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 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 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.
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.
This early-stage trial tests VGN-R09b, a gene therapy, given to children with AADC deficiency who are not doing well on standard medicines. It aims to improve symptoms and quality of life by addressing the underlying cause of AADC deficiency.
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