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Идёт набор NCT06708468

Personalized Training for People With Rare Neuromuscular Disorders

Без фазы С лечением Neuromuscular Diseases (NMD) Charcot Marie Tooth Disease (CMT) Facioscapulohumeral Muscular Dystrophy Myotonic Dystrophy Type 1 (DM1)

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: Exercise, Usual Care.
Кому может быть актуально
Состояния в реестре: Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1). Базовые параметры: 18 лет — 70 лет · 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
Норвегия
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

Personalized Exercise Training for People With Rare Neuromuscular Disorders: a Multi-center, Evaluator-blinded, Two Arm, Randomized Controlled Study to Assess the Effects on Physical Function From Personalized Strength and Balance Exercise in a Rehabilitation Setting.

Обзор

The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders: Charcot-Marie-Tooth (CMT), Facioscapulohumeral Muscular Dystrophy (FSHD), and Myotonic Dystrophy Type 1 (DM1). The key objectives are: 1. To investigate if the intervention group experiences improvements in dynamic balance that are superior to the control group 2. To investigate if the intervention group experiences long-term improvements in dynamic balance that are superior to the control group during the follow-up 3. To investigate if improvements in dynamic balance are associated with improvements in physical activity, body composition, estimated motor units, metabolomics, muscle echnogenecity and volume, and other indicators of health and quality of life. This is a national study and will involve 120 individuals with rare-neuromuscular disorders from Norway's four health regions.

Вмешательства

  • Другое Exercise
    The intervention includes 12-day personalized exercise program set in a rehabilitation center, followed by a three-month digital intervention and then a second 12-day personalized exercise program in a rehabilitation center.
  • Другое Usual Care
    Usual care includes appointments at hospitals and in the community health system according to what has been recommended to that individual

Первичные конечные точки

  • Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest) [Срок оценки: Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup)]
Вторичные конечные точки (9)
  • Fatigue severity scale [Срок оценки: Baseline, 4-month, 10-month and 16-month (only intervention-group)]
  • Clinical measures of strength and function [Срок оценки: Baseline 4-month, 10-month and 16-month (only intervention group)]
  • Global Metabolomics [Срок оценки: Baseline, 4-month, 10-month]
  • Dual Energy X-ray Absorptiometry [Срок оценки: 4-month, 10-month and 16-month]
  • Neuromuscular ultrasound [Срок оценки: 4-month, 10-month and 16-month]
  • Physical activity [Срок оценки: 4-month, 10-month and 16-month]
  • Motor unit number estimation [Срок оценки: Baseline 4-month, 10-month and 16-month (only intervention-group)]
  • ABC-scale [Срок оценки: Basleine, 4 month, 10 month, 16 month (only intervention group)]
  • PROMIS-29 [Срок оценки: Baseline, 4 month, 10 month and 16 month (only intervention group)]

Критерии участия

Критерии включения

  • A confirmed diagnosis of either FSHD, DM1 or CMT
  • 18-70 years of age at the time of signing the informed consent.
  • Any gender
  • Ability to stand, rise from a chair and walk at least 10 meters with or without any need of assistive devices
  • Indication for rehabilitation as confirmed by the treating neurologist or physiotherapist
  • Ability to understand and follow instructions in Norwegian
  • Capable of giving signed informed consent

Критерии исключения

  • Pregnancy or planning to become pregnant
  • Any other neurological or non-neurological disorders affecting physical capacity, such as disabling arthritis, severe heart-failure/cardiomyopathy, on-going cancer treatment
  • Alcohol or drug abuse as per their medical chart
  • History of non-compliance to medical advice/follow-up

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Рандомизированное
Модель
Параллельные группы
Маскирование
Простое слепое
Основная цель
Лечение

Центры проведения

Норвегия · 5 центров
  • Haukeland University Hospital — Bergen
  • Helse Møre and Romsdal HF (Molde Hospital) — Molde
  • Namsos Hospital — Namsos
  • Oslo University Hospital — Oslo
  • University Hospital of North Norway — Tromsø

Идентификаторы

NCT: NCT06708468 · 760722

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