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 objective of this trial is to collect additional safety data.The further objective of this trial is to collect long-term efficacy measures as well as to assess the treatment effect on:- disability and productivity- functional impairment- health-…
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…
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…
primaryTo evaluate the effect of MEDI6570 on non-calcified coronary atherosclerotic plaques compared with placebosecundaryTo evaluate the effect of MEDI6570 on a surrogate biomarker of HF compared with placeboTo evaluate the effect of MEDI6570 on…
The primary objective of this trial in CIAS is to assess the efficacy in improving cognitive impairment using MCCB in patients with schizophrenia treated for 26 weeks with Iclepertin 10mg as compared with placebo.