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Multi-omics Profiling of Patients With Aplastic Anemia Before and After CD7-CAR-T Therapy

NCT07139600 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 5

Last updated 2025-08-24

No results posted yet for this study

Summary

Aplastic anemia (AA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and hypocellular bone marrow, with immune-mediated destruction of hematopoietic stem and progenitor cells (HSPCs) playing a central role in its pathogenesis. Although immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT) have improved survival, a significant proportion of patients remain refractory, relapse after treatment, or lack suitable donors for transplantation. Therefore, novel therapeutic strategies are urgently needed.

Chimeric antigen receptor T (CAR-T) cell therapy has demonstrated remarkable efficacy in hematologic malignancies. CD7 is an early surface marker of T-lineage cells and is dispensable for T cell development and function, making it a promising therapeutic target. This exploratory study aims to investigate the molecular and cellular mechanisms of CD7-CAR-T therapy in AA patients by analyzing multi-omics changes before and after treatment.

This is a prospective, single-center, single-arm, open-label study enrolling patients with relapsed or refractory severe aplastic anemia. Participants will receive autologous CD7-CAR-T cells following lymphodepletion. Multi-omics profiling, including genomics, transcriptomics, proteomics, and immunophenotyping, will be performed on patient samples before and after infusion. The primary objective is to explore dynamic molecular changes associated with treatment response and disease progression. Secondary objectives include safety evaluation and preliminary assessment of efficacy.

Findings from this study may provide mechanistic insights into CD7-CAR-T therapy in AA and inform the development of innovative immunotherapies for bone marrow failure syndromes.

Conditions

  • Aplastic Anaemia
  • CAR-T
  • CD7 Positive

Interventions

BIOLOGICAL

CD7 CAR-T cells infusion

Autologous T cells are collected by leukapheresis, activated, and transduced with a retroviral vector encoding a CD7-specific CAR. Following fludarabine/cyclophosphamide lymphodepletion, CD7-CAR-T cells (3 × 10\^6/kg) are infused intravenously. This intervention is distinguished by its CD7 target, aiming to suppress aberrant T-cell-mediated marrow destruction and restore hematopoiesis in aplastic anemia.

Sponsors & Collaborators

  • Xuzhou Medical University

    lead OTHER

Principal Investigators

  • Hai Cheng · The Affiliated Hospital oh Xuzhou Medical University

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-08-01
Primary Completion
2027-07-31
Completion
2027-07-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07139600 on ClinicalTrials.gov