Regular Physical Exercise in Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne Clinical Trial

Official title:

To Train -or Not to Train? The Role of Physical Exercise as Part of Management in Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03963453
• Other study ID #: 2019/260 part II
• Status: Completed
• Start date: February 1, 2021
• Est. completion date: April 30, 2022

Study information

• Verified date: December 2021
• Source: Haukeland University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: April 30, 2022
• Est. primary completion date: March 30, 2022
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Other:
• Physical Exercise
Physical exercise are prescribed to be performed three times per week for a year

Locations

Country	Name	City	State
Norway	Department of Physiotherapy. Haukeland University Hospital	Bergen

Sponsors (2)

Lead Sponsor	Collaborator
Haukeland University Hospital	Extrastiftelsen

Country where clinical trial is conducted

Norway, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).	Change from Baseline Physical Activity level at 12 months.
Secondary	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: "Spare time activities". Score "no activity" being a 1, "7 times or more" being a 5.; "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).; 9. 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).	Change from Baseline PAQ-C score at 12 months
Secondary	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.	Day 1 (daily for seven days)
Secondary	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.	Change from Baseline quality of life at 12 months
Secondary	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.	Change from Baseline functioning at 12 months
Secondary	"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	Change from Baseline functioning at 12 months
Secondary	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.	Change from Baseline muscular strength at 12 months
Secondary	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.	Change from Baseline muscular strength at 12 months
Secondary	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.	Change from Baseline physical capacity at 12 months
Secondary	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.	Day 1, day 3 and day 5.
Secondary	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.	Change from Baseline FVC at 12 months
Secondary	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.	Change from Baseline FEV1 at 12 months
Secondary	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.	Change from Baseline FEV1/FVC ratio at 12 months.
Secondary	Lung function - Peak Expiratory Flow (PEF).	Spirometry assessment. The maximal airflow achieved during expiration maneuver, measured as liters per second.	Change from Baseline PEF at 12 months.
Secondary	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.	Change from Baseline SVC at 12 months
Secondary	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.	Change from Baseline MIP at 12 months
Secondary	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.	Change from Baseline MEP at 12 months
Secondary	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.	Change from Baseline PCF at 12 months
Secondary	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	Change from baseline body fat tissue mass at 12 months
Secondary	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	Change from baseline body fat tissue mass at 12 months
Secondary	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	Change from baseline body fat tissue mass at 12 months
Secondary	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	Change from baseline body fat tissue mass at 12 months
Secondary	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	Change from baseline body lean tissue mass at 12 months
Secondary	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	Change from baseline body lean tissue mass at 12 months
Secondary	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	Change from baseline body lean tissue mass at 12 months
Secondary	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	Change from baseline body lean tissue mass at 12 months
Secondary	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	Change from Baseline bone mineral density at 12 months
Secondary	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	Change from Baseline bone mineral density at 12 months
Secondary	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	Change from Baseline bone mineral density at 12 months
Secondary	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	Change from Baseline bone mineral density at 12 months
Secondary	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.	Change from Baseline bone mineral density at 12 months
Secondary	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.	Change from Baseline bone mineral density at 12 months
Secondary	Anthropometric measure - age	The participants age measured in years of age will be registered at start and end of study period	Day 1
Secondary	Anthropometric measure - body height	The Participant's height measured in centimeter (cm) will be registered at each hospital visit during study period.	Day 1
Secondary	Anthropometric measure - body weight	The Participant's body weight measured as kilograms will be measured and registered at each hospital visit during study period	Day 1
Secondary	Anthropometric measure - Body Mass Index (BMI)	The Participant's BMI will be calculated by (kg/m2). at each hospital visit during the study period.	Day 1
Secondary	Cardiac function - Blood Pressure	The participants systolic and diastolic blood pressure will be registered (mmHg) at start and end of study period	Day 2
Secondary	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.	Day 2
Secondary	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.	Day 2
Secondary	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.	Day 2
Secondary	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.	Day 2
Secondary	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.	Day 2
Secondary	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.	Day 2
Secondary	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.	Day 2
Secondary	Cardiac function. Electro cardiogram (ECG), heart rate (HR)	Participants will undergo ECG, measuring HR beats per minute at start and end of study.	Day 2
Secondary	Cardiac function. Electro cardiogram (ECG) - QRS duration	Participants will undergo ECG, measuring QRS by milliseconds at start and end of study.	Day 2
Secondary	Cardiac function. Electro cardiogram (ECG) - PR interval	Participants will undergo ECG, measuring PR interval by milliseconds at start and end of study.	Day 2
Secondary	Cardiac function. Electro cardiogram (ECG) - QT time	Participants will undergo ECG, measuring PR interval by milliseconds at start and end of study.	Day 2
Secondary	Cardiac function. Electro cardiogram (ECG) - QT time corrected	Participants will undergo ECG, measuring QT time corrected by milliseconds at start and end of study.	Day 2
Secondary	Cardiac function. Electro cardiogram (ECG) - QRS axis	Participants will undergo ECG, measuring QRS axis in grades at start and end of study.	Day 2