With CMTA support of $1.2M, David Herrmann, MB, BCh, MD, and his team at the University of Rochester are moving forward with the next phase of the Accelerate Clinical Trials in CMT (ACT-CMT) study. Early efforts focused on preparing all clinical centers to begin, and included protocol updates, IRB approvals, and refresher training to ensure consistent assessments across sites. The CMTA Center of Excellence at Massachusetts General Hospital in Boston, MA, has joined as a new clinical site, bringing the total number of sites to four. Each participating center is a CMTA Center of Excellence and an INC clinical site.
Enrollment is ongoing at the University of Rochester, with the University of Pennsylvania, Massachusetts General Hospital, and the University of Iowa preparing to begin. The study includes expanded clinical assessments to track how CMT1A changes over time, along with blood and skin sample collection to support biomarker development. At the University of Wisconsin, CMTA Strategy To Accelerate Research (CMTA-STAR) Advisory Board Chairperson John Svaren, PhD, is preparing to measure PMP22 and MPZ levels in plasma. This could offer a new way to monitor treatment effects in future CMT1A clinical trials.
October 2025 Update
Over the past six months, study visits have focused on individuals with CMT1A who were part of the original ACT-CMT study and are now completing year 4 and year 5 follow-ups. These visits provide important information on how CMT1A changes over time. Sites are also enrolling additional participants with CMT1A who were not involved in the first phase of ACT-CMT, expanding the reach of the study.
Each visit includes expanded clinical assessments to map the long-term progression of CMT1A. Blood and skin biopsy samples continue to be collected to refine potential biomarkers (biological signs that can show how the disease is changing or responding to treatment). At the University of Wisconsin, CMTA Strategy To Accelerate Research (CMTA-STAR) Advisory Board Chairperson John Svaren, PhD, is beginning to measure PMP22 levels in plasma as a possible new way to track treatment effects in future clinical trials.
By extending ACT-CMT, this project is strengthening the foundation for biomarker-driven trials and helping accelerate the path toward treatments for people living with CMT1A.
