Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD)

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ClinicalTrials.gov Identifier: NCT01603407

Recruitment Status : Completed

First Posted : May 23, 2012

Results First Posted : August 12, 2022

Last Update Posted : August 12, 2022

View this study on the modernized ClinicalTrials.gov

Sponsor:

Collaborators:

Newcastle University

University Medical Center Freiburg

National Institute of Neurological Disorders and Stroke (NINDS)

Information provided by (Responsible Party):

Robert Griggs, MD, University of Rochester

Study Type	Interventional
Study Design	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition	Duchenne Muscular Dystrophy
Interventions	Drug: Prednisone Drug: Deflazacort
Enrollment	196

Participant Flow

Recruitment Details
Pre-assignment Details	229 were screened for eligibility. 33 failed screening


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Period Title: Overall Study
Started	65	65	66
Completed	54	54	56
Not Completed	11	11	10
Reason Not Completed
Adverse Event	1	1	1
Protocol Violation	2	2	0
Withdrawal by Subject	8	5	7
Lost to Follow-up	0	3	2

Baseline Characteristics

Arm/Group Title		Daily Prednisone	Daily Deflazacort	Intermittent Prednisone	Total
Arm/Group Description		daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...	Total of all reporting groups
Arm/Group Description		daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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	Total of all reporting groups
Overall Number of Baseline Participants		65	65	66	196
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Continuous Mean (Standard Deviation) Unit of measure: Years
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
		5.8 (1.0)	5.8 (1.0)	5.9 (1.1)	5.9 (1.0)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
	Female	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Male	65 100.0%	65 100.0%	66 100.0%	196 100.0%
Ethnicity (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
	Hispanic or Latino	9 13.8%	9 13.8%	13 19.7%	31 15.8%
	Not Hispanic or Latino	56 86.2%	56 86.2%	53 80.3%	165 84.2%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%	0 0.0%
Race/Ethnicity, Customized Measure Type: Count of Participants Unit of measure: Participants
Caucasian	Number Analyzed	65 participants	65 participants	66 participants	196 participants
Caucasian		59 90.8%	58 89.2%	49 74.2%	166 84.7%
race other than Caucasian	Number Analyzed	65 participants	65 participants	66 participants	196 participants
race other than Caucasian		6 9.2%	7 10.8%	17 25.8%	30 15.3%
Region of Enrollment Measure Type: Number Unit of measure: Participants
Canada	Number Analyzed	65 participants	65 participants	66 participants	196 participants
Canada		5	4	4	13
United States	Number Analyzed	65 participants	65 participants	66 participants	196 participants
United States		27	27	28	82
Italy	Number Analyzed	65 participants	65 participants	66 participants	196 participants
Italy		8	7	7	22
United Kingdom	Number Analyzed	65 participants	65 participants	66 participants	196 participants
United Kingdom		19	20	20	59
Germany	Number Analyzed	65 participants	65 participants	66 participants	196 participants
Germany		6	7	7	20
Mean weight Mean (Standard Deviation) Unit of measure: Kg
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
		20.0 (3.0)	19.9 (4.3)	20.2 (3.5)	20.0 (3.6)
Mean height ^[1] Mean (Standard Deviation) Unit of measure: Cm
	Number Analyzed	65 participants	65 participants	65 participants	195 participants
		109.9 (7.0)	109.5 (7.6)	110.9 (7.1)	110.1 (7.2)
		[1] Measure Analysis Population Description: Data was not collected for one participant in the intermittent prednisone arm.
Mean body mass index ^[1] Mean (Standard Deviation) Unit of measure: Kg/m^2
	Number Analyzed	65 participants	65 participants	65 participants	195 participants
		16.5 (1.3)	16.5 (2.0)	16.4 (1.6)	16.4 (1.6)
		[1] Measure Analysis Population Description: Data was not collected for one participant in the intermittent prednisone arm.
Mean rise from floor velocity Mean (Standard Deviation) Unit of measure: Rise/sec
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
		0.19 (0.09)	0.19 (0.07)	0.18 (0.08)	0.18 (0.08)
Mean 10 meter walk/run velocity Mean (Standard Deviation) Unit of measure: 10 m/s
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
		0.17 (0.04)	0.17 (0.04)	0.19 (0.04)	0.17 (0.04)
Mean NSAA total score ^[1] Mean (Standard Deviation) Unit of measure: Units on a scale
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
		22.0 (5.7)	21.1 (5.7)	21.1 (4.9)	21.4 (5.4)
		[1] Measure Description: 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 Six-minute walk test distance ^[1] Mean (Standard Deviation) Unit of measure: Meters
	Number Analyzed	63 participants	65 participants	65 participants	193 participants
		342.4 (58.0)	330.4 (65.1)	331.6 (63.6)	334.7 (62.2)
		[1] Measure Analysis Population Description: Data was not collected for two participants in the daily prednisone arm and one participant in the intermittent prednisone arm.
Mean forced vital capacity Mean (Standard Deviation) Unit of measure: Liters
	Number Analyzed	65 participants	65 participants	66 participants	196 participants
		1.0 (0.4)	1.1 (0.4)	1.1 (0.4)	1.1 (0.4)
Mean percentage of predicted normal forced vital capacity ^[1] Mean (Standard Deviation) Unit of measure: Percentage predicted normal
	Number Analyzed	65 participants	65 participants	65 participants	195 participants
		85.4 (29.8)	87.2 (36.2)	88.1 (34.0)	86.9 (33.3)
		[1] Measure Analysis Population Description: Data was not collected for one participant in the intermittent prednisone arm.

