BridgeMD
Tests treatment safety and results for Niemann-Pick Type C Disease
Official title A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
ClinicalTrials.gov ID: NCT07054515
What this study is testing
What is AZ-3102?
AZ-3102 is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for niemann-pick type c disease.
Also referred to as nizubaglustat.
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
- An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed are presented...
- Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of...
You likely can't join if
- Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
- Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Minneapolis, Minnesota, United States
- Rochester, Minnesota, United States
- Dallas, Texas, United States
- Fairfax, Virginia, United States
- Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
- Córdoba, Córdoba Province, Argentina
- North Adelaide, South Australia, Australia
- Parkville, Victoria, Australia
- Rio de Janeiro, Rio de Janeiro, Brazil
- Porto Alegre, Rio Grande do Sul, Brazil
- Curitiba, Brazil
- Calgary, Alberta, Canada
- Edmonton, Alberta, Canada
- Montreal, Quebec, Canada
- Paris, France
- Höchheim, Germany
- Ernākulam, Kerala, India
- New Delhi, National Capital Territory of Delhi, India
- Jaipur, Rajasthan, India
+ 17 more site(s).
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, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Minneapolis, Minnesota, United States; Rochester, Minnesota, United States; Dallas, Texas, United States; Fairfax, Virginia, United States; Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina and 31 more location(s). 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.