Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled
Read the full statement from Azafaros here:
Here is a summary of the article:
Azafaros’ RAINBOW study: A Phase 2 clinical trial evaluating nizubaglustat, a potential treatment for rare lysosomal storage disorders with neurological involvement, such as GM1 and GM2 gangliosidoses, and Niemann-Pick disease type C (NPC)1.
Trial enrollment: The study exceeded the target number of patients, recruiting participants with GM2 gangliosidosis and NPC from Brazil. The study is randomized, double-blind, placebo-controlled, and multi-center.
Trial objectives: The study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of two doses of nizubaglustat over 12 weeks2. The results will support two Phase 3 pivotal trials in GM1 and GM2 gangliosidoses, and NPC.
Trial significance: The study is an important step towards developing a new therapeutic solution for these rare and severe diseases, where patients have no or limited treatment options. Nizubaglustat has received multiple regulatory designations and incentives from the FDA, EMA, and MHRA.
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