Outcome Measures

1.Primary Outcome


Title	Forced Vital Capacity
Description	Forced vital capacity was measured during a spirometry test. Forced ex...
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	61	62	65
Least Squares Mean (Standard Error) Unit of Measure: liters
	1.4 (0.03)	1.4 (0.03)	1.5 (0.03)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.3904
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.5690
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.1518
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

2.Primary Outcome


Title	Rise From the Floor Velocity
Description	Reciprocal of time to rise from the floor
Description	Reciprocal of time to rise from the floor
Time Frame	Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	63	65
Least Squares Mean (Standard Error) Unit of Measure: rise/sec
	0.24 (0.01)	0.24 (0.01)	0.18 (0.01)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.7365
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

3.Primary Outcome


Title	Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
Description	The TSQM Global Satisfaction with Treatment is a 14-item questionnaire...
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	62	65
Least Squares Mean (Standard Error) Unit of Measure: score on a scale
	71.2 (2.3)	67.8 (2.3)	65.1 (2.3)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.2456
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0369
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.3589
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

4.Secondary Outcome


Title	North Star Ambulatory Assessment (NSAA) Score
Description	The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale ...
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	63	65
Least Squares Mean (Standard Error) Unit of Measure: score on a scale
	23.7 (0.6)	24.0 (0.6)	20.7 (0.6)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.7335
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0004
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0001
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

5.Secondary Outcome


Title	6 Minute Walk Test
Description	Measures the total distance walked in 6 minutes averaged over all post...
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	60	63	64
Least Squares Mean (Standard Error) Unit of Measure: meters
	384.95 (9.6)	384.17 (9.7)	346.81 (9.4)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.9532
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0040
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0046
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

6.Secondary Outcome


Title	Range of Motion (Goniometry) of Left Ankle
Description	Range of motion at the ankle joint in dorsiflexion measured in degrees...
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	62	65
Mean (Standard Deviation) Unit of Measure: degrees
	4.39 (0.83)	3.29 (0.85)	2.67 (0.82)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.3320
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.1248
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.5854
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

7.Secondary Outcome


Title	Range of Motion (Goniometry) of Right Ankle
Description	Range of motion at the ankle joint in dorsiflexion measured in degrees...
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	62	65
Mean (Standard Deviation) Unit of Measure: degrees
	4.05 (0.89)	2.81 (0.92)	2.29 (0.89)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.30814
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.1405
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.6640
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

8.Secondary Outcome


Title	Number of Participants Who Tolerated the Regimen
Description	The number of participants who completed 36 months of follow-up on the...
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	65	65	66
Measure Type: Count of Participants Unit of Measure: Participants
	36 55.4%	36 55.4%	37 56.1%

9.Secondary Outcome


Title	Heart Rate
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame	36 months

Outcome Measure Data

Analysis Population Description

Data was not collected on 1 participant in the daily prednisone arm, and 2 participants in the intermittent prednisone arm.


