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Clinical trial

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

NCT03173521 is registered in ClinicalTrials.gov with Fondazione Telethon as lead sponsor.

NCT NCT03173521
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Registry record

As filed in ClinicalTrials.gov.

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
Lead sponsor
Fondazione Telethon
Phase
Phase 1/2
Overall status
Completed
Start date
July 17, 2017
Completion date
July 16, 2024
Enrollment
9 (actual)
Sites
2

Conditions

As the registry lists them; a condition with its own page is linked.

mucopolysaccharidosis type vi