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Clinical Trial Results:
Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen

Summary
EudraCT number	2010-023744-33
Trial protocol	GB IT
Global end of trial date	31 Aug 2023

Results information
Results version number	v1(current)
This version publication date	19 Jun 2024
First version publication date	19 Jun 2024
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

Top of page

Sponsor protocol code

4.0

ISRCTN number

ISRCTN46102316

US NCT number

NCT01603407

WHO universal trial number (UTN)

Sponsor organisation name

University of Rochester

Sponsor organisation address

518 Hylan Building, Rochester, United States, NY14627

Public contact

Kim Hart, University of Rochester, 001 585 275 3767, Kim_Hart@URMC.Rochester.edu

Scientific contact

Kim Hart, University of Rochester, 001 585 275 3767, Kim_Hart@URMC.Rochester.edu

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

15 Nov 2019

Is this the analysis of the primary completion data?

Yes

Primary completion date

15 Nov 2019

Global end of trial reached?

Yes

Global end of trial date

31 Aug 2023

Was the trial ended prematurely?

Main objective of the trial

The proposed randomized controlled trial will compare three corticosteroid regimens to address the pragmatic hypothesis that daily corticosteroids (prednisone or deflazacort) will be of greater benefit in terms of function and subject/parent satisfaction than intermittent corticosteroids (prednisone).

Protection of trial subjects

None

Background therapy

None

Evidence for comparator

The comparators used in the trial are daily prednisone, intermittent prednisone, and daily deflazacort. All three regimens are in common use in boys with Duchenne muscular dystrophy. Prednisone is a corticosteroid licenced for use in a wide range of conditions, and has been shown to improve muscle function in boys with DMD. Prednisone, rather than prednisolone, is the formulation of choice for the purposes of this trial. Prednisone is not currently licenced for use in DMD and will therefore be considered an Investigational Medicinal Product (IMP) for the purposes of this trial. Deflazacort is a glucocorticoid derived from prednisolone, and is licenced in Europe for use in a wide range of conditions. Deflazacort is not currently licenced for the indication of muscular dystrophy and there has been less exposure of children to deflazacort compared to prednisone in clinical trials. Moreover, deflazacort is not approved for any indication in the USA. Therefore for the purposes of this trial it will be considered an IMP. Despite the publication of consensus statements on the topic of the use of steroids to treat DMD, patient experience continues to be that widely different corticosteroid regimens remain in use.

Actual start date of recruitment

30 Jan 2013

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

United Kingdom: 59

Country: Number of subjects enrolled

Germany: 20

Country: Number of subjects enrolled

Italy: 22

Country: Number of subjects enrolled

United States: 82

Country: Number of subjects enrolled

Canada: 13

Worldwide total number of subjects

196

EEA total number of subjects

101

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

196

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

Top of page

Recruitment details

The recruitment period ran from 30 January 2013 until 17 September 2016, at 32 sites in the USA, Canada, the UK, Germany and Italy.

Screening details

All DMD patients’ families were told of the trial and, if eligible, could consider participation. Clinical staff identified eligible patients via direct contact and electronic records. Potential participants were given brief trial information, and could agree to approach by research staff. Interested patients gave consent and were randomised.

Period 1 title

Baseline

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Are arms mutually exclusive

Yes

Daily prednisone

Arm description

0.75 mg/kg/day prednisone, administered to participants daily, in the morning at home.

Arm type

Experimental

Investigational medicinal product name

Prednisone

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Once daily administration, at 0.75 mg/kg/day

Daily deflazacort

Arm description

0.9 mg/kg/day deflazacort, administered to participants daily, in the morning at home.

Arm type

Experimental

Investigational medicinal product name

Deflazacort

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Once daily administration, at 0.9 mg/kg/day

Intermittent prednisone

Arm description

0.75 mg/kg/d prednisone for 10 days alternating with 10 days off, administered to participants daily, in the morning at home.

Arm type

Experimental

Investigational medicinal product name

Prednisone

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Once daily administration, at 0.75 mg/kg/day, 10 days on, 10 days off (placebo on 'off' days)

Number of subjects in period 1	Daily prednisone	Daily deflazacort	Intermittent prednisone
Started	65	65	66
Completed	65	65	66

Period 2 title

Months 3-36

Is this the baseline period?

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Are arms mutually exclusive

Yes

Daily prednisone

Arm description

0.75 mg/kg/day prednisone, administered to participants daily, in the morning at home.

Arm type

Experimental

Investigational medicinal product name

Prednisone

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Once daily administration, at 0.75 mg/kg/day

Daily deflazacort

Arm description

0.9 mg/kg/day deflazacort, administered to participants daily, in the morning at home.

Arm type

Experimental

Investigational medicinal product name

Deflazacort

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Once daily administration, at 0.9 mg/kg/day

Intermittent prednisone

Arm description

0.75 mg/kg/d prednisone for 10 days alternating with 10 days off, administered to participants daily, in the morning at home.

Arm type

Experimental

Investigational medicinal product name

Prednisone

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Once daily administration, at 0.75 mg/kg/day, 10 days on, 10 days off (placebo on 'off' days)

Number of subjects in period 2	Daily prednisone	Daily deflazacort	Intermittent prednisone
Started	65	65	66
Completed	54	54	56
Not completed	11	11	10
Consent withdrawn by subject	5	3	7
Adverse event, non-fatal	1	1	1
Unknown	1	-	-
Non-compliance with protocol	2	2	-
Entered a different trial	2	2	-
Lost to follow-up	-	3	2

Baseline characteristics

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Reporting group title

Daily prednisone

Reporting group description

0.75 mg/kg/day prednisone, administered to participants daily, in the morning at home.

