With CMTA support of $1.2M, CMTA Strategy To Accelerate Research (CMTA-STAR) Advisory Board member David Herrmann, MB, BCh, MD, and his team at the University of Rochester are continuing the Accelerate Clinical Trials in CMT (ACT-CMT) study to better understand the long-term impact of CMT1A, advance CMT1A biomarker development, and prepare for future clinical trials.
Enrollment is ongoing at the University of Rochester, with the University of Pennsylvania, Massachusetts General Hospital, and the University of Iowa. The study includes expanded clinical assessments to track how CMT1A changes over time, along with blood and skin sample collection to advance CMT1A biomarker development. At the University of Wisconsin-Madison, 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.
April 2026 Update
The study remains focused on completing initial visits for the ACT-CMT extension, including year 4 and year 5 follow-ups for participants from the original cohort. Sites have also continued enrolling individuals with CMT1A who were not part of the earlier ACT-CMT phase. Initial extension enrollment is expected to close in May 2026. End-of-study visits for early enrollees are now underway.
These visits include expanded clinical assessments to define the long-term pattern of CMT1A progression. Blood and skin biopsy samples continue to be collected to support CMT1A biomarker development for clinical trials.
Dr. Svaren has initiated plasma PMP22 measurements as a potential way to monitor treatment effects in future trials. In parallel, his lab is also performing broader plasma RNA sequencing to identify changes in gene expression that may provide new insights into how CMT1A leads to nerve degeneration.
By extending ACT-CMT, this project strengthens the foundation for biomarker-driven trials and accelerates the path toward treatments for people living with CMT1A.
