Gene therapy for propionic acidemia in infants
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy treatment designed to help the body process certain amino acids better in infants with propionic acidemia. The therapy uses a modified virus to deliver a working copy of a faulty gene, potentially reducing dangerous acid buildups and hospital visits.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be between 6 months and 2 years old at the time of treatment
- Your child must have confirmed propionic acidemia (a genetic test showing mutations in both copies of the PCCA gene)
- Your child must have had at least one serious episode of the disease (such as confusion, poor feeding, vomiting, seizures, or hospitalization)
- Your child's liver and blood counts must be in relatively good condition (blood levels meet certain thresholds)
- Your child must not have active viral infections or a history of liver transplant
- Your family must be willing to share your child's participation with your primary care doctor and other doctors
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 trial tests an mRNA medicine (mRNA-3927) in people with propionic acidemia (PA). It aims to see whether this treatment is safe and can help the body when PA is confirmed by genetic testing.
This is a “chart review” study that looks at how people with propionic acidemia have symptoms and medical events over time. It uses existing medical records (not an experimental drug) to better understand the disease and care needs.
This study follows people with propionic acidemia to better understand how the body, diet, gut bacteria, and blood chemistry change over time. It may help researchers learn more about the condition and how pregnancy, transplant, and daily life affect health.
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 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.
This trial tests a gene therapy given as a nose spray for people with cerebral palsy or HIE. It aims to see if the treatment is safe and helps improve movement over two years.
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