Clinical trial NCT03963453: Regular Physical Exercise in Duchenne Muscular Dystrophy
Official title: To Train -or Not to Train? the Role of Physical Exercise As Part of Management in Duchenne Muscular Dystrophy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Muscular Dystrophy, Duchenne
Keywords: Physical Therapy Modalities
Exercise
Physical Activity
Exercise Program
Muscular Dystrophy, Duchenne
Lead sponsor: Haukeland University Hospital
Sponsor class: OTHER
Collaborators: name: Extrastiftelsen
First posted: 2019-05-24
Enrollment: 12
Sex: MALE
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Boys with DMD from Hordaland county, attending the pediatric rehabilitation centre at Haukeland university hospital, will be recruited for intervention.

For comparison, boys with DMD, attending other Norwegian pediatric rehabilitation centres will be invited as control group. Assessment of their physical activity level and self-reported physical activity level will be examined at start and after 12 months. In addition, functional abilities by use of North Star ambulatory assessment or EK2 scale will be assessed.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Physical Activity Level
timeFrame: Change from Baseline Physical Activity level at 12 months.
Secondary outcomes:
measure: Physical Activity Questionnaire for children (PAQ-C)
timeFrame: Change from Baseline PAQ-C score at 12 months
measure: Physical Activity Diary
timeFrame: Day 1 (daily for seven days)
measure: Pediatric Quality of Life Inventory (PedsQL version 4). Child Report.
timeFrame: Change from Baseline quality of life at 12 months
measure: North Star Ambulatory Assessment
timeFrame: Change from Baseline functioning at 12 months
measure: "Egen Klassification 2- scale" (EK2 scale)
timeFrame: Change from Baseline functioning at 12 months
measure: Muscular strength- abdominal muscles
timeFrame: Change from Baseline muscular strength at 12 months
measure: Muscular strength - hand grip
timeFrame: Change from Baseline muscular strength at 12 months
measure: The 6 minutes assisted bicycling test
timeFrame: Change from Baseline physical capacity at 12 months
measure: Blood sample - Creatin kinase (CK) value
timeFrame: Day 1, day 3 and day 5.
measure: Lung function - Forced Vital Capacity (FVC).
timeFrame: Change from Baseline FVC at 12 months
measure: Lung function - Forced Expiratory Flow first second (FEV1).
timeFrame: Change from Baseline FEV1 at 12 months
measure: Lung function - FEV1/FVC ratio.
timeFrame: Change from Baseline FEV1/FVC ratio at 12 months.
measure: Lung function - Peak Expiratory Flow (PEF).
timeFrame: Change from Baseline PEF at 12 months.
measure: Lung function - Slow Vital Capacity (SVC).
timeFrame: Change from Baseline SVC at 12 months
measure: Respiratory muscle function - Maximal Inspiratory Pressure (MIP)
timeFrame: Change from Baseline MIP at 12 months
measure: Respiratory muscle function - Maximal Expiratory Pressure (MEP)
timeFrame: Change from Baseline MEP at 12 months
measure: Lung function - Peak Cough Flow (PCF)
timeFrame: Change from Baseline PCF at 12 months
measure: Body composition - total body fat tissue mass
timeFrame: Change from baseline body fat tissue mass at 12 months
measure: Body composition - fat tissue mass of the trunk
timeFrame: Change from baseline body fat tissue mass at 12 months
measure: Body composition - fat tissue mass of the arms
timeFrame: Change from baseline body fat tissue mass at 12 months
measure: Body composition - fat tissue mass of the legs
timeFrame: Change from baseline body fat tissue mass at 12 months
measure: Body composition, Participant's lean tissue mass, total body
timeFrame: Change from baseline body lean tissue mass at 12 months
measure: Body composition, Participant's lean tissue mass of the trunk
timeFrame: Change from baseline body lean tissue mass at 12 months
measure: Body composition, Participant's lean tissue mass of the arms
timeFrame: Change from baseline body lean tissue mass at 12 months
measure: Body composition, Participant's lean tissue mass of the legs
timeFrame: Change from baseline body lean tissue mass at 12 months
measure: Body composition - Bone Mineral Content (BMC), total body
timeFrame: Change from Baseline bone mineral density at 12 months
measure: Body composition - Bone Mineral Content (BMC) of the trunk
timeFrame: Change from Baseline bone mineral density at 12 months
measure: Body composition - Bone Mineral Content (BMC) of the arms
timeFrame: Change from Baseline bone mineral density at 12 months
measure: Body composition - Bone Mineral Content (BMC) of the legs
timeFrame: Change from Baseline bone mineral density at 12 months
measure: Body composition - bone mineral density. Lumbal column
timeFrame: Change from Baseline bone mineral density at 12 months
measure: Body composition - bone mineral density. Hip bone
timeFrame: Change from Baseline bone mineral density at 12 months
measure: Anthropometric measure - age
timeFrame: Day 1
measure: Anthropometric measure - body height
timeFrame: Day 1
measure: Anthropometric measure - body weight
timeFrame: Day 1
measure: Anthropometric measure - Body Mass Index (BMI)
timeFrame: Day 1
measure: Cardiac function - Blood Pressure
timeFrame: Day 2
measure: Cardiac function. Echocardiography - Left ventricular mass
timeFrame: Day 2
measure: Cardiac function. Echocardiography - Left ventricular (LV) systolic function by ejection fraction
timeFrame: Day 2
measure: Cardiac function. Echocardiography - Left ventricular (LV) systolic function by biplane Simpson
timeFrame: Day 2
measure: Cardiac function. Echocardiography - Left ventricular (LV) systolic function by tissue doppler
timeFrame: Day 2
measure: Cardiac function. Echocardiography - Left ventricular (LV) systolic function by speckle tracking
timeFrame: Day 2
measure: Cardiac function. Echocardiography - Left ventricular (LV) diastolic function by tissue doppler
timeFrame: Day 2
measure: Cardiac function. Echocardiography - Left ventricular (LV) diastolic function by isovolumic relaxation time (IVRT)
timeFrame: Day 2
measure: Cardiac function. Electro cardiogram (ECG), heart rate (HR)
timeFrame: Day 2
measure: Cardiac function. Electro cardiogram (ECG) - QRS duration
timeFrame: Day 2
measure: Cardiac function. Electro cardiogram (ECG) - PR interval
timeFrame: Day 2
measure: Cardiac function. Electro cardiogram (ECG) - QT time
timeFrame: Day 2
measure: Cardiac function. Electro cardiogram (ECG) - QT time corrected
timeFrame: Day 2
measure: Cardiac function. Electro cardiogram (ECG) - QRS axis
timeFrame: Day 2
Other outcomes:
Locations:
city: Bergen
country: Norway
facility: Department of Physiotherapy. Haukeland University Hospital
Study officials:
affiliation: Orthopedic Clinik, Haukeland University Hospital, PB1400, 5021 Bergen/Norway
name: Kjell Matre, PhD
role: STUDY_CHAIR
References: