Gene therapy for Type 1 Gaucher disease
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 60 years old.
- You have been diagnosed with Type 1 Gaucher disease through genetic testing and a blood test showing very low enzyme activity.
- You are either currently on stable treatment for Gaucher disease, or if untreated, you have low blood counts, an enlarged liver, or an enlarged spleen.
- You have not received any gene therapy before.
- You do not have serious liver disease, active hepatitis B or C, or a history of cancer in the last 5 years.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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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 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 tests an injection called LY-M001 for adults with Gaucher disease type I. The goal is to see if it can safely help replace the missing enzyme and improve symptoms.
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.
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 early-stage study tests an IV gene therapy that delivers a working copy of an enzyme gene to help the body make beta-galactosidase in GM1 gangliosidosis. It may help slow or improve disease symptoms and looks closely at safety and how well the treatment works.
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