This study is a new international research project funded by the National Institutes of Health (NIH) called Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT). Dr. David Herrmann at the University of Rochester is the study’s Principal Investigator. There are 3 participating centers in the USA.
※ University of Rochester, NY (Dr. David Herrmann, shown in photo below)
※ University of Pennsylvania, PA (Dr. Steve Scherer) recruitment completed
※ University of Iowa, IA (Dr. Mike Shy)
The purpose of this study is to determine the best way to measure the progression of Charcot-Marie-Tooth Disease Type 1A (CMT1A) over time. This study is important to help us to prepare for clinical trials. We are looking at new ways of measuring changes in CMT1A progression over a short period of time and assessing their usefulness in measuring effects of treatments in future clinical trials. In addition to measurements that are currently used to measure progression of CMT1A such as strength and sensation, this study would also include the following:
※ Two questionnaires (the CMT Health Index and Overall Neuropathy Limitation Scale) that explore how you think CMT1A affects you
※ CMT Functional Outcome Measure (CMT-FOM) which assesses balance, walking, standing up from sitting in a chair, and manual dexterity
※ We will also test your ability to feel nylon monofilaments (like fishing line) and photographing a small area of your hand using a non-invasive microscope that can look into the skin and see nerve endings
※ Nerve conduction studies on one arm and one leg
※ For some participants, an MRI scan of your lower leg muscles The study visits are approximately 4 hours (with an additional hour for optional MRI).
The study visits are at 6-12 month intervals for up to 3 years. Participants will receive an honorarium and reimbursement of qualified travel expenses. For more information about participating, please contact:
University of Rochester
Steffen Behrens-Spraggins (585) 275-5760
University of Iowa
Alexa Bacha (319) 467-5006