8 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 evaluate the effect of multiple doses of azithromycin, clarithromycin and ritonavir on the pharmacokinetics, safety and tolerability of a single oral 150 mg dose of ivacaftor in healthy controls and in…
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…
To determine the effectiveness of the currently recommended treatment regimens.
The primary outcome is the first attempt success ratio of peripheral intravenous catheter placement with the use of the Veinplicity®, when compared to the traditional landmark technique, in patients with a medium-risk profile according to the A-DIVA…
1. To assess whether azithromycin enhances resolution of systemic inflammation in patients with drug susceptible pulmonary TB receiving HRZE treatment. 2. To assess whether azithromycin on top of HRZE treatment in patients with drug susceptible…
The primary objective is to evaluate the potential efficacy of personalized adjunctive antibiotic therapy in maintaining clinical remission in pediatric subjects undergoing SOC induction therapy for mild to moderate Crohn*s disease who have a…