Reporting group title

Daily deflazacort

Reporting group description

0.9 mg/kg/day deflazacort, administered to participants daily, in the morning at home.

Reporting group title

Intermittent prednisone

Reporting group description

0.75 mg/kg/d prednisone for 10 days alternating with 10 days off, administered to participants daily, in the morning at home.

Reporting group values	Daily prednisone	Daily deflazacort	Intermittent prednisone	Total
Number of subjects	65	65	66	196
Age categorical
Units: Subjects
Children (2-11 years)	65	65	66	196
Age continuous
Units: years
arithmetic mean (standard deviation)	5.9 ( 1.0 )	5.8 ( 1.0 )	6.0 ( 1.1 )	-
Gender categorical
Units: Subjects
Female	0	0	0	0
Male	65	65	66	196
Country
Units: Subjects
USA	27	27	28	82
Canada	5	4	4	13
UK	19	20	20	59
Germany	6	7	7	20
Italy	8	7	7	22
Race
Units: Subjects
White	59	58	49	166
Black	2	0	1	3
Asian	1	2	6	9
Mixed	1	1	3	5
Other	1	2	4	7
Missing	1	2	3	6
Ethnicity
Units: Subjects
Non-hispanic	56	51	50	157
Hispanic	9	9	13	31
Missing	0	5	3	8
Mutation
Units: Subjects
No	1	1	0	2
Yes	64	64	66	194
Mother DMD Carrier
Units: Subjects
No	18	13	16	47
Yes	24	32	27	83
Missing	23	20	23	66
Family history of diabetes
Units: Subjects
No	42	34	36	112
Yes	21	30	29	80
Missing	2	1	1	4
Family history of tuberculosis
Units: Subjects
No	60	55	58	173
Yes	1	2	3	6
Missing	4	8	5	17
Speech and language difficulties
Units: Subjects
No	41	40	39	120
Yes	24	24	27	75
Missing	0	1	0	1
Presence of learning difficulties
Units: Subjects
No	47	43	41	131
Yes	13	17	20	50
Missing	5	5	5	15
Severity of learning difficulties
Units: Subjects
None	47	43	41	131
Mild	10	13	10	33
Moderate	2	2	10	14
Severe	1	0	0	1
Missing	5	7	5	17
Autism diagnosis
Units: Subjects
No	65	64	64	193
Yes	0	1	2	3
ADHD diagnosis
Units: Subjects
No	63	65	63	191
Yes	2	0	3	5
Previous surgery for DMD
Units: Subjects
No	63	60	64	187
Yes	2	5	2	9
Weight band
Units: Subjects
Band A	38	41	39	118
Band B	26	18	25	69
Band C	1	6	2	9
Initiation of Vitamin D
Units: Subjects
Yes	1	1	1	3
Missing	64	64	65	193
NSAA1 (Standing)
Units: Subjects
Unable	0	1	0	1
Modified	3	9	2	14
Able	60	55	62	177
Missing	2	0	2	4
NSAA2 (Walk (10 meters))
Units: Subjects
Modified	9	14	11	34
Able	54	51	53	158
Missing	2	0	2	4
NSAA3 (Stand up from chair)
Units: Subjects
Unable	0	1	0	1
Modified	22	20	20	62
Able	41	44	44	129
Missing	2	0	2	4
NSAA4 (Stand on one leg (Right))
Units: Subjects
Unable	3	3	3	9
Modified	37	43	40	120
Able	23	19	21	63
Missing	2	0	2	4
NSAA5 (Stand on one leg (Left))
Units: Subjects
Unable	2	3	2	7
Modified	38	41	40	119
Able	23	21	22	66
Missing	2	0	2	4
NSAA6 (Climb step (Right))
Units: Subjects
Unable	1	4	5	10
Modified	30	27	25	82
Able	32	34	34	100
Missing	2	0	2	4
NSAA7 (Climb step (Left))
Units: Subjects
Unable	3	5	2	10
Modified	33	28	35	96
Able	27	32	27	86
Missing	2	0	2	4
NSAA8 (Descend step (Right))
Units: Subjects
Unable	1	3	0	4
Modified	45	42	46	133
Able	17	20	18	55
Missing	2	0	2	4
NSAA9 (Descend step (Left))
Units: Subjects
Unable	2	3	3	8
Modified	40	46	47	133
Able	21	16	14	51
Missing	2	0	2	4
NSAA10 (Gets to sitting)
Units: Subjects
Unable	1	1	0	2
Modified	42	50	55	147
Able	20	14	9	43
Missing	2	0	2	4
NSAA11 (Rise from the floor)
Units: Subjects
Unable	2	1	3	6
Modified	59	64	58	181
Able	2	0	3	5
Missing	2	0	2	4
NSAA12 (Lifts head from supine)
Units: Subjects
Unable	15	16	20	51
Modified	34	37	37	108
Able	14	12	7	33
Missing	2	0	2	4
NSAA13 (Stand on heels)
Units: Subjects
Unable	10	17	16	43
Modified	33	37	35	105
Able	20	11	13	44
Missing	2	0	2	4
NSAA14 (Jump)
Units: Subjects
Unable	10	10	11	31
Modified	22	20	13	55
Able	31	35	40	106
Missing	2	0	2	4
NSAA15 (Hop (Right))
Units: Subjects
Unable	28	31	24	83
Modified	27	29	37	93
Able	8	5	3	16
Missing	2	0	2	4
NSAA16 (Hop (Left))
Units: Subjects
Unable	29	30	29	88
Modified	24	30	32	86
Able	10	5	3	18
Missing	2	0	2	4
NSAA17 (Walk/Run 10 meters)
Units: Subjects
Unable	4	4	3	11
Modified	33	38	42	113
Able	26	23	19	68
Missing	2	0	2	4
Number of brothers with DMD
Units: Subjects
None	43	48	56	147
One	8	9	8	25
Two	2	0	0	2
Missing	12	8	2	22
IOWA Conners I/O Subscale Score >= 10
Units: Subjects
No	57	58	55	170
Yes	4	7	8	19
Missing	4	0	3	7
IOWA Conners O/D Subscale Score >=9
Units: Subjects
No	58	58	56	172
Yes	3	7	6	16
Missing	4	0	4	8
PARS-III Total Score <72
Units: Subjects
No	61	62	57	180
Yes	1	3	6	10
Missing	3	0	3	6

End points

Top of page

Reporting group title

Daily prednisone

Reporting group description

0.75 mg/kg/day prednisone, administered to participants daily, in the morning at home.

