Clinical Trials/NCT06633328

NCT06633328

Recruiting

Early Phase 1

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

Zhejiang University1 site in 1 country30 target enrollmentOctober 20, 2024

ConditionsAplastic Anemia

InterventionsCD7 CAR-T cells injection Allogeneic hematopoietic stem cell transplantation

DrugsCD7 CAR-T cells injection

Overview

Phase: Early Phase 1
Intervention: CD7 CAR-T cells injection
Conditions: Aplastic Anemia
Sponsor: Zhejiang University
Enrollment: 30
Locations: 1
Primary Endpoint: Dose-limiting toxicity (DLT)
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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).

Registry

clinicaltrials.gov

Start Date

October 20, 2024

End Date

October 20, 2027

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Zhejiang University

Responsible Party

Principal Investigator

He Huang

Clinical Professor

Zhejiang University

Eligibility Criteria

Inclusion Criteria

•Chinese expert consensus on the diagnosis and treatment of aplastic anemia（2017）, Diagnosis of severe aplastic anemia ，
•The degree of bone marrow cell proliferation \< 25%, or 25%-50% but residual hematopoietic cells \< 30%；
•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;

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.

Arms & Interventions

Treatment Group

Aplastic Anemia

Intervention: CD7 CAR-T cells injection

Treatment Group

Aplastic Anemia

Intervention: Allogeneic hematopoietic stem cell transplantation

Outcomes

Primary Outcomes

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 \[Safety and Tolerability\]

Secondary Outcomes

Allogeneic hematopoietic stem cell transplant implantation rate(Up to 100 days after Treatment)
Time to neutrophil and platelet engraftment(Up to 30 days after Treatment)
Disease-feesurvival，DFS(Up to 2 years after Treatment)
Overall survival, OS(Up to 2 years after Treatment)