To Train -or Not to Train? the Role of Physical Exercise As Part of Management in Duchenne Muscular Dystrophy
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 12
- Locations
- 1
- Primary Endpoint
- Physical Activity Level
Study Overview
Brief Summary
This study examine whether an evidence-based individual user-preferred exercise program will increase the physical activity level in boys with Duchenne muscular Dystrophy (DMD).
Detailed Description
Regular physical activity is recommended in DMD. However, due to progression of muscle weakness and loss of function, a sedentary lifestyle is normal, and disuse of still functioning muscles leads to a secondary deterioration. Use of steroids have proven to decrease the rate of progression, and also leads to adverse advents like obesity and osteoporosis.
Use of muscle strength training and aerobe exercise in DMD, may optimize muscle function, cardio-respiratory fitness and overall physical activity level, in addition to decrease possible adverse advents.
In this study, children with DMD attending Haukeland University Hospital will be invited to participate (N≈14). The participant's physical activity level is registered during a four week baseline period and follow up. At start, 6 months and 12 months, a five day hospital stay will be conducted, both for testing and to prescribe an individual user preferred physical exercise program. The exercise program will be performed at home between the hospital visits. Benefits, safety and feasibility of regular physical exercise will be examined during follow-up and end of study. For comparison of the physical activity level and motor function, DMD children attending standards of care follow-up at others Norwegian regional pediatric rehabilitation clinics will be invited to participate in a control group.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 6 Years to 18 Years (Child, Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Patients with conclusive DMD diagnosis
- •Written consent
- •Able to perform physical exercise and answer questions
Exclusion Criteria
- •Lack of consent
- •Cognitive disabled unable to answer questionnaire, understand instructions, and able to know what they participate in.
- •Language difficulties
Outcomes
Primary Outcomes
Physical Activity Level
Time Frame: Change from Baseline Physical Activity level at 12 months.
The participants physical activity level will be monitored by use of an ActiGraph for seven days including a weekend. Two registrations will take place during a four week baseline period, followed by additional registrations at 3, 6, 9 and 12 months (intervention period).
Secondary Outcomes
- Physical Activity Questionnaire for children (PAQ-C)(Change from Baseline PAQ-C score at 12 months)
- Blood sample - Creatin kinase (CK) value(Day 1, day 3 and day 5.)
- Body composition - total body fat tissue mass(Change from baseline body fat tissue mass at 12 months)
- Body composition, Participant's lean tissue mass of the arms(Change from baseline body lean tissue mass at 12 months)
- Cardiac function. Electro cardiogram (ECG) - PR interval(Day 2)
- Body composition - Bone Mineral Content (BMC) of the arms(Change from Baseline bone mineral density at 12 months)
- Body composition - fat tissue mass of the trunk(Change from baseline body fat tissue mass at 12 months)
- North Star Ambulatory Assessment(Change from Baseline functioning at 12 months)
- Muscular strength - hand grip(Change from Baseline muscular strength at 12 months)
- Respiratory muscle function - Maximal Inspiratory Pressure (MIP)(Change from Baseline MIP at 12 months)
- Respiratory muscle function - Maximal Expiratory Pressure (MEP)(Change from Baseline MEP at 12 months)
- Body composition - Bone Mineral Content (BMC) of the legs(Change from Baseline bone mineral density at 12 months)
- Lung function - Forced Vital Capacity (FVC).(Change from Baseline FVC at 12 months)
- Lung function - FEV1/FVC ratio.(Change from Baseline FEV1/FVC ratio at 12 months.)
- Lung function - Peak Expiratory Flow (PEF).(Change from Baseline PEF at 12 months.)
- Lung function - Peak Cough Flow (PCF)(Change from Baseline PCF at 12 months)
- Body composition, Participant's lean tissue mass, total body(Change from baseline body lean tissue mass at 12 months)
- Body composition - Bone Mineral Content (BMC) of the trunk(Change from Baseline bone mineral density at 12 months)
- Anthropometric measure - body height(Day 1)
- Cardiac function. Echocardiography - Left ventricular mass(Day 2)
- Cardiac function. Electro cardiogram (ECG) - QRS duration(Day 2)
- Cardiac function. Electro cardiogram (ECG) - QT time(Day 2)
- Physical Activity Diary(Day 1 (daily for seven days))
- "Egen Klassification 2- scale" (EK2 scale)(Change from Baseline functioning at 12 months)
- Pediatric Quality of Life Inventory (PedsQL version 4). Child Report.(Change from Baseline quality of life at 12 months)
- Body composition - fat tissue mass of the arms(Change from baseline body fat tissue mass at 12 months)
- Body composition - fat tissue mass of the legs(Change from baseline body fat tissue mass at 12 months)
- Body composition, Participant's lean tissue mass of the trunk(Change from baseline body lean tissue mass at 12 months)
- Body composition, Participant's lean tissue mass of the legs(Change from baseline body lean tissue mass at 12 months)
- Anthropometric measure - body weight(Day 1)
- Cardiac function. Echocardiography - Left ventricular (LV) systolic function by ejection fraction(Day 2)
- Cardiac function. Echocardiography - Left ventricular (LV) systolic function by biplane Simpson(Day 2)
- Cardiac function. Echocardiography - Left ventricular (LV) systolic function by tissue doppler(Day 2)
- Cardiac function. Echocardiography - Left ventricular (LV) systolic function by speckle tracking(Day 2)
- Cardiac function. Echocardiography - Left ventricular (LV) diastolic function by tissue doppler(Day 2)
- Cardiac function. Electro cardiogram (ECG), heart rate (HR)(Day 2)
- Muscular strength- abdominal muscles(Change from Baseline muscular strength at 12 months)
- The 6 minutes assisted bicycling test(Change from Baseline physical capacity at 12 months)
- Lung function - Forced Expiratory Flow first second (FEV1).(Change from Baseline FEV1 at 12 months)
- Lung function - Slow Vital Capacity (SVC).(Change from Baseline SVC at 12 months)
- Body composition - Bone Mineral Content (BMC), total body(Change from Baseline bone mineral density at 12 months)
- Anthropometric measure - age(Day 1)
- Cardiac function. Electro cardiogram (ECG) - QT time corrected(Day 2)
- Cardiac function. Electro cardiogram (ECG) - QRS axis(Day 2)
- Body composition - bone mineral density. Lumbal column(Change from Baseline bone mineral density at 12 months)
- Body composition - bone mineral density. Hip bone(Change from Baseline bone mineral density at 12 months)
- Anthropometric measure - Body Mass Index (BMI)(Day 1)
- Cardiac function - Blood Pressure(Day 2)
- Cardiac function. Echocardiography - Left ventricular (LV) diastolic function by isovolumic relaxation time (IVRT)(Day 2)