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| ID | Type | Description | Link |
|---|---|---|---|
| 2010-023744-33 | EudraCT Number | ||
| U01NS061799 | U.S. NIH Grant/Contract | View source | |
| 46102316 | Registry Identifier | ISRCTN |
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| Name | Class |
|---|---|
| Newcastle University | OTHER |
| University Medical Center Freiburg | OTHER |
| National Institute of Neurological Disorders and Stroke (NINDS) | NIH |
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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.
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:
The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Daily prednisone | Experimental | daily prednisone (0.75 mg/kg/day) |
|
| Intermittent prednisone | Experimental | intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) |
|
| Daily deflazacort | Experimental | daily deflazacort (0.9 mg/kg/day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Prednisone | Drug | daily prednisone (0.75 mg/kg/day) tablets for 36-60 months |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| Rise From the Floor Velocity | Reciprocal of time to rise from the floor | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| 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. | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Robert C. Griggs, MD | University of Rochester | Principal Investigator |
| Kate Bushby, MD | Newcastle University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Los Angeles (UCLA) Medical Center | Los Angeles | California | 90095 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 8170484 | Background | Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C. Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle Nerve. 1994 Apr;17(4):386-91. doi: 10.1002/mus.880170405. | |
| 14987333 | Background | Atkinson MJ, Sinha A, Hass SL, Colman SS, Kumar RN, Brod M, Rowland CR. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004 Feb 26;2:12. doi: 10.1186/1477-7525-2-12. |
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229 were screened for eligibility. 33 failed screening
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| ID | Title | Description |
|---|---|---|
| FG000 | Daily Prednisone | daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months |
| FG001 | Daily Deflazacort | daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 18, 2019 | May 11, 2022 |
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| Prednisone |
| Drug |
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months |
|
| Deflazacort | Drug | daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months |
|
| Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| 6 Minute Walk Test | Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36. | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| 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. | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| 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. | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| 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. | 3 years |
| Heart Rate | Measured by trans-thoracic echocardiogram and 12-lead ECG. | 36 months |
| 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. | Average of Months 12, 24, and 36 visits |
| 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. | Average of Months 12, 24, and 36 visits |
| Left Ventricular Ejection Fraction Percent | Measured by trans-thoracic echocardiogram and 12-lead ECG. | 36 months |
| Fractional Shortening Percent | Measured by trans-thoracic echocardiogram and 12-lead ECG. | 36 months |
| PR Interval | Measured by trans-thoracic echocardiogram and 12-lead ECG. | 36 months |
| Participant Weight | 36 months |
| Participant Height | 36 months |
| Participant Body Mass Index | 36 months |
| University of California Davis Medical Center |
| Sacramento |
| California |
| 95817 |
| United States |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States |
| Nemours Children's Hospital | Orlando | Florida | 32827 | United States |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60614 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of New Mexico Health Science Center | Albuquerque | New Mexico | 87131 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Vanderbilt Children's Hospital | Nashville | Tennessee | 37232 | United States |
| University of Utah Medical Center | Salt Lake City | Utah | 84132 | United States |
| Alberta Children's Hospital | Calgary | Alberta | T3B 6A8 | Canada |
| Children's Hospital London Health Sciences Centre | London | Ontario | N6A 4G5 | Canada |
| Children's Hospital of Eastern Ontario (CHEO) | Ottawa | Ontario | K1H 8L1 | Canada |
| Children's Hospital, Technical University Dresden | Dresden | 01307 | Germany |
| University Hospital of Essen | Essen | 45122 | Germany |
| University Medical Center Freiburg | Freiburg im Breisgau | 79110 | Germany |
| Children's University Hospital, Göttingen | Göttingen | 37075 | Germany |
| University of Messina AOU Policlinico Gaetano Martino | Messina | 98125 | Italy |
| C. Besta Neurological Institute Foundation | Milan | 20133 | Italy |
| University of Padova School of Medicine | Padova | 35128 | Italy |
| Neuromuscular Center University of Turin | Torino | 10126 | Italy |
| The General Infirmary at Leeds | Leeds | England | LS2 9NS | United Kingdom |
| Greater Glasgow and Clyde NHS Yorkhill Hospital | Glasgow | Scotland | G3 8SJ | United Kingdom |
| Heart of England NHS Foundation Trust Birmingham Heartland's Hospital | Birmingham | B9 5SS | United Kingdom |
| Alder Hey Children's Hospital NHS Trust | Liverpool | L12 2AP | United Kingdom |
| Great Ormond Street Hospital for Children NHS Trust | London | WC1N 3JH | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | M13 9WL | United Kingdom |
| Institute of Human Genetics, International Centre for Life | Newcastle upon Tyne | NE1 3BZ | United Kingdom |
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| 42249273 | Derived | Naveed A, Recinos E, Chow D, Degan C, Tsonaka R, Guglieri M, Al-Khalili Szigyarto C, Spitali P, van der Burgt YEM, Diaz-Manera J, Dang UJ; FOR-DMD investigators of the Muscle Study Group; Hathout Y. Quantitative tandem mass tag-based serum proteomics for longitudinal biomarker monitoring in Duchenne muscular dystrophy. Clin Proteomics. 2026 Jun 6. doi: 10.1186/s12014-026-09613-4. Online ahead of print. |
