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…
The aim of the study is twofold. The first is the impact of DRL-21994 and the registered product on the release of vessels studied and compared. The second is the speed with which DRL-21994 is included in the body examined, as well as the degree of…
The aim of the study is twofold. The first is the impact of the investigational drugs and the registered product on safety, tolerability and the release of vessels studied and compared. The second is the speed with which the investigational drugs…
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 evaluate the efficacy and durability of KBP-5074 in reducing SBP.The primary endpoint for efficacy is change in seated trough cuff SBP from baseline to Week 12.The second key endpoint for durability is…