation ≥90%. Patients must have suitable venous access for leukapheresis and comply with contraception requirements if of reproductive potential. Exclusion criteria include active infections, severe cardiovascular disease, recent malignancy (except certain adequately treated cancers), pregnancy or breastfeeding, HIV, HBV, HCV above specified thresholds, or hypersensitivity to CAR-T components.
The study consists of four stages: patient screening and enrollment; preconditioning including leukapheresis, CAR-T cell manufacture, and chemotherapy; CD7-CAR-T cell infusion with inpatient monitoring; and long-term follow-up. Patients receive a lymphodepletion regimen with fludarabine and cyclophosphamide prior to a single infusion of CD7-CAR-T cells at 3×10^6 cells/kg. Safety assessments, including monitoring for cytokine release syndrome and other adverse events, will be conducted closely during hospitalization. Efficacy assessments include complete blood counts, bone marrow evaluation, and multi-omics analysis of immune cell subsets and cytokine profiles at defined intervals.
Participants will be followed up for up to 3 years post-infusion, with intensive monitoring in the first 6 months and periodic assessments thereafter to evaluate disease status, CAR-T cell persistence, long-term safety, and quality of life. The primary endpoint is overall response rate (ORR), with secondary endpoints including progression-free survival (PFS), overall survival (OS), and safety outcomes. This study aims to provide the first evaluation of CD7-CAR-T therapy in refractory or relapsed SAA patients, potentially offering a novel therapeutic option for this high-risk population.
- Conditions
- Aplastic Anaemia, CAR-T, CD7 Positive
- Being tested
- CD7 CAR-T cells infusion
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD7 CAR-T cells infusionBiological
Autologous T cells are collected by leukapheresis, activated, and transduced with a retroviral vector encoding a CD7-specific CAR. Following fludarabine/cyclophosphamide lymphodepletion, CD7-CAR-T cells (3 × 10^6/kg) are infused intravenously. This intervention is distinguished by its CD7 target, aiming to suppress aberrant T-cell-mediated marrow destruction and restore hematopoiesis in aplastic anemia.
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
The proportion of patients achieving Complete Response (CR) or Partial Response (PR)
Measured: Up to 36 months post-infusion.
Progression-Free Survival (PFS)
Time from CAR-T cell infusion to disease progression, relapse, or death from any cause, whichever occurs first.
Measured: Up to 36 months post-infusion.
Overall Survival (OS)
Time from CAR-T cell infusion to death from any cause.
Measured: Up to 36 months post-infusion.
Event-Free Survival (EFS)
Time from CAR-T cell infusion to the occurrence of any of the following events: failure to achieve response, disease progression or relapse, initiation of alternative therapy, or death from any cause.
Measured: Up to 36 months post-infusion.
Secondary outcomes (1)
Safety and Tolerability
Incidence, severity, and type of treatment-emergent adverse events (TEAEs), including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). Graded according to CTCAE v5.0.
Measured: From infusion through study completion (up to 36 months).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2025
- Study start1 Aug 2024
- Primary completion31 Jul 2027 (estimated)
- Study completion31 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnaemiaStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 5 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Xuzhou Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kai Lin Xu，MD, Xuzhou Medical University
- Investigators
- Hai Cheng, Study director, The Affiliated Hospital oh Xuzhou Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07139600
- Sponsor's protocol ID
- XYFY2024-KL365-01
- Status last verified
- Aug 2025
- First submitted
- 17 Aug 2025
- Last updated
- 24 Aug 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 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Multi-omics Changes in Aplastic Anemia Patients Receiving CD7-CAR-T Therapy
- ClinicalTrials.gov ID
- NCT07139600
- Acronym / sponsor ID
- XYFY2024-KL365-01
- Conditions
- Aplastic Anaemia, CAR-T, CD7 Positive
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Xuzhou Medical UniversityUniversity, hospital or other
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 31 Jul 2027 (estimated)
- Study completion
- 31 Jul 2027 (estimated)
- First posted
- 24 Aug 2025 (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.