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	64	65	64
Least Squares Mean (Standard Error) Unit of Measure: bpm
	94.10 (1.96)	93.52 (2.09)	91.65 (2.10)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.8345
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.3762
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.5059
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

10.Secondary Outcome


Title	Quality of Life - Parent
Description	Quality of life was measured by parent/guardian self-report for all ch...
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	64	64
Least Squares Mean (Standard Error) Unit of Measure: score on a scale
	64.88 (1.67)	63.71 (1.69)	61.33 (1.69)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.5947
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.1098
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.2803
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

11.Secondary Outcome


Title	Quality of Life- Child
Description	Quality of life was measured by child self-report in children age 5 an...
Description	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

Outcome Measure Data

Analysis Population Description

Only children over the age of 5 self-reported quality of life.


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	54	52	47
Least Squares Mean (Standard Error) Unit of Measure: score on a scale
	67.39 (2.16)	64.96 (2.17)	65.07 (2.17)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.3875
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.4240
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.9683
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

12.Secondary Outcome


Title	Left Ventricular Ejection Fraction Percent
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame	36 months

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	63	62	61
Least Squares Mean (Standard Error) Unit of Measure: percentage of ejection fraction
	61.88 (1.09)	62.65 (1.17)	62.45 (1.23)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.6161
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.7302
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.9007
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

13.Secondary Outcome


Title	Fractional Shortening Percent
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame	36 months

Outcome Measure Data

Analysis Population Description

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/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	61	61	58
Least Squares Mean (Standard Error) Unit of Measure: percentage of fractional shortening
	33.74 (0.81)	34.01 (0.87)	34.33 (0.87)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.8138
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.5995
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.7616
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

14.Secondary Outcome


Title	PR Interval
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Description	Measured by trans-thoracic echocardiogram and 12-lead ECG.
Time Frame	36 months

Outcome Measure Data

Analysis Population Description

Data was not collected on 3 participants in the daily prednisone arm, and 3 participants in the intermittent prednisone arm.


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	65	63
Least Squares Mean (Standard Error) Unit of Measure: milliseconds
	115.59 (2.30)	116.87 (2.33)	117.90 (2.22)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.6800
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.4365
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.7281
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

15.Secondary Outcome


Title	Participant Weight
Description	[Not Specified]
Description	[Not Specified]
Time Frame	36 months

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	62	65
Least Squares Mean (Standard Error) Unit of Measure: kilograms
	26.3 (0.36)	24.9 (0.36)	26.3 (0.35)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0041
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	-1.545
	Confidence Interval	(2-Sided) 98.3% -2.6611 to -0.2479
	Estimation Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.9076
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	0.9076
	Confidence Interval	(2-Sided) 98.3% -1.248 to 1.1331
	Estimation Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0054
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	1.3971
	Confidence Interval	(2-Sided) 98.3% 0.2014 to 2.5927
	Estimation Comments	[Not Specified]

16.Secondary Outcome


Title	Participant Height
Description	[Not Specified]
Description	[Not Specified]
Time Frame	36 months

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	62	65
Least Squares Mean (Standard Error) Unit of Measure: centimeters
	116.8 (0.26)	115.3 (0.27)	119.9 (0.26)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	-1.48
	Confidence Interval	(2-Sided) 98.3% -2.3242 to -0.6358
	Estimation Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	3.054
	Confidence Interval	(2-Sided) 98.3% 2.2222 to 3.8857
	Estimation Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	4.534
	Confidence Interval	(2-Sided) 98.3% 3.6941 to 5.3738
	Estimation Comments	[Not Specified]

17.Secondary Outcome


Title	Participant Body Mass Index
Description	[Not Specified]
Description	[Not Specified]
Time Frame	36 months

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description:	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
Overall Number of Participants Analyzed	62	62	65
Least Squares Mean (Standard Error) Unit of Measure: kilograms/square meter
	18.9 (0.27)	18.3 (0.26)	18.1 (0.25)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Daily Deflazacort
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0853
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	-0.6205
	Confidence Interval	(2-Sided) 98.3% -1.4845 to 0.2434
	Estimation Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Daily Prednisone, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0143
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	-0.876
	Confidence Interval	(2-Sided) 98.3% -1.7292 to -0.0229
	Estimation Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	Daily Deflazacort, Intermittent Prednisone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.4731
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	-0.2555
	Confidence Interval	(2-Sided) 98.3% -1.1117 to 0.6007
	Estimation Comments	[Not Specified]

