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
Delivery System 🔄	Viral transduction
Vector Type 🔄	LV
Dose 1 🔄	1-2E9 MOI/ml

No.of Trial Sites	1
Locations	China