Aplastic anemia (AA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and hypocellular bone marrow, with immune-mediated destruction of hematopoietic stem and progenitor cells (HSPCs) playing a central role in its pathogenesis. Although immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT) have improved survival, a significant proportion of patients remain refractory, relapse after treatment, or lack suitable donors for transplantation. Therefore, novel therapeutic strategies are urgently needed.
Chimeric antigen receptor T (CAR-T) cell therapy has demonstrated remarkable efficacy in hematologic malignancies. CD7 is an early surface marker of T-lineage cells and is dispensable for T cell development and function, making it a promising therapeutic target. This exploratory study aims to investigate the molecular and cellular mechanisms of CD7-CAR-T therapy in AA patients by analyzing multi-omics changes before and after treatment.
This is a prospective, single-center, single-arm, open-label study enrolling patients with relapsed or refractory severe aplastic anemia. Participants will receive autologous CD7-CAR-T cells following lymphodepletion. Multi-omics profiling, including genomics, transcriptomics, proteomics, and immunophenotyping, will be performed on patient samples before and after infusion. The primary objective is to explore dynamic molecular changes associated with treatment response and disease progression. Secondary objectives include safety evaluation and preliminary assessment of efficacy.
Findings from this study may provide mechanistic insights into CD7-CAR-T therapy in AA and inform the development of innovative immunotherapies for bone marrow failure syndromes.
Detailed description
This is a single-center, single-arm, prospective clinical study evaluating the safety and efficacy of CD7-directed CAR-T cell therapy in adult patients with refractory or relapsed severe aplastic anemia (SAA). SAA is an acquired bone marrow failure disorder characterized by pancytopenia and hypocellular marrow without malignant infiltration. Standard immunosuppressive therapy (IST) has improved survival, but approximately 30% of patients fail initial treatment, and relapse or progression to myelodysplastic syndrome or acute myeloid leukemia remains a clinical challenge. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) improves outcomes for younger patients but is limited by donor availability and graft-versus-host disease risks.
Eligible participants are adults (≥18 years) with ECOG performance status 0-1, life expectancy ≥3 months, and refractory or relapsed SAA after IST, or ineligible for, lacking access to, or refusing allo-HSCT. Key laboratory criteria include creatinine ≤2.5× upper limit of normal, total bilirubin ≤3× ULN, ALT and AST ≤3× ULN, and baseline oxygen saturation ≥90%. Patients must have suitable venous access for leukapheresis and comply with contraception requirements if of reproductive potential. Exclusion criteria include active infections, severe cardiovascular disease, recent malignancy (except certain adequately treated cancers), pregnancy or breastfeeding, HIV, HBV, HCV above specified thresholds, or hypersensitivity to CAR-T components.
The study consists of four stages: patient screening and enrollment; preconditioning including leukapheresis, CAR-T cell manufacture, and chemotherapy; CD7-CAR-T cell infusion with inpatient monitoring; and long-term follow-up. Patients receive a lymphodepletion regimen with fludarabine and cyclophosphamide prior to a single infusion of CD7-CAR-T cells at 3×10^6 cells/kg. Safety assessments, including monitoring for cytokine release syndrome and other adverse events, will be conducted closely during hospitalization. Efficacy assessments include complete blood counts, bone marrow evaluation, and multi-omics analysis of immune cell subsets and cytokine profiles at defined intervals.
Participants will be followed up for up to 3 years post-infusion, with intensive monitoring in the first 6 months and periodic assessments thereafter to evaluate disease status, CAR-T cell persistence, long-term safety, and quality of life. The primary endpoint is overall response rate (ORR), with secondary endpoints including progression-free survival (PFS), overall survival (OS), and safety outcomes. This study aims to provide the first evaluation of CD7-CAR-T therapy in refractory or relapsed SAA patients, potentially offering a novel therapeutic option for this high-risk population.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Adult patients (≥18 years) with refractory or relapsed severe aplastic anemia (SAA) after standard immunosuppressive therapy, or ineligible for/allogeneic hematopoietic stem cell transplantation. Participants must have ECOG 0-1, life expectancy ≥3 months, suitable venous access for leukapheresis, and meet basic lab criteria. Key exclusions include active infections, severe cardiovascular disease, recent malignancy, pregnancy/breastfeeding, or known HIV, HBV, or HCV infection.
