Aplastic AnemiaEarly Phase 1NCT06633328
CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia
A Early Phase 1 study testing CD7 CAR-T cells injection and Allogeneic hematopoietic stem cell transplantation in people with Aplastic Anemia, run by Zhejiang University at 1 site in China.
Trial summary
This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with severe aplastic anemia Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- CD7 CAR-T cells injection and Allogeneic hematopoietic stem cell transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 20 Oct 2024to 20 Oct 2027
- 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
- Chinese expert consensus on the diagnosis and treatment of aplastic anemia（2017）, Diagnosis of severe aplastic anemia ，1. The degree of bone marrow cell proliferation < 25%, or 25%-50% but residual hematopoietic cells < 30%；2. With pancytopenia (at least two of the following peripheral blood parameters) : (1) absolute neutrophil <0.5×10^9/L; (2) Platelet count< 20×10^9/L; (3) The absolute value of reticulocytes <20×109/L;
- Suitable donors (relatives) with allogeneic HSCT indications and at least haploid allogeneic transplantation;
- Patients who are not suitable or unwilling to undergo traditional allogeneic hematopoietic stem cell transplantation;
- creatinine clearance > 60ml/min; without liver invasion, serum total bilirubin ≤ 1.5 times the upper limit of normal, and serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were both ≤ 3 times the upper limit of the normal range. If there is liver invasion, serum erythrambirubin ≤ 3 times the upper limit of normal, and serum ALT and AST are both ≤ 5 times the upper limit of the normal range;
- Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;
- No active infection in the lungs, blood oxygen saturation in indoor air is ≥ 92%;
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- Estimated survival time ≥ 3 months;
- ECOG performance status 0 to 1;
- Females and males of childbearing potential must agree to use adequate contraception prior to study entry, during study participation, and for 6 months after infusion (the safety of this therapy for unborn children is not known and has unknown risks);
- Those who voluntarily participated in this trial and provided informed consent;
Who cannot join
- Allergy to pre-treatment measures；
- Those with acute graft versus host disease (GvHD) or moderate to severe chronic GvHD within 4 weeks before screening; Those who have received systemic drug therapy for GvHD within 4 weeks before the reinfusion；
- History of epilepsy or other central nervous system disorders；
- Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythmia in the past;
- Less than 100 days after allogeneic hematopoietic stem cell transplantation；
- Patients with HIV infection，Active infection of hepatitis B virus or hepatitis C virus，Uncured active infection；
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- The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- Received anti-cancer chemotherapy or other drug treatment within 2 weeks prior to screening;
- Any condition that, in the opinion of the investigator, may increase the risk to the subject or interfere with the results of the study.
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 Zhejiang University School of Medicine
Hanzhou, 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 Donor Derived CD7 CAR-T Cell Bridging Allogeneic Hematopoietic Stem Cell Transplantation for the for Patients With Severe Aplastic Anemia
This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with severe aplastic anemia
Read the detailed description
This is a prospective, open-label, single-center clinical trial. This study will evaluate the safety and efficacy of CD7 CAR-T Bridging to allo-HSCT in the treatment of severe aplastic anemia.The primary endpoints are dose limiting toxicity (DLT) and the incidence of treatment emergent adverse event (TEAE).
- Conditions
- Aplastic Anemia
- Being tested
- CD7 CAR-T cells injection, Allogeneic hematopoietic stem cell transplantation
- Keywords
- allo-HSCT, CD7 CAR-T
- 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 injectionDrug
CD7 CAR T cells treat patients with severe aplastic anemia
Allogeneic hematopoietic stem cell transplantationOther
In this study, Allogeneic hematopoietic stem cell transplantation is used as a bridge therapy to CD7 CAR T cells infusion to treat patients with severe aplastic anemia
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | Aplastic Anemia Receives: Drug: CD7 CAR-T cells injection; Other: Allogeneic hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-limiting toxicity (DLT)
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Measured: Up to 28 days after Treatment
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events [Safety and Tolerability]
Measured: Up to 2 years 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 posted9 Oct 2024
- Study start20 Oct 2024 (estimated)
- Primary completion20 Oct 2027 (estimated)
- Study completion20 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Zhejiang UniversityThe sponsor's other registered studies.
