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Autologous Hematopoietic Stem Cell Transplantation for Neurological Damage Associated With Hereditary Homocysteine Remethylation Disorders

NCT07757685 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2026-08-11

No results posted yet for this study

Summary

This study aims to evaluate the safety, feasibility, and preliminary efficacy of autologous hematopoietic stem cell transplantation (ASCT) in the treatment of neurological damage associated with hereditary homocysteine remethylation disorders. Meanwhile, peripheral blood, cerebrospinal fluid, and related clinical samples will be prospectively collected before and after transplantation to dynamically monitor changes in immune reconstitution and neuroinflammatory biomarkers. The study intends to explore the impact of immune system resetting on disease progression and central nervous system immune microenvironment, providing evidence for subsequent precise patient stratification and optimized therapeutic strategies.

Conditions

  • Hereditary Homocysteine Remethylation Disorder

Interventions

OTHER

Autologous Hematopoietic Stem Cell Transplantation

ASCT Regimen 1. Autologous hematopoietic stem cell collection Mobilization with chemotherapy plus cytokines: * Cyclophosphamide: 30 mg/kg/d, administered intravenously for 2 consecutive days. * G-CSF: 5-10 μg/kg/d subcutaneous injection for 5 consecutive days. Collection target: CD34+ cells ≥ 2×10⁶/kg; repeat leukapheresis permitted to meet target dose. 2. Conditioning Regimen: * Thiotepa: 5 mg/kg/d Day -7 * Fludarabine: 5 mg/kg/d Days -6 to -2 (5 total days) * Rituximab: 375 mg/m² Day -1 3. Stem cell infusion: Day 0 Infusion dose: ≥ 2×10⁶ CD34+ cells/kg

Sponsors & Collaborators

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2026-08-01
Primary Completion
2030-07-31
Completion
2030-07-31

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 NCT07757685 on ClinicalTrials.gov