Actively Recruiting

Early Phase 1
All Genders
ID06633328

Clinical Study on the Safety and Efficacy of Donor Derived CD7 CAR-T Cell Bridging Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

Led by Zhejiang University · Updated on 2024-10-09

30

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

Sponsors

Z

Zhejiang University

Lead Sponsor

Y

Yake Biotechnology Ltd.

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of CD7 CAR-T cell therapy followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with severe aplastic anemia. This early phase 1, open-label study aims to understand the dose-limiting toxicities and adverse events related to this treatment approach, which is designed for patients who are not suitable or unwilling to undergo traditional stem cell transplantation. The trial is conducted at a single center and sponsored by Zhejiang University. Participants will receive CD7 CAR-T cells as a novel treatment, followed by allo-HSCT as a bridging therapy. The study focuses on patients with severe aplastic anemia meeting specific diagnostic criteria and having suitable donors. The treatment and follow-up periods include monitoring for toxicities up to 28 days after treatment and adverse events up to two years post-treatment. Secondary goals include evaluating transplant success, blood cell recovery times, disease-free survival, and overall survival. Throughout the study, participants will undergo various assessments including blood tests, echocardiograms, and evaluations of organ function. Researchers will track treatment-emergent adverse events, transplant implantation success, and survival outcomes over a two-year period. Patients must meet performance status and organ function requirements and agree to contraception use if of childbearing potential. Participation includes regular monitoring and safety follow-up to evaluate the therapy's impact and safety profile.

CONDITIONS

Brief Title

CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of severe aplastic anemia based on Chinese expert consensus (2017) with bone marrow proliferation less than 25% or 25%-50% with less than 30% residual hematopoietic cells
  • Pancytopenia with at least two of these: absolute neutrophil count <0.5 x 10^9/L; platelet count <20 x 10^9/L; reticulocyte count <20 x 10^9/L
  • Suitable donors (relatives) available for at least haploidentical allogeneic hematopoietic stem cell transplantation
  • Not suitable or unwilling to undergo traditional allogeneic stem cell transplantation
  • Creatinine clearance greater than 60 ml/min; liver function within specified limits including total bilirubin ≤1.5 times upper limit of normal
  • Left ventricular ejection fraction ≥50% by echocardiogram
  • No active lung infection; blood oxygen saturation ≥92% on room air
  • Estimated survival time of at least 3 months
  • ECOG performance status of 0 to 1
  • Females and males of childbearing potential agree to use adequate contraception prior to and during the study and for 6 months after infusion
  • Voluntary participation with informed consent provided
Not Eligible

You will not qualify if you...

  • Allergy to pre-treatment measures
  • Acute graft versus host disease (GvHD) or moderate to severe chronic GvHD within 4 weeks before screening
  • Systemic drug therapy for GvHD within 4 weeks before reinfusion
  • History of epilepsy or other central nervous system disorders
  • Electrocardiogram showing prolonged QT interval or severe heart disease such as severe arrhythmia
  • Less than 100 days since previous allogeneic hematopoietic stem cell transplantation
  • HIV infection, active hepatitis B or C infection, or uncured active infection
  • Proliferation rate less than 5 times response to CD3/CD28 co-stimulation
  • Anti-cancer chemotherapy or other drug treatment within 2 weeks prior to screening
  • Any condition that may increase risk or interfere with study results as judged by the investigator

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

screening and enrollment visit

Treatment

Duration - Up to 28 days after Treatment

Participants receive CD7 CAR-T cell injection followed by allogeneic hematopoietic stem cell transplantation as a bridge therapy for severe aplastic anemia.

1 baseline visit and multiple visits during treatment

Follow-up

Duration - Up to 2 years after Treatment

Participants are monitored for treatment-emergent adverse events and treatment outcomes including engraftment and survival.

Regular visits up to 2 years after treatment

Trial Site Locations

Total: 1 location

1

The First Affiliated Hospital of Zhejiang University School of Medicine

Hanzhou, Zhejiang, China, 310003

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Research Team

H

He Z Huang, MD

Y

Yongxian Hu, MD

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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