5 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 primary objectives of this study are to assess the safety and estimate the radiation dose of the radioligand [18F]FB610, and to assess the optimal kinetic model for quantification of the tracer uptake. Secondary objectives are to determine…
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• To evaluate the effect of ravulizumab on adjudicated On-Trial Relapses in adult patients with NMOSDSecondary• To evaluate the safety of ravulizumab in adult patients with NMOSD• To evaluate the effect of ravulizumab on adjudicated…