Bone Marrow Failure SyndromeEarly Phase 1NCT06787560
CD7 CAR-T Cell Sequential Allo-HSCT for Non-malignant Blood and Immune System Diseases
A Early Phase 1 study testing CD7 CAR-T cells injection and Allo-HSCT in people with Bone Marrow Failure Syndrome, run by Zhejiang University at 1 site in China.
Trial summary
A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Bone Marrow Failure Syndrome
- What is being tested?
- CD7 CAR-T cells injection and Allo-HSCT
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 31 Jan 2025to 31 Jan 2028
- Phase
- Early 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Non-malignant blood and immune system diseases include: hereditary bone marrow failure, congenital immune deficiency, hemoglobinopathy and other non-malignant blood and immune system diseases,
- Confirmed hereditary bone marrow failure syndrome. Including: Fanconi anemia, congenital pure red cell aplastic anemia, congenital dyskeratosis, Scheux-Day syndrome, congenital neutropenia, various bone marrow failure related congenital thrombocytopenia and other unclassified congenital bone marrow exhaustion diseases;
- It meets the criteria of clinical manifestation, immune function and gene diagnosis of immune deficiency disease;
- Diagnosed with hemoglobinopathy and dependent on blood transfusions; serum ferritin levels are < 3000 μg/L, with cardiac and hepatic iron content indicating moderate or lower iron overload; documentation of iron chelation therapy (including prescriptions or invoices) for at least three months prior to screening is available; no hydroxyurea, ruxolitinib, decitabine, or cytarabine has been administered in the three months preceding enrollment. The spleen size must not extend beyond the umbilical horizontal line or the midline of the abdomen. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is indicated, and suitable donors for related allo-HSCT are available.
- 2. Serum total bilirubin ≤1.5 times the upper limit of normal value, serum Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 3 times the upper limit of normal value range;
- 3. Echocardiography showed Left ventricular ejection fraction (LVEF) >50%;
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- 4. Pulse oxygen saturation ≥92% (non-oxygen state);
- 5. The estimated survival is more than 3 months;
- 6. ECOG score 0-1;
- 7. Abdominal B-ultrasonography and other examinations were performed to evaluate spleen size. Splenectomy should be evaluated before transplantation for patients with giant spleen;
- 8. Women and men who are fertile must consent to the use of appropriate contraception before entering the study, during study participation, and for 6 months after transfusion (the safety of this therapy for the unborn child is not known, with unknown risks);
- 9. Subjects who are willing to participate in the study are able to understand and have the ability to sign informed consent.
Who cannot join
- 1. People with a history of epilepsy or other central nervous system disorders;
- 2. Epstein-Barr virus (EBV) DNA positive;
- 3. People with a history of prolonged QT interval or serious heart disease;
- 4. People with active hepatitis B or C virus;
- 5. Tuberculosis, AIDS and other major infectious diseases;
- 6. Sepsis, pulmonary infection, intestinal infection and other major organ infection and poor control, and/or hypersensitive C-reactive protein, procalcitonin significantly elevated;
- 7. People who have previously received other clinical studies and gene therapy;
- 8. Any situation that the investigator believes may increase the risk to the subject or interfere with the test 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
The first affiliated hospital of medical college of zhejiang university
Hangzhou, Zhejiang, 310003
He Huang, MD, 0571-87233772, hehuangyu@126.com
Yongxian Hu, MD, 0571-87233772, huyongxian2000@aliyun.com
About this study
Official title: Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases
A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases
Read the detailed description
This is a single-arm, open-label clinical trial to evaluate the safety and efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases. It is planned to enroll 12-20 participants in this trial.
- Conditions
- Bone Marrow Failure Syndrome
- Being tested
- CD7 CAR-T cells injection, Allo-HSCT
- Keywords
- CAR-T, Allo-HSCT, Immuno Deficiency Disorders
- Medical subject headings
- Bone Marrow Failure Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD7 CAR-T cells injectionBiological
Each subject receive CD7 CAR T-cells by intravenous infusion
Allo-HSCTProcedure or surgery
allogeneic hematopoietic stem cell transplantation
| Study group | Role | What they receive |
|---|---|---|
| CD7 CAR-T cells（ CD7 chimeric antigen receptor T cells） | Experimental | Patients or donors undergo leukapheresis. Patients will receive a lymphodepletion chemotherapy with CTX、Flu、VP-16 before CAR-T cells infusion. CAR-T cells could be transfused after 48 hours of preconditioning. Receives: Biological: CD7 CAR-T cells injection; Procedure: Allo-HSCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events
Measured: Up to 2 years after Treatment
Transplant related mortality rate
The proportion of patients who died after transplantation to the total number of transplant patients during the same period
Measured: Up to 100 days after Treatment
Secondary outcomes (4)
Allogeneic hematopoietic stem cell transplant implantation rate
The proportion of the number of patients who achieved hematopoietic reconstitution to the total number of allogeneic hematopoietic stem cell transplantation patients in the same period.