Adverse Events

Time Frame	36 months
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
Arm/Group Description	daily prednisone (0.75 mg/kg/day) ...	daily deflazacort (0.9 mg/kg/day ...	intermittent prednisone (0.75 mg/kg...
Arm/Group Description	daily prednisone (0.75 mg/kg/day) Prednisone: daily prednisone (0.75 mg/kg/day) tablets for 36-60 months	daily deflazacort (0.9 mg/kg/day Deflazacort: daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months	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
All-Cause Mortality
	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/65 (0.00%)	0/65 (0.00%)	0/66 (0.00%)
Serious Adverse Events Serious Adverse Events
	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	5/65 (7.69%)	10/65 (15.38%)	5/66 (7.58%)
Blood and lymphatic system disorders
Lymphadenopathy ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Tonsillitis ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Gastrointestinal disorders
Abdominal pain ^†	1/65 (1.54%)	0/65 (0.00%)	1/66 (1.52%)
Constipation ^†	1/65 (1.54%)	0/65 (0.00%)	0/66 (0.00%)
Appendicitis ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Diarrhea ^†	0/65 (0.00%)	1/65 (1.54%)	1/66 (1.52%)
Vomiting ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
General disorders
adenotonsillectomy ^†	1/65 (1.54%)	0/65 (0.00%)	0/66 (0.00%)
Fall ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Epistaxis ^†	0/65 (0.00%)	0/65 (0.00%)	1/66 (1.52%)
Pyrexia ^†	0/65 (0.00%)	0/65 (0.00%)	1/66 (1.52%)
Infections and infestations
lower respiratory tract infection ^†	1/65 (1.54%)	0/65 (0.00%)	1/66 (1.52%)
Ear infection ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Gastrointestinal infection ^†	0/65 (0.00%)	1/65 (1.54%)	1/66 (1.52%)
Oral Infection ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Upper respiratory tract infection ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Injury, poisoning and procedural complications
Head injury ^†	0/65 (0.00%)	0/65 (0.00%)	1/66 (1.52%)
Metabolism and nutrition disorders
Joint pain ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Musculoskeletal and connective tissue disorders
Intervertebral disc compression ^†	1/65 (1.54%)	0/65 (0.00%)	0/66 (0.00%)
Rhabdomyolysis ^†	1/65 (1.54%)	1/65 (1.54%)	1/66 (1.52%)
Tentomy ^†	1/65 (1.54%)	1/65 (1.54%)	1/66 (1.52%)
Bone fracture ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Nervous system disorders
Headache ^†	0/65 (0.00%)	0/65 (0.00%)	1/66 (1.52%)
Renal and urinary disorders
Fetal Incontinence ^†	1/65 (1.54%)	0/65 (0.00%)	0/66 (0.00%)
mildly increase urine calcium/creatinine ratio ^†	1/65 (1.54%)	0/65 (0.00%)	0/66 (0.00%)
Chromaturia ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Myoglobinuria ^†	0/65 (0.00%)	1/65 (1.54%)	0/66 (0.00%)
Reproductive system and breast disorders
Circumsion ^†	0/65 (0.00%)	0/65 (0.00%)	1/66 (1.52%)
Orchidopexy ^†	0/65 (0.00%)	0/65 (0.00%)	1/66 (1.52%)
Surgical and medical procedures
Umbilical hernia repair ^†	0/65 (0.00%)	0/65 (0.00%)	1/66 (1.52%)
† Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Daily Prednisone	Daily Deflazacort	Intermittent Prednisone
