Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Conditions: GM2 Gangliosidosis; Niemann-Pick Disease, Type C Interventions: Drug: AZ-3102 (Dose 1); Drug: Placebo; Drug: AZ-3102 (Dose 2) Sponsor: Azafaros A.G. Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials