Actively Recruiting
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
Led by Zhejiang University · Updated on 2025-01-22
20
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 studying the safety and effectiveness of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for people with non-malignant blood and immune system diseases. These conditions include hereditary bone marrow failure, congenital immune deficiencies, hemoglobinopathy, and other related diseases. This early phase 1 clinical trial aims to evaluate this treatment approach to improve care for these patients. Participants will undergo leukapheresis to collect cells from patients or donors. Before receiving the CD7 CAR-T cell infusion, patients will have lymphodepletion chemotherapy with drugs called CTX, Flu, and VP-16. The CAR-T cells are given intravenously about 48 hours after this preconditioning. The treatment combines the CAR-T cells infusion followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). During the study, participants will be monitored for safety and effectiveness up to two years after treatment. Assessments include tracking adverse events, transplant-related mortality within 100 days, engraftment of stem cells, and survival outcomes. Various tests such as echocardiography, blood tests, and ultrasonography will be done, along with clinical evaluations and informed consent processes. The study plans to enroll 12 to 20 participants and includes ongoing follow-up to understand treatment impact.
CONDITIONS
Brief Title
CD7 CAR-T Cell Sequential Allo-HSCT for Non-malignant Blood and Immune System Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with non-malignant blood and immune system diseases such as hereditary bone marrow failure syndrome, congenital immune deficiency, or hemoglobinopathy
- Serum total bilirubin less than or equal to 1.5 times the upper limit of normal
- Serum ALT and AST less than or equal to 3 times the upper limit of normal
- Left ventricular ejection fraction greater than 50% by echocardiography
- Pulse oxygen saturation 92% or higher without oxygen support
- Estimated survival time more than 3 months
- ECOG score between 0 and 1
- Evaluation of spleen size by abdominal ultrasound; splenectomy considered for giant spleen
- Willingness to use contraception during the study and for 6 months after treatment
- Ability to understand and sign informed consent
You will not qualify if you...
- History of epilepsy or other central nervous system disorders
- Epstein-Barr virus DNA positive
- History of prolonged QT interval or serious heart disease
- Active hepatitis B or C infection
- Tuberculosis, AIDS, or other major infectious diseases
- Sepsis, pulmonary or intestinal infection, or other major uncontrolled organ infections
- Previous participation in other clinical studies or gene therapy
- Any condition increasing risk or interfering with study results as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 100 days
Participants receive lymphodepletion chemotherapy followed by CD7 CAR-T cell infusion and then undergo allogeneic hematopoietic stem cell transplantation.
Multiple visits during chemotherapy, CAR-T infusion, and transplant period
Duration - Up to 2 years after treatment
Participants are monitored for safety and efficacy outcomes including adverse events, engraftment, survival, and other clinical assessments.
Regular follow-up visits up to 2 years
Trial Site Locations
Total: 1 location
1
The first affiliated hospital of medical college of zhejiang university
Hangzhou, Zhejiang, China, 310003
Actively Recruiting
Research Team
H
He 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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