- 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
- 30 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, Zhejiang University
- Investigators
- He Huang, MD, Principal investigator, Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06633328
- Sponsor's protocol ID
- TXB2024009
- Status last verified
- Aug 2024
- First submitted
- 8 Oct 2024
- Last updated
- 9 Oct 2024
- 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 9 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on the Safety and Efficacy of Donor Derived CD7 CAR-T Cell Bridging Allogeneic Hematopoietic Stem Cell Transplantation for the for Patients With Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT06633328
- Acronym / sponsor ID
- TXB2024009
- Conditions
- Aplastic Anemia
- Keywords
- allo-HSCT, CD7 CAR-T
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Study start
- 20 Oct 2024 (estimated)
- Primary completion
- 20 Oct 2027 (estimated)
- Study completion
- 20 Oct 2027 (estimated)
- First posted
- 9 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 is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with severe aplastic anemia
Detailed description
This is a prospective, open-label, single-center clinical trial. This study will evaluate the safety and efficacy of CD7 CAR-T Bridging to allo-HSCT in the treatment of severe aplastic anemia.The primary endpoints are dose limiting toxicity (DLT) and the incidence of treatment emergent adverse event (TEAE).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Drug: CD7 CAR-T cells injection, Other: Allogeneic hematopoietic stem cell transplantationAplastic Anemia |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Chinese expert consensus on the diagnosis and treatment of aplastic anemia（2017）, Diagnosis of severe aplastic anemia ，1. The degree of bone marrow cell proliferation < 25%, or 25%-50% but residual hematopoietic cells < 30%；2. With pancytopenia (at least two of the following peripheral blood parameters) : (1) absolute neutrophil <0.5×10^9/L; (2) Platelet count< 20×10^9/L; (3) The absolute value of reticulocytes <20×109/L;
- Suitable donors (relatives) with allogeneic HSCT indications and at least haploid allogeneic transplantation;
- Patients who are not suitable or unwilling to undergo traditional allogeneic hematopoietic stem cell transplantation;
- creatinine clearance > 60ml/min; without liver invasion, serum total bilirubin ≤ 1.5 times the upper limit of normal, and serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were both ≤ 3 times the upper limit of the normal range. If there is liver invasion, serum erythrambirubin ≤ 3 times the upper limit of normal, and serum ALT and AST are both ≤ 5 times the upper limit of the normal range;
- Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;
Key exclusion criteria (9 listed)
- Allergy to pre-treatment measures；
- Those with acute graft versus host disease (GvHD) or moderate to severe chronic GvHD within 4 weeks before screening; Those who have received systemic drug therapy for GvHD within 4 weeks before the reinfusion；
- History of epilepsy or other central nervous system disorders；
- Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythmia in the past;
- Less than 100 days after allogeneic hematopoietic stem cell transplantation；
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 injectionCD7 CAR T cells treat patients with severe aplastic anemia | Drug | Treatment Group |
| Allogeneic hematopoietic stem cell transplantationIn this study, Allogeneic hematopoietic stem cell transplantation is used as a bridge therapy to CD7 CAR T cells infusion to treat patients with severe aplastic anemia | Other | Treatment Group |
Primary outcome
Dose-limiting toxicity (DLT)
Time frame: Up to 28 days after Treatment
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Up to 2 years after Treatment
Incidence of treatment-emergent adverse events [Safety and Tolerability]
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 9 Oct 2024.
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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 Allogeneic hematopoietic stem cell transplantation in people with Aplastic Anemia, 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 10 inclusion and 9 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 9 Oct 2024. 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 Z Huang, MD (13605714822, hehuangyu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose-limiting toxicity (DLT) (time frame: Up to 28 days 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 9 Oct 2024.