NEW CMT Summit Alert! Launch into CMT Clinical Trial Readiness with HNF!

by | Apr 1, 2024 | 0 comments

View Agenda Friday, June 7

8:00am Registration & Breakfast

9:00 – 9:20am Keynote Address: “From NEW Gene to Phase 3 Clinical Trial – Treatment is Within Reach!”

Shoshana Shendelman, PhD

CEO & Founder, Applied Therapeutics

9:30 – 10:45pm: Panel Discussion: CMT Biobank & Biomarkers for CMT Therapy Development

A biomarker may be used to predict which patients will respond to treatment or see how well the body responds to a treatment for a disease. Without them, clinical trials may enroll patients who will have a lower chance to respond, or they rely on clinical evaluations that may not be sensitive enough to capture change (good or bad). In this session, you’ll hear from experts in the field on the importance of identifying and validating biomarkers for use in CMT therapeutic development. What’s on the horizon, and how is a patient-led biorepository advancing the development of evidence-based biomarkers for all CMT subtypes?

  • Matthew Jarpe, PhD, HNF Scientific Advisor (Presenter/Moderator)
  • Anna Pfalzer, PhD,  CSO, COMBINEDBrain (Presenter)
  • Fireside Chat Panelist
    • Anna Pfalzer, PhD, CSO, COMBINEDBrain
    • Shoshana Shendelman, PhD, CEO & Founder, Applied Therapeutics
    • Allison Moore, Founder/CEO, Hereditary Neuropathy Foundation,

Roundtable discussions & Feedback Sharing

10:45 – 11:00am Break

11:00 – 12:15pm Panel Discussion: Innovating Digital Endpoints with Wearable Technology

The identification and development of digital measures are vital to remote monitoring CMT disease progression or improvement in real-world settings. By collecting remote data (not in a clinical setting), researchers, industry, and regulatory agencies can better understand the daily impact of CMT on people living with the disease. This panel session will cover the development of methods to identify digital measures of CMT disease from wearables to generate clinically meaningful patient outcomes. Participants will have the option to participate in a digital wearable pilot study sponsored by BioSensics and HNF.

  • Klaus Romero, MD, MS, FCP, CEO, Critical Path Institute (Moderator)
  • Kayla Cornett, PhD, University of Sydney (Presenter/Panelist)
  • Ashkan Vaziri, PhD, Founder and CEO, BioSensics™ (Presenter/Panelist)

CMT Patient, TBD

Audience participation Q & A

12:15 – 1:00pm Lunch

1:00 – 2:15pm “Making Sense & Science of CMT Symptoms”

“Is this symptom related to CMT?” This is a frequent question asked by many individuals living with CMT and their loved ones. Most of us are familiar with the most common symptoms of CMT, such as drop foot & hand weakness. However, many less-recognized symptoms can be just as disruptive to our lives but have not yet been studied or linked to CMT. This is why in 2013, HNF started the Global Registry for Inherited Neuropathies (GRIN) and the CMT Natural History Study.

How do we know if our symptoms are CMT-related, and how can we drive research to answer this question better?

  • Joy Aldrich, Grin Registry Coordinator, HNF (Presenter & Panelist)
  • Jason Colquitt, CEO, AcrossHealthcare
  • Robert Moore, GRIN Data Manager, HNF

Roundtable discussions & Feedback Sharing

2:15 – 2:45pm: CMT Lounges: In-Person Support, Vendor Resources & Socializing!

CMT Social – Meet & Greet w/ Vendor Tables

CMT Adaptive Exercise – Julie Stone, Founder CMTdefy

2:45 – 3:15pm: Presentation: Is CMT Surgery Right for You?
Glenn Pfeffer, MD

Join us for an enlightening summit session titled “Is Surgery Right for You” with renowned orthopedic surgeon Glenn Pfeffer, MD. As Director of the Foot and Ankle Center at Cedars-Sinai Medical Center in Los Angeles, Dr. Pfeffer brings over 30 years of experience in treating foot and ankle problems in patients with CMT and has performed over 1000 CMT foot surgeries.

3:20 – 4:45pm: Panel Discussion: Identifying Roadblocks to Success for CMT Clinical Trials

This interactive panel will shed light on the unique challenges faced by CMT clinical trials, including the diverse subtypes & symptoms, limited clinical experience, patient access, investment, and complementary outcome measures that are sensitive enough to measure change in a relatively short period of time. You’ll learn about evidence-based, innovative strategies and solutions that may apply to CMT for enhanced efficacy and the future success of CMT clinical trials. The open format will allow for patient engagement interaction to better define how these potential solutions, such as adaptive and decentralized trials, including telemedicine, patients & caregiver needs, real-world evidence (RWE) data capture (patient registries, collaborative networks & wearable & tech devices) may have an impact in supporting the success of these trials. 

  • Moderator: Shannon Strom, PhD, RAC, Drug Development Expert
  • Michelle Campbell, PhD FDA, Associate Director, Stakeholder Engagement & Clinical Outcomes, Office of Neuroscience
  • Dottie Caplan – SVP Patient Advocacy and Engagement, Applied Therapeutics (Panelist)
  • CMT Patient – TBD


4:45 – 5:00pm: Closing Remarks

View Agenda Saturday, June 8

8:30am: Check in & Breakfast

9:00 – 10:00am Panel Discussion: Revolutionizing Clinical Trials with Video – If a Picture is Worth a Thousand Words, a Video is Worth a Million

To make clinical trials for CMT successful, we need new ways to measure how the disease affects people. One idea is to use videos to see how it affects things like walking and step length. Using videos could allow us to use footage from the past or even videos taken by regular people, not just clinic experts. Given the increased use of telemedicine, video may be a useful addition to clinical trial assessments.

