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Muscular Dystrophy, DuchenneNCT03963453

Regular Physical Exercise in Duchenne Muscular Dystrophy

An observational study of Muscular Dystrophy, Duchenne, run by Haukeland University Hospital at 1 site in Norway.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 6 to 18 years, men only
Conditions the study is for
Muscular Dystrophy, Duchenne
What is being tested?
Physical Exercise
How many people
12 participants
Where
1 siteNorway
Study dates
1 Feb 2021to 30 Apr 2022
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 18 years
  • Men only
  • No healthy volunteers
  • Child, Adult

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.

Who may be able to join

  • Patients with conclusive DMD diagnosis
  • Written consent
  • Able to perform physical exercise and answer questions

Who cannot join

  • Lack of consent
  • Cognitive disabled unable to answer questionnaire, understand instructions, and able to know what they participate in.
  • Language difficulties

Trial locations and contacts

1 site in 1 country.

Norway1 site

  • Department of Physiotherapy. Haukeland University Hospital

    Bergen, Norway, 5021

Other trials in Norway

About this study

Official title: To Train -or Not to Train? the Role of Physical Exercise As Part of Management in Duchenne Muscular Dystrophy

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).

Read the 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.

Being tested
Physical Exercise
Keywords
Physical Therapy Modalities, Exercise, Physical Activity, Exercise Program, Muscular Dystrophy, Duchenne
Medical subject headings
Muscular Dystrophy, Duchenne, Motor Activity

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Physical ExerciseOther

    Physical exercise are prescribed to be performed three times per week for a year

Study groups in NCT03963453
Study groupRoleWhat they receive
InterventionalRegular physical exercise group
Receives: Other: Physical Exercise
ControlStandards of care treatment

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Physical Activity Level

    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).

    Measured: Change from Baseline Physical Activity level at 12 months.

Secondary outcomes (49)

  1. Physical Activity Questionnaire for children (PAQ-C)

    The PAQ-C is a self-administrated, 7 day recall instrument, developed to assess general levels of physical activity throughout the elementary school year. The PAQ-C provides a summary physical activity score derived from nine items, each scored on a 5-point scale. The nine items and scores are:

    1. "Spare time activities". Score "no activity" being a 1, "7 times or more" being a 5.
    2. "Physical education". 3. "Recess".4. "Lunch". 5 "Right after school". 6 "Evening". 7 "Weekend". 8. "Describe you best". Each of these 7 item start from lowest activity response (score 1) to the highest activity response (score 5).
    3. How often did you do physical activities for each weekday? None=score 1 Little bit = score 2, Medium = score 3, Often = score 4, Very often = Score 5. Item 9 mean score is reported. Total score 45 represents the participant's general level of physical activity. The participants perform the PAQ-C at baseline and at every hospital visit (start, 6 and 12 months).

    Measured: Change from Baseline PAQ-C score at 12 months

  2. Physical Activity Diary

    During physical activity registration with use of ActiGraph monitor, the participants and parents are asked to fill out a diary, describing type of physical activity been performed, for how long the physical activity was performed, how tired the participants became, and how did the participant enjoy the activity being performed. In addition the participants is asked to give a summary of the week regarding to name the most enjoyable activity this week and the reason why, and to describe if there occurred something unusual that increased or decreased their physical activity level more than regular.

    Measured: Day 1 (daily for seven days)

  3. Pediatric Quality of Life Inventory (PedsQL version 4). Child Report.

    The Pediatric Quality of Life Inventory TM (PedsQL TM) is a questionnaire measuring measures quality of life in children, adolescents and young adults. The questionnaire have four subscales with a total of 23 items. Each items with score 0 to 4. The subscales are Physical functioning (eight items), Emotional functioning (five items), Social functioning (five items) and School functioning (five items). A score can be calculated for each subscale. Total score 100 points indicate optimal quality of life. A Psychosocial score can also be calculated (based on the subscales Emotional, Social and School functioning). Participants will report from PedQoL at start and end of study.

    Measured: Change from Baseline quality of life at 12 months

  4. North Star Ambulatory Assessment

    Standardised test for individual with DMD and Spinal Muscular Atrophy able to ambulate. The following physical activities and functions being quantified: Stand, Walk, Stand up from chair, Stand on one leg (right and left), Climb box step (right and left), descend box step (right and left), Get to sitting, Rise from floor, lifts head, Stands on heels, jump, hop on one leg (right and left), run (10 meter timed test). Performed at every three hospital visit during study period.

    Measured: Change from Baseline functioning at 12 months

  5. "Egen Klassification 2- scale" (EK2 scale)

    Standardised functional assessment for individuals with DMD not able to walk. The following functions and activities are quantified: Ability to use wheelchair, ability to transfer from wheelchair, ability to stand, ability to balance in the wheelchair, ability to move arms, ability to use the hands and arms for eating, ability to turn in bed, ability to cough, ability to speak, physical well-being, Daytime fatigue, head control, ability to control joystick, food textures, eating a meal, swallowing, hand function which of these activities can you do. A total score possible to achieve is 51 points (0-3 point grading on each function), higher score indicating lower degree of functioning. The test will be performed at each hospital visit

    Measured: Change from Baseline functioning at 12 months

  6. Muscular strength- abdominal muscles

    Isometric testing of abdominal muscles. The force being developed will be recorded as Nm and Kg. The participants will be tested at start, after 6 and 12 months.

