Impact of a Coordinated Dietetic-adapted Physical Activity Program on the Percentage of Lean Body Mass in Adults With Cystic Fibrosis Treated With Elexacaftor-Tezacaftor-Ivacaftor: Multicentre Randomised Controlled Trial

Официальное название

Impact d'un Programme coordonné diététique-activité Physique adaptée Sur le Pourcentage de Masse Maigre d'Adultes Atteints de Mucoviscidose traités Par Elexacaftor-Tezacaftor-Ivacaftor : Essai contrôlé randomisé Multicentrique

Обзор

Cystic fibrosis is an autosomal recessive inherited disease linked to various mutations in the gene coding for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, with respiratory and digestive disorders conditioning the prognosis. Digestive damage may be responsible for malnutrition of multifactorial origin (insufficient energy intake, increased energy losses, increased basal metabolic rate), and studies show a correlation between reduced lean body mass and respiratory function. In 2019, the French National Authority for Health (HAS) redefined undernutrition by including "quantified reduction in muscle mass and/or function" as a phenotypic diagnostic criterion. Elexacaftor-Tezacaftor-Ivacaftor, an innovative therapy (authorization in 2021) for this population, aims to restore the function of CFTR protein. Significant improvements in lung function and weight gain were observed from the first weeks of treatment. These improvements have also led to the emergence of lesser-known nutritional problems in these patients, such as overweight and the development of metabolic complications. Nonetheless, new management options in terms of dietary adjustments and adapted physical activity for these patients are possible, given the development of their abilities. Adapted Physical Activity (APA) helps to improve general muscular function by strengthening respiratory and skeletal muscles, improving aerobic capacity, and aiding bronchial drainage through muscle strengthening and endurance work. Maintaining or even increasing muscle mass depends not only on appropriate food intake and optimal dietary management, but also on regular physical activity, as recommended by the HAS. Our hypothesis is therefore that a structured dietetic/adapted physical activity program (DIAPASOM program) can increase the percentage of lean body mass at 12 months in adult cystic fibrosis patients treated with Elexacaftor-Tezacaftor-Ivacaftor.

Подробное описание

Cystic fibrosis is an autosomal recessive inherited disease linked to various mutations in the gene coding for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, with respiratory and digestive disorders conditioning the prognosis.

Digestive damage may be responsible for malnutrition of multifactorial origin (insufficient energy intake, increased energy losses, increased basal metabolic rate), and studies show a correlation between reduced lean body mass and respiratory function.

In 2019, the French National Authority for Health (HAS) redefined undernutrition by including "quantified reduction in muscle mass and/or function" as a phenotypic diagnostic criterion.

Elexacaftor-Tezacaftor-Ivacaftor, an innovative therapy (authorization in 2021) for this population, aims to restore the function of CFTR protein. Significant improvements in lung function and weight gain were observed from the first weeks of treatment. These improvements have also led to the emergence of lesser-known nutritional problems in these patients, such as overweight and the development of metabolic complications. Nonetheless, new management options in terms of dietary adjustments and adapted physical activity for these patients are possible, given the development of their abilities.

Adapted Physical Activity (APA) helps to improve general muscular function by strengthening respiratory and skeletal muscles, improving aerobic capacity, and aiding bronchial drainage through muscle strengthening and endurance work. Maintaining or even increasing muscle mass depends not only on appropriate food intake and optimal dietary management, but also on regular physical activity, as recommended by the HAS.

Our hypothesis is therefore that a structured dietetic/adapted physical activity program (DIAPASOM program) can increase the percentage of lean body mass at 12 months in adult cystic fibrosis patients treated with Elexacaftor-Tezacaftor-Ivacaftor.

Вмешательства

• Другое DIAPASOM program
  A program of adapted physical activity carried out remotely by a specialist instructor, combined with personalized dietetic care by a dietician for one year.

Первичные конечные точки

• Evolution of percentage of patients lean mass as a percentage of body mass [Срок оценки: From randomization, up to 12 months]

Вторичные конечные точки (12)

• Evolution of percentage of patients fat mass as a percentage of body mass [Срок оценки: From randomization, up to 12 months]
• Weight evolution [Срок оценки: From randomization, up to 12 months]
• Body Mass Index (BMI) evolution [Срок оценки: From randomization, up to 12 months]
• Cardio-respiratory endurance [Срок оценки: From randomization, up to 12 months]
• Bilateral Handgrip strength [Срок оценки: From randomization, up to 12 months]
• Lower limb muscle power [Срок оценки: From randomization, up to 12 months]
• Upper limb muscle power [Срок оценки: From randomization, up to 12 months]
• Static trunck extensors muscle endurance [Срок оценки: From randomization, up to 12 months]
• Static trunck flexors muscle endurance [Срок оценки: From randomization, up to 12 months]
• Balance between static muscular endurance of extensors and flexors [Срок оценки: From randomization, up to 12 months]
• Posterior chain flexibility (Hamstring, hips and lower back) [Срок оценки: From randomization, up to 12 months]
• Upper limb flexibility [Срок оценки: From randomization, up to 12 months]

Критерии участия

Критерии включения

• Subject aged 18 or over
• Suffering from cystic fibrosis
• Treated with Elexacaftor-Tezacaftor-Ivacaftor for at least 6 months
• Affiliated to a social security scheme
• with a signed Informed Consent form.

Критерии исключения

• Pregnant and breast-feeding women
• Subject under legal protection, guardianship or curatorship
• Subject whose physical activity is not medically authorised or whose physical and motor capacities do not allow them to take part in physical activity.
• Subject who is unable to comply with the requirements of the DIAPASOM program
• Difficulty in understanding the self-questionnaires
• Wearing a pacemaker or metal prosthesis
• Fluid retention

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

Открытое

Основная цель

Поддерживающая терапия

Центры проведения

Франция · 4 центра

• Cystic Fibrosis Resource and Competence Centre, University Hospital, Angers — Angers
• Cystic Fibrosis Resource and Competence Centre, Fondation Ildys, Roscoff — Roscoff
• Cystic Fibrosis Resource and Competence Centre, University Hospital, Tours — Tours
• Cystic Fibrosis Resource and Competence Centre, Hospital, Tours — Vannes

Идентификаторы

NCT: NCT06457451 · 2023-A02715-40 · DR220270

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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