The Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT) study is an international research project funded by the National Institutes of Health (NIH). Dr. David Herrmann at the University of Rochester is the Principal Investigator. Three centers in the United States are participating:

  • University of Rochester, NY (Dr. David Herrmann)
  • University of Pennsylvania, PA(Dr. Steve Scherer)
  • University of Iowa, IA (Dr. Mike Shy)

The study is enrolling people with CMT1A between the ages of 18 and 75.

Study Purpose

The goal of ACT-CMT is to determine the best way to measure the progression of CMT Type 1A (CMT1A) over time, in preparation for future clinical trials. Researchers are evaluating new approaches to detecting changes in CMT1A progression over a short period and assessing how useful those measures would be for testing treatments in future trials.

In addition to currently used measures of progression (such as strength and sensation tests), the study includes:

  • Two questionnaires (the CMT Health Index and the Overall Neuropathy Limitation Scale) that capture how participants experience the effects of CMT1A on daily life
  • The CMT Functional Outcome Measure (CMT-FOM), which evaluates balance, walking, rising from a seated position, and hand dexterity
  • A sensory test using nylon monofilaments (thin filaments similar to fishing line) placed on the skin to measure the ability to feel light touch
  • Skin microscopy: a non-invasive imaging technique that photographs a small area of the hand to visualize nerve endings just beneath the skin surface
  • Nerve conduction studies on one arm and one leg
  • For some participants, an MRI scan of the lower leg muscles

What to Expect

Study visits are approximately four hours each (with an additional hour for the optional MRI). Visits occur at 6 to 12 month intervals for up to three years. Participants receive an honorarium and reimbursement for qualified travel expenses.

How to Participate

For more information or to express interest in enrolling, contact the site closest to you:

University of Rochester
Steffen Behrens-Spraggins
(585) 275-5760
Steffen_Behrens-spraggins@URMC.Rochester.edu

University of Iowa
Alexa Bacha
(319) 467-5006
alexa-bacha@uiowa.edu