Oral Nizubaglustat for Niemann-Pick Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new oral treatment, nizubaglustat (AZ-3102), for two rare conditions: Niemann-Pick type C disease and GM1 or GM2 gangliosidosis. The goal is to determine if this treatment is safe and effective for individuals with the late-infantile or juvenile forms of these diseases. Participants will be randomly assigned to receive either the treatment or a placebo (a pill with no active drug) for comparison. The trial seeks individuals aged 4 and older who have been diagnosed with one of these conditions. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. Please refer to the detailed subprotocols or consult with the trial coordinators for more information.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that nizubaglustat is safe and well-tolerated. One study found that people with Niemann-Pick disease type C and GM2 gangliosidosis experienced no major side effects from the treatment. This suggests nizubaglustat is generally manageable for patients.
Although minor side effects might occur, the data supports further development of this treatment. Nizubaglustat has demonstrated sufficient safety evidence to continue in clinical research. Prospective trial participants may find this information reassuring. However, discussing any concerns with a doctor is always important.12345Why do researchers think this study treatment might be promising for Niemann-Pick disease?
Unlike the standard treatments for Niemann-Pick Disease, which often focus on managing symptoms or slowing disease progression, nizubaglustat targets the disease at a molecular level. It works by inhibiting an enzyme involved in the abnormal lipid storage that characterizes this condition. This novel mechanism of action offers the potential to address the root cause of the disease, rather than just alleviating symptoms. Researchers are excited because this could lead to more effective and long-lasting outcomes for patients.
What evidence suggests that this treatment might be an effective treatment for Niemann-Pick disease?
This trial will evaluate nizubaglustat for Niemann-Pick disease type C and GM1/GM2 gangliosidoses. Studies have shown that nizubaglustat, the treatment being tested, has promising early results for these conditions. In previous trials, patients demonstrated positive signs that the treatment might help manage these diseases. Nizubaglustat targets specific processes in the body involved in these diseases, potentially slowing their progression. Research from earlier studies indicates it is generally safe and well-tolerated by patients. While more information is needed, these initial findings offer hope that nizubaglustat could be an effective option for these rare diseases.26789
Are You a Good Fit for This Trial?
This trial is for children and young adults with late-infantile and juvenile forms of Niemann-Pick Type C disease, GM1 Gangliosidosis, or GM2 Gangliosidosis. Specific eligibility criteria are not provided but typically include a confirmed diagnosis and meeting certain health standards.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive oral nizubaglustat or placebo for 18 months in a double-blind, randomized, placebo-controlled setting
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AZ-3102
Find a Clinic Near You
Who Is Running the Clinical Trial?
Azafaros A.G.
Lead Sponsor