Study of LY-M001 for Gaucher Disease Type I
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 60 years old.
- You have a confirmed diagnosis of Gaucher disease type I (with specific genetic testing and low enzyme activity).
- If you've had treatment before, enough time has passed since your last dose.
- You are not pregnant and will use effective birth control during the study and for 6 months after.
- You have not had gene therapy, cell therapy, or a spleen removal in the past.
- You do not have serious liver, heart, immune, or other medical conditions that could interfere.
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 looks at people with Gaucher disease (types I, II, or III) to better “group” patients for understanding and future research. If you (or your child) have confirmed Gaucher disease, you may be asked to share information and follow a study schedule.
This study measures inflammation and “oxidative stress” (cell stress caused by imbalance in the body) using blood biomarkers in adults with Gaucher disease. It may help researchers understand what’s happening in the body and how treatment status relates to these blood signals.
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 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 whether a new drug called VGN-R08b can safely help children with Type III Gaucher disease—a rare genetic disorder affecting the brain and body. The drug is being tested in children aged 2–17 who have already tried other treatments but still have neurological symptoms, particularly eye movement problems.
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.
Hear when a new Gaucher Disease Type I trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.