7-week study looking at the medication ACER-001 and its effects on fasting and reduction in blood glucose levels.

Children’s Hospital of Pittsburgh of UPMC Division of Genetic and Genomic Medicine is looking for patients 10 years of age or older with a diagnosis of Medium-chain acyl-CoA dehydrogenase (MCAD) deficiency to participate in a 7-week research study looking at the medication ACER-001 and its effects on fasting and reduction in blood glucose levels.  Participants must have a confirmed diagnosis of MCAD deficiency based on DNA sequencing and be willing to come to Pittsburgh three or four times during the study. The study will involve a screening visit, an overnight admission to monitor fasting glucose levels and other metabolites for up to 32 hours, dosing of study medication for 4 weeks, a second overnight admission for up to 24 hours to monitor fasting glucose levels and other metabolites, and a final end-of-study visit. Participants will also receive multiple phone calls during the study to check on symptoms while taking the study medication. Participants will be asked to take the study medication 1 or 2 times daily. Travel and a participant stipend will be covered by the study site.  If you are interested in learning more about this trial, please contact Elizabeth McCracken at 412-692-5662.