5 results
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…
The primary objective of this study is to assess the hemostatic efficacy and safety of rVWF with or without ADVATE in subjects (* 18 years) diagnosed with hereditary severe VWD undergoing major and minor elective surgical procedures.
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 Objective(s)* To evaluate, relative to placebo, the effect of intravenous (IV) FCM on repeated heart failure (HF) hospitalisations and cardiovascular (CV) death.Secondary Objective(s)* To evaluate, relative to placebo, the effect of IV FCM…