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

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. After approval by the country health authorities, a double-blind extension period was proposed to the patients who complete the 12-week study.

Timeline

Start

2023-04-24

Primary completion

2024-04-19

Completion

2026-05-05

Outcome

Met primary endpoint

release “the RAINBOW study met its primary objective of demonstrating that nizubaglustat was safe and well tolerated.” azafaros.com ↗

Drugs

Indications