Clinical Trial: 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
Study Status: ACTIVE_NOT_RECRUITING
Recruit Status: ACTIVE_NOT_RECRUITING
Condition: GM2 Gangliosidosis
Study Type: INTERVENTIONAL
Official Title: Randomized, Double Blind, Placebo Controlled, Multicenter, 12 Weeks Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangl
Brief Summary:
This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C) patients.If approved by the country health authorities, a double-blind extension period will be proposed to the patients who complete the 12-week study.
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