CMT2C: What is Your Experience?
Living with Charcot-Marie-Tooth Disease Type 2C (CMT2C) can be an isolating journey, especially with limited research focusing on the lived experiences of those with this specific type of CMT. Most research typically focuses on clinical outcomes, but living with CMT is about more than just symptoms—it’s about the daily experiences and impacts on a person’s life. This study seeks to hear and learn from your personal experiences with CMT2C.
What is This CMT2C Research Opportunity?
The overall aim of this study is to understand peoples’ experience of living with CMT2C and identify relevant/important symptoms and impacts of the disease that should be assessed in clinical trial studies. This will partly be done by conducting 1:1 interviews with adolescents and adults with CMT2C to understand the symptoms and impacts they experience and to see if certain questionnaires that may be used in clinical trials are relevant and make sense to your CMT2C experience.
Little research has been done to understand the symptoms experienced by people living with CMT2C and how these symptoms impact peoples’ lives. It is important to understand these experiences to help develop new treatments and make sure clinical trials for CMT2C are set up correctly.
Who is the Sponsor of This CMT2C Research Study?
This CMT2C research study is sponsored by a CMTA-STAR Alliance Partner, the pharmaceutical company Actio Biosciences and is conducted by Clarivate, a healthcare research company independent of Actio Biosciences and CMTA. Clarivate will conduct all interviews to gather insights directly from participants.
What is Involved in Participation?
This CMT2C research will take place virtually from the comfort of your home. Participants will be asked to engage in one 60-minute interview conducted online over Microsoft Teams with a researcher from Clarivate. People who have trouble hearing, talking, typing on a computer, or have other concerns about taking part are encouraged to consider participating; the interview process will be adapted to ensure these individuals can provide their experiences and views.
During the interview, people will be asked about the symptoms they experience because of CMT2C and the ways in which these symptoms impact their lives. The interview will also ask peoples’ views/opinions on some questionnaires that aim to assess CMT2C symptoms and impacts.
Will I Be Paid to Participate in This Study?
Participants will be compensated $150 by the study sponsor for their time and valuable insights. Caregivers who assist a person with CMT2C to complete study forms and an interview will also receive $50 for their time. Participation is voluntary, and this compensation is a small token of appreciation for sharing your lived experience with CMT2C. Note: CMTA does not provide compensation for study participation.
Who is Eligible to Participate in This CMT2C Research?
The study aims to recruit twelve people (aged 12 years and above) of all abilities living with CMT2C (resulting from a TRPV4 mutation), as confirmed by a healthcare professional. Individuals currently taking part in an interventional clinical trial for CMT2C treatments are not eligible to take part in this study. Please note, that previous CMT2C clinical trial participation is permitted.
If you decide to take part in this study, you can still take part in future clinical trials for CMT2C.
Your experiences and insights could help shape future research and treatments if you meet these criteria.
How Do I Participate in This CMT2C Research Opportunity?
If you or someone you know is eligible and interested in participating in this CMT2C research study, or if you would like more information about the study/how the interview will be adapted for people of all abilities, please contact the Clarivate research team at CMT2CStudy@clarivate.com to express your interest. Be sure to include all your contact information and put “CMTA Patients as Partners in Research Opportunity” in the subject line of your email. The Clarivate team are happy to talk through the study and make sure your questions are answered.
What Does This CMT2C Research Opportunity Mean for Me?
By sharing your story, you can help expand the understanding of CMT2C and contribute to developing effective treatments. This is an opportunity to ensure your voice is heard and to shape the future of CMT2C clinical research.
CMTA’s Commitment to Patient-Centered Research
CMTA remains committed to supporting patient-centered research, and along with our CMTA-STAR Alliance Partner Actio Biosciences, we are excited to partner with the CMT2C community to drive progress towards treatments and a cure. Your participation could bring us one step closer to these goals.
CMTA is the global leader in innovative approaches to understanding CMT while working to develop treatments and, ultimately, a cure for this disease. This Patients as Partners in Research opportunity is another example of our leadership, collaborative reach, and commitment to creating and exploring new ways to carry out our mission to improve the lives of everyone living with CMT.
Register With Patients as Partners in Research Today
Not yet registered with Patients as Partners in Research? Click the button below to register today! Registering ensures you are the first in line for any CMT research opportunity you might be eligible for and are interested in. Registration will also keep you updated on all the latest news and developments in CMT research. Registration is free, and everyone with CMT is invited to join.
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Published on: January 23, 2025