IT and IV Lentiviral Gene Therapy for X-ALD

NCTID	NCT03727555 (View at clinicaltrials.gov)
Description	This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the intrathecal and intravenous lentiviral gene transfer clinical protocol. (Show More)
Development Status	Active
Indication	X-linked Adrenoleukodystrophy
Disease Ontology Term	DOID:10588
Compound Name	(LV) TYF-ABCD1
Sponsor	Shenzhen Geno-Immune Medical Institute
Funder Type	Other
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	30 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	ABCD1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intrathecal, intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	1-2E9 MOI/ml
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China