App-based Study Seeks Real-world Data on the Personal Impact of CMT

An international observational study using a smartphone-based app called CMT&Me is exploring the impact of Charcot-Marie-Tooth disease (CMT) on patients and caregivers.

The study (NCT03782883) was designed by Vitaccess in collaboration with Pharnext. Its goal is to collect detailed patient-reported data to help researchers and clinicians better understand the unmet needs and the burdens of CMT from the perspectives of those living with the disease. The study is still recruiting and those interested in participating may do so here.

The detailed trial protocol, “Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study,” was published recently in the journal Neurodegenerative Disease Management.

Patient-reported outcomes are increasingly seen as a key component of disease research and management. Although these data often are generated in the course of clinical trials, relatively little data comes from patients managing their conditions in so-called “real-world” settings.

Real-world evidence can provide more detailed and longer-term data from a more diverse patient population than would be typical of a trial setting. This can be especially true for rare conditions like CMT, where recruiting enough patients for an adequately sized clinical trial can be challenging.

The study seeks approximately 2,000 adults with CMT in France, Germany, Italy, Spain, the U.K. and the U.S. Volunteers will be asked periodically over a two-year period to provide data about how CMT affects their lives.

CMT&Me app users first create a profile that includes data on demographics, lifestyle characteristics, diagnosis, and treatments. Participants then will complete a series of standard and study-specific questionnaires that assess health-related quality of life, specific symptoms such as fatigue, pain, and cramps, work limitations, and physical function.

These surveys will be taken upon enrollment, and then either monthly or quarterly, depending on the questionnaire.

Investigators running the trial will perform an interim analysis upon registration of the 1,000th patient and at 12 months intervals from study launch. A final analysis will occur upon study completion, roughly two years from launch.

While the study is ongoing, participants will receive regular communication from trial researchers, via email, social media, and in-app communications.

Pharnext and its collaborators intend to publish the results in academic journals and to present them at scientific conferences.

As this is an entirely digital study, measures have been taken to ensure data safety and confidentiality. Personally identifiable information is not maintained on users’ devices, and neither the study’s sponsors nor any external researchers view it. All data is stored in an aggregated and de-identified form in a central database.

In concluding their protocol, the investigators wrote that “this international, longitudinal, real-world digital [patient-reported outcome] study — the first of its kind — will undoubtedly help researchers and clinicians understand the real-world impact of CMT and the unmet needs of patients.”