  • Moderator: Klaus Romero, MD, MS, FCP, CEO, Critical Path Institute
  • Allison Moore, Founder/CEO, Hereditary Neuropathy Foundation,
  • PT, DPT, CMT Centers of Excellence (pending approval)
  • Michelle Campbell, PhD FDA, Associate Director, Stakeholder Engagement & Clinical Outcomes, Office of Neuroscience

Patient Video Breakout Session & Feedback Sharing

10:00 – 10:25am CMT Lounges: In-Person Support, Vendor Resources & Socializing!

CMT Social – Meet & Greet w/ Vendor Tables

CMT Adaptive Exercise – Julie Stone, Founder CMTdefy

10:30 – 10:50am Presentation: What Does it Take? A Fighter Mom’s Journey from Kitchen Table to Injection!

Lori Sames, Founder, Hannah’s Hope Fund

Learn how “fighter mom” Lori Sames raised the necessary funds and curated the qualified experts to tackle a gene therapy for Giant Axonal Neuropathy (GAN), an inherited neuropathy. Topics covered will include lessons learned, accelerating progress, cost-effective strategies, and regulatory considerations. Hear from Lori about the next steps needed to cross the finish line and how this will transform the future of gene therapies in CMT.

11:00 – 12:25pm: Panel Discussion: What’s Next for Gene Therapies in CMT?

How does the drug development process work and how long does it take for a drug to get from a research lab to your pharmacy? What is gene therapy and why is it important to develop treatments for specific CMT types? What are the pros and cons of gene therapy? What is currently in the gene therapy pipeline for CMT?  

You’ll learn the good, the bad and the ugly of gene therapy during this overview of the various approaches. Topics will include targets and delivery options and which subtypes have the best chance of success, Conventional therapeutic approaches vs. gene therapy, can patients participate in both?  Other questions answered during this session will include: How does the FDA view gene therapies for CMT? What are the pros & cons, what are the obstacles, and is it right for you? Patients and stakeholders will walk away with the basic knowledge & understanding of what it takes to develop a gene therapy for CMT.

  • Sharon Hesterlee, PhD, Chief Research Officer, MDA
  • Sabrina Yum, MD, CHOP
  • Matt Jarpe, PhD, Science Advisor, HNF
  • Brian Lin, PhD, MDA

Audience Q & A

12:30 – 1:15pm Lunch

1:15pm – 2:15pm 

For Industry & Researchers Only: Surrogate Biomarkers

  • Matthew Jarpe, PhD, HNF Scientific Advisor
  • Anna Pfalzer, MD, COMBINEDBrain

For Patients & Caregivers: Workshop Managing Mental Health & CMT

Estela Lugo, Program Development Manager, Hereditary Neuropathy Foundation

Julie Stone, Founder CMTdefy, Founder

2:30 – 4:00pm Presentation: Overview: Upcoming CMT Pipeline & Clinical Trials

Hear from HNF’s Therapeutic Research in Accelerated Discovery (TRIAD) partners about their latest research milestones and what’s next in the pipeline for CMT. 

  • Matthew Jarpe, PhD, HNF Scientific Advisor
  • Gideon Shapiro, PhD, Founder/CSO, Miralinc Pharma – HDAC6 to treat CMT
  • TBD: pending approvals
  • TBD: pending approvals

4:00- 4:30pm Philanthropic & Investment Opportunities

4:30pm Closing Remarks

5:30pm HNF Cocktail & Dinner Party

Thank you Sponsors!

Applied Therapeutics Logo
Helios Bracing

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 derisking upcoming trials by bridging the gap between clinical trial study teams and patient perspectives and experiences.

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. As HNF focuses on accelerating access to critical research data and specimens, this summit will also include on-site CMT Biobank collection.

Our aim is to create an all-in-one event for CMT Clinical Trial readiness with patients at the heart and center!

Session Topics include:

  • Patient and caregiver engagement
  • Patient experience
  • Designing a clinical trial protocol
  • Technology integration for clinical trials
  • Collaboration to share non-competitive patient insights

Onsite CMT Research activities:

HNF will maximize CMT data collection, including collecting biospecimen samples for our CMT Biobank and participating in HNF’s Global Registry for Inherited Neuropathies (GRIN).

What is the CMT Biobank?

HNF is excited to announce that we are continuing to collect CMT biospecimens to support therapy development. All CMT patient subtypes are welcome to participate!  There are still gaps in understanding the natural history of the disease correlation of genotype / phenotype, easy access to samples and data sharing to support translational research, and the validation of drug candidates and biomarkers for CMT.

**You will have a scheduled time during the summit for a phlebotomist to take your samples.

Learning Objectives:

  • Come together to share common ideas and open dialogue about the challenges we need to overcome so that we can work together and amplify the CMT patient voice and plan successful clinical trials.
  • Discover valuable insights into the patient’s experiences, challenges, and perspectives in navigating clinical trials, contributing to shaping future progress.
  • Bridge the gap between clinical trial study teams and patient perspectives and experiences.

#FacesofCMT will offer a unique opportunity to connect with patients or caregivers-some of whom have actively participated in clinical trials-in 1:1 meetings. Attendees can sign up on-site to meet with the participating patient advocates.

Learn more on this topic

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Do you want to make a difference in CMT research? HNF is looking for patients with a confirmed CMT diagnosis to participate in the CMT Biobank

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