Duchenne Muscular Dystrophy
Conditions
Keywords
prednisone, deflazacort, muscular dystrophy, neuromuscular disease, multi-center
Brief summary
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.
Detailed description
Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up. Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems. Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all. The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects: 1. Prednisone 0.75mg/kg/day 2. Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment 3. Deflazacort 0.9mg/kg/day. The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.
Interventions
DRUGPrednisone
daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
DRUGDeflazacort
daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
Sponsors
Newcastle University
University Medical Center Freiburg
National Institute of Neurological Disorders and Stroke (NINDS)
University of Rochester
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
MALE
Age
4 Years to 7 Years
Healthy volunteers
No
Inclusion criteria
* Evidence of signed and dated informed consent form. * Confirmed diagnosis of Duchenne muscular dystrophy * Age greater than or equal to 4 years and less than 8 years old * Ability to rise independently from floor, from supine to standing * Willingness and ability to comply with scheduled visits, drug administration plan and study procedures * Ability to maintain reproducible FVC measurements.
Exclusion criteria
* History of major renal or hepatic impairment, immunosuppression or other contraindications to corticosteroid therapy. * History of chronic systemic fungal or viral infections. Acute bacterial infection(including TB) would exclude from enrolment until the infection had been appropriately treated and resolved. * Diabetes mellitus. * Idiopathic hypercalcuria. * Lack of chicken pox immunity and refusal to undergo immunization. * Evidence of symptomatic cardiomyopathy at screening assessment (one to three months prior to the baseline visit). Asymptomatic cardiac abnormality on investigation would not be an exclusion. * Current or previous treatment (greater than four consecutive weeks of oral therapy) with corticosteroids or other immunosuppressive treatments for DMD or other recurrent indications (e.g., asthma), unless approved by FOR-DMD Team (i.e., concurrent participation in another allowed DMD trial). * Inability to take tablets, as assessed by the site investigator by the end of the screening period (the screening period ranges from one to three months prior to the baseline visit). * Allergy/sensitivity to study drugs or their formulations including lactose and/or sucrose intolerance. * Severe behavioral problems, including severe autism. * Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow up will be correctly completed or impair the assessment of study results, in the judgment of the site investigator. * Weight of less than 13 kilograms. * Exposure to any investigational drug currently or within 3 months prior to start of study treatment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Forced Vital Capacity | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test. |
| Rise From the Floor Velocity | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Reciprocal of time to rise from the floor |
| Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Range of Motion (Goniometry) of Right Ankle | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. |
| Number of Participants Who Tolerated the Regimen | 3 years | The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication. |
| Heart Rate | 36 months | Measured by trans-thoracic echocardiogram and 12-lead ECG. |
| Quality of Life - Parent | Average of Months 12, 24, and 36 visits | Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child. |
| Quality of Life- Child | Average of Months 12, 24, and 36 visits | Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life. |
| North Star Ambulatory Assessment (NSAA) Score | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects. The activities are graded as follows: 2 - Normal - no obvious modification of activity 1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function. |
| Fractional Shortening Percent | 36 months | Measured by trans-thoracic echocardiogram and 12-lead ECG. |
| PR Interval | 36 months | Measured by trans-thoracic echocardiogram and 12-lead ECG. |
| Participant Weight | 36 months | — |
| Participant Height | 36 months | — |
| Participant Body Mass Index | 36 months | — |
| Left Ventricular Ejection Fraction Percent | 36 months | Measured by trans-thoracic echocardiogram and 12-lead ECG. |
| 6 Minute Walk Test | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36. |
