Enzyme replacement therapy for children with Gaucher disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are under 12 years old.
- You have a confirmed diagnosis of type 1 or type 3 Gaucher disease.
- Your spleen is enlarged (felt 2-3 cm below the ribs), and you also have anemia, an enlarged liver, or low platelets.
- You have not had enzyme replacement therapy or substrate reduction therapy for at least 3 months before starting.
- You have not had surgery to remove the spleen or liver, and you are not planning such surgery during the study.
- You have not had a stem cell transplant or other organ transplant.
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 trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
This study tests a new gene therapy called LY-M001 for adults with Type 1 Gaucher disease. It aims to help the body produce the missing enzyme and reduce symptoms like low blood counts or enlarged organs.
This study looks at how enzyme replacement therapy affects heart function in children with Gaucher disease type 3. It aims to see if the therapy helps keep the heart healthy.
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 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.
This study looks for tiny particles in blood (called extracellular vesicles) that may help identify Gaucher disease and could guide future treatments. It also studies people who have Gaucher gene carrier status but do not have the disease, using careful genetic testing.
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