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Gene Therapy Trial Browser
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
Evaluating the safety and efficacy of Lentiviral Hematopoietic Stem Cell Gene Therapy for advanced stage of Metachromatic Leukodystrophy and adrenoleukodystrophy.
(Show More)
Indication
Metachromatic Leukodystrophy, Adrenoleukodystrophy
Compound Name
CD34+ hematopoietic stem cells transduced with ABCD1
Sponsor
Shenzhen Second People's Hospital
Funder Type
Other
Status
Recruiting
Enrollment Count
50
Therapy Information
Target Gene/Variant
ABCD1
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
Eligible Sex
ALL
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
Recent Updates
Resources/Links
Resources/Links
No External Links Available.