| Range of Motion (Goniometry) of Left Ankle | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. |
Countries
Canada, Germany, Italy, United Kingdom, United States
Participant flow
Pre-assignment details
229 were screened for eligibility. 33 failed screening
Participants by arm
| Arm | Count |
|---|---|
| Daily Prednisone daily prednisone (0.75 mg/kg/day)
Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months | 65 |
| Daily Deflazacort daily deflazacort (0.9 mg/kg/day
Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months | 65 |
| Intermittent Prednisone intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
Prednisone: intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months | 66 |
| Total | 196 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 1 | 1 | 1 |
| Overall Study | Lost to Follow-up | 0 | 3 | 2 |
| Overall Study | Protocol Violation | 2 | 2 | 0 |
| Overall Study | Withdrawal by Subject | 8 | 5 | 7 |
Baseline characteristics
| Characteristic | Total | Daily Deflazacort | Intermittent Prednisone | Daily Prednisone |
|---|---|---|---|---|
| Age, Continuous | 5.9 years STANDARD_DEVIATION 1 | 5.8 years STANDARD_DEVIATION 1 | 5.9 years STANDARD_DEVIATION 1.1 | 5.8 years STANDARD_DEVIATION 1 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 31 Participants | 9 Participants | 13 Participants | 9 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 165 Participants | 56 Participants | 53 Participants | 56 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Mean 10 meter walk/run velocity | 0.17 10 m/s STANDARD_DEVIATION 0.04 | 0.17 10 m/s STANDARD_DEVIATION 0.04 | 0.19 10 m/s STANDARD_DEVIATION 0.04 | 0.17 10 m/s STANDARD_DEVIATION 0.04 |
| Mean body mass index | 16.4 kg/m^2 STANDARD_DEVIATION 1.6 | 16.5 kg/m^2 STANDARD_DEVIATION 2 | 16.4 kg/m^2 STANDARD_DEVIATION 1.6 | 16.5 kg/m^2 STANDARD_DEVIATION 1.3 |
| Mean forced vital capacity | 1.1 liters STANDARD_DEVIATION 0.4 | 1.1 liters STANDARD_DEVIATION 0.4 | 1.1 liters STANDARD_DEVIATION 0.4 | 1.0 liters STANDARD_DEVIATION 0.4 |
| Mean height | 110.1 cm STANDARD_DEVIATION 7.2 | 109.5 cm STANDARD_DEVIATION 7.6 | 110.9 cm STANDARD_DEVIATION 7.1 | 109.9 cm STANDARD_DEVIATION 7 |
| Mean NSAA total score | 21.4 units on a scale STANDARD_DEVIATION 5.4 | 21.1 units on a scale STANDARD_DEVIATION 5.7 | 21.1 units on a scale STANDARD_DEVIATION 4.9 | 22.0 units on a scale STANDARD_DEVIATION 5.7 |
| Mean percentage of predicted normal forced vital capacity | 86.9 percentage predicted normal STANDARD_DEVIATION 33.3 | 87.2 percentage predicted normal STANDARD_DEVIATION 36.2 | 88.1 percentage predicted normal STANDARD_DEVIATION 34 | 85.4 percentage predicted normal STANDARD_DEVIATION 29.8 |
| Mean rise from floor velocity | 0.18 rise/sec STANDARD_DEVIATION 0.08 | 0.19 rise/sec STANDARD_DEVIATION 0.07 | 0.18 rise/sec STANDARD_DEVIATION 0.08 | 0.19 rise/sec STANDARD_DEVIATION 0.09 |
| Mean Six-minute walk test distance | 334.7 meters STANDARD_DEVIATION 62.2 | 330.4 meters STANDARD_DEVIATION 65.1 | 331.6 meters STANDARD_DEVIATION 63.6 | 342.4 meters STANDARD_DEVIATION 58 |
| Mean weight | 20.0 kg STANDARD_DEVIATION 3.6 | 19.9 kg STANDARD_DEVIATION 4.3 | 20.2 kg STANDARD_DEVIATION 3.5 | 20.0 kg STANDARD_DEVIATION 3 |
| Race/Ethnicity, Customized Caucasian | 166 Participants | 58 Participants | 49 Participants | 59 Participants |
| Race/Ethnicity, Customized race other than Caucasian | 30 Participants | 7 Participants | 17 Participants | 6 Participants |
| Region of Enrollment Canada | 13 participants | 4 participants | 4 participants | 5 participants |
| Region of Enrollment Germany | 20 participants | 7 participants | 7 participants | 6 participants |
| Region of Enrollment Italy | 22 participants | 7 participants | 7 participants | 8 participants |
| Region of Enrollment United Kingdom | 59 participants | 20 participants | 20 participants | 19 participants |
| Region of Enrollment United States | 82 participants | 27 participants | 28 participants | 27 participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 196 Participants | 65 Participants | 66 Participants | 65 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 65 | 0 / 65 | 0 / 66 |
| other Total, other adverse events | 61 / 65 | 58 / 65 | 55 / 66 |
| serious Total, serious adverse events | 5 / 65 | 10 / 65 | 5 / 66 |
Outcome results
Primary
Forced Vital Capacity
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Population: Data was not collected on 4 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Forced Vital Capacity | 1.4 liters | Standard Error 0.03 |
| Daily Deflazacort | Forced Vital Capacity | 1.4 liters | Standard Error 0.03 |
| Intermittent Prednisone | Forced Vital Capacity | 1.5 liters | Standard Error 0.03 |