Reporting group title

Daily deflazacort

Reporting group description

0.9 mg/kg/day deflazacort, administered to participants daily, in the morning at home.

Reporting group title

Intermittent prednisone

Reporting group description

0.75 mg/kg/d prednisone for 10 days alternating with 10 days off, administered to participants daily, in the morning at home.

Reporting group title

Daily prednisone

Reporting group description

0.75 mg/kg/day prednisone, administered to participants daily, in the morning at home.

Reporting group title

Daily deflazacort

Reporting group description

0.9 mg/kg/day deflazacort, administered to participants daily, in the morning at home.

Reporting group title

Intermittent prednisone

Reporting group description

0.75 mg/kg/d prednisone for 10 days alternating with 10 days off, administered to participants daily, in the morning at home.

Primary: Reciprocal of time to rise from the floor averaged over months 3-36

Top of page

End point title

Reciprocal of time to rise from the floor averaged over months 3-36

End point description

End point type

Primary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	63	65
Units: rises per second
least squares mean (standard error)	0.2362 ( 0.008621 )	0.2401 ( 0.008893 )	0.1800 ( 0.008589 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7365

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.003939

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.02423

upper limit

0.0321

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.0562

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.08392

upper limit

-0.02848

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.06013

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.08807

upper limit

-0.0322

Primary: Forced Vital Capacity (FVC)

Top of page

End point title

Forced Vital Capacity (FVC)

End point description

End point type

Primary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	61	62	65
Units: litre(s)
least squares mean (standard error)	1.4374 ( 0.03103 )	1.4015 ( 0.03191 )	1.4608 ( 0.03097 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

123

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3904

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.03586

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.1362

upper limit

0.06451

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.569

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.02343

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.07551

upper limit

0.1224

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1518

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.05929

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.03995

upper limit

0.1585

Primary: TSQM Global Satisfaction Subscale Score

Top of page

End point title

TSQM Global Satisfaction Subscale Score

End point description

End point type

Primary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	65
Units: Score
least squares mean (standard error)	71.2278 ( 2.2508 )	67.7708 ( 2.3229 )	65.0729 ( 2.2550 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily deflazacort v Daily prednisone

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2456

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.457

Confidence interval

level

98.3%

sides

2-sided

lower limit

-10.6077

upper limit

3.6937

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0369

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-6.1549

Confidence interval

level

98.3%

sides

2-sided

lower limit

-13.2091

upper limit

0.8993

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3589

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.6979

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.7651

upper limit

4.3693

Secondary: Forced Vital Capacity (FVC) %

Top of page

End point title

Forced Vital Capacity (FVC) %

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	61	62	65
Units: percent
least squares mean (standard error)	96.7870 ( 1.8937 )	98.0438 ( 1.9580 )	92.7105 ( 1.8979 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

123

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.619

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

1.2568

Confidence interval

level

98.3%

sides

2-sided

lower limit

-4.8233

upper limit

7.3368

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1031

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-4.0765

Confidence interval

level

98.3%

sides

2-sided

lower limit

-10.0725

upper limit

1.9194

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0341

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-5.3333

Confidence interval

level

98.3%

sides

2-sided

lower limit

-11.3498

upper limit

0.6831

Secondary: 10 meter walk/run velocity

Top of page

End point title

10 meter walk/run velocity

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	63	65
Units: second
least squares mean (standard error)	0.2035 ( 0.004880 )	0.2009 ( 0.005008 )	0.1718 ( 0.004839 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6976

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.00262

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.01886

upper limit

0.01362

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.03165

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.04762

upper limit

-0.01567

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.02903

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.04515

upper limit

-0.0129

Secondary: NSAA Total Score

Top of page

End point title

NSAA Total Score

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	63	65
Units: Score
least squares mean (standard error)	23.7361 ( 0.6016 )	24.0198 ( 0.6101 )	20.7441 ( 0.5933 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7335

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.2836

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.7216

upper limit

2.2889

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0004

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.992

Confidence interval

level

98.3%

sides

2-sided

lower limit

-4.9712

upper limit

-1.0129

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.2757

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.2581

upper limit

-1.2933

Secondary: Six-minute Walk Test distance

Top of page

End point title

Six-minute Walk Test distance

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	60	63	64
Units: meter
least squares mean (standard error)	384.95 ( 9.5798 )	384.17 ( 9.6568 )	346.81 ( 9.4059 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

123

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9532

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.7782

Confidence interval

level

98.3%

sides

2-sided

lower limit

-32.6647

upper limit

31.1083

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.004

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-38.1321

Confidence interval

level

98.3%

sides

2-sided

lower limit

-69.5948

upper limit

-6.6694

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0046

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-37.3538

Confidence interval

level

98.3%

sides

2-sided

lower limit

-68.7344

upper limit

-5.9733

Secondary: TSQM Effectiveness Subscale

Top of page

End point title

TSQM Effectiveness Subscale

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	65
Units: Score
least squares mean (standard error)	69.5998 ( 1.8774 )	66.1078 ( 1.9212 )	65.4464 ( 1.8872 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1618

