The A083-03 Study

We are conducting clinical research to find a new treatment for muscle weakness in people with Charcot-Marie-Tooth Disease (CMT).

What is the purpose of the A083-03 study?

The main purpose of the A083-03 study is to see if the investigational medicine, ACE-083, can safely increase muscle size and strength in people with CMT1 or CMTX. Currently, there are no approved therapies for people with CMT.

Who can take part in the A083-03 study?

You may qualify to participate in the A083-03 study if you meet the following criteria*:
  • 18 years of age or older
  • Genetically confirmed diagnosis of CMT1 or CMTX or have a first-degree relative (parent, sibling, or child) with a genetically confirmed diagnosis of CMT1 or CMTX, and show clinical signs and symptoms of CMT1 or CMTX
  • Mild to moderate ankle weakness (ability to lift left and right toes)
*Other inclusion and exclusion criteria will apply. Visit the Study A083-03 page at for more information regarding inclusion and exclusion criteria.

What will happen during the study?

If you qualify and decide to participate in Study A083-03, your participation will last approximately 15 months. The first 6 months of the study will be double-blind, and the remainder will be open-label. “Double-blind” means neither you nor your study doctor will know if you are receiving ACE-083 or placebo (saline). Open-label means all participants receive ACE-083. No placebo (saline) is given during the open-label part of the study.

Qualified study participants will be randomly assigned to receive the study drug (ACE-083) or a placebo (saline) for the first 6 months. All study participants will receive study drug (ACE-083) after the first 6 months. The study drug or placebo (saline) is given as injections into the muscle in the front of each lower leg once every three (3) weeks.

During the study, you will be asked to do the following:
  • Come to the study site for study drug injections
  • Answer questions about your health
  • Have physical exams, blood draws, an ECG (heart test), MRI scans, and physical performance tests to assess changes in your strength, balance and muscle function
  • Attend all scheduled study visits
Experienced study staff will monitor your health throughout the study.

Study Drug ACE-083 Is the A083-03 study an option for you? Answer a few questions to find out.
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ACE-083 is an investigational product that is currently being studied and has not been approved by any regulatory agency for any use.