Fetal enzyme therapy trial for certain inherited storage disorders
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This Phase 1 trial tests an enzyme replacement treatment given to a developing baby before birth for specific inherited “lysosomal storage” diseases. The goal is to see if the treatment is safe and can improve outcomes for babies diagnosed in pregnancy.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Pregnancy is between 18 weeks 0 days and 34 weeks 6 days gestation, with a live baby
- The fetus must have a confirmed diagnosis of one of the listed lysosomal storage disorders during pregnancy tests (amniotic fluid, fetal blood, placenta, or similar samples)
- You (the pregnant patient) must be between 18 and 50 years old and be able to give consent for the study
- The fetus should not have a severe structural birth defect found at diagnosis
- The pregnancy should not have serious complications that make the fetal procedure unsafe or impossible (such as significant heart/lung disease, active preterm labor, or ruptured membranes)
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 is a registry that collects information from people diagnosed with lysosomal storage diseases. It helps researchers better understand these conditions and support future studies.
This early-stage trial tests JR-446, a new treatment for MPS IIIB, a rare genetic disorder that affects the brain and body. The treatment is delivered directly into the fluid around the spinal cord and aims to slow or improve symptoms in young children.
This trial tests how well a new enzyme treatment (cipaglucosidase alfa with miglustat) works and how safe it is in children and babies with infant-onset Pompe disease. It may help by improving muscle function after earlier treatment or, for some babies, starting treatment when no prior enzyme therapy has been given.
This study tests a new gene therapy called JWK008 for adults with MPS type I. The goal is to see if it is safe and can help improve symptoms. You may be able to join if you are 18 or older and have not had serious side effects from enzyme replacement therapy.
This early-stage trial tests a new gene therapy for adults with late-onset Pompe disease who have already tried enzyme replacement therapy (ERT). The goal is to see if the gene therapy is safe and can help improve muscle strength and breathing.
This trial tests if enzyme replacement therapy works well and is safe for Fabry disease. It is for people who are already prescribed this treatment.
Hear when a new MPS I trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.