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.492

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.4813

upper limit

2.4972

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0914

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-4.1534

Confidence interval

level

98.3%

sides

2-sided

lower limit

-10.0501

upper limit

1.7433

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7883

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.6614

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.5868

upper limit

5.2641

Secondary: TSQM Side effects Subscale

Top of page

End point title

TSQM Side effects Subscale

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	64
Units: Score
least squares mean (standard error)	85.3830 ( 2.1500 )	83.1366 ( 2.2278 )	85.9917 ( 2.1602 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.4322

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.2463

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.124

upper limit

4.6313

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.829

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.6087

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.1734

upper limit

7.3909

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3135

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

2.8551

Confidence interval

level

98.3%

sides

2-sided

lower limit

-3.9524

upper limit

9.6625

Secondary: Participant weight

Top of page

End point title

Participant weight

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	65
Units: kilogram(s)
least squares mean (standard error)	26.3098 ( 0.3573 )	24.8552 ( 0.3611 )	26.2523 ( 0.3518 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0041

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.4545

Confidence interval

level

98.3%

sides

2-sided

lower limit

-2.6611

upper limit

-0.2479

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9076

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.05745

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.248

upper limit

1.1331

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0054

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

1.3971

Confidence interval

level

98.3%

sides

2-sided

lower limit

0.2014

upper limit

2.5927

Secondary: Participant height

Top of page

End point title

Participant height

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	65
Units: centimeter
least squares mean (standard error)	116.81 ( 0.2584 )	115.33 ( 0.2669 )	119.87 ( 0.2617 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.48

Confidence interval

level

98.3%

sides

2-sided

lower limit

-2.3242

upper limit

-0.6358

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Intermittent prednisone v Daily prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

3.054

Confidence interval

level

98.3%

sides

2-sided

lower limit

2.2222

upper limit

3.8857

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

4.534

Confidence interval

level

98.3%

sides

2-sided

lower limit

3.6941

upper limit

5.3738

Secondary: Body Mass Index (BMI)

Top of page

End point title

Body Mass Index (BMI)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	65
Units: kilogram(s)/square meter
least squares mean (standard error)	18.9337 ( 0.2564 )	18.3132 ( 0.2592 )	18.0577 ( 0.2524 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0853

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.6205

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.4845

upper limit

0.2434

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0143

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.876

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.7292

upper limit

-0.02285

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.4731

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.2555

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.1117

upper limit

0.6007

Secondary: IOWA Conners Total Score

Top of page

End point title

IOWA Conners Total Score

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	60	62	62
Units: Score
least squares mean (standard error)	8.0970 ( 0.6029 )	8.5777 ( 0.6173 )	9.2830 ( 0.6070 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

122

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5456

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.4807

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.4327

upper limit

2.394

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

122

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1358

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

1.186

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.7215

upper limit

3.0936

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Intermittent prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3713

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.7053

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.1914

upper limit

2.6021

Secondary: PARS-III Total Score

Top of page

End point title

PARS-III Total Score

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	61	62	63
Units: Score
least squares mean (standard error)	85.8447 ( 0.9183 )	85.0151 ( 0.9406 )	84.8066 ( 0.9340 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

123

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.4933

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.8296

Confidence interval

level

98.3%

sides

2-sided

lower limit

-3.7417

upper limit

2.0825

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3917

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.0381

Confidence interval

level

98.3%

sides

2-sided

lower limit

-3.9519

upper limit

1.8757

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.8627

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.2085

Confidence interval

level

98.3%

sides

2-sided

lower limit

-3.1099

upper limit

2.6929

Secondary: SDQ Total Difficulties Score

Top of page

End point title

SDQ Total Difficulties Score

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	60	64
Units: Score
least squares mean (standard error)	11.5469 ( 0.5082 )	12.1498 ( 0.5303 )	11.7749 ( 0.5108 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

122

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3742

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.6029

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.0281

upper limit

2.2338

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7314

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.228

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.3704

upper limit

1.8264

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5776

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.3749

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.9944

upper limit

1.2446

Secondary: Revised Rutter Scale Score

Top of page

End point title

Revised Rutter Scale Score

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	60	62	65
Units: Score
least squares mean (standard error)	3.5921 ( 0.2799 )	3.7675 ( 0.2866 )	3.9236 ( 0.2780 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

122

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6371

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.1754

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.7192

upper limit

1.0701

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3657

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.3315

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.5494

upper limit

1.2125

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6692

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.1561

Confidence interval

level

98.3%

sides

2-sided

lower limit

-0.7229

upper limit

1.0351

Secondary: Systolic Blood Pressure

Top of page

End point title

Systolic Blood Pressure

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	65
Units: mm Hg
least squares mean (standard error)	105.22 ( 0.8427 )	105.40 ( 0.8747 )	103.47 ( 0.8461 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.8758

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.174

Confidence interval

level

98.3%

sides

2-sided

lower limit

-2.5056

upper limit

2.8536

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1099

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.755

Confidence interval

level

98.3%

sides

2-sided

lower limit

-4.3868

upper limit

0.8769

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.081

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.929

Confidence interval

level

98.3%

sides

2-sided

lower limit

-4.5772

upper limit

0.7193

Secondary: Diastolic Blood Pressure

Top of page

End point title

Diastolic Blood Pressure

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	62	64
Units: mm Hg
least squares mean (standard error)	63.3740 ( 0.7083 )	61.6113 ( 0.7324 )	61.9813 ( 0.7144 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0616

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.7628

Confidence interval

level

98.3%

sides

2-sided

lower limit

-4.0205

upper limit

0.495

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Intermittent prednisone v Daily prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1316

