Exercise Therapy for Patients With Muscle Dystrophies

Effects of Exercise Therapy on Functional Capacity, Muscle Strength and Patient-reported Outcomes in Patients With Muscle Dystrophies

Muscular Dystrophies (MD) are a heterogeneous group of diseases characterised by muscle wasting that lead to progressive loss of function. Although exercise training has been traditionally neglected to these patients due to concerns regarding muscle damage, research has shown that exercise therapy is safe and tolerable in this population and can lead to potential gains in endurance and muscle strength, as well as other patient-reported outcomes such as health-related quality of life.Therefore, in this study, the investigators aim to examine the feasibility, tolerability and safety as well as the effects of a 12-week, exercise-training program in patients with MD on functional capacity, muscle strength, and health-related quality of life.

Study Overview

• Status: Recruiting
• Conditions: Muscular Dystrophies
• Intervention / Treatment: Behavioral: Exercise Training

• Study Type: Interventional
• Enrollment (Estimated): 42
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Raquel Sebio, PhD; Phone Number: +34679336288; Email: sebio@clinic.cat
• Study Contact Backup: Name: Sara Laxe, PhD; Email: laxe@clinic.cat

Study Locations

• Spain
  • Barcelona, Spain, 08036
    • Recruiting
    • Hospital Clinic de Barcelona
    • Contact: Raquel Sebio, PhD; Phone Number: +34634787194

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Clinical or Genetically diagnosis of Muscular Dystrophy including Limb-Girdle type I and type II, Myotonic Dystrophy type I and type II and facioscapulohumeral dystrophy.
• Age > 18 years old
• Ability to walk independently for 10 or more metres

Exclusion Criteria:

• Inability to understand or read Spanish/Catalan.
• Patients engaging in a similar exercise-based program in the past 12 months
• Patients with severe cognitive, neurological or musculoskeletal impairment unable to participate in the exercise program
• Unstable respiratory, cardiovascular or metabolic disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Exercise Training; This group will engage in a 12-week exercise training program	Behavioral: Exercise Training; 12-week exercise training program including a combination of endurance and resistance training for 45 minutes, twice a week.
No Intervention: Control group; Usual care. Patients in this group will be offered the same program at the end of the study

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Timed Up and Go	Time consumed by the patient to raise from a chair, walk 3 meters, turn 180º, walk 3 metres back and sit down on the chair	1 week post-intervention

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Short Physical Performance Battery	Score in this test is derived from three assessments: balance, gait speed and lower limb muscle performance	1 week post-intervention
Upper Body Muscle Strength	Muscle strength of the biceps brachii will be assessed with a hand-held dynamometer	1 week post-intervention
Lower Body Muscle Strength	Muscle strength of the quadriceps will be assessed with a hand-held dynamometer	1 week post-intervention
Handgrip Strength	Handgrip strength will be assessed with a hydraulic dynamometer.	1 week post-intervention
Health-Related Quality of Life	HRQoL will be measured with the Short-Form Health Survey (SF-12). The score of the scale goes from 0 - 100, with higher scores representing better health-related quality of life.	1 week post-intervention
Body Weight	Body weight (kg)	1 week post-intervention
Body Mass Index	Body weight and height will be combined to report the results on body mass index (kg/m2)	1 week post-intervention
Percentage of fat-free mass	Percentage of fat-free mass will be obtained through bio-impedance analysis	1 week post-intervention
Percentage of fat mass	Percentage of fat mass will be obtained through bio-impedance analysis	1 week post-intervention
Fatigue	Fatigue will be quantified with the Brief Fatigue Inventory. The scale has 9 items with a maximal score of 10 points. Higher scores represent heavier fatigue.	1 week post-intervention
Respiratory Muscle Strength	Maximal Inspiratory and Expiratory Muscle Strength will be measured according to international guidelines (ATS/ERS) with a MicroRPM.	1 week post-intervention
Physical Activity Levels	Accelerometry-assessment of physical activity will be measured in terms of steps per day and minutes spent in light, moderate and vigorous physical activity	1 week post-intervention

Collaborators and Investigators

• Sponsor: Universitat Pompeu Fabra
• Collaborators: Hospital Clinic of Barcelona

Study record dates

Study Major Dates

• Study Start (Actual): July 12, 2022
• Primary Completion (Estimated): July 1, 2024
• Study Completion (Estimated): December 31, 2024

Study Registration Dates

• First Submitted: September 22, 2021
• First Submitted That Met QC Criteria: October 5, 2021
• First Posted (Actual): October 18, 2021

Study Record Updates

• Last Update Posted (Actual): July 28, 2023
• Last Update Submitted That Met QC Criteria: July 27, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Keywords: muscle strength; exercise tolerance; exercise therapy; muscular dystrophies
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies
• Other Study ID Numbers: HCLINIC - MD - 2021

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No