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Clinical Trial Report

Gene Therapy Trial Report

Summary

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

NCTID NCT02559830 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Metachromatic Leukodystrophy, Adrenoleukodystrophy
Disease Ontology Term DOID:10588; DOID:10581
Compound Name CD34+ hematopoietic stem cells transduced with ABCD1 or ARSA
Sponsor Shenzhen Second People's Hospital
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 50
Results Posted Not Available

Therapy Information

Target Gene/Variant ABCD1 or ARSA
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 2E6 transduced CD34+ cells/kg (maximum 20E6)
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2015-08-12
Completion Date 2025-10
Last Update 2022-05-31

Participation Criteria

Eligible Age 1 Year - 16 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations China

Regulatory Information

Has US IND
FDA Designations
Recent Updates

Resources/Links

Resources/Links

No External Links Available.