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.3927

Confidence interval

level

98.3%

sides

2-sided

lower limit

-3.6085

upper limit

0.823

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.691

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.37

Confidence interval

level

98.3%

sides

2-sided

lower limit

-1.8699

upper limit

2.61

Secondary: Pulse

Top of page

End point title

Pulse

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	64	65
Units: bpm
least squares mean (standard error)	98.8095 ( 1.1039 )	99.6571 ( 1.1453 )	97.0617 ( 1.1040 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5605

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.8476

Confidence interval

level

98.3%

sides

2-sided

lower limit

-2.6545

upper limit

4.3496

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Intermittent prednisone v Daily prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2252

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.7478

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.2092

upper limit

1.7135

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

129

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0735

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.5954

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.0693

upper limit

0.8785

Secondary: PedsQL Physical functioning (Parent)

Top of page

End point title

PedsQL Physical functioning (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	64	64
Units: Score
least squares mean (standard error)	60.552123 ( 2.456210 )	56.667290 ( 2.438880 )	53.787578 ( 2.448008 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2376

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.884834

Confidence interval

level

98.3%

sides

2-sided

lower limit

-11.7375

upper limit

3.9678

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0362

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-6.764545

Confidence interval

level

98.3%

sides

2-sided

lower limit

-14.4738

upper limit

0.94469

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3583

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.879712

Confidence interval

level

98.3%

sides

2-sided

lower limit

-10.3624

upper limit

4.60301

Secondary: PedsQL Emotional functioning (Parent)

Top of page

End point title

PedsQL Emotional functioning (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	64	64
Units: Score
least squares mean (standard error)	71.123574 ( 2.183412 )	69.699984 ( 2.212084 )	69.480768 ( 2.191095 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6251

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.42359

Confidence interval

level

98.3%

sides

2-sided

lower limit

-8.37755

upper limit

5.53037

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.569

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.642806

Confidence interval

level

98.3%

sides

2-sided

lower limit

-8.52941

upper limit

5.2438

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9382

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.219216

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.97003

upper limit

6.53159

Secondary: PedsQL Social functioning (Parent)

Top of page

End point title

PedsQL Social functioning (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	64	64
Units: Score
least squares mean (standard error)	63.581032 ( 1.927473 )	66.024577 ( 1.967497 )	60.028922 ( 1.954941 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3372

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

2.443545

Confidence interval

level

98.3%

sides

2-sided

lower limit

-3.6344

upper limit

8.5215

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.163

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.55211

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.6311

upper limit

2.52689

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0168

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-5.995655

Confidence interval

level

98.3%

sides

2-sided

lower limit

-11.983

upper limit

-0.00827

Secondary: PedsQL School functioning (Parent)

Top of page

End point title

PedsQL School functioning (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	64	64
Units: Score
least squares mean (standard error)	68.097830 ( 1.900524 )	66.653218 ( 1.914070 )	66.397809 ( 1.953206 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5652

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.444611

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.44113

upper limit

4.5519

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5022

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.700021

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.74901

upper limit

4.34897

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9178

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.25541

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.16229

upper limit

5.65148

Secondary: PedsQL Psychological health (Parent)

Top of page

End point title

PedsQL Psychological health (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	64	64
Units: Score
least squares mean (standard error)	67.442758 ( 1.652715 )	67.495323 ( 1.659289 )	65.522673 ( 1.679620 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9806

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.052566

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.11134

upper limit

5.21647

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3807

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.920085

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.1495

upper limit

3.30933

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3616

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.972651

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.13475

upper limit

3.18944

Secondary: PedsQL Total Score (Parent)

Top of page

End point title

PedsQL Total Score (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	62	64	64
Units: Score
least squares mean (standard error)	64.878617 ( 1.671003 )	63.711112 ( 1.690016 )	61.334318 ( 1.689153 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5947

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.167505

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.40628

upper limit

4.07127

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Intermittent prednisone v Daily prednisone

Number of subjects included in analysis

126

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1098

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.544299

Confidence interval

level

98.3%

sides

2-sided

lower limit

-8.83474

upper limit

1.74614

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

128

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2803

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.376794

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.63231

upper limit

2.87872

Secondary: PedsQL Physical functioning (Child)

Top of page

End point title

PedsQL Physical functioning (Child)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	54	52	49
Units: Score
least squares mean (standard error)	64.802642 ( 2.818842 )	61.626840 ( 2.569129 )	60.024621 ( 2.669149 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

106

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3501

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.175801

Confidence interval

level

98.3%

sides

2-sided

lower limit

-11.2954

upper limit

4.94375

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

103

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1923

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-4.778021

Confidence interval

level

98.3%

sides

2-sided

lower limit

-13.5339

upper limit

3.97783

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

101

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6332

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.602219

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.6192

upper limit

6.41478

Secondary: PedsQL Emotional functioning (Child)

Top of page

End point title

PedsQL Emotional functioning (Child)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	54	52	47
Units: Score
least squares mean (standard error)	68.783278 ( 2.707352 )	65.931150 ( 2.645838 )	70.129170 ( 2.703500 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

106

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.396

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.852128

Confidence interval

level

98.3%

sides

2-sided

lower limit

-10.8762

upper limit

5.17198

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

101

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6982

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

1.345892

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.9442

upper limit

9.63594

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2158

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

4.19802

Confidence interval

level

98.3%

sides

2-sided

lower limit

-3.8992

upper limit

12.2952

Secondary: PedsQL Social functioning (Child)

Top of page

End point title

PedsQL Social functioning (Child)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	54	52	47
Units: Score
least squares mean (standard error)	66.183571 ( 2.734419 )	67.903617 ( 2.701055 )	67.103568 ( 2.639465 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily deflazacort v Daily prednisone

