BridgeMD
Tests treatment safety and results for GM2 Gangliosidosis
Official title A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
ClinicalTrials.gov ID: NCT07399704
What this study is testing
What is AZ-3102?
AZ-3102 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for gm2 gangliosidosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with stable, full-dose Miglustat (Cohort 2).
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- Cohort 1 (NPC and GM2 patients):
- Have been assigned by chance into Phase 2 Study AZA-001-5A2-01. OR Cohort 2 (NPC patients):
- Be male or female aged ≥12 years
- Have a genetically-confirmed diagnosis of NPC disease
- Have received full-dose Miglustat treatment for at least 12 months and experienced disease stabilization or worsening with treatment over the 2...
You likely can't join if
- A positive serum pregnancy test (only tested for women of childbearing potential).
- Female planning to breastfeed during the study.
- Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.
- Participation in another treatment or non-treatment study or early access program.
See the full eligibility criteria
- Cohort 1 (NPC and GM2 patients):
- Have been assigned by chance into Phase 2 Study AZA-001-5A2-01. OR Cohort 2 (NPC patients):
- Be male or female aged ≥12 years
- Have a genetically-confirmed diagnosis of NPC disease
- Have received full-dose Miglustat treatment for at least 12 months and experienced disease stabilization or worsening with treatment over the 2 previous clinic visits. Patients experiencing clinical improvement with...
- Wish to change treatment to Nizubaglustat for their NPC disease.
- Participants from Phase 2 Study AZA-001-5A2-01 (RAINBOW) who transitioned to Miglustat may be eligible for Cohort 2 if they meet all other criteria. Participation is supported and deemed beneficial by the Principal...
- A positive serum pregnancy test (only tested for women of childbearing potential).
- Female planning to breastfeed during the study.
- Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.
- Participation in another treatment or non-treatment study or early access program.
The study team makes the final eligibility decision.
Where it's taking place
- Água Verde, Curitiba, Brazil
- Porto Alegre, Rio Grande do Sul, Brazil
- Rio de Janeiro, Brazil
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Água Verde, Curitiba, Brazil; Porto Alegre, Rio Grande do Sul, Brazil; Rio de Janeiro, Brazil. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.