Clinical trial infrastructure
A CMT trial does not fail for lack of a good idea. It fails for lack of groundwork: natural history data showing how the disease actually progresses, measures sensitive enough to detect change, patients identified and ready to enroll, and outcome measures the FDA recognizes. HNF has spent years building exactly that infrastructure for Charcot-Marie-Tooth disease. A sponsor bringing a CMT program forward does not have to build it from scratch, and does not have to wait years to start.
What HNF brings to a trial program
HNF has been instrumental in advancing CMT trials by providing patient insight, developing data collection methods, consulting on industry-sponsored trial design, and informing the FDA throughout drug development. In practice, a partner draws on six things.
Registry data has been essential in clinical-trial protocol design, site selection, and efficient patient recruitment for a pivotal Phase III study in the most common type of CMT.
A coordinated initiative
CMT DEPLOY
CMT DEPLOY is HNF's effort to remove every roadblock between a promising therapy and the patients who need it. It brings patients, advocacy leaders, researchers, biopharma, and regulators under one mission, on the premise that even the best therapy can fail without clean, consistent, longitudinal data.
Built as a public-private partnership with:
- Critical Path Institute (C-Path)
- BioSensics
- Across Healthcare / Matrix
- TRIAD academic, research & industry partners
- Global universities & patient leaders
By the numbers
As of July 2026.
Frequently asked questions
What does HNF provide to a CMT clinical trial program?
A sponsor can draw on GRIN natural history and patient-reported outcome data, recruitment-ready genotyped cohorts, validated CMT outcome measures, the CMT Biobank for wet science, trial-design consulting grounded in registry data, and FDA-aligned regulatory groundwork. These components are connected, so alignment on paper links directly to data, samples, and validated models.
Why is natural history data required before a trial?
A natural history study characterizes how a CMT subtype presents and progresses over time. It is a prerequisite for any human trial because it lets sponsors define meaningful endpoints, identify eligible patients, and design a study that can succeed. GRIN supplies the longitudinal patient data that backs endpoint selection and regulatory review.
Are there validated CMT outcome measures and trial-ready sites?
Yes. Sites can be trained in validated CMT functional measures (CMTES, CMTNSv2, ONLS, and CMTPedS), alongside pilots of digital and wearable endpoints. Through CMT DEPLOY, HNF works to make sites CMT Trial Ready and to develop outcome measures in alignment with the Critical Path Institute (C-Path) and the FDA.
Does HNF have CMT animal models for preclinical work?
HNF has developed transgenic rat models for CMT4A, SORD, and CMT2A, produced with the rat-model vendor Inotiv. The CMT2A work is developing the first CMT2A rat model with optic atrophy, a qualified first distinguished by the optic-atrophy phenotype. The CMT Biobank supplies patient biospecimens that anchor biomarker discovery.
How do we start a partnership?
Contact a research partner and tell us where your program sits. HNF will point you to the right resource, whether that is GRIN data access, biobank samples, trial-ready sites, or a regulatory strategy, and help you build the program around it.
Move a CMT program forward
Whether you need natural history data, biobank samples, trial-ready sites, or a regulatory strategy, HNF can help you build the program around it. Tell us where you are and we will point you to the right resource.
See the science behind the pathway on our Clinical Trial Readiness program page, or explore the TRIAD partnership model →