Идёт набор NCT07409142

BetterLife FSHD: A Patient-driven Health and Research Platform

— Наблюдательное FSH FSH Muscular Dystrophy FSHD - Facioscapulohumeral Muscular Dystrophy FSHD1

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Observational.
Кому может быть актуально: Состояния в реестре: FSH, FSH Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1. Базовые параметры: от 1 год · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

Не всё понятно в терминах? Прочитайте наш гид для пациентов →

Обзор

BetterLife FSHD is a registry platform built to support people living with FSHD. It connects patients with personalized resources, tools, and research opportunities that match their health experiences and needs. At the same time, BetterLife collects secure health and experience data from patients to support research. This data is shared with researchers to help better understand FSHD and work toward improved care, treatments, and outcomes for the community. Learn more and enroll at: www.BetterLifeFSHD.org

Подробное описание

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder that causes relentless weakening of skeletal muscles. BetterLife FSHD is a patient-driven health platform and research registry that aims to help FSHD patients live their best lives while also powering research.

In BetterLife, participants are prompted to respond to a series of short surveys spread out over a quarterly and yearly basis. Survey topics include demographics, health history, FSHD diagnosis and progression, FSHD management strategies, and quality of life domains like pain, fatigue, and mental health.

As participants provide survey data, they receive a personalized feed of resources relevant to them from the FSHD Society's library of articles, blogs, videos, and webinars. Survey data is also used to inform participants which clinical trials and other research studies they may be eligible for.

The information that BetterLife FSHD collects is stored and managed in a modern and secure real-world data infrastructure. De-identified data is made available upon request to researchers, clinicians, biopharmaceutical companies, regulator/payor bodies, and other organizations involved in FSHD research and therapeutic development with approval from a steering committee. BetterLife can also be used to conduct and support research initiatives external to the FSHD Society.

Вмешательства

Другое Observational
Observational study

Первичные конечные точки

Longitudinal Health Data [Срок оценки: Assessed annually from enrollment until study completion (10 years)]
Self Reported FSHD Progression [Срок оценки: Assessed every 6 months, until study completion (10 years)]

Вторичные конечные точки (12)

Anxiety Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Depression Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Pain Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Sleep Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Fatigue Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Upper Body Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Mobility Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Physical Activity Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Falls Patient Reported Outcome Measure [Срок оценки: Quarterly, until study completion (10 years)]
Research Preferences [Срок оценки: Yearly until study completion (10 years)]
Diagnostic Journey [Срок оценки: One time at Baseline]
Healthcare Experiences [Срок оценки: Yearly, until study completion (10 years)]

Критерии участия

Критерии включения

Individuals with a clinical or genetic diagnosis of FSHD, or individuals with a family history of FSHD who are showing symptoms
Age 1 year or older
Residing in the United States or its territories
If age 18 or older, individual must be able and willing to provide consent
If under age 18, individual must be able and willing to provide assent, when applicable, and have a parent or legal guardian register and provide consent