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	61/65 (93.85%)	58/65 (89.23%)	55/66 (83.33%)
Eye disorders
Cataracts ^†	1/65 (1.54%)	5/65 (7.69%)	0/66 (0.00%)
Gastrointestinal disorders
Vomiting ^†	19/65 (29.23%)	16/65 (24.62%)	15/66 (22.73%)
Diarrhea ^†	9/65 (13.85%)	7/65 (10.77%)	10/66 (15.15%)
Abdominal pain ^†	8/65 (12.31%)	8/65 (12.31%)	8/66 (12.12%)
Abdominal pain upper ^†	10/65 (15.38%)	4/65 (6.15%)	10/66 (15.15%)
Constipation ^†	7/65 (10.77%)	9/65 (13.85%)	7/66 (10.61%)
Nausea ^†	5/65 (7.69%)	4/65 (6.15%)	1/66 (1.52%)
Gastroenteritis ^†	4/65 (6.15%)	3/65 (4.62%)	1/66 (1.52%)
Abdominal discomfort ^†	4/65 (6.15%)	1/65 (1.54%)	2/66 (3.03%)
Gastrointestinal disorders ^†	4/65 (6.15%)	2/65 (3.08%)	0/66 (0.00%)
General disorders
Pyrexia ^†	12/65 (18.46%)	11/65 (16.92%)	10/66 (15.15%)
Malaise ^†	2/65 (3.08%)	4/65 (6.15%)	2/66 (3.03%)
Infections and infestations
Ear infection ^†	4/65 (6.15%)	7/65 (10.77%)	8/66 (12.12%)
Influenza ^†	8/65 (12.31%)	1/65 (1.54%)	2/66 (3.03%)
Nasopharyngitis ^†	4/65 (6.15%)	2/65 (3.08%)	2/66 (3.03%)
Injury, poisoning and procedural complications
Fall ^†	10/65 (15.38%)	7/65 (10.77%)	12/66 (18.18%)
Ligament sprain ^†	1/65 (1.54%)	4/65 (6.15%)	1/66 (1.52%)
Metabolism and nutrition disorders
Cushingoid ^†	6/65 (9.23%)	6/65 (9.23%)	3/66 (4.55%)
Abnormal weight gain ^†	9/65 (13.85%)	4/65 (6.15%)	5/66 (7.58%)
Vitamin D deficiency ^†	7/65 (10.77%)	6/65 (9.23%)	5/66 (7.58%)
Musculoskeletal and connective tissue disorders
Pain in extremity ^†	8/65 (12.31%)	10/65 (15.38%)	5/66 (7.58%)
Back pain ^†	5/65 (7.69%)	6/65 (9.23%)	8/66 (12.12%)
Arthralgia ^†	3/65 (4.62%)	6/65 (9.23%)	2/66 (3.03%)
Muscle spasms ^†	1/65 (1.54%)	5/65 (7.69%)	3/66 (4.55%)
Intervertebral disc space narrowing ^†	4/65 (6.15%)	0/65 (0.00%)	0/66 (0.00%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma ^†	5/65 (7.69%)	1/65 (1.54%)	0/66 (0.00%)
Nervous system disorders
Headache ^†	9/65 (13.85%)	9/65 (13.85%)	6/66 (9.09%)
Psychiatric disorders
Abnormal behavior ^†	12/65 (18.46%)	17/65 (26.15%)	16/66 (24.24%)
Mood altered ^†	4/65 (6.15%)	7/65 (10.77%)	2/66 (3.03%)
Aggression ^†	4/65 (6.15%)	4/65 (6.15%)	2/66 (3.03%)
Insomnia ^†	1/65 (1.54%)	4/65 (6.15%)	2/66 (3.03%)
Psychomotor hyperactivity ^†	2/65 (3.08%)	1/65 (1.54%)	4/66 (6.06%)
Renal and urinary disorders
Pollakiuria ^†	4/65 (6.15%)	2/65 (3.08%)	5/66 (7.58%)
Respiratory, thoracic and mediastinal disorders
Cough ^†	13/65 (20.00%)	12/65 (18.46%)	11/66 (16.67%)
Naspharyngitis ^†	9/65 (13.85%)	9/65 (13.85%)	11/66 (16.67%)
Upper respiratory tract infection ^†	11/65 (16.92%)	11/65 (16.92%)	7/66 (10.61%)
Rhinitis ^†	4/65 (6.15%)	5/65 (7.69%)	6/66 (9.09%)
Rhinorrhoea ^†	2/65 (3.08%)	5/65 (7.69%)	2/66 (3.03%)
Skin and subcutaneous tissue disorders
Hypertrichosis ^†	5/65 (7.69%)	7/65 (10.77%)	0/66 (0.00%)
Rash ^†	3/65 (4.62%)	4/65 (6.15%)	2/66 (3.03%)
† Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The PI cannot publish any manuscript without review and written consent of the FOR-DMD study steering committee.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Robert Griggs
Organization:	University of Rochester
Phone:	585-275-6072
EMail:	robert_griggs@urmc.rochester.edu

