Clinical Trial Readiness

A promising therapy cannot reach patients without the groundwork that makes a trial possible: natural history data, validated ways to measure change, trial-trained sites, and protocols regulators recognize. HNF has spent years building that readiness for Charcot-Marie-Tooth disease, so a CMT trial starts on solid ground rather than from scratch.

Natural history dataLongitudinal evidence on how each subtype progresses.

Validated measuresCommon outcome tools so studies can be compared.

CMT Trial Ready sitesCertified training to a shared standard.

FDA-alignedProtocols co-created with C-Path and the FDA.

What clinical trial readiness means

"Trial-ready" is not one thing. It is a set of pieces that all have to be in place before a sponsor can run a CMT study that can succeed. HNF builds and connects six of them.

Natural history studies GRIN characterizes how each CMT subtype presents and progresses over time, the prerequisite for any human trial.

Validated outcome measures Sites trained in CMTES, CMTNSv2, ONLS, and CMTPedS, so change can be measured consistently across studies.

CMT Trial Ready sites Certified clinical-trial training that prepares Centers of Excellence to run CMT studies to a common standard.

Aggregated data A CMT data warehouse that pulls fragmented studies into one analysis-ready, regulatory-aligned resource.

Patient material The CMT Biobank supplies the biospecimens that anchor biomarker discovery before a candidate reaches patients.

Regulatory alignment Protocols co-created with leading CMT experts, the Critical Path Institute (C-Path), and the FDA.

Why readiness decides whether a trial succeeds

Trials fail for avoidable reasons: poor design, missing data, patients who were never represented in the first place. Even the best therapy can fail without clean, consistent, longitudinal data to show whether it works.

HNF's readiness work attacks those failure points directly. Natural history data defines what progression looks like. Validated measures make change visible. Certified sites run studies to a common standard. Aggregated data and FDA-aligned protocols keep the evidence usable all the way through review.

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, care partners, advocacy leaders, researchers, biopharma, and regulators under one shared mission, on the premise that progress should be measured in months, not decades.

Certified trial trainingMaking sites "CMT Trial Ready."

Wearable pilot studiesReal-world monitoring for better outcome measures.

CMT data warehouseAggregated, analysis-ready data for regulatory alignment.

Natural history protocolsCo-created with leading CMT experts, C-Path, and the FDA.

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

The readiness HNF has built

As of July 2026.

6,000+GRIN natural history participants

4,282+biospecimens banked

31Centers of Excellence

20of the 31 have run a Phase II / III CMT trial

Frequently asked questions

What does “clinical trial ready” mean for CMT?

It means the pieces a sponsor needs to run a CMT study are already in place: natural history data on how each subtype progresses, validated ways to measure change, clinical sites trained to a common standard, aggregated regulatory-aligned data, patient biospecimens, and protocols recognized by the FDA.

What is CMT DEPLOY?

CMT DEPLOY is HNF’s public-private initiative to remove the roadblocks between a promising therapy and the patients who need it. It brings patients, advocacy leaders, researchers, biopharma, and regulators together around certified trial training, wearable studies, a CMT data warehouse, and natural history protocols.

Which outcome measures does HNF use?

HNF trains sites in validated CMT measures including the CMTES, CMTNSv2, ONLS, and CMTPedS, so change can be measured consistently across studies. Wearable pilots add objective, real-world measures alongside them.

How is HNF’s readiness work aligned with the FDA?

Natural history protocols and outcome measures are co-created with leading CMT experts, the Critical Path Institute (C-Path), and the FDA, so the evidence stays usable all the way through regulatory review.

Build a CMT trial on ready ground

Whether you need natural history data, validated outcome measures, trial-ready sites, or a regulatory strategy, HNF can help you build the program around it. The infrastructure is already connected.

See the trial infrastructure Contact a research partner

Readiness starts with patient data

The single most useful thing a person with CMT can do to make the next trial possible is to be counted. Join GRIN, HNF's patient registry, and your de-identified story becomes the natural history data every trial is built on.

Join GRIN, HNF's patient registry