Q and A on the RAINBOW study
This is a RAndomized, double blINd, placeBo-controlled, multicenter, 12-week phase II study to evaluate the safety, tolerability pharmacokinetics, and pharmacodynamics of Oral AZ-3102 in patients diagnosed With GM2 Gangliosidosis or Niemann-Pick Type C disease.
AZ-3102 is an Investigational Medicinal Product which is being developed for the treatment of Niemann-Pick Type C and GM2 Gangliosidosis. At present, AZ-3102 has not been approved in any country for the treatment of Niemann-Pick Type C and GM2 Gangliosidosis. AZ-3102 belongs to the small molecules drug class. With their ability to pass through cell membranes and the blood-brain barrier, these small molecules offer the opportunity to reach targets inside the cells of the body, including in the brain and spinal cord (central nervous system). In Niemann-Pick Type C and GM2 Gangliosidosis, the lysosomes (components of cells) start to malfunction. This causes certain fats called lipids to accumulate. The storage of these lipids induces the dysregulation of cellular functions and the symptoms of these diseases. AZ-3102 is designed with the aim of reaching the central nervous system and reducing the accumulation of fats or lipids (known as Substrate Reduction Therapy). Through this mechanism, AZ-3102 is designed with the aim of reducing the impact of the impaired lysosome on cell function. AZ-3102 comes in capsule form and can be swallowed with fluids, but the capsules cannot be opened.
For the latest update on current study centers please click on the following link: https://clinicaltrials.gov/ct2/show/NCT05758922
Eligibility to participate in the study can only be decided by the study doctor or physician at the study center, in discussion with you and your family. Azafaros employees do not make this decision.
In general terms, you should:
- Have a genetic diagnosis confirmed with either disease (GM2 Gangliosidosis or Niemann-Pick disease type C)
- Be aged between 12 and 20 years old
- Be neurologically symptomatic
- Be able to swallow capsules
- Not be treated with Miglustat or have stopped at least a month ago because of tolerability issues
- Not be treated with other investigational drugs or have stopped at least three months ago
The complete enrollment criteria can be found at ClinicalTrials.gov, but please contact your study doctor if you are interested in participating. The study doctor can also explain if the exclusion criteria apply to you.