Search clinical trials by condition, location and status
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
The natural history study of patients with Sanfilippo disease(s) (MPS3)
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.