| 42202243 | Derived | Clemens PR, Berglund A, Schiava M, James MK, McDermott MP, Bushby K, Lampa E, Rochford ETJ, Ward LM, Griggs RC, Hoffman EP, Guglieri M. Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial. Neurology. 2026 Jul 14;107(1):e214756. doi: 10.1212/WNL.0000000000214756. Epub 2026 May 27. |
| 42139655 | Derived | Gadaleta G, Geagan C, Schiava M, Riguzzi P, Hendriksen J, Campbell C, McDermott MP, Martens WB, Gregory SJ, Mongini TE, Griggs RC, Guglieri M; FOR-DMD Investigators of the Muscle Study Group. Neurobehavioral Profiles in Young Steroid-Naive Boys With Duchenne Muscular Dystrophy: A Baseline Data Analysis From the FOR-DMD Trial. Neurology. 2026 Jun 9;106(11):e218094. doi: 10.1212/WNL.0000000000218094. Epub 2026 May 15. |
| 35381069 | Derived | Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Speed C, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Griggs RC; FOR-DMD Investigators of the Muscle Study Group; Straub V, van Ruiten H, Childs AM, Ciafaloni E, Shieh PB, Spinty S, Maggi L, Baranello G, Butterfield RJ, Horrocks IA, Roper H, Alhaswani Z, Flanigan KM, Kuntz NL, Manzur A, Darras BT, Kang PB, Morrison L, Krzesniak-Swinarska M, Mah JK, Mongini TE, Ricci F, von der Hagen M, Finkel RS, O'Reardon K, Wicklund M, Kumar A, McDonald CM, Han JJ, Joyce N, Henricson EK, Schara-Schmidt U, Gangfuss A, Wilichowski E, Barohn RJ, Statland JM, Campbell C, Vita G, Vita GL, Howard JF Jr, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T. Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA. 2022 Apr 19;327(15):1456-1468. doi: 10.1001/jama.2022.4315. |
| 34857536 | Derived | Schiava M, Amos R, VanRuiten H, McDermott MP, Martens WB, Gregory S, Mayhew A, McColl E, Tawil R, Willis T, Bushby K, Griggs RC, Guglieri M; FOR-DMD Investigators of the Muscle Study Group. Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy. Neurology. 2022 Jan 24;98(4):e390-e401. doi: 10.1212/WNL.0000000000013122. |
| FG002 | 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 |
| COMPLETED |
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| NOT COMPLETED |
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Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Daily Prednisone | daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months |
| BG001 | Daily Deflazacort | daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months |
| BG002 | 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 |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
| |||||||||||
| Mean weight | Mean | Standard Deviation | kg |
| ||||||||||
| Mean height | Data was not collected for one participant in the intermittent prednisone arm. | Mean | Standard Deviation | cm |
| |||||||||
| Mean body mass index | Data was not collected for one participant in the intermittent prednisone arm. | Mean | Standard Deviation | kg/m^2 |
| |||||||||
| Mean rise from floor velocity | Mean | Standard Deviation | rise/sec |
| ||||||||||
| Mean 10 meter walk/run velocity | Mean | Standard Deviation | 10 m/s |
| ||||||||||
| Mean NSAA total 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. | Mean | Standard Deviation | units on a scale |
| |||||||||
| Mean Six-minute walk test distance | Data was not collected for two participants in the daily prednisone arm and one participant in the intermittent prednisone arm. | Mean | Standard Deviation | meters |
| |||||||||
| Mean forced vital capacity | Mean | Standard Deviation | liters |
| ||||||||||
| Mean percentage of predicted normal forced vital capacity | Data was not collected for one participant in the intermittent prednisone arm. | Mean | Standard Deviation | percentage predicted normal |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | 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. | Posted | Least Squares Mean | Standard Error | liters | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
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| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Rise From the Floor Velocity | Reciprocal of time to rise from the floor | 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. | Posted | Least Squares Mean | Standard Error | rise/sec | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
|
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| 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. | 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. | Posted | Least Squares Mean | Standard Error | score on a scale | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Least Squares Mean | Standard Error | score on a scale | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 6 Minute Walk Test | Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36. | 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. | Posted | Least Squares Mean | Standard Error | meters | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Mean | Standard Deviation | degrees | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Mean | Standard Deviation | degrees | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | Posted | Count of Participants | Participants | 3 years |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Heart Rate | Measured by trans-thoracic echocardiogram and 12-lead ECG. | Data was not collected on 1 participant in the daily prednisone arm, and 2 participants in the intermittent prednisone arm. | Posted | Least Squares Mean | Standard Error | bpm | 36 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | 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. | Posted | Least Squares Mean | Standard Error | score on a scale | Average of Months 12, 24, and 36 visits |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. | Only children over the age of 5 self-reported quality of life. | Posted | Least Squares Mean | Standard Error | score on a scale | Average of Months 12, 24, and 36 visits |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Left Ventricular Ejection Fraction Percent | Measured by trans-thoracic echocardiogram and 12-lead ECG. | 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. | Posted | Least Squares Mean | Standard Error | percentage of ejection fraction | 36 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Fractional Shortening Percent | Measured by trans-thoracic echocardiogram and 12-lead ECG. | 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. | Posted | Least Squares Mean | Standard Error | percentage of fractional shortening | 36 months |