Publications:

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. Erratum In: Muscle Nerve 1994 Jul;17(7):833.

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.

Backman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 1995 May;5(3):233-41. doi: 10.1016/0960-8966(94)00048-e.

Bianchi ML. How to manage osteoporosis in children. Best Pract Res Clin Rheumatol. 2005 Dec;19(6):991-1005. doi: 10.1016/j.berh.2005.06.006.

Biggar WD, Bachrach LK, Henderson RC, Kalkwarf H, Plotkin H, Wong BL. Bone health in Duchenne muscular dystrophy: a workshop report from the meeting in Cincinnati, Ohio, July 8, 2004. Neuromuscul Disord. 2005 Jan;15(1):80-5. doi: 10.1016/j.nmd.2004.09.010. No abstract available.

Biggar WD, Gingras M, Fehlings DL, Harris VA, Steele CA. Deflazacort treatment of Duchenne muscular dystrophy. J Pediatr. 2001 Jan;138(1):45-50. doi: 10.1067/mpd.2001.109601.

Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord. 2006 Apr;16(4):249-55. doi: 10.1016/j.nmd.2006.01.010. Epub 2006 Mar 20.

Biggar WD, Politano L, Harris VA, Passamano L, Vajsar J, Alman B, Palladino A, Comi LI, Nigro G. Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols. Neuromuscul Disord. 2004 Sep;14(8-9):476-82. doi: 10.1016/j.nmd.2004.05.001.

Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Miller JP, Province MA. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve. 1983 Feb;6(2):91-103. doi: 10.1002/mus.880060204.

Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley RT 3rd, Miller JP, Kaiser KK, Florence JM, Pandya S, Signore L, et al. Clinical investigation of Duchenne muscular dystrophy. Interesting results in a trial of prednisone. Arch Neurol. 1987 Aug;44(8):812-7. doi: 10.1001/archneur.1987.00520200016010.

Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Florence J, King WM, Pandya S, Robison J, Schierbecker J, et al. Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy. Neurology. 1989 Apr;39(4):475-81. doi: 10.1212/wnl.39.4.475.

Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord. 2004 Sep;14(8-9):526-34. doi: 10.1016/j.nmd.2004.05.006. No abstract available.

Bushby K, Muntoni F, Bourke JP. 107th ENMC international workshop: the management of cardiac involvement in muscular dystrophy and myotonic dystrophy. 7th-9th June 2002, Naarden, the Netherlands. Neuromuscul Disord. 2003 Feb;13(2):166-72. doi: 10.1016/s0960-8966(02)00213-4. No abstract available.

Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27.

Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. 2010 Feb;9(2):177-89. doi: 10.1016/S1474-4422(09)70272-8. Epub 2009 Nov 27. Erratum In: Lancet Neurol. 2010 Mar;9(3):237.

Carter GT, McDonald CM. Preserving function in Duchenne dystrophy with long-term pulse prednisone therapy. Am J Phys Med Rehabil. 2000 Sep-Oct;79(5):455-8. doi: 10.1097/00002060-200009000-00009.

Collett BR, Ohan JL, Myers KM. Ten-year review of rating scales. V: scales assessing attention-deficit/hyperactivity disorder. J Am Acad Child Adolesc Psychiatry. 2003 Sep;42(9):1015-37. doi: 10.1097/01.CHI.0000070245.24125.B6.

Collett BR, Ohan JL, Myers KM. Ten-year review of rating scales. VI: scales assessing externalizing behaviors. J Am Acad Child Adolesc Psychiatry. 2003 Oct;42(10):1143-70. doi: 10.1097/00004583-200310000-00006.

Connolly AM, Schierbecker J, Renna R, Florence J. High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2002 Dec;12(10):917-25. doi: 10.1016/s0960-8966(02)00180-3.

Duboc D, Meune C, Lerebours G, Devaux JY, Vaksmann G, Becane HM. Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy. J Am Coll Cardiol. 2005 Mar 15;45(6):855-7. doi: 10.1016/j.jacc.2004.09.078.

Dubowitz V, Kinali M, Main M, Mercuri E, Muntoni F. Remission of clinical signs in early duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy. Eur J Paediatr Neurol. 2002;6(3):153-9. doi: 10.1053/ejpn.2002.0583.

Eagle M, Baudouin SV, Chandler C, Giddings DR, Bullock R, Bushby K. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscul Disord. 2002 Dec;12(10):926-9. doi: 10.1016/s0960-8966(02)00140-2.

Fenichel GM, Florence JM, Pestronk A, Mendell JR, Moxley RT 3rd, Griggs RC, Brooke MH, Miller JP, Robison J, King W, et al. Long-term benefit from prednisone therapy in Duchenne muscular dystrophy. Neurology. 1991 Dec;41(12):1874-7. doi: 10.1212/wnl.41.12.1874.

