A study for children and adults living with MCADD (a rare metabolic condition)
This study is looking at whether a study medication called ACER-001 can help manage blood sugar levels in people with Medium-chain acyl-CoA dehydrogenase deficiency (MCADD) — a rare condition that affects how the body uses fat for energy. The study involves three outpatient visits over about five weeks at UPMC Children's Hospital of Pittsburgh. It is open to children and adults age 4 and older across the United States. Travel and lodging costs are reimbursed for those coming from outside the Pittsburgh area.
What to expect
Three outpatient visits over approximately five weeks at UPMC Children's Hospital of Pittsburgh
Wearing a continuous glucose monitor during the study period
Blood draws at each visit, and an IV line placed at two of the three visits
Taking the study medication twice daily for about two weeks
Travel and lodging costs are reimbursed for participants coming from outside the Pittsburgh area
You may be a fit if
You have a confirmed diagnosis of MCADD (Medium-chain acyl-CoA dehydrogenase deficiency)
You are 4 years of age or older
You are willing and able to attend outpatient study visits, including blood draws and continuous glucose monitoring
Additional criteria may apply — the study team is happy to talk through your specific situation
A few quick questions. No obligation, and your answers stay private.