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)
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 (Click here for study contact information)
Enrollment Count	50
Results Posted	Not Available

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

No.of Trial Sites	1
Locations	China

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