Fenichel GM, Mendell JR, Moxley RT 3rd, Griggs RC, Brooke MH, Miller JP, Pestronk A, Robison J, King W, Signore L, et al. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Arch Neurol. 1991 Jun;48(6):575-9. doi: 10.1001/archneur.1991.00530180027012.

Follmann D. Multivariate tests for multiple endpoints in clinical trials. Stat Med. 1995 Jun 15;14(11):1163-75. doi: 10.1002/sim.4780141103.

Griggs RC, Moxley RT 3rd, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, Miller JP, Cwik VA, Pandya S, Robison J, et al. Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology. 1993 Mar;43(3 Pt 1):520-7. doi: 10.1212/wnl.43.3_part_1.520.

Griggs RC, Moxley RT 3rd, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, Miller JP. Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group. Arch Neurol. 1991 Apr;48(4):383-8. doi: 10.1001/archneur.1991.00530160047012.

Hinton VJ, Nereo NE, Fee RJ, Cyrulnik SE. Social behavior problems in boys with Duchenne muscular dystrophy. J Dev Behav Pediatr. 2006 Dec;27(6):470-6. doi: 10.1097/00004703-200612000-00003.

Hopke PK, Liu C, Rubin DB. Multiple imputation for multivariate data with missing and below-threshold measurements: time-series concentrations of pollutants in the Arctic. Biometrics. 2001 Mar;57(1):22-33. doi: 10.1111/j.0006-341x.2001.00022.x.

Kinali M, Mercuri E, Main M, Muntoni F, Dubowitz V. An effective, low-dosage, intermittent schedule of prednisolone in the long-term treatment of early cases of Duchenne dystrophy. Neuromuscul Disord. 2002 Oct;12 Suppl 1:S169-74. doi: 10.1016/s0960-8966(02)00097-4.

Lippuner K, Casez JP, Horber FF, Jaeger P. Effects of deflazacort versus prednisone on bone mass, body composition, and lipid profile: a randomized, double blind study in kidney transplant patients. J Clin Endocrinol Metab. 1998 Nov;83(11):3795-802. doi: 10.1210/jcem.83.11.5235.

Little R, Yau L. Intent-to-treat analysis for longitudinal studies with drop-outs. Biometrics. 1996 Dec;52(4):1324-33.

Loftus J, Allen R, Hesp R, David J, Reid DM, Wright DJ, Green JR, Reeve J, Ansell BM, Woo PM. Randomized, double-blind trial of deflazacort versus prednisone in juvenile chronic (or rheumatoid) arthritis: a relatively bone-sparing effect of deflazacort. Pediatrics. 1991 Sep;88(3):428-36.

Lunceford JK, Davidian M. Stratification and weighting via the propensity score in estimation of causal treatment effects: a comparative study. Stat Med. 2004 Oct 15;23(19):2937-60. doi: 10.1002/sim.1903. Erratum In: Stat Med. 2017 Jun 30;36(14 ):2320.

Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2004;(2):CD003725. doi: 10.1002/14651858.CD003725.pub2.

Markham A, Bryson HM. Deflazacort. A review of its pharmacological properties and therapeutic efficacy. Drugs. 1995 Aug;50(2):317-33. doi: 10.2165/00003495-199550020-00008.

Markham LW, Spicer RL, Khoury PR, Wong BL, Mathews KD, Cripe LH. Steroid therapy and cardiac function in Duchenne muscular dystrophy. Pediatr Cardiol. 2005 Nov-Dec;26(6):768-71. doi: 10.1007/s00246-005-0909-4.

Mayhew A, Cano S, Scott E, Eagle M, Bushby K, Muntoni F; North Star Clinical Network for Paediatric Neuromuscular Disease. Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol. 2011 Jun;53(6):535-42. doi: 10.1111/j.1469-8749.2011.03939.x. Epub 2011 Mar 17.

Mendell JR, Moxley RT, Griggs RC, Brooke MH, Fenichel GM, Miller JP, King W, Signore L, Pandya S, Florence J, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med. 1989 Jun 15;320(24):1592-7. doi: 10.1056/NEJM198906153202405.

Merlini L, Cicognani A, Malaspina E, Gennari M, Gnudi S, Talim B, Franzoni E. Early prednisone treatment in Duchenne muscular dystrophy. Muscle Nerve. 2003 Feb;27(2):222-7. doi: 10.1002/mus.10319.

