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)
Indication	Mucopolysaccharidosis Type IV
Compound Name	AAV2/8.TBG.hARSB
Sponsor	Fondazione Telethon
Funder Type	Other
Status	Active not recruiting
Enrollment Count	9

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
Dose 1	6 x 10^11 gc/kg
Dose 2	2 x 10^12 gc/kg
Dose 3	6 x 10^12 gc/kg
Dose 4
Dose 5

No.of Trial Sites	2
Locations	Turkey,Italy

Has US IND	False
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