Number of subjects included in analysis

106

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6174

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

1.720046

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.50173

upper limit

9.94182

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

101

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7909

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.919997

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.36573

upper limit

9.20572

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Intermittent prednisone v Daily deflazacort

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.8141

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.800049

Confidence interval

level

98.3%

sides

2-sided

lower limit

-8.92431

upper limit

7.32421

Secondary: PedsQL School functioning (Child)

Top of page

End point title

PedsQL School functioning (Child)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	53	52	47
Units: Score
least squares mean (standard error)	66.997459 ( 2.571557 )	64.376071 ( 2.561826 )	65.459647 ( 2.537007 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

105

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.4193

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.621388

Confidence interval

level

98.3%

sides

2-sided

lower limit

-10.3734

upper limit

5.13058

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

100

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.639

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.537811

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.3671

upper limit

6.29145

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.7334

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

1.083577

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.5135

upper limit

8.68066

Secondary: PedsQL Psychological health (Child)

Top of page

End point title

PedsQL Psychological health (Child)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	54	52	47
Units: Score
least squares mean (standard error)	68.030401 ( 2.220309 )	66.535611 ( 2.172845 )	67.787745 ( 2.291333 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

106

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.5906

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.49479

Confidence interval

level

98.3%

sides

2-sided

lower limit

-8.13045

upper limit

5.14088

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

101

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9353

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.242656

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.38778

upper limit

6.90247

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6666

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

1.252134

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.69057

upper limit

8.19483

Secondary: PedsQL Total Score (Child)

Top of page

End point title

PedsQL Total Score (Child)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	54	52	47
Units: Score
least squares mean (standard error)	67.393595 ( 2.163314 )	64.964480 ( 2.167527 )	65.074246 ( 2.173933 )

MMRM (daily prednisone vs daily deflazacort

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

106

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3875

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.429115

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.14448

upper limit

4.28625

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Intermittent prednisone v Daily prednisone

Number of subjects included in analysis

101

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.424

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.319348

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.2513

upper limit

4.6126

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9683

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.109766

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.48556

upper limit

6.70509

Secondary: PedsQL Neuromuscular Disease (NMD) (Parent)

Top of page

End point title

PedsQL Neuromuscular Disease (NMD) (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	61	63	64
Units: Score
least squares mean (standard error)	80.451773 ( 1.297970 )	78.932563 ( 1.341342 )	77.958755 ( 1.329188 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.38

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.51921

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.65055

upper limit

2.61213

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.1474

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.493018

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.60089

upper limit

1.61485

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.574

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.973808

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.10986

upper limit

3.16224

Secondary: PedsQL Communication (Parent)

Top of page

End point title

PedsQL Communication (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	61	63	64
Units: Score
least squares mean (standard error)	70.761931 ( 2.905224 )	70.401908 ( 2.991088 )	67.041237 ( 3.072087 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.9256

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.360024

Confidence interval

level

98.3%

sides

2-sided

lower limit

-9.5599

upper limit

8.83985

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3416

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.720694

Confidence interval

level

98.3%

sides

2-sided

lower limit

-13.0598

upper limit

5.61846

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3832

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.360671

Confidence interval

level

98.3%

sides

2-sided

lower limit

-12.5607

upper limit

5.83933

Secondary: PedsQL Family resources (Parent)

Top of page

End point title

PedsQL Family resources (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	61	63	64
Units: Score
least squares mean (standard error)	81.638630 ( 1.948499 )	79.053570 ( 2.008589 )	74.842625 ( 1.988352 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.3145

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-2.58506

Confidence interval

level

98.3%

sides

2-sided

lower limit

-8.7193

upper limit

3.5492

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0079

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-6.796005

Confidence interval

level

98.3%

sides

2-sided

lower limit

-12.9005

upper limit

-0.69148

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Intermittent prednisone v Daily deflazacort

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0977

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-4.210945

Confidence interval

level

98.3%

sides

2-sided

lower limit

-10.2804

upper limit

1.8585

Secondary: PedsQL Neuromuscular Module Total (Parent)

Top of page

End point title

PedsQL Neuromuscular Module Total (Parent)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	61	63	64
Units: Score
least squares mean (standard error)	79.573350 ( 1.388228 )	77.934436 ( 1.442132 )	76.000414 ( 1.422186 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily prednisone v Daily deflazacort

Number of subjects included in analysis

124

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.37

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.638914

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.0025

upper limit

2.72468

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

125

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0521

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.572935

Confidence interval

level

98.3%

sides

2-sided

lower limit

-7.96408

upper limit

0.81821

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

127

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2927

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-1.934022

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.32235

upper limit

2.4543

Secondary: PedsQL Neuromuscular Disease (Child)

Top of page

End point title

PedsQL Neuromuscular Disease (Child)

End point description

End point type

Secondary

End point timeframe

Months 3-36

End point values	Daily prednisone	Daily deflazacort	Intermittent prednisone
Number of subjects analysed	52	49	48
Units: Score
least squares mean (standard error)	80.418850 ( 1.802410 )	79.495976 ( 1.785707 )	79.977143 ( 1.754817 )

MMRM (daily prednisone vs daily deflazacort)

Comparison groups

Daily deflazacort v Daily prednisone

Number of subjects included in analysis

101

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.6849

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.922874

Confidence interval

level

98.3%

sides

2-sided

lower limit

-6.35415

upper limit

4.5084

MMRM (daily prednisone vs intermittent prednisone)

Comparison groups

Daily prednisone v Intermittent prednisone

Number of subjects included in analysis

100

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.8478

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.441707

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.93813

upper limit

5.05472

MMRM (daily deflazacort vs intermittent prednisone

Comparison groups

Daily deflazacort v Intermittent prednisone

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.8348

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

0.481167

Confidence interval

level

98.3%

sides

2-sided

lower limit

-5.02832

upper limit

5.99066

Adverse events

Top of page

Timeframe for reporting adverse events

All non-SAEs/SARs occurring during drug treatment were reported on the eCRF system within four weeks of the form being due.