Moxley RT 3rd, Ashwal S, Pandya S, Connolly A, Florence J, Mathews K, Baumbach L, McDonald C, Sussman M, Wade C; Quality Standards Subcommittee of the American Academy of Neurology; Practice Committee of the Child Neurology Society. Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology. 2005 Jan 11;64(1):13-20. doi: 10.1212/01.WNL.0000148485.00049.B7.

O'Brien PC. Procedures for comparing samples with multiple endpoints. Biometrics. 1984 Dec;40(4):1079-87.

Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.

Pocock SJ, Geller NL, Tsiatis AA. The analysis of multiple endpoints in clinical trials. Biometrics. 1987 Sep;43(3):487-98.

Quinlivan R, Roper H, Davie M, Shaw NJ, McDonagh J, Bushby K. Report of a Muscular Dystrophy Campaign funded workshop Birmingham, UK, January 16th 2004. Osteoporosis in Duchenne muscular dystrophy; its prevalence, treatment and prevention. Neuromuscul Disord. 2005 Jan;15(1):72-9. doi: 10.1016/j.nmd.2004.09.009. Epub 2004 Dec 10. No abstract available.

Reitter B. Deflazacort vs. prednisone in Duchenne muscular dystrophy: trends of an ongoing study. Brain Dev. 1995;17 Suppl:39-43.

Sansome A, Royston P, Dubowitz V. Steroids in Duchenne muscular dystrophy; pilot study of a new low-dosage schedule. Neuromuscul Disord. 1993 Sep-Nov;3(5-6):567-9. doi: 10.1016/0960-8966(93)90117-3.

Silversides CK, Webb GD, Harris VA, Biggar DW. Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy. Am J Cardiol. 2003 Mar 15;91(6):769-72. doi: 10.1016/s0002-9149(02)03429-x. No abstract available.

Simonds AK, Muntoni F, Heather S, Fielding S. Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy. Thorax. 1998 Nov;53(11):949-52. doi: 10.1136/thx.53.11.949.

Stein RE, Jessop DJ. Functional status II(R). A measure of child health status. Med Care. 1990 Nov;28(11):1041-55. doi: 10.1097/00005650-199011000-00006. Erratum In: Med Care 1991 May;29(5):following 489.

Tang DI, Geller NL, Pocock SJ. On the design and analysis of randomized clinical trials with multiple endpoints. Biometrics. 1993 Mar;49(1):23-30.

Varni JW, Seid M, Knight TS, Uzark K, Szer IS. The PedsQL 4.0 Generic Core Scales: sensitivity, responsiveness, and impact on clinical decision-making. J Behav Med. 2002 Apr;25(2):175-93. doi: 10.1023/a:1014836921812.

Varni JW, Seid M, Kurtin PS. PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations. Med Care. 2001 Aug;39(8):800-12. doi: 10.1097/00005650-200108000-00006.

Varni JW, Seid M, Rode CA. The PedsQL: measurement model for the pediatric quality of life inventory. Med Care. 1999 Feb;37(2):126-39. doi: 10.1097/00005650-199902000-00003.

Witt WP, Kasper JD, Riley AW. Mental health services use among school-aged children with disabilities: the role of sociodemographics, functional limitations, family burdens, and care coordination. Health Serv Res. 2003 Dec;38(6 Pt 1):1441-66. doi: 10.1111/j.1475-6773.2003.00187.x.

Xie H, Heitjan DF. Sensitivity analysis of causal inference in a clinical trial subject to crossover. Clin Trials. 2004 Feb;1(1):21-30. doi: 10.1191/1740774504cn005oa. Erratum In: Clin Trials. 2004;1(5):2 p following 474. Clin Trials. 2005;2(2):194.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

Layout table for additonal information

Responsible Party:	Robert Griggs, MD, University of Rochester
ClinicalTrials.gov Identifier:	NCT01603407
Other Study ID Numbers:	U01NS061799 ( U.S. NIH Grant/Contract ) 2010-023744-33 ( EudraCT Number ) U01NS061799 ( U.S. NIH Grant/Contract ) 46102316 ( Registry Identifier: ISRCTN )
First Submitted:	April 3, 2012
First Posted:	May 23, 2012
Results First Submitted:	May 12, 2022
Results First Posted:	August 12, 2022
Last Update Posted:	August 12, 2022

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