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Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

NCT02559830 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2022-05-31

No results posted yet for this study

Summary

Evaluating the safety and efficacy of Lentiviral Hematopoietic Stem Cell Gene Therapy for advanced stage of Metachromatic Leukodystrophy and adrenoleukodystrophy.

Conditions

  • Metachromatic Leukodystrophy
  • Adrenoleukodystrophy

Interventions

GENETIC

transduced CD34+ hematopoietic stem cell

Autologous hematopoietic stem cells (HSCs) collected from the mobilized peripheral blood and transduced ex vivo with a Lentiviral vector encoding the human ARSA(for MLD)/ABCD1(for ALD) cDNA（complementary DNA）. Dose: ≥ 2x10\^6 transduced CD34+ cells/Kg (maximum 20x10\^6) at bedside for infusion.

Sponsors & Collaborators

  • Shenzhen University

    collaborator OTHER

  • Guangzhou Women and Children's Medical Center

    collaborator OTHER

  • Shenzhen Second People's Hospital

    lead OTHER

Principal Investigators

  • Qizhou Lian, M.D.,Ph.D. · The University of Hong Kong

  • Jiacai Zhuo · Shenzhen Second People's Hospital

  • Xin Du, M.D.,Ph.D. · Shenzhen Second People's Hospital

  • Hua Jiang, M.D,Ph.D · Guangzhou Women and Children's Medical Center

  • GuangFu Chen, M.D.,Ph.D · Shenzhen Second People's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-01-31
Primary Completion
2025-10-31
Completion
2025-10-31

Countries

  • China

Study Locations

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Entities

Companies

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 NCT02559830 on ClinicalTrials.gov