CD7 CAR-T Cell Sequential Allo-HSCT for Non-malignant Blood and Immune System Diseases

NCT ID: NCT06787560

Last Updated: 2025-01-22

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-01-31
• Study Completion Date: 2028-01-31

Brief Summary

A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases

Detailed Description

This is a single-arm, open-label clinical trial to evaluate the safety and efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation for Non-malignant Blood and Immune System Diseases. It is planned to enroll 12-20 participants in this trial.

Conditions

Bone Marrow Failure Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CD7 CAR-T cells（ CD7 chimeric antigen receptor T cells）

Patients or donors undergo leukapheresis. Patients will receive a lymphodepletion chemotherapy with CTX、Flu、VP-16 before CAR-T cells infusion. CAR-T cells could be transfused after 48 hours of preconditioning.

Group Type: EXPERIMENTAL

CD7 CAR-T cells injection

Intervention Type: BIOLOGICAL

Each subject receive CD7 CAR T-cells by intravenous infusion

Allo-HSCT

Intervention Type: PROCEDURE

allogeneic hematopoietic stem cell transplantation

Interventions

CD7 CAR-T cells injection

Each subject receive CD7 CAR T-cells by intravenous infusion

Intervention Type: BIOLOGICAL

Allo-HSCT

allogeneic hematopoietic stem cell transplantation

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

• 1. Non-malignant blood and immune system diseases include: hereditary bone marrow failure, congenital immune deficiency, hemoglobinopathy and other non-malignant blood and immune system diseases,

1. Confirmed hereditary bone marrow failure syndrome. Including: Fanconi anemia, congenital pure red cell aplastic anemia, congenital dyskeratosis, Scheux-Day syndrome, congenital neutropenia, various bone marrow failure related congenital thrombocytopenia and other unclassified congenital bone marrow exhaustion diseases;

2. It meets the criteria of clinical manifestation, immune function and gene diagnosis of immune deficiency disease;

3. Diagnosed with hemoglobinopathy and dependent on blood transfusions; serum ferritin levels are < 3000 μg/L, with cardiac and hepatic iron content indicating moderate or lower iron overload; documentation of iron chelation therapy (including prescriptions or invoices) for at least three months prior to screening is available; no hydroxyurea, ruxolitinib, decitabine, or cytarabine has been administered in the three months preceding enrollment. The spleen size must not extend beyond the umbilical horizontal line or the midline of the abdomen. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is indicated, and suitable donors for related allo-HSCT are available.

• 2. Serum total bilirubin ≤1.5 times the upper limit of normal value, serum Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 3 times the upper limit of normal value range;
• 3. Echocardiography showed Left ventricular ejection fraction (LVEF) >50%;
• 4. Pulse oxygen saturation ≥92% (non-oxygen state);
• 5. The estimated survival is more than 3 months;
• 6. ECOG score 0-1;
• 7. Abdominal B-ultrasonography and other examinations were performed to evaluate spleen size. Splenectomy should be evaluated before transplantation for patients with giant spleen;
• 8. Women and men who are fertile must consent to the use of appropriate contraception before entering the study, during study participation, and for 6 months after transfusion (the safety of this therapy for the unborn child is not known, with unknown risks);
• 9. Subjects who are willing to participate in the study are able to understand and have the ability to sign informed consent.

Exclusion Criteria

• 1. People with a history of epilepsy or other central nervous system disorders;
• 2. Epstein-Barr virus (EBV) DNA positive;
• 3. People with a history of prolonged QT interval or serious heart disease;
• 4. People with active hepatitis B or C virus;
• 5. Tuberculosis, AIDS and other major infectious diseases;
• 6. Sepsis, pulmonary infection, intestinal infection and other major organ infection and poor control, and/or hypersensitive C-reactive protein, procalcitonin significantly elevated;
• 7. People who have previously received other clinical studies and gene therapy;
• 8. Any situation that the investigator believes may increase the risk to the subject or interfere with the test results.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Yake Biotechnology Ltd.

INDUSTRY

Sponsor Role: collaborator

Zhejiang University

OTHER

Sponsor Role: lead

Responsible Party

He Huang

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

He Huang, MD

Role: PRINCIPAL_INVESTIGATOR

Zhejiang University

Locations

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, China

Site Status: RECRUITING

Countries

China

Central Contacts

He Huang, MD

Role: CONTACT

hehuangyu@126.com

0571-87233772

Yongxian Hu, MD

Role: CONTACT

huyongxian2000@aliyun.com

0571-87233772

Facility Contacts

He Huang, MD

Role: primary

hehuangyu@126.com

0571-87233772

Yongxian Hu, MD

Role: backup

huyongxian2000@aliyun.com

0571-87233772

Other Identifiers

TXB2024023

Identifier Type: -

Identifier Source: org_study_id