A nationwide clinical research study is enrolling children and adults with MCAD Deficiency

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About The Study

Children’s Hospital of Pittsburgh of UPMC Division of Genetic and Genomic Medicine is conducting a clinical research study evaluating ACER-001 and its effects on blood glucose levels in individuals with Medium-chain acyl-CoA dehydrogenase deficiency (MCADD).

The purpose of this study is to better understand how this investigational medication affects blood glucose levels in children and adults living with MCADD.

Participants may travel from anywhere within the United States.

You May Recognize Yourself Here

My Child Has Been
Diagnosed

Your child may have been diagnosed with (MCADD) through newborn screening or metabolic testing.
Some children are followed closely in specialty clinics, while others are stable and doing well with routine management.
Even children who are not currently experiencing symptoms may be eligible for this research study.

I Was Diagnosed as a
Child

Some individuals were diagnosed with MCADD through newborn screening or testing in early childhood.
As people grow older, they may not see a metabolic specialist regularly or may feel their condition is well managed.
Adults with a confirmed diagnosis may still be eligible to participate in this study.

I Remember a Metabolic Condition

You may remember being told as a child to avoid long periods without food due to a metabolic condition.
If you have a confirmed diagnosis of (MCAD) deficiency, this research study may apply to you —
even if you are currently feeling well.

Who May Qualify?
  • Are 4 years of age or older
  • Have a confirmed diagnosis of Medium-chain acyl-CoA dehydrogenase deficiency (MCADD)
  • Are able to complete study procedures including CGM use and blood draws
  • Are willing to maintain continuous glucose monitoring during the study

Additional medical criteria apply.
The study team will review full eligibility with you.

WHAT PARTICIPATION INVOLVES

Attend 3 outpatient study visits over approximately 5 weeks
Wear a continuous glucose monitor (CGM)
Have blood drawn at each visit
Have an IV placed at 2 of the 3 visits
Take ACER-001 twice daily for approximately 2 weeks
Keep a basic log of medication symptoms and fasting times

Travel & Costs

Participants can travel from anywhere in the United States.
The study covers reasonable travel costs including airfare and ground transportation. Hotel accommodations and meals are arranged when overnight travel is required.
All study-related procedures and materials are provided at no cost.
Participants receive $100 per visit for three in-person visits (total $300). Compensation is paid per completed visit.

WHY PARTICIPATE?

Research helps improve understanding of how MCADD affects blood glucose levels over time.

see if you qualify
FAQ

We’ve pulled together some basics to help you

Even individuals who are currently stable can contribute to advancing future care and scientific understanding.

Do I need to live near Pittsburgh?

No. This is a nationwide study. Travel is arranged when needed.

What if my child feels well?

Children who are stable may still qualify. Researchers are studying blood glucose levels in individuals with confirmed MCADD diagnoses regardless of current symptoms.

Do I need genetic paperwork?

A confirmed diagnosis of MCADD is required. The study team will discuss documentation during screening.

Will I receive the study medication?

Yes. All participants will receive ACER-001 taken twice daily for approximately 2 weeks.