Our Phase II Study - RAINBOW

RAndomized, double-blINd, placeBo-controlled, multicenter, 12-week study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of Oral AZ-3102 in patients With GM2 gangliosidosis or Niemann-Pick type C (NPC) diseases.

This study aims to assess the correct doses of AZ-3102 to treat GM2 and NPC diseases by evaluating the clearance of AZ3102 from the body and the effect of two different doses in a small number of patients with those diseases.


Study Design

RAINBOW is designed as a short, multinational study in up to two countries, with 12 participants (six per disease) between the ages of 12 and 20 years, with late-infantile or juvenile onsets. Enrollment will be initiated in early 2023, with a duration study time of three months.

We are currently selecting sites in Brazil and in the US.