Measured: Up to 100 days after Treatment
Time to neutrophil and platelet engraftment
The time for neutrophils and platelets to reach the implantation criteria after stem cell reinfusion
Measured: Up to 30 days after Treatment
Disease-feesurvival，DFS
The proportion of disease-free patients who survived to the total number of patients who transplanted allogeneic hematopoietic stem cells during the same period.
Measured: Up to 2 years after Treatment
Overall survival, OS
After transplantation until death from any cause.
Measured: Up to 2 years after Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2025
- Study start31 Jan 2025 (estimated)
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bone Marrow Failure SyndromeStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Responsible party
- Principal investigator, He Huang, First Affiliated Hospital of Zhejiang University
- Investigators
- He Huang, MD, Principal investigator, Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06787560
- Sponsor's protocol ID
- TXB2024023
- Status last verified
- Jan 2025
- First submitted
- 16 Jan 2025
- Last updated
- 22 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases
- ClinicalTrials.gov ID
- NCT06787560
- Acronym / sponsor ID
- TXB2024023
- Conditions
- Bone Marrow Failure Syndrome
- Keywords
- CAR-T, Allo-HSCT, Immuno Deficiency Disorders
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Study start
- 31 Jan 2025 (estimated)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Jan 2028 (estimated)
- First posted
- 22 Jan 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.
A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases
Detailed description
This is a single-arm, open-label clinical trial to evaluate the safety and efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases. It is planned to enroll 12-20 participants in this trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD7 CAR-T cells（ CD7 chimeric antigen receptor T cells） | Experimental | Biological: CD7 CAR-T cells injection, Procedure: Allo-HSCTPatients or donors undergo leukapheresis. Patients will receive a lymphodepletion chemotherapy with CTX、Flu、VP-16 before CAR-T cells infusion. CAR-T cells could be transfused after 48 hours of preconditioning. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- 1. Non-malignant blood and immune system diseases include: hereditary bone marrow failure, congenital immune deficiency, hemoglobinopathy and other non-malignant blood and immune system diseases,
- 2. Serum total bilirubin ≤1.5 times the upper limit of normal value, serum Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 3 times the upper limit of normal value range;
- 3. Echocardiography showed Left ventricular ejection fraction (LVEF) >50%;
- 4. Pulse oxygen saturation ≥92% (non-oxygen state);
- 5. The estimated survival is more than 3 months;
Key exclusion criteria (8 listed)
- 1. People with a history of epilepsy or other central nervous system disorders;
- 2. Epstein-Barr virus (EBV) DNA positive;
- 3. People with a history of prolonged QT interval or serious heart disease;
- 4. People with active hepatitis B or C virus;
- 5. Tuberculosis, AIDS and other major infectious diseases;
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 injectionEach subject receive CD7 CAR T-cells by intravenous infusion | Biological | CD7 CAR-T cells（ CD7 chimeric antigen receptor T cells） |
| Allo-HSCTallogeneic hematopoietic stem cell transplantation | Procedure or surgery | CD7 CAR-T cells（ CD7 chimeric antigen receptor T cells） |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Up to 2 years after Treatment
Incidence of treatment-emergent adverse events
Transplant related mortality rate
Time frame: Up to 100 days after Treatment
The proportion of patients who died after transplantation to the total number of transplant patients during the same period
4 secondary outcomes
Allogeneic hematopoietic stem cell transplant implantation rate
Time frame: Up to 100 days after Treatment
Time to neutrophil and platelet engraftment
Time frame: Up to 30 days after Treatment
Disease-feesurvival，DFS
Time frame: Up to 2 years after Treatment
Overall survival, OS
Time frame: Up to 2 years after Treatment
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 22 Jan 2025.
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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.
He Huang, MD principal investigator
Zhejiang University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CD7 CAR-T cells injection and Allo-HSCT in people with Bone Marrow Failure Syndrome, run by Zhejiang 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 12 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: looking for participants now, as of the sponsor's last update on 22 Jan 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 He Huang, MD (0571-87233772, hehuangyu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) (time frame: Up to 2 years after Treatment). The study lists 2 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 22 Jan 2025.