5 results
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…
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…
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…
Primary Objective: To determine whether a four week treatment with vildagliptin compared to acarbose improves endothelial dysfunction in patients with type 2 diabetes mellitus.Secondary Objective(s):-To determine the effect of vildagliptin on plasma…
To determine the objective response rate of entrectinib, as assessed by BICR, in each patient population basket of solid tumors that harbor anNTRK1/2/3, ROS1, or ALK gene rearrangement