Key inclusion criteria (9 listed)
- Age ≥18 years at the time of informed consent, of Chinese nationality. ECOG performance status 0-1, with an expected survival time ≥3 months.
- Relapsed or refractory after standard therapy without achieving complete remission.
- Ineligible for, without access to, or refusing allogeneic hematopoietic stem cell transplantation.
- Serum creatinine ≤2.5 × upper limit of normal (ULN). Oxygen saturation ≥90% at baseline. Total bilirubin ≤3 × ULN. ALT and AST ≤3 × ULN. Adequate venous access for mononuclear cell collection and no contraindications to leukapheresis.
- Female participants of childbearing potential must have a negative high-sensitivity serum pregnancy test (β-hCG) at screening and before the first dose of fludarabine/cyclophosphamide. Male and female participants of childbearing potential must agree to use effective contraception from informed consent through at least 36 months after CD7-CAR-T infusion.
Key exclusion criteria (8 listed)
- History of or treatment for other malignancies within 5 years prior to screening, except adequately treated cervical carcinoma in situ, basal or squamous cell skin cancer, curatively treated localized prostate cancer, or ductal carcinoma in situ.
- NYHA class III or IV congestive heart failure. Stroke, myocardial infarction, or hemodynamically unstable arrhythmia within 6 months prior to consent.
- Left ventricular ejection fraction (LVEF) <50% by echocardiography. Severe or uncontrolled concomitant illness within 14 days prior to consent, including active infection.
- Pregnant or breastfeeding women. Positive serology for HIV. Positive for hepatitis B surface antigen or detectable HBV DNA. Positive for hepatitis C antibody with detectable HCV RNA. Positive syphilis serology (TP-Ab and RPR). Positive CMV DNA.
- History of life-threatening hypersensitivity to CD7-CAR-T cells or any excipients (including DMSO), or known hypersensitivity to biologics such as antibodies or cytokines.
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 |
|---|---|---|
| CD7 CAR-T cells infusionAutologous T cells are collected by leukapheresis, activated, and transduced with a retroviral vector encoding a CD7-specific CAR. Following fludarabine/cyclophosphamide lymphodepletion, CD7-CAR-T cells (3 × 10^6/kg) are infused intravenously. This intervention is distinguished by its CD7 target, aiming to suppress aberrant T-cell-mediated marrow destruction and restore hematopoiesis in aplastic anemia. | Biological | – |
Primary outcome
Objective Response Rate (ORR)
Time frame: Up to 36 months post-infusion.
The proportion of patients achieving Complete Response (CR) or Partial Response (PR)
Progression-Free Survival (PFS)
Time frame: Up to 36 months post-infusion.
Time from CAR-T cell infusion to disease progression, relapse, or death from any cause, whichever occurs first.
Overall Survival (OS)
Time frame: Up to 36 months post-infusion.
Time from CAR-T cell infusion to death from any cause.
Event-Free Survival (EFS)
Time frame: Up to 36 months post-infusion.
Time from CAR-T cell infusion to the occurrence of any of the following events: failure to achieve response, disease progression or relapse, initiation of alternative therapy, or death from any cause.
1 secondary outcome
Safety and Tolerability
Time frame: From infusion through study completion (up to 36 months).
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 Aug 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. This study is not recruiting at the moment.
Hai Cheng study director
The Affiliated Hospital oh Xuzhou Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Aplastic Anaemia and CAR-T, run by Xuzhou Medical University 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 9 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 24 Aug 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: Up to 36 months post-infusion.). The study lists 4 primary outcomes in all. 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 Aug 2025.