Sodium Phenylbutyrate for MCADD in Children and Adults
Treatments studied
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a medicine called sodium phenylbutyrate (ACER-001) to see if it helps people with MCADD, a genetic condition that affects how your body breaks down fats. If you have MCADD and the common gene change, you might be able to join.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have been diagnosed with MCADD and have the common c.985A>G gene change.
- You must be at least 4 years old.
- You must be able to wear a continuous glucose monitor (a small sensor on your skin that checks blood sugar) and have blood draws and an IV placed.
- If you could become pregnant, you must have a negative pregnancy test and use birth control during the study. If you could get someone pregnant, you must also use birth control.
- You cannot have certain medical conditions like kidney problems, diabetes, or a serious infection.
- You cannot be taking certain medicines (like valproate, corticosteroids, or some chemotherapy drugs) that could interfere with the trial.
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 how medium-chain fatty acids (a type of fat found in coconut oil) affect metabolism in people with MCADD, a genetic condition that affects fat breakdown, compared to healthy people. The results may help guide dietary advice for those with MCADD.
This trial tests a drug called ACER-001 in a single patient with a rare metabolic disorder. The goal is to see if the drug helps manage symptoms.
This trial tests an oil called triheptanoin for children with a rare genetic condition (PDCD) that affects how their bodies use energy. The study aims to see if triheptanoin can help improve their health.
This trial tests a medicine called glycerol phenylbutyrate in children with urea cycle disorders, which are genetic conditions that make it hard for the body to get rid of extra ammonia. The goal is to see if the medicine can help lower ammonia levels and manage the condition safely.
This study follows people with MADD to learn how the condition changes over time. It aims to improve understanding and future care.
This trial tests whether a supplement called NMN is safe and well-tolerated in children and adults with a rare genetic condition called DHDDS-CDG. NMN may help support cellular energy and protein production, which are affected by this condition.
Hear when a new Medium-chain Acyl-CoA Dehydrogenase Deficiency trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.