p-value: 0.3904Mixed Models Analysis
p-value: 0.569Mixed Models Analysis
p-value: 0.1518Mixed Models Analysis
Primary
Rise From the Floor Velocity
Reciprocal of time to rise from the floor
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Population: Data was not collected on 3 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Rise From the Floor Velocity | 0.24 rise/sec | Standard Error 0.01 |
| Daily Deflazacort | Rise From the Floor Velocity | 0.24 rise/sec | Standard Error 0.01 |
| Intermittent Prednisone | Rise From the Floor Velocity | 0.18 rise/sec | Standard Error 0.01 |
p-value: 0.7365Mixed Models Analysis
p-value: <0.0001Mixed Models Analysis
p-value: <0.0001Mixed Models Analysis
Primary
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Population: Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | 71.2 score on a scale | Standard Error 2.3 |
| Daily Deflazacort | Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | 67.8 score on a scale | Standard Error 2.3 |
| Intermittent Prednisone | Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | 65.1 score on a scale | Standard Error 2.3 |
p-value: 0.2456Mixed Models Analysis
p-value: 0.0369Mixed Models Analysis
p-value: 0.3589Mixed Models Analysis
Secondary
6 Minute Walk Test
Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Population: Data was not collected on 5 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 2 participant in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | 6 Minute Walk Test | 384.95 meters | Standard Error 9.6 |
| Daily Deflazacort | 6 Minute Walk Test | 384.17 meters | Standard Error 9.7 |
| Intermittent Prednisone | 6 Minute Walk Test | 346.81 meters | Standard Error 9.4 |
p-value: 0.9532Mixed Models Analysis
p-value: 0.004Mixed Models Analysis
p-value: 0.0046Mixed Models Analysis
Secondary
Fractional Shortening Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
Population: Data was not collected on 4 participants in the daily prednisone arm, 4 participants in the deflazacort arm and 8 participants in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Fractional Shortening Percent | 33.74 percentage of fractional shortening | Standard Error 0.81 |
| Daily Deflazacort | Fractional Shortening Percent | 34.01 percentage of fractional shortening | Standard Error 0.87 |
| Intermittent Prednisone | Fractional Shortening Percent | 34.33 percentage of fractional shortening | Standard Error 0.87 |
p-value: 0.8138Mixed Models Analysis
p-value: 0.5995Mixed Models Analysis
p-value: 0.7616Mixed Models Analysis
Secondary
Heart Rate
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
Population: Data was not collected on 1 participant in the daily prednisone arm, and 2 participants in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Heart Rate | 94.10 bpm | Standard Error 1.96 |
| Daily Deflazacort | Heart Rate | 93.52 bpm | Standard Error 2.09 |
| Intermittent Prednisone | Heart Rate | 91.65 bpm | Standard Error 2.1 |
p-value: 0.8345Mixed Models Analysis
p-value: 0.3762Mixed Models Analysis
p-value: 0.5059Mixed Models Analysis
Secondary
Left Ventricular Ejection Fraction Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
Population: Data was not collected on 2 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 5 participants in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Left Ventricular Ejection Fraction Percent | 61.88 percentage of ejection fraction | Standard Error 1.09 |
| Daily Deflazacort | Left Ventricular Ejection Fraction Percent | 62.65 percentage of ejection fraction | Standard Error 1.17 |
| Intermittent Prednisone | Left Ventricular Ejection Fraction Percent | 62.45 percentage of ejection fraction | Standard Error 1.23 |
p-value: 0.6161Mixed Models Analysis
p-value: 0.7302Mixed Models Analysis
p-value: 0.9007Mixed Models Analysis
Secondary
North Star Ambulatory Assessment (NSAA) Score
The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects. The activities are graded as follows: 2 - Normal - no obvious modification of activity 1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Population: Data was not collected on 3 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | North Star Ambulatory Assessment (NSAA) Score | 23.7 score on a scale | Standard Error 0.6 |
| Daily Deflazacort | North Star Ambulatory Assessment (NSAA) Score | 24.0 score on a scale | Standard Error 0.6 |
| Intermittent Prednisone | North Star Ambulatory Assessment (NSAA) Score | 20.7 score on a scale | Standard Error 0.6 |
p-value: 0.7335Mixed Models Analysis
p-value: 0.0004Mixed Models Analysis
p-value: 0.0001Mixed Models Analysis
Secondary
Number of Participants Who Tolerated the Regimen
The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication.
