6 results
The primary objectives of this study is:* To determine the safety and tolerability of rhHNS via ascending doses administered via a surgically implanted intrathecal drug device (IDDD) once monthly for 6 months, in patientswith MPS IIIA.The secondary…
The primary objective of this study is to assess the potential clinical efficacy of HGT-1410administered via a surgically implanted IDDD in patients with MPS IIIA. Efficacy will bemeasured as a meaningful amelioration in the progression of cognitive…
Part 1In Part 1 of the study, no study compound will be administered, but a bone marrow aspiration and a blood sample will be taken to evaluate an assay that will be used for Part 2 and Part 3 of the study. Because no nirogacestat is given, we can…
Primary Efficacy Objective The primary efficacy objective is to determine the long-term efficacy of NCAP (Neurostimulation of the Cholinergic Anti-inflammatory Pathway) delivered by the implanted device as assessed by the Crohn*s Disease Activity…
Primary Objective(s): To evaluate long-term safety in patients with mucopolysaccharidosis type IIIA disease (MPS IIIA or Sanfilippo Type A) who received HGT-1410Secondary Objective(s):To evaluate:• The long-term cognitive function as measured by the…
Primary Efficacy ObjectiveThe primary efficacy objective is to determine the effect of NCAP delivered by the implanted device on clinical signs and symptoms of Crohn*s disease, as assessed by the Crohn*s Disease Activity Index (CDAI)Secondary…