|
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| Secondary | PR Interval | Measured by trans-thoracic echocardiogram and 12-lead ECG. | Data was not collected on 3 participants in the daily prednisone arm, and 3 participants in the intermittent prednisone arm. | Posted | Least Squares Mean | Standard Error | milliseconds | 36 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Participant Weight | Posted | Least Squares Mean | Standard Error | kilograms | 36 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Participant Height | Posted | Least Squares Mean | Standard Error | centimeters | 36 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Participant Body Mass Index | Posted | Least Squares Mean | Standard Error | kilograms/square meter | 36 months |
|
|
36 months
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Daily Prednisone | daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months | 0 | 65 | 5 | 65 | 61 | 65 |
| EG001 | Daily Deflazacort | daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months | 0 | 65 | 10 | 65 | 58 | 65 |
| EG002 | 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 | 0 | 66 | 5 | 66 | 55 | 66 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| adenotonsillectomy | General disorders | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Fetal Incontinence | Renal and urinary disorders | Systematic Assessment |
| ||
| mildly increase urine calcium/creatinine ratio | Renal and urinary disorders | Systematic Assessment |
| ||
| Intervertebral disc compression | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| lower respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Tentomy | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Appendicitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Bone fracture | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Chromaturia | Renal and urinary disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Ear infection | Infections and infestations | Systematic Assessment |
| ||
| Fall | General disorders | Systematic Assessment |
| ||
| Gastrointestinal infection | Infections and infestations | Systematic Assessment |
| ||
| Joint pain | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Lymphadenopathy | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Myoglobinuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Tonsillitis | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Oral Infection | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Circumsion | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Epistaxis | General disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Head injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Orchidopexy | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Umbilical hernia repair | Surgical and medical procedures | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cushingoid | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Cataracts | Eye disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal pain upper | Gastrointestinal disorders | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastroenteritis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal discomfort | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastrointestinal disorders | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Malaise | General disorders | Systematic Assessment |
| ||
| Ear infection | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Ligament sprain | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Abnormal weight gain | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Vitamin D deficiency | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Muscle spasms | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Intervertebral disc space narrowing | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Abnormal behavior | Psychiatric disorders | Systematic Assessment |
| ||
| Mood altered | Psychiatric disorders | Systematic Assessment |
| ||
| Aggression | Psychiatric disorders | Systematic Assessment |
| ||
| Insomnia | Psychiatric disorders | Systematic Assessment |
| ||
| Psychomotor hyperactivity | Psychiatric disorders | Systematic Assessment |
| ||
| Pollakiuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Naspharyngitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Upper respiratory tract infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rhinitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Hypertrichosis | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
The PI cannot publish any manuscript without review and written consent of the FOR-DMD study steering committee.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Robert Griggs | University of Rochester | 585-275-6072 | robert_griggs@urmc.rochester.edu |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 16, 2021 | May 11, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D009136 | Muscular Dystrophies |
| D009468 | Neuromuscular Diseases |
| ID | Term |
|---|---|
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| D011241 | Prednisone |
| C021988 | deflazacort |
| ID | Term |
|---|---|
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
Not provided
Not provided
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| race other than Caucasian |
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| United States |
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| Italy |
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| United Kingdom |
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| Germany |
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| Superiority |
| Mixed Models Analysis | 0.1518 | Superiority |
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| Units | Counts |
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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
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