Gene Therapy in Patients With Mucopolysaccharidosis Disease

NCTID	NCT03173521 (View at clinicaltrials.gov)
Description	This study investigated the safety and efficacy of gene therapy approaches for Mucopolysaccharidosis type VI disease caused by the deficiency of arylsulfatase B (ARSB) enzyme. The aim of the study is to evaluate the safety and efficacy of the treatment. (Show More)
Development Status	Active
Indication	Mucopolysaccharidosis Type VI
Disease Ontology Term	DOID:12800
Compound Name	AAV2/8.TBG.hARSB
Sponsor	Fondazione Telethon
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	9
Results Posted	Not Available

Target Gene/Variant	ARSB
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2/8
Editor Type	none
Dose 1	6E11 gc/kg
Dose 2	2E12 gc/kg
Dose 3	6E12 gc/kg
Dose 4
Dose 5

No.of Trial Sites	2
Locations	Turkey,Italy