New daily pill for Gaucher disease type 3
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new daily pill called nizubaglustat (AZ-3102) for Gaucher disease type 3 (GD3). The pill aims to treat the disease by reducing the buildup of fatty substances in cells, which may help improve symptoms like anemia, low platelets, and enlarged spleen.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Gaucher disease type 3 (GD3).
- You need to be between 4 and 30 years old.
- You must have at least two areas of the disease affecting your daily life (for example, movement, thinking, or eye movement).
- You should have some typical GD3 symptoms like low red blood cells (anemia), low platelets (thrombocytopenia), or an enlarged spleen.
- You cannot have had enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the last 12 months.
- You cannot have had prior gene therapy, stem cell transplant, or a splenectomy (surgery to remove the spleen).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study tests a new gene therapy, VGN-R08b, for people with type 3 Gaucher disease. It aims to see if the treatment is safe and can help with neurological symptoms like eye movement problems.
This trial tests a treatment called velaglucerase beta, which replaces an enzyme that people with Gaucher disease lack. It may help reduce enlarged spleen and liver, improve anemia, and increase platelet counts in children under 12.
This study looks at MRI-based lab measurements (similar to a special type of scan) to find markers that reflect health in people with type 3 Gaucher disease. It may help researchers track the disease over time and improve future treatments.
This special access program provides an experimental oral medicine called venglustat (taken as a tablet) to patients with Type 3 Gaucher disease, which affects the brain. It is designed for patients who are already on stable enzyme replacement therapy and aims to see if the drug can help manage neurological symptoms.
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.
This trial tests a new gene therapy (VGN-R08b) for infants with Type 2 Gaucher disease, a rare genetic condition that affects the brain and body. The goal is to see if the treatment is safe and can help improve symptoms.
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