A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
This study is testing a new medicine, AZ-3102, for children and young people who have GM1 or GM2 gangliosidosis, which are rare genetic conditions. The main goal is to find out if this medicine can safely improve problems with balance and coordination that are common with these conditions. For 18 months, some participants will receive the active medicine, while others will receive a placebo (a dummy pill with no active ingredients). This is to help doctors understand the true effect of AZ-3102. Researchers will also look at other symptoms, how the body handles the medicine, and any side effects. This is a large, important study taking place at multiple hospitals.
At a glance
What is this study about?
This study is designed to learn more about a new medicine called AZ-3102. It's for children and young people who have a rare genetic condition called GM1 or GM2 gangliosidosis. These conditions can cause various problems, including difficulties with movement and balance, which doctors call 'ataxia'. The main purpose of this study is to see if AZ-3102 can help improve these balance and movement issues safely.
To make sure we get clear results, the study will last 18 months and involve two groups of participants. One group will receive the new medicine, AZ-3102, and the other group will receive a 'placebo'. A placebo looks exactly like the medicine but doesn't contain any active ingredients. Neither the participants, their families, nor the study doctors will know who is getting the medicine and who is getting the placebo – this is called a 'double-blind' study. This method helps ensure that any improvements or changes observed are truly due to the medicine and not just other factors.
Beyond just looking at balance problems, the researchers will also check if the medicine helps with other symptoms of GM1 or GM2 gangliosidosis. They will also carefully monitor how the body uses and processes the medicine (this is called pharmacokinetics) and how it affects the body (pharmacodynamics). Most importantly, they will be closely watching for any side effects or safety concerns to make sure the medicine is well-tolerated. This is a Phase 3 study, which means it's a critical step before a new medicine can potentially become widely available.
Key takeaways
- This study is testing a new medicine (AZ-3102) for GM1 and GM2 gangliosidosis.
- The main goal is to see if it helps with balance and movement problems.
- It's a long study (18 months of treatment) with regular hospital visits.
- Participants will receive either the medicine or a dummy pill, and no one will know which.
- Safety and potential side effects will be closely monitored throughout the study.
- Participation helps advance understanding of these rare conditions.
Who may be eligible?
To be considered for this study, your child must have been diagnosed with either GM1 gangliosidosis or a specific type of GM2 gangliosidosis (Tay-Sachs, Sandhoff, or GM2AB variant). They need to be at least 4 years old and have started showing neurological symptoms of their condition between the ages of 1 and 10 years.
Crucially, your child also needs to have noticeable problems with balance and coordination. Doctors will use a special scoring system, and your child's score needs to be within a certain range to qualify. If your child is a girl who could become pregnant, or a boy with a partner who could become pregnant, you'll need to agree to use effective contraception during the study.
There are also some reasons why your child might not be able to join the study. For example, if they have another serious medical condition that isn't GM1 or GM2, or certain kidney or liver problems. Children weighing less than 10kg, or those who have recently changed their anti-epilepsy medicine, or have taken other new experimental drugs recently, would also not be able to participate. This is all to ensure the study is as safe and accurate as possible for everyone involved.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Has my child been diagnosed with GM1 or a specific type of GM2 gangliosidosis (Tay-Sachs, Sandhoff, or GM2AB variant)?
- Is my child 4 years old or older?
- Did my child's neurological symptoms start between the ages of 1 and 10 years?
- Does my child have noticeable balance and coordination problems?
- Does my child weigh at least 10 kg?
What does participation involve?
If your child takes part, they will receive either the study medicine (AZ-3102) or a dummy pill (placebo) by mouth every day for 18 months. You and the study team won't know which one they are getting. Throughout this period, your child will have regular visits to the hospital or clinic. These visits will involve various tests and assessments to check their balance, movement, and other symptoms. Blood and urine samples will also be taken to see how the medicine is working and to monitor their health. The study team will closely monitor for any side effects. After the 18 months of treatment, there will likely be follow-up appointments to check on your child's health after they stop taking the study medicine. The total duration of active participation, including screening and follow-up, will be longer than 18 months.
Potential risks and benefits
Locations (36)
- UCSF Children's Hospital and Research Center at OaklandVerified postcodeOakland, United States· Recruiting
- University of Minnesota Medical SchoolVerified postcodeMinneapolis, United States· Not yet recruiting
- Mayo Clinic Children's Center - PINVerified postcodeRochester, United States· Recruiting
- Children's Medical Center DallasVerified postcodeDallas, United States· Not yet recruiting
- Lysosomal Rare Disorders Research and Treatment CenterVerified postcodeFairfax, United States· Recruiting
- Hospital Universitario AustralUnverifiedCiudad Autónoma Buenos Aires, Argentina· Recruiting
- Hospital de Niños de La Santisima TrinidadVerified postcodeCórdoba, Argentina· Recruiting
- Women's and Children's HospitalVerified postcodeNorth Adelaide, Australia· Recruiting
- Royal Children's Hospital Melbourne - PINVerified postcodeParkville, Australia· Not yet recruiting
- Instituto Fernandes FigueiraVerified postcodeRio de Janeiro, Brazil· Recruiting
- Hospital de Clinicas de Porto Alegre (HCPA) - PPDSVerified postcodePorto Alegre, Brazil· Recruiting
- Hospital Pequeno PrincipeVerified postcodeCuritiba, Brazil· Not yet recruiting
Common questions
What is GM1 or GM2 gangliosidosis?
These are rare genetic conditions that can affect the brain and body, often causing problems with movement, balance, and other symptoms.
What does 'double-blind, placebo-controlled' mean?
It means neither the patient, their family, nor the doctors will know who is getting the real medicine and who is getting a dummy pill. This helps us get clearer results.
Is AZ-3102 a cure for these conditions?
This study is testing if AZ-3102 can safely improve symptoms, especially balance problems. It's not yet known if it can cure the conditions.
How long will my child need to take part?
The main treatment part of the study lasts for 18 months, but there will be screening and follow-up visits before and after, making the total involvement longer.
What if my child experiences side effects?
The study team will carefully monitor your child's health and any side effects. You should always tell them about any new symptoms or concerns right away.
How to find out more
Patient Advocacy Representative
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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