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 program provides access to an investigational treatment (ATB200/AT2221) for children with infantile-onset Pompe disease who are not doing well on standard enzyme replacement therapy. The goal is to see if this new combination can help slow or improve symptoms.
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 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 program provides early access to an experimental enzyme therapy given directly into the brain for children with Sanfilippo syndrome type B, a genetic condition that affects the nervous system. The treatment aims to replace a missing enzyme to slow or stabilize symptoms.
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.
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.