Clinical trials
Niemann-Pick Diseases clinical trials
Below are recruiting niemann-pick diseases clinical trials, each written for real people, not researchers. We’re tracking 9 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05588167Recruiting
Niemann-Pick type C patient database and genetic data study
This study collects health information and genetic samples from people diagnosed with Niemann-Pick disease type C. The goal is to build a large database that can help researchers better understand the condition and improve future care.
Bethesda, MarylandAges 3 months+ - NCT07545473Recruiting
Eye Imaging to Detect Brain Disease Markers
This study uses a special camera to photograph the back of your eye in detail. Researchers believe changes in the eye may reflect brain diseases like Alzheimer's or Parkinson's, and this imaging could help detect these conditions earlier.
Melbourne, VictoriaAges 30 years+ - NCT07082725RecruitingPhase 3
Study of oral nizubaglustat for Niemann-Pick type C
This study tests an oral medication called nizubaglustat for people with Niemann-Pick type C disease who cannot take or are not satisfied with the current treatment miglustat. It aims to see if the drug can help with symptoms like coordination and movement problems.
Oakland, CaliforniaAges 4 years+ - NCT07054515RecruitingPhase 3
Study of oral nizubaglustat for rare genetic disorders
This trial tests a new oral medicine, nizubaglustat (AZ-3102), for children and adults (4+) with late-infantile or juvenile Niemann-Pick type C, GM1, or GM2 gangliosidosis. It aims to see if the drug is safe and helps slow disease progression.
Oakland, CaliforniaAges 4 years+ - NCT06192576Recruiting
Long-term safety study of olipudase alfa in children under 2 with ASMD
This study looks at the long-term safety and effects of olipudase alfa in children under 2 years old with acid sphingomyelinase deficiency (ASMD), a rare genetic condition. It aims to see how the treatment works over time and if it is safe for young children.
Chicago, IllinoisAges Up to 2 years - NCT05368038Enrolling by invitation
Newborn screening program for babies up to 4 weeks old
This trial tests a flexible newborn screening process that looks for certain conditions early in life. It may help by catching problems sooner, when treatment can be started earlier.
Brooklyn, New YorkAges Up to 4 weeks - NCT05163288RecruitingPhase 3
N-acetyl-leucine trial for Niemann-Pick disease type C
This Phase 3 trial tests whether N-acetyl-leucine can improve symptoms in people with Niemann-Pick disease type C (NPC). If you qualify, you will be asked to take study medicine and complete symptom and movement tests over time.
Rochester, MinnesotaAges 4 years+ - NCT03333200Recruiting
Study of genetic brain diseases over time
This trial follows people with genetic neurodegenerative (brain-wasting) disorders over time to better understand how the condition changes. The goal is to learn patterns that could help future treatments or care plans.
Pittsburgh, PennsylvaniaAges Any age - NCT00344331Recruiting
Testing blood and MRI markers for Niemann-Pick type C
This study measures biochemical (blood/biologic) markers and may use clinical evaluation and MRI to better understand Niemann-Pick disease type C. It could help improve how the condition is tracked and understood over time.
Bethesda, MarylandAges 1 day–120 years
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Common questions
- Are there clinical trials for niemann-pick diseases?
- Yes. Clin2 currently lists 9 recruiting niemann-pick diseases studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a niemann-pick diseases trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a niemann-pick diseases trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.