Gene Therapy in Patients With Mucopolysaccharidosis Disease

Mucopolysaccharidosis Type VI Clinical Trial

Official title: A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects With Mucopolysaccharidosis Type VI (MPS VI) Using Adeno-Associated Viral Vector 8 to Deliver the Human ARSB Gene to Liver

Status Active, not recruiting

Clinical Trial Summary

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.

Clinical Trial Description

Mucopolysaccharidosis type VI disease is involved in Lysosomal Storage Disorder. The MPS VI disease is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement. ;

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis Type VI
• Mucopolysaccharidosis VI

• NCT number: NCT03173521
• Study type: Interventional
• Source: Fondazione Telethon
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: July 17, 2017
• Completion date: September 30, 2025