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 (NPC)
This study is testing a new medicine called Nizubaglustat for children and young people (aged 4 and above) who have Niemann-Pick Type C (NPC) disease. NPC is a rare condition that affects the brain and other body parts, often causing problems with movement and balance. For 18 months, some participants will receive Nizubaglustat, while others will receive a dummy pill (placebo). Neither the participants nor their doctors will know who is getting which. The main goal is to find out if Nizubaglustat can improve movement difficulties, like unsteadiness, more than the dummy pill. The study will also check for other improvements, look at how the drug works in the body, and make sure it is safe and well-tolerated. It's a key step in understanding potential new treatments for NPC.
At a glance
What is this study about?
This research study is about a new medicine called Nizubaglustat, being tested for Niemann-Pick Type C (NPC) disease. NPC is a very rare genetic condition. It causes a build-up of certain fats in the body's cells, which can lead to a range of symptoms affecting the brain, nerves, and other organs. This study focuses on children and young people who have NPC, specifically those whose symptoms started between the ages of 2 and 15, and who have movement and balance problems, often called 'ataxia'.
The study aims to see if Nizubaglustat can help improve these movement difficulties more effectively than a dummy pill (placebo). It's designed to be 'double-blind', meaning neither you nor your doctor will know if you're receiving the actual drug or the placebo. This helps to ensure the results are as fair and accurate as possible. The study will last for 18 months, and researchers will carefully monitor participants to understand how the drug works, if it helps with other symptoms, and importantly, if it is safe to use.
Finding new treatments for rare conditions like NPC is extremely important. This study is in 'Phase 3', which means it's one of the final stages of testing before a new medicine might become widely available. By taking part, participants could contribute significantly to scientific understanding and potentially help open the door to a new treatment option for others living with NPC.
Key takeaways
- Tests Nizubaglustat for Niemann-Pick Type C (NPC) in children and young people.
- Aims to improve movement problems (ataxia) compared to a dummy pill.
- Study lasts 18 months and carefully checks for safety and benefits.
- Neither you nor your doctor will know if you receive the active drug or placebo.
- Participation could help find new treatments for NPC disease.
- Open to individuals aged 4 and older with specific NPC symptoms.
Who may be eligible?
To be considered for this study, you would need to have been officially diagnosed with Niemann-Pick Type C (NPC) disease. You must be at least 4 years old and have started experiencing neurological symptoms (like problems with movement or balance) between the ages of 2 and 15. The study is looking for people who currently have some movement difficulties, specifically those related to unsteadiness or coordination.
It's important that you are either not currently taking a similar drug called miglustat, or if you are, that it hasn't been working well for you. If you are taking miglustat and are considering joining, you would need to stop taking it for one month before starting the study medication. For women who could become pregnant, and male participants whose partners could become pregnant, there are specific guidelines about using contraception that you would need to follow.
You would not be able to join if you have other serious medical conditions that might interfere with the study's results, or if your weight is less than 10kg. Also, if you have significant liver or kidney problems, very low blood platelet counts, or if you've recently had changes to your epilepsy medication, you would likely not be able to participate. Having received certain other experimental treatments or stem cell transplants within the last one to two years would also exclude you.
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.
- Do you have a confirmed diagnosis of Niemann-Pick Type C (NPC) disease?
- Are you 4 years old or older?
- Did your current movement problems (neurological symptoms) start between ages 2 and 15?
- Are you currently having some difficulties with movement or balance?
- Are you not receiving another similar treatment (miglustat) or is it not working well for you?
- Do you not have other serious health conditions like severe liver or kidney problems?
What does participation involve?
If you decide to take part, you'll be one of two groups: one receiving the new drug, Nizubaglustat, and the other receiving a dummy pill (placebo). Neither you nor your study doctor will know which you're taking. The study will last for 18 months, during which you'll take the study medication by mouth every day. You'll have regular visits to the clinic so the study team can check how you're doing, assess your movement, and monitor your health through various tests. This will help them understand if the drug is working and if it's safe. After the 18 months of treatment, there will likely be follow-up appointments to continue monitoring your health and any effects of the medication.
Potential risks and benefits
Locations (34)
- 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 Melbourne HospitalVerified postcodeParkville, 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
Common questions
What is Niemann-Pick Type C disease?
It's a rare genetic condition where the body can't properly process certain fats, leading to problems in the brain and other organs, often causing movement difficulties.
What does 'double-blind' mean?
It means neither you nor your doctor will know if you're taking the actual study medicine or a dummy pill (placebo). This helps make the study results more reliable.
Why might I need to stop my current medicine if I join?
If you're taking a similar medicine called miglustat and it's working well, you might not be eligible. If you're on it but it's not working, you'd need to stop taking it for a month before starting the study drug to get clearer results.
How long will the study last?
The main part of the study where you take medication will last for 18 months. There may be follow-up checks after this period.
Can I leave the study if I change my mind?
Yes, you are free to withdraw from the study at any time, for any reason, without it affecting your standard medical care.
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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