Time frame: 3 years
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Daily Prednisone | Number of Participants Who Tolerated the Regimen | 36 Participants |
| Daily Deflazacort | Number of Participants Who Tolerated the Regimen | 36 Participants |
| Intermittent Prednisone | Number of Participants Who Tolerated the Regimen | 37 Participants |
Secondary
Participant Body Mass Index
Time frame: 36 months
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Participant Body Mass Index | 18.9 kilograms/square meter | Standard Error 0.27 |
| Daily Deflazacort | Participant Body Mass Index | 18.3 kilograms/square meter | Standard Error 0.26 |
| Intermittent Prednisone | Participant Body Mass Index | 18.1 kilograms/square meter | Standard Error 0.25 |
p-value: 0.085398.3% CI: [-1.4845, 0.2434]Mixed Models Analysis
p-value: 0.014398.3% CI: [-1.7292, -0.0229]Mixed Models Analysis
p-value: 0.473198.3% CI: [-1.1117, 0.6007]Mixed Models Analysis
Secondary
Participant Height
Time frame: 36 months
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Participant Height | 116.8 centimeters | Standard Error 0.26 |
| Daily Deflazacort | Participant Height | 115.3 centimeters | Standard Error 0.27 |
| Intermittent Prednisone | Participant Height | 119.9 centimeters | Standard Error 0.26 |
p-value: <0.000198.3% CI: [-2.3242, -0.6358]Mixed Models Analysis
p-value: <0.000198.3% CI: [2.2222, 3.8857]Mixed Models Analysis
p-value: <0.000198.3% CI: [3.6941, 5.3738]Mixed Models Analysis
Secondary
Participant Weight
Time frame: 36 months
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Participant Weight | 26.3 kilograms | Standard Error 0.36 |
| Daily Deflazacort | Participant Weight | 24.9 kilograms | Standard Error 0.36 |
| Intermittent Prednisone | Participant Weight | 26.3 kilograms | Standard Error 0.35 |
p-value: 0.004198.3% CI: [-2.6611, -0.2479]Mixed Models Analysis
p-value: 0.907698.3% CI: [-1.248, 1.1331]Mixed Models Analysis
p-value: 0.005498.3% CI: [0.2014, 2.5927]Mixed Models Analysis
Secondary
PR Interval
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time frame: 36 months
Population: Data was not collected on 3 participants in the daily prednisone arm, and 3 participants in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | PR Interval | 115.59 milliseconds | Standard Error 2.3 |
| Daily Deflazacort | PR Interval | 116.87 milliseconds | Standard Error 2.33 |
| Intermittent Prednisone | PR Interval | 117.90 milliseconds | Standard Error 2.22 |
p-value: 0.68Mixed Models Analysis
p-value: 0.4365Mixed Models Analysis
p-value: 0.7281Mixed Models Analysis
Secondary
Quality of Life- Child
Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life.
Time frame: Average of Months 12, 24, and 36 visits
Population: Only children over the age of 5 self-reported quality of life.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Quality of Life- Child | 67.39 score on a scale | Standard Error 2.16 |
| Daily Deflazacort | Quality of Life- Child | 64.96 score on a scale | Standard Error 2.17 |
| Intermittent Prednisone | Quality of Life- Child | 65.07 score on a scale | Standard Error 2.17 |
p-value: 0.3875Mixed Models Analysis
p-value: 0.424Mixed Models Analysis
p-value: 0.9683Mixed Models Analysis
Secondary
Quality of Life - Parent
Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child.
Time frame: Average of Months 12, 24, and 36 visits
Population: Data was not collected on 3 participants in the daily prednisone arm, 1 participants in the deflazacort arm and 2 participant in the intermittent prednisone arm.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Quality of Life - Parent | 64.88 score on a scale | Standard Error 1.67 |
| Daily Deflazacort | Quality of Life - Parent | 63.71 score on a scale | Standard Error 1.69 |
| Intermittent Prednisone | Quality of Life - Parent | 61.33 score on a scale | Standard Error 1.69 |
p-value: 0.5947Mixed Models Analysis
p-value: 0.1098Mixed Models Analysis
p-value: 0.2803Mixed Models Analysis
Secondary
Range of Motion (Goniometry) of Left Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Population: Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Range of Motion (Goniometry) of Left Ankle | 4.39 degrees | Standard Deviation 0.83 |
| Daily Deflazacort | Range of Motion (Goniometry) of Left Ankle | 3.29 degrees | Standard Deviation 0.85 |
| Intermittent Prednisone | Range of Motion (Goniometry) of Left Ankle | 2.67 degrees | Standard Deviation 0.82 |
p-value: 0.332Mixed Models Analysis
p-value: 0.1248Mixed Models Analysis
p-value: 0.5854Mixed Models Analysis
Secondary
Range of Motion (Goniometry) of Right Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Population: Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Daily Prednisone | Range of Motion (Goniometry) of Right Ankle | 4.05 degrees | Standard Deviation 0.89 |
| Daily Deflazacort | Range of Motion (Goniometry) of Right Ankle | 2.81 degrees | Standard Deviation 0.92 |
| Intermittent Prednisone | Range of Motion (Goniometry) of Right Ankle | 2.29 degrees | Standard Deviation 0.89 |
p-value: 0.30814Mixed Models Analysis
p-value: 0.1405Mixed Models Analysis
p-value: 0.664Mixed Models Analysis