RecruitingPHASE3INTERVENTIONAL

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

This research study is testing a new medicine called AZ-3102 for rare genetic conditions that affect children and young people. These conditions are Niemann-Pick Type C disease, GM1 gangliosidosis, and GM2 gangliosidosis. The study wants to find out if AZ-3102 is safe to use and if it can help improve the health of those with these conditions. For 18 months, some participants will receive the new medicine, while others will receive a dummy pill (placebo). This helps us understand if any changes are due to the medicine itself. Doctors and participants won't know who is getting which treatment. Researchers will carefully monitor everyone involved to understand the medicine's effects.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Azafaros A.G.

Enrolment target

147

Start

30 Jun 2025

Estimated completion

04 Nov 2027

Niemann-Pick Type C Disease GM1 Gangliosidosis GM2 Gangliosidosis

What is this study about?

This study is a research project looking into a new medicine called AZ-3102. It's designed for people, mainly children and young adults, who have one of three rare genetic conditions: Niemann-Pick Type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. These conditions can cause problems with how the body processes certain fats and sugars, leading to various health issues. The main goal is to see if AZ-3102 can safely help improve the health of individuals living with these conditions.

This kind of study involves different groups of people. Some will receive the new medicine, AZ-3102, while others will receive a 'placebo', which looks exactly like the medicine but contains no active ingredients. This is a common way to test new treatments, as it helps researchers figure out if any improvements are genuinely due to the medicine being tested, rather than other factors. Neither the participants, their families, nor their doctors will know who is getting the medicine and who is getting the placebo until the study is over.

The study will last for 18 months, and participants will be closely monitored by a team of medical professionals. They will gather information about the health of everyone involved to see if AZ-3102 makes a difference. Because these conditions have specific features, the study is set up so that information is collected carefully for each condition, even though they are all part of the same overall research programme.

Key takeaways

This study is testing a new medicine (AZ-3102) for rare genetic conditions.
It's for children and young people aged 4 and over with specific diagnoses.
The study lasts 18 months and involves regular clinic visits.
Participants will receive either the new medicine or a dummy pill (placebo).
Neither you nor your doctor will know which treatment you are getting during the study.
Your safety and well-being will be closely monitored throughout.

Who may be eligible?

This study is looking for children and young people who have been diagnosed with certain forms of Niemann-Pick Type C disease, GM1 Gangliosidosis, or GM2 Gangliosidosis.

To be considered, you must be at least 4 years old and have a diagnosis of one of these conditions, specifically the 'late-infantile' or 'juvenile' type. There are also other specific health requirements for each condition that your doctor would need to check.

Generally, those who don't meet these age or diagnosis criteria, or have other certain health issues that might affect the study results, would not be able to take part. Your doctor will have more detailed information about who can and cannot join based on your specific health situation.

Quick self-check

Are you or your child diagnosed with Niemann-Pick Type C, GM1 Gangliosidosis, or GM2 Gangliosidosis?
Is the diagnosis specifically the 'late-infantile' or 'juvenile' form?
Is the person wanting to take part aged 4 years or older?
Are you able to attend regular clinic visits for 18 months?
Are you willing to potentially receive either the study medicine or a placebo?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you would be involved for a total of 18 months. During this time, you would regularly take the study medication, either AZ-3102 or the placebo, by mouth as instructed by the study doctor. You would have a schedule of visits to the study clinic where the medical team will carry out various checks and assessments.

These assessments might include physical examinations, blood tests, and tests to measure how your condition is affecting you. The exact number and type of visits and tests will depend on which specific condition you have, but they are all designed to keep you safe and to see if the medicine is working. The study team will explain the full schedule of visits and what each involves before you make a decision.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. A potential benefit of this study is that AZ-3102 might help improve your health condition, or it could help researchers learn more about these rare diseases and potential future treatments, which could benefit others. However, there's also a chance the medicine might not help, or you might experience side effects. It's important to remember that you can choose to leave the study at any time, for any reason, without it affecting your medical care.

Locations (37)

UCSF Children's Hospital and Research Center at Oakland
Oakland, United States· Recruiting
University of Minnesota Medical School
Minneapolis, United States· Not yet recruiting
Mayo Clinic Childrens Center - PIN
Rochester, United States· Recruiting
Children's Medical Center Dallas
Dallas, United States· Not yet recruiting
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, United States· Recruiting
Hospital Universitario Austral
Ciudad Autónoma Buenos Aires, Argentina· Recruiting
Hospital de Niños de La Santisima Trinidad
Córdoba, Argentina· Recruiting
Women's and Children's Hospital
North Adelaide, Australia· Recruiting
Royal Melbourne Hospital
Parkville, Australia· Recruiting
Royal Children's Hospital Melbourne - PIN
Parkville, Australia· Not yet recruiting
Instituto Fernandes Figueira
Rio de Janeiro, Brazil· Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Brazil· Recruiting

+25 more sites — see the official record for the full list.

Common questions

What is AZ-3102?

AZ-3102 is a new medicine being tested in this study to see if it can help people with Niemann-Pick Type C, GM1 Gangliosidosis, or GM2 Gangliosidosis.

How long will the study last?

If you join, your active participation in the study will last for 18 months.

Will I know if I'm getting the medicine or a dummy pill?

No, neither you, your family, nor your doctors will know whether you are receiving AZ-3102 or the placebo until the study is over.

Can I stop taking part in the study?

Yes, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Why is a placebo used in this study?

A placebo helps researchers determine if any changes or improvements are truly due to the new medicine, AZ-3102, rather than other factors.

How to find out more

Patient Advocacy Representative

info@azafaros.com Please reach out by email Official trial record

Always speak to your GP or specialist before deciding to take part in a study.