    Measured: Change from Baseline muscular strength at 12 months

  7. Muscular strength - hand grip

    Isometric testing of hand grip. The force being developed will be recorded as Nm and Kg. The participants will be tested at start, after 6 and 12 months.

    Measured: Change from Baseline muscular strength at 12 months

  8. The 6 minutes assisted bicycling test

    Standardized test where participants will perform a 6 minutes arm cycling test, and the distance, heart rate and perceived exhaustion scored at OMNI scale will be recorded. The test will be performed at each of the three hospital visits.

    Measured: Change from Baseline physical capacity at 12 months

  9. Blood sample - Creatin kinase (CK) value

    Venous blood samples, as biomarker for muscle inflammation or tissue damage, measured by U/L.

    Assessment of CK will be performed for safety reasons due to intervention. Blood samples will be performed three mornings during the hospital visits.

    Measured: Day 1, day 3 and day 5.

  10. Lung function - Forced Vital Capacity (FVC).

    Performed by use of spirometry assessment. Measured by liters in absolute value and percent predicted value. Lung function will be measured at each of the three times during study period.

    Measured: Change from Baseline FVC at 12 months

  11. Lung function - Forced Expiratory Flow first second (FEV1).

    Performed by use of spirometry assessment. Measured by liters in absolute value and percent predicted value. Lung function will be measured at each of the three times during study period.

    Measured: Change from Baseline FEV1 at 12 months

  12. Lung function - FEV1/FVC ratio.

    Spirometry assessment. The Ratio is calculated in percent value. All participants will be examined at all three hospital visits during study period.

    Measured: Change from Baseline FEV1/FVC ratio at 12 months.

  13. Lung function - Peak Expiratory Flow (PEF).

    Spirometry assessment. The maximal airflow achieved during expiration maneuver, measured as liters per second.

    Measured: Change from Baseline PEF at 12 months.

  14. Lung function - Slow Vital Capacity (SVC).

    The Participant's SVC measured as liters / absolute value will be registered at each hospital visit during study period.

    Measured: Change from Baseline SVC at 12 months

  15. Respiratory muscle function - Maximal Inspiratory Pressure (MIP)

    The Participant's MIP will be measured and registered as absolute value cm water pressure (cm H20) at each hospital visits during the study period.

    Measured: Change from Baseline MIP at 12 months

  16. Respiratory muscle function - Maximal Expiratory Pressure (MEP)

    The Participant's MEP will be measured and registered as absolute value cm water pressure (cm H20) at each hospital visits during the study period.

    Measured: Change from Baseline MEP at 12 months

  17. Lung function - Peak Cough Flow (PCF)

    The Participant's PCF will be measured and recorded as absolute value liters per minutes at each hospital visits during the study period.

    Measured: Change from Baseline PCF at 12 months

  18. Body composition - total body fat tissue mass

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify total fat mass (kilogram and percent) at start and end of study period

    Measured: Change from baseline body fat tissue mass at 12 months

  19. Body composition - fat tissue mass of the trunk

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify fat mass of the trunk (kilogram and percent) at start and end of study period

    Measured: Change from baseline body fat tissue mass at 12 months

  20. Body composition - fat tissue mass of the arms

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify fat mass of the arms (kilogram and percent) at start and end of study period

    Measured: Change from baseline body fat tissue mass at 12 months

  21. Body composition - fat tissue mass of the legs

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify fat mass of the legs (kilogram and percent) at start and end of study period

    Measured: Change from baseline body fat tissue mass at 12 months

  22. Body composition, Participant's lean tissue mass, total body

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify both total lean tissue mass (kilogram and percent) at start and end of study period

    Measured: Change from baseline body lean tissue mass at 12 months

  23. Body composition, Participant's lean tissue mass of the trunk

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify lean tissue mass of the trunk (kilogram and percent) at start and end of study period

    Measured: Change from baseline body lean tissue mass at 12 months

  24. Body composition, Participant's lean tissue mass of the arms

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify lean tissue mass of the arms (kilogram and percent) at start and end of study period

    Measured: Change from baseline body lean tissue mass at 12 months

  25. Body composition, Participant's lean tissue mass of the legs

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify lean tissue mass of the legs (kilogram and percent) at start and end of study period

    Measured: Change from baseline body lean tissue mass at 12 months

  26. Body composition - Bone Mineral Content (BMC), total body

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify total BMC (kilogram and percent) at start and end of study period

    Measured: Change from Baseline bone mineral density at 12 months

  27. Body composition - Bone Mineral Content (BMC) of the trunk

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify the trunk BMC (kilogram and percent) at start and end of study period

    Measured: Change from Baseline bone mineral density at 12 months

  28. Body composition - Bone Mineral Content (BMC) of the arms

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify the arm's BMC (kilogram and percent) at start and end of study period

    Measured: Change from Baseline bone mineral density at 12 months

  29. Body composition - Bone Mineral Content (BMC) of the legs

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify the leg's BMC (kilogram and percent) at start and end of study period

    Measured: Change from Baseline bone mineral density at 12 months

  30. Body composition - bone mineral density. Lumbal column

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify mineral (grams/ square centimeter) of the lumbar column at start and end of study period.

