Page 33 - 2021 Spring CMTA Report - Special Research Edition

Wondering How You Can Get Involved?

If reading about the CMTA’s exciting research agenda has inspired
you to get involved, there are lots of ways to do it.

Join the INC Patient Registry, which makes it possible for study is to collect real-time data directly from patients, who
researchers to find new treatments, create new studies and describe what it is like to live with CMT. The study also aims
work to improve the lives of everyone with CMT. Enrollees to find out how treatment can improve patient quality of life
in the patient registry are contacted when there are and slow CMT progression.
opportunities to participate in clinical trials and other The CMT&Me app collects real-world data using “bring your
research studies such as 6601: Natural History Evaluation own device” (BYOD) technology—participants use their own
of Charcot-Marie-Tooth Disease. smartphones to complete questionnaires or surveys at their
INC 6601 is a longitudinal study of individuals with CMT convenience. The app immediately submits the information
to see how it changes over time. Participants are invited patients provide to a central database. A Scientific Advisory
back on a yearly basis to determine how the changes are Board oversees the CMT&Me study, and includes
occurring. During the first visit, patients are assessed clinicians who care for CMT patients, representatives
for eligibility, fill out consent forms and have minimal from patient advocacy organizations (PAO) and experts in
demographics and a treatment history taken. At each visit, patient-reported outcomes (PRO) and data management.
your medications and medical history will be assessed, and
you will be asked to have a neurological examination, have
nerve conduction studies and fill out a questionnaire
about your health. Impact of CMT on work and mental health
Enrollment in the patient contact registry is being facilitated
through INC-affiliated CMTA Centers of Excellence.
We strongly encourage anyone who wants to be
considered for clinical trials to visit a CMTA Center of
Excellence where people with CMT can participate in
clinical research. More than 2000 patients from 6 countries, aged 18 or above,
Another great way to participate is to join our Patients as were asked to enter data about CMT, its management and its
Partners in Research (PPR) initiative, which is enrolling the impact on their lives over at least two years via a smartphone
patient community in STAR’s critical work of finding treat- app, CMT&Me.
ments and ultimately a cure for CMT. IMPACT ON MENTAL HEALTH
Ways to get involved in PPR include completing surveys
about your symptoms and experiences with CMT, 1/3
participating in focus groups with the CMTA and Reported diagnosis Of these, 45% use,
our strategic partners in the biotechnology and of depression in or had previously used,
pharmaceutical fields, enrolling in CMTA funded addition to CMTlA* anti-depressants*
research studies with our clinical and
scientific partners and joining clinical trials for IMPACT ON WORKING LIFE
which you may be eligible. For a list of open trials,
visit cmtausa.org/patient-partners. 74%
Finally, it’s not too late to sign up for the CMT & ME of salaried employees 36 days per year
digital app, which is not just an app, but an confirmed their absent from work due
international research study being conducted on working life was to CMTlA*
an app to determine the burden of disease in CMT. impacted by CMTIA*
The French company Pharnext is sponsoring the
study over two years in six countries: the US, UK, To sign up or learn more,
Germany, France, Italy and Spain. The aim of the visit vitaccess.com/cmt-and-me

*Data from digital lifestyle study CMT&Me 33