MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Clinical trials for MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY explained in plain language.
Never miss a new study
Get alerted when new MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY trials appear
Sign up with your email to follow new studies for MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Could a urea cycle drug help MCAD patients fast longer?
Disease control TerminatedThis study tests whether sodium phenylbutyrate, a drug already approved for another condition, can help people with MCAD deficiency (a genetic disorder that affects fat breakdown). About 24 participants aged 10 and older will take the drug for 4 weeks. Researchers will check for …
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
-
Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 04:00 UTC
-
Could a fatty acid drug stop dangerous sugar crashes in rare disease?
Prevention TerminatedThis study tests a drug called triheptanoin in 8 adults with MCADD, a rare condition that can cause dangerously low blood sugar. The goal is to see if the drug is safe and can prevent hypoglycemia during fasting. Participants will stay overnight at a hospital for monitoring and b…
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Prevention
Last updated Jun 27, 2026 12:23 UTC