    Measured: Change from Baseline bone mineral density at 12 months

  31. Body composition - bone mineral density. Hip bone

    Participants will be assessed using a Lunar iDXA dual x-ray scanner (dxa-scan), to quantify mineral (grams/ square centimeter) of the hip bone at start and end of study period.

    Measured: Change from Baseline bone mineral density at 12 months

  32. Anthropometric measure - age

    The participants age measured in years of age will be registered at start and end of study period

    Measured: Day 1

  33. Anthropometric measure - body height

    The Participant's height measured in centimeter (cm) will be registered at each hospital visit during study period.

    Measured: Day 1

  34. Anthropometric measure - body weight

    The Participant's body weight measured as kilograms will be measured and registered at each hospital visit during study period

    Measured: Day 1

  35. Anthropometric measure - Body Mass Index (BMI)

    The Participant's BMI will be calculated by (kg/m2). at each hospital visit during the study period.

    Measured: Day 1

  36. Cardiac function - Blood Pressure

    The participants systolic and diastolic blood pressure will be registered (mmHg) at start and end of study period

    Measured: Day 2

  37. Cardiac function. Echocardiography - Left ventricular mass

    Participants will undergo Ultrasound of the heart, using the cube formula indexed by Height (centimeter^2.7), derevid from two dimensional linear left ventricular measurements (gram per meter ^2.7) at start and end of study.

    Measured: Day 2

  38. Cardiac function. Echocardiography - Left ventricular (LV) systolic function by ejection fraction

    Participants will undergo Ultrasound of the heart, measuring LV ejection fraction (%) derived from 2D linear LV measurements (Teichholz) at start and end of study.

    Measured: Day 2

  39. Cardiac function. Echocardiography - Left ventricular (LV) systolic function by biplane Simpson

    Participants will undergo Ultrasound of the heart, measuring LV ejection fraction (%) derived from 2D linear LV measurements (Teichholz) at start and end of study.

    Measured: Day 2

  40. Cardiac function. Echocardiography - Left ventricular (LV) systolic function by tissue doppler

    Participants will undergo Ultrasound of the heart, measuring LV systolic tissue Doppler (meter/second) at start and end of study.

    Measured: Day 2

  41. Cardiac function. Echocardiography - Left ventricular (LV) systolic function by speckle tracking

    Participants will undergo Ultrasound of the heart, measuring LV global, longitudinal 2D speckle tracking (%) at start and end of study.

    Measured: Day 2

  42. Cardiac function. Echocardiography - Left ventricular (LV) diastolic function by tissue doppler

    Participants will undergo Ultrasound of the heart, measuring the ratio of doppler transmitral flow (E) and tissue doppler derived early diastolic velocity (E merk) (without any unit) at start and end of study.

    Measured: Day 2

  43. Cardiac function. Echocardiography - Left ventricular (LV) diastolic function by isovolumic relaxation time (IVRT)

    Participants will undergo Ultrasound of the heart, measuring IVRT in milliseconds (ms) at start and end of study.

    Measured: Day 2

  44. Cardiac function. Electro cardiogram (ECG), heart rate (HR)

    Participants will undergo ECG, measuring HR beats per minute at start and end of study.

    Measured: Day 2

  45. Cardiac function. Electro cardiogram (ECG) - QRS duration

    Participants will undergo ECG, measuring QRS by milliseconds at start and end of study.

    Measured: Day 2

  46. Cardiac function. Electro cardiogram (ECG) - PR interval

    Participants will undergo ECG, measuring PR interval by milliseconds at start and end of study.

    Measured: Day 2

  47. Cardiac function. Electro cardiogram (ECG) - QT time

    Participants will undergo ECG, measuring PR interval by milliseconds at start and end of study.

    Measured: Day 2

  48. Cardiac function. Electro cardiogram (ECG) - QT time corrected

    Participants will undergo ECG, measuring QT time corrected by milliseconds at start and end of study.

    Measured: Day 2

  49. Cardiac function. Electro cardiogram (ECG) - QRS axis

    Participants will undergo ECG, measuring QRS axis in grades at start and end of study.

    Measured: Day 2

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 May 2019
  2. Study start1 Feb 2021
  3. Primary completion30 Mar 2022
  4. Study completion30 Apr 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03963453
Sponsor's protocol ID
2019/260 part II
Status last verified
Dec 2021
First submitted
9 May 2019
Last updated
5 Mar 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

An observational study of Muscular Dystrophy, Duchenne, run by Haukeland University Hospital at 1 site in Norway.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Norway. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Physical Activity Level (time frame: Change from Baseline Physical Activity level at 12 months.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03963453.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2025.

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