Back to search
Sanfilippo Syndrome A
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Sanfilippo Syndrome A trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Sanfilippo Syndrome A trials may be worth asking aboutNorth America
Europe
Asia-Pacific
Rest of World
Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandin…
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with…
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhH…
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysacchar…