CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia

Early Phase 1

Recruiting

• Conditions: Aplastic Anemia
• Interventions: Drug: CD7 CAR-T cells injection; Other: Allogeneic hematopoietic stem cell transplantation

• Registration Number: NCT06633328
• Lead Sponsor: Zhejiang University

Brief 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

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).

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Status Verified: 2024-08
• Study First Submitted: 2024-10-08
• Study First Submitted QC: 2024-10-08
• Last Update Submitted: 2024-10-08
• Study First Posted: 2024-10-09
• Last Update Posted: 2024-10-09
• Study Start: 2024-10-20
• Primary Completion: 2027-10-20
• Study Completion: 2027-10-20

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• 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%;
• 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;

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Exclusion Criteria

• 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；
• 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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment Group	CD7 CAR-T cells injection	Aplastic Anemia
Treatment Group	Allogeneic hematopoietic stem cell transplantation	Aplastic Anemia

Primary Outcome Measures

Name	Time	Method
Dose-limiting toxicity (DLT)	Up to 28 days after Treatment	Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of treatment-emergent adverse events (TEAEs)	Up to 2 years after Treatment	Incidence of treatment-emergent adverse events \[Safety and Tolerability\]

Secondary Outcome Measures

Name	Time	Method
Allogeneic hematopoietic stem cell transplant implantation rate	Up to 100 days after Treatment	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.
Time to neutrophil and platelet engraftment	Up to 30 days after Treatment	The time for neutrophils and platelets to reach the implantation criteria after stem cell reinfusion
Disease-feesurvival，DFS	Up to 2 years after Treatment	The proportion of disease-free patients who survived to the total number of patients who transplanted allogeneic hematopoietic stem cells during the same period.
Overall survival, OS	Up to 2 years after Treatment	After transplantation until death from any cause.

Trial Locations

Locations (1)

The First Affiliated Hospital of Zhejiang University School of Medicine; 🇨🇳 Hanzhou, Zhejiang, China