Adverse event reporting additional description

All Adverse Events were recorded. PIs were responsible for managing all AEs/ARs according to local protocols.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

2.1

Reporting group title

Daily prednisone

Reporting group description

Participants receiving daily prednisone.

Reporting group title

Daily deflazacort

Reporting group description

Participants receiving daily deflazacort.

Reporting group title

Intermittent prednisone

Reporting group description

Participants receiving prednisone, 10 days on, 10 days off.

Serious adverse events	Daily prednisone	Daily deflazacort	Intermittent prednisone
Total subjects affected by serious adverse events
subjects affected / exposed	7 / 65 (10.77%)	10 / 65 (15.38%)	5 / 66 (7.58%)
number of deaths (all causes)	0	0	0
number of deaths resulting from adverse events	0	0	0
Injury, poisoning and procedural complications
Fracture
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Fall
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Head injury
subjects affected / exposed	0 / 65 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 2
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Surgical and medical procedures
Adenotonsillectomy
subjects affected / exposed	1 / 65 (1.54%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Tenotomy
subjects affected / exposed	1 / 65 (1.54%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Umbilical hernia repair
subjects affected / exposed	0 / 65 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Circumcision
subjects affected / exposed	0 / 65 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Orchidopexy
subjects affected / exposed	0 / 65 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Nervous system disorders
Headache
subjects affected / exposed	0 / 65 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Blood and lymphatic system disorders
Lymphadenopathy
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	0 / 65 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Diarrhoea
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Vomiting
subjects affected / exposed	0 / 65 (0.00%)	2 / 65 (3.08%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Appendicitis
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Infection
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	2 / 66 (3.03%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 2
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Incontinence
subjects affected / exposed	1 / 65 (1.54%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Constipation
subjects affected / exposed	1 / 65 (1.54%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Abdominal pain
subjects affected / exposed	1 / 65 (1.54%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 2
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Infection
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Epistaxis
subjects affected / exposed	0 / 65 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Renal and urinary disorders
Chromaturia
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Myoglobinuria
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Hypercalciuria
subjects affected / exposed	1 / 65 (1.54%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
subjects affected / exposed	1 / 65 (1.54%)	2 / 65 (3.08%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 3	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Joint pain
subjects affected / exposed	0 / 65 (0.00%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Intervertebral disc compression
subjects affected / exposed	2 / 65 (3.08%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	2 / 2	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Infections and infestations
Infection
subjects affected / exposed	1 / 65 (1.54%)	4 / 65 (6.15%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 1	0 / 5	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Daily prednisone	Daily deflazacort	Intermittent prednisone
Total subjects affected by non serious adverse events
subjects affected / exposed	61 / 65 (93.85%)	58 / 65 (89.23%)	55 / 66 (83.33%)
Injury, poisoning and procedural complications
Fall
subjects affected / exposed	10 / 65 (15.38%)	7 / 65 (10.77%)	12 / 66 (18.18%)
occurrences all number	15	9	16
Nervous system disorders
Headache
subjects affected / exposed	9 / 65 (13.85%)	9 / 65 (13.85%)	6 / 66 (9.09%)
occurrences all number	25	17	8
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	12 / 65 (18.46%)	11 / 65 (16.92%)	10 / 66 (15.15%)
occurrences all number	21	12	13
Gastrointestinal disorders
Vomiting
subjects affected / exposed	19 / 65 (29.23%)	16 / 65 (24.62%)	15 / 66 (22.73%)
occurrences all number	34	25	21
Diarrhoea
subjects affected / exposed	9 / 65 (13.85%)	7 / 65 (10.77%)	10 / 66 (15.15%)
occurrences all number	11	8	11
Abdominal pain
subjects affected / exposed	8 / 65 (12.31%)	8 / 65 (12.31%)	8 / 66 (12.12%)
occurrences all number	14	10	10
Abdominal pain upper
subjects affected / exposed	10 / 65 (15.38%)	4 / 65 (6.15%)	10 / 66 (15.15%)
occurrences all number	11	4	12
Constipation
subjects affected / exposed	7 / 65 (10.77%)	9 / 65 (13.85%)	7 / 66 (10.61%)
occurrences all number	9	11	9
Nausea
subjects affected / exposed	5 / 65 (7.69%)	4 / 65 (6.15%)	1 / 66 (1.52%)
occurrences all number	5	4	1
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	13 / 65 (20.00%)	12 / 65 (18.46%)	11 / 66 (16.67%)
occurrences all number	20	14	22
Nasopharyngitis
subjects affected / exposed	9 / 65 (13.85%)	9 / 65 (13.85%)	11 / 66 (16.67%)
occurrences all number	23	18	18
Upper respiratory tract infection
subjects affected / exposed	11 / 65 (16.92%)	11 / 65 (16.92%)	7 / 66 (10.61%)
occurrences all number	19	11	10
Rhinitis
subjects affected / exposed	4 / 65 (6.15%)	5 / 65 (7.69%)	6 / 66 (9.09%)
occurrences all number	5	6	8
Skin and subcutaneous tissue disorders
Hypertrichosis
subjects affected / exposed	5 / 65 (7.69%)	7 / 65 (10.77%)	0 / 66 (0.00%)
occurrences all number	7	7	0
Psychiatric disorders
Abnormal behaviour
subjects affected / exposed	12 / 65 (18.46%)	17 / 65 (26.15%)	16 / 66 (24.24%)
occurrences all number	14	20	20
Mood altered
subjects affected / exposed	4 / 65 (6.15%)	7 / 65 (10.77%)	2 / 66 (3.03%)
occurrences all number	4	8	2
Aggression
subjects affected / exposed	4 / 65 (6.15%)	4 / 65 (6.15%)	2 / 66 (3.03%)
occurrences all number	5	4	3
Renal and urinary disorders
Pollakiuria
subjects affected / exposed	4 / 65 (6.15%)	2 / 65 (3.08%)	5 / 66 (7.58%)
occurrences all number	8	3	6
Musculoskeletal and connective tissue disorders
Pain in extremity
subjects affected / exposed	8 / 65 (12.31%)	10 / 65 (15.38%)	5 / 66 (7.58%)
occurrences all number	9	15	7
Back pain
subjects affected / exposed	5 / 65 (7.69%)	6 / 65 (9.23%)	8 / 66 (12.12%)
occurrences all number	5	6	8
Arthralgia
subjects affected / exposed	3 / 65 (4.62%)	6 / 65 (9.23%)	2 / 66 (3.03%)
occurrences all number	3	8	2
Infections and infestations
Ear infection
subjects affected / exposed	4 / 65 (6.15%)	7 / 65 (10.77%)	8 / 66 (12.12%)
occurrences all number	6	7	10
Influenza
subjects affected / exposed	8 / 65 (12.31%)	1 / 65 (1.54%)	2 / 66 (3.03%)
occurrences all number	10	1	2
Metabolism and nutrition disorders
Abnormal weight gain
subjects affected / exposed	9 / 65 (13.85%)	4 / 65 (6.15%)	5 / 66 (7.58%)
occurrences all number	12	4	5
Vitamin D deficiency
subjects affected / exposed	7 / 65 (10.77%)	6 / 65 (9.23%)	5 / 66 (7.58%)
occurrences all number	9	9	7
Cushingoid
subjects affected / exposed	6 / 65 (9.23%)	6 / 65 (9.23%)	3 / 66 (4.55%)
occurrences all number	7	6	3

