Prenatal gene therapy for GM1 gangliosidosis types I and II
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests whether giving a gene therapy before birth can help babies with GM1 gangliosidosis, a rare genetic disorder that damages the brain and body. It uses a harmless virus to deliver a working copy of the missing enzyme, aiming to slow or prevent disease progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are at least 18 years old and pregnant.
- Your pregnancy is between 28 weeks and 35 weeks and 6 days.
- Your baby has been diagnosed with Type I or Type II GM1 gangliosidosis through genetic testing.
- You have low levels of antibodies against the virus used in the therapy (anti-AAV9 antibodies less than 1:50).
- Your baby does not have any other serious birth defects or genetic conditions.
- You agree to sign consent forms and follow the study requirements.
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 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-phase study tests a gene therapy meant to treat people who have IGHMBP2 gene changes. It aims to improve the way the nervous system works and to see if the treatment is safe, especially in young children.
This trial tests a gene therapy for people with a specific mutation (S295L) in the SLC6A1 gene, which causes a neurodevelopmental disorder. The therapy aims to correct the genetic issue, and participants will be monitored closely for safety and effectiveness.
This study explores whether a gene therapy approach for GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) can work by testing cells from your blood. It aims to understand how this treatment might help your body.
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.