NCT06787560

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

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for early_phase_1

Timeline
22mo left

Started Jan 2025

Typical duration for early_phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress42%
Jan 2025Jan 2028

First Submitted

Initial submission to the registry

January 16, 2025

Completed
6 days until next milestone

First Posted

Study publicly available on registry

January 22, 2025

Completed
9 days until next milestone

Study Start

First participant enrolled

January 31, 2025

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 31, 2028

Last Updated

January 22, 2025

Status Verified

January 1, 2025

Enrollment Period

3 years

First QC Date

January 16, 2025

Last Update Submit

January 16, 2025

Conditions

Bone Marrow Failure Syndrome

Keywords

CAR-TAllo-HSCTImmuno Deficiency Disorders

Outcome Measures

Primary Outcomes (2)

  • Incidence of treatment-emergent adverse events (TEAEs)

    Incidence of treatment-emergent adverse events

    Up to 2 years after Treatment

  • Transplant related mortality rate

    The proportion of patients who died after transplantation to the total number of transplant patients during the same period

    Up to 100 days after Treatment

Secondary Outcomes (4)

  • 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

Study Arms (1)

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

EXPERIMENTAL

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.

Biological: CD7 CAR-T cells injectionProcedure: Allo-HSCT

Interventions

CD7 CAR-T cells injectionBIOLOGICAL

Each subject receive CD7 CAR T-cells by intravenous infusion

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

allogeneic hematopoietic stem cell transplantation

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

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • \. 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,
  • 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;
  • It meets the criteria of clinical manifestation, immune function and gene diagnosis of immune deficiency disease;
  • 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.
  • \. 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;
  • \. Echocardiography showed Left ventricular ejection fraction (LVEF) \>50%;
  • \. Pulse oxygen saturation ≥92% (non-oxygen state);
  • \. The estimated survival is more than 3 months;
  • \. ECOG score 0-1;
  • \. Abdominal B-ultrasonography and other examinations were performed to evaluate spleen size. Splenectomy should be evaluated before transplantation for patients with giant spleen;
  • \. 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);
  • \. Subjects who are willing to participate in the study are able to understand and have the ability to sign informed consent.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, 310003, China

RECRUITING

MeSH Terms

Conditions

Bone Marrow Failure Disorders

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • He Huang, MD

    Zhejiang University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

He Huang, MD

CONTACT

0571-87233772hehuangyu@126.com

Yongxian Hu, MD

CONTACT

0571-87233772huyongxian2000@aliyun.com

Study Design

Study Type
interventional
Phase
early phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

January 16, 2025

First Posted

January 22, 2025

Study Start

January 31, 2025

Primary Completion (Estimated)

January 31, 2028

Study Completion (Estimated)

January 31, 2028

Last Updated

January 22, 2025

Record last verified: 2025-01

Locations

CN(1)