Clinical Trial Readiness Summit
HNF’s patient-centered CMT Summit puts patients, caregivers, clinicians, researchers, and industry in the same room and gives patients a hand in setting the agenda. The result is a working meeting that builds the shared understanding, and the readiness, that Charcot-Marie-Tooth clinical trials depend on.
What makes it different is who is at the front of the room. Patients and caregivers are not an audience here, they help choose the topics and sit on the panels, so the questions that get asked are the ones families actually live with. Researchers and drug developers hear that directly rather than second hand.
It is also a working meeting rather than a conference. Patients can take part in research on the day, clinicians can train toward becoming trial ready, and the conversations between sessions are where a surprising amount of the real progress happens. For many people who come, the part that stays with them is simpler than any of that: a room full of people who did not need CMT explained to them.
A patient-centered format
At an HNF summit, “patient-centered” is not a slogan. It means the meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders together: in topic selection, in who sits on the panels, in audience engagement during Q&A, and in the conversations between sessions and at the evening reception.
That format matters because CMT trial readiness is not only a scientific problem. It depends on clinicians, sponsors, and patients agreeing on what is worth measuring and what patients are willing to take on. Putting everyone in one room is how that alignment gets built.
Who attends
The summit is built around bringing every stakeholder in CMT together, with patients and caregivers at the center.
What the summit accomplishes
- 1 Patients help set the agenda Topic selection, panelist participation, and Q&A are shaped by patients and caregivers, not chosen for them. The summit is patient-centered by design.
- 2 Everyone is in one room Patients, caregivers, clinicians, researchers, and industry meet together, including in the conversations between sessions and at the evening reception, where a lot of the real connection happens.
- 3 It builds trial readiness The summit helps the community and the field get ready for clinical trials: aligning on what matters to patients, what to measure, and how sites can become CMT trial ready.
- 4 Patients participate in on-site research The summit is not only conversation. Patients can take part in research right there at the meeting, from contributing biobank samples that fuel subtype studies to trying digital wearable measures, completing hearing tests, and more. It is a chance to advance the science on the day itself, and to experience the kinds of assessments a future clinical trial may rely on.
- 5 It carries the patient voice forward What is heard at the summit does not stay in the room. It feeds HNF’s patient-focused research and the evidence HNF brings to industry and the FDA, where decisions get made about which treatments are developed and how they are judged. The priorities patients raise here shape what gets measured in a trial, and a measure that reflects real daily life is what allows a therapy to prove it works. This is how a conversation in a hotel ballroom turns into leverage with a regulator.
Scroll for more, or select any photo to enlarge it.
Agenda themes
Programs vary year to year, but summit conversations tend to return to the same core themes.
- The state of CMT research and the road to clinical trials
- Natural history, outcome measures, and trial design
- Genetic testing and understanding your CMT subtype
- Patient-reported outcomes and the patient voice in drug development
- Living well with CMT: care, mobility, and daily life
- Emerging technology, wearables, and digital biomarkers
From the room to the clinic
Building “CMT Trial Ready” sites
Trial readiness extends beyond the summit. Through CMT DEPLOY, HNF supports certified clinical trial training that helps sites become “CMT Trial Ready,” so that when a sponsor opens a study, the sites, the measures, and the patient community are already prepared. The summit is where much of that shared preparation begins.
Frequently asked questions
What is the CMT Summit?
The CMT Summit is HNF’s patient-centered meeting that brings patients, caregivers, clinicians, researchers, and industry into the same room to build readiness for Charcot-Marie-Tooth disease clinical trials. Patients and caregivers help set the agenda, choose panelists, and shape the conversation.
Who can attend the CMT Summit?
The summit is built for everyone in the CMT community: people living with CMT, caregivers, clinicians, researchers, industry and gene therapy developers, and patient advocates. Patients and caregivers are at the center of every session.
What does "trial readiness" mean?
Trial readiness is the shared preparation that lets a clinical trial start well: aligning on what matters to patients, what to measure, and how clinical sites can become "CMT Trial Ready." Through CMT DEPLOY, HNF supports certified trial training so that sites, measures, and the patient community are prepared before a sponsor opens a study.
How do I find out about the next summit?
Add your details to the interest form on this page and HNF will reach out when registration for the next CMT Summit opens.
Take part in the next summit
Be the first to know about registration and early-bird pricing for the April 2027 summit. Add your details, whether you want to attend, speak, or bring your organization, and we will reach out the moment registration opens.
Curious about the bigger picture? See how the patient voice reaches the FDA at the 2018 PFDD meeting, or read about patient-reported outcomes →