HNF to host CMT disease summit on readiness for clinical trials

Two-day event will offer perspectives on studies, advice about participating

Mary Chapman avatar

by Mary Chapman |

Share this article:

Share article via email
A speaker leads a presentation at a conference.

The Hereditary Neuropathy Foundation (HNF) will present a patient-centered Charcot-Marie-Tooth disease (CMT) summit June 7-8 that will address clinical trial readiness.

The event will happen at the Loews Coronado Bay Resort, San Diego, and a limited number of discounted rooms are available. Tickets are $200 for patients and guests, and $300 for clinicians and researchers. Industry members must pay $400 and nonprofit organization members, $200.

Patients will be able to share their perspectives to help inform future clinical studies, learn about participating in a trial, and connect with others living with CMT, a group of inherited conditions of the peripheral nervous system that affects an estimated 126,000 U.S. residents and 2.6 million people globally.

Those with CMT will also be able to participate in HNF’s Global Registry for Inherited Neuropathies and donate specimens to the foundation’s CMT Biobank, the first biorepository for Charcot-Marie-Tooth disease. The new biobank collects and stores patient samples including blood, tissue, and skin cells with the aim of helping to advance treatment research.

“As NEW clinical trials emerge for CMT, the importance of optimizing measures, endpoints, and the participant experience has never been more urgent! HNF is committed to de-risking upcoming trials by bridging the gap between clinical trial study teams and patient perspectives and experiences,” the HNF summit webpage reads. “This impact-driven summit is a chance for all patients, caregivers, clinicians, industry, and regulatory to connect with one another to exchange ideas, and engage in open discussions about the obstacles we face in designing and conducting CMT clinical trials.”

Recommended Reading
An illustration showing a hand surrounded by coins and dollars.

CMTRF supporting work needed to move HDAC6 inhibitor into trials

About the clinical trial summit

The summit is designed to let the community share its challenges in clinical studies and ideas for overcoming them.

The agenda for June 7 will include a keynote presentation followed by panel discussions titled “CMT Biobank & Biomarkers for CMT Therapy Development,” “Innovating Digital Endpoints with Wearable Technology,” “Revolutionizing Clinical Trials,” and “What’s Blocking CMT Clinical Trials & Investments?” A workshop on managing mental health in CMT will also be held.

Glenn Pfeffer, MD, a surgeon who specializes in CMT, will be available for by-appointment surgical consultations. He will also present “New Techniques in Foot Corrective Surgeries for CMT” and discuss surgery as an option. In CMT, weakness in lower leg and foot muscles can lead to foot deformations such as foot drop and cavovarus foot, or high arch, which can potentially be corrected with surgery.

Presentations for June 8 will include “Turning Symptoms into Science,” “Repurposing Drugs for CMT,” “What’s Next for Gene Therapies in CMT, “What Does it Take? A Fighter Mom’s Journey from Kitchen Table to Injection!” and “What Does it Take to Participate in Future CMT Clinical Trials?” There will also be an overview of therapies being developed and upcoming clinical trials, followed by a question-and-answer period. A dinner party will wind up the day.

Both days will feature breakout sessions, social lounges, opportunities for surgical consultations, genetic testing support, and biobank and registry participation. A “meet and greet” for children and teenagers will also be hosted.