More information

Top of page

Were there any global substantial amendments to the protocol? Yes

12 Jun 2012

Changes to: Study protocol Dear Colleagues EU Letter FAQ FOR DMD – EU GP Notification Letter Parent Consent Form Child PIS age four to five Parent PIS New documents: Instructions about study drug for families Letter for patients’ associations/registries Document changed since previous submission: IOWA-Conners questionnaire Biobank information: Biobank FOR DMD Information Biobank Consent Form Adult Parents Biobank Information Sheet (adult) Biobank Information Sheet (child 10-16) Biobank Information Sheet (child <10)

09 Dec 2015

Substantial Amendment to set up Addenbrooke's Hospital, Cambridge, as a trial site.

24 Aug 2016

Substantial Amendment covering: • Protocol changes, as detailed in the enclosed document, covering: o A change to allow FOR-DMD trial participants to take part in other trials, after 36 months’ participation in FOR-DMD o A change to allow FOR-DMD trial participants to take part in FOR-DMD for a minimum of 36 rather than 60 months, with the option to remain on the trial to 60 months o Only primary outcome measures and safety variables will be tracked at years four and five o Participants will be unblended at 60 months, with unblinding after 36 months only to allow the participant to take part in another trial, or for clinical need, rather than other reasons eg request of participant or his family o Removal of some tests after 36 months’ participation o Changes to the Schedule of Evaluations, and screening procedures o Changes regarding documentation of concomitant medication o A decrease in the sample size from 300 to 225 participants, at 35, rather than 40, trial sites o An increase in the recruitment period, from two and a half years to three years o Clarification of terms, update of an address, and correction of typos • A change to the Reference Safety Information (RSI) for the trial From SmPC for deflazacort, 22 July 2009, and prednisone, 30 April 2009. To Section 4.8 of SmPCs for deflazacort, 17 May 2015, and prednisone, 4 March 2013

14 Nov 2016

Substantial Amendment covering: • A change to the UK Co-ordinating Investigator (CI (UK term); PI (US term)). Dr Michela Guglieri Replaced: Prof Kate Bushby • A change to site of importation into the EU • A change to site of QP Certification From: Catalent (Deeside) to Catalent (Bathgate)

21 Feb 2017

This Amendment is to cover the requirements for Campaign 4 of the trial medication: - Change in manufacturer, from Catalent to Piramal - New IB (Edition 3) – new stability and dissolution information and undesirable effects lists updated for new versions of SmPCs - New IMPDs for Deflazacort and Prednisone (Version 3), with information on QP certification and updated information on undesirable effects from new SmPCs - Expiry date changes – bringing the MHRA up to date on what changed, and when

23 Jun 2017

Relates to the Urgent Safety Measure implemented to bridge trial participants between batches of trial medication, with interim prednisone/prednisolone Current trial medication expires 30 June 2017. New stocks not available until mid- to late-July 2017. ALSO includes implementation of Amendment 7 (substantial) – provision of new batch of trial medication with later expiry date (Campaign 4, split to Campaigns 4a and 4b, to allow provision of the most urgently-needed kits first).

25 Apr 2019

This relates to a change in the PI at the Birmingham, UK (GBR03), trial site.

Were there any global interruptions to the trial? Yes

Date	Interruption	Restart date
01 Jul 2017	Transfer of all participants in the FOR-DMD trial, who still receive trial medication, to open-label prednisone or prednisolone. The reason for this was that the stock current at the time expired on 30 June 2017, and new supplies of trial medication were not available immediately.	18 Jul 2017

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results: Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Reciprocal of time to rise from the floor averaged over months 3-36

Primary: Reciprocal of time to rise from the floor averaged over months