Found 81 matching trials
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
PHASE2The primary objective of this study was to evaluate the safety of a 2.0 mg/...
Location
Multiple Locations
A Dose-Ranging Study of 50 µg to 100 µg LSD in Healthy Volunteers
PHASE1This study with low-dose LSD comprised 2 substudies in healthy subjects. Su...
Location
PAREXEL, Early Phase Clinical Unit
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
PHASE2The primary objective of this study is to evaluate the effect of 2.0 mg/kg/...
Location
Children's Hospital & Research Center Oakland
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
N/AThis is a prospective non-therapeutic observational study in NP-C patients....
Location
University Hospital Copenhagen (Rigshospitalet)
Personality and Drug Use
N/AThis is an observational, naturalistic study that aims to assess whether pe...
Location
Multiple Locations
LSD-Perceptual-Choice-Study
NAThe aim of the study is to characterize the effects of low doses of LSD (d-...
Location
University Psychiatric Clinics Basel
Investigating Lysosomal Storage Diseases in Minority Groups
N/AAlthough lysosomal storage disorders, such as Fabry disease, Gaucher diseas...
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
PHASE1Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosoma...
Location
Emma Children's Hospital, Academic Medical Center
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
PHASE2The purpose of this study is to evaluate the safety, tolerability and clini...
Location
Multiple Locations
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
PHASE1This multicenter, open-label extension study is designed to assess long-ter...
Location
Multiple Locations