Gene therapy before birth for GM1 gangliosidosis
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests giving a gene therapy directly to a baby before birth to treat GM1 gangliosidosis, a rare genetic condition that damages the brain and body. The treatment aims to replace the missing enzyme to help the baby develop healthier.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 years or older and pregnant.
- Your baby is between 28 weeks and 35 weeks and 6 days of pregnancy (measured from the first day of your last period).
- Your baby has been diagnosed with GM1 gangliosidosis (Type I or II) through genetic testing during pregnancy.
- You do not have antibodies to the virus used in the gene therapy (the study will test for this).
- You agree that if your baby passes away during the study, you will allow an autopsy to learn more.
- Your baby does not have other severe health problems (like major heart defects or chromosomal disorders) that could shorten life.
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 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.
This trial tests a new gene therapy (VGM-R02b) for children with Glutaric Acidemia Type 1 (GA-1) whose symptoms are not well controlled by standard treatment. The goal is to see if the therapy is safe and can help improve neurological symptoms.
This study looks at the potential of gene therapy to treat GM2 gangliosidosis, a rare inherited disease that affects the brain and nerves. By studying blood samples, researchers hope to learn more about how the disease works and whether gene therapy might be a future treatment option.
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 early-stage study tests whether a one-time gene therapy can safely improve outcomes for babies with the most severe type of spinal muscular atrophy (SMA type 1). It focuses on safety and early signs of benefit, and participation requires specific genetic and health criteria.
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.
Hear when a new GM1 Gangliosidoses trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.