MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Clinical trials for MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY explained in plain language.
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Could a liver drug help kids and adults with rare metabolic disorder?
Disease control Recruiting nowThis study tests whether sodium phenylbutyrate (ACER-001), a drug already approved for another condition, can help people with MCAD deficiency caused by a specific gene mutation. About 24 participants aged 4 and older will take the drug and be monitored for safety and how well it…
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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Could a fatty acid drug stop dangerous sugar crashes in rare disease?
Disease control Recruiting nowThis study tests a drug called triheptanoin, already approved for similar conditions, to see if it can prevent dangerously low blood sugar in people with MCADD, a rare inherited disorder. About 24 participants aged 4 and older will take the medication and be monitored for safety …
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Jun 27, 2026 09:11 UTC
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Fatty acid study aims to unlock secrets of rare metabolic disease
Knowledge-focused Recruiting nowThis study looks at how medium-chain and long-chain fatty acids affect metabolism in people with Medium-Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) and healthy individuals. Researchers will measure ketone bodies, resting metabolic rate, and other factors after participants co…
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Phase: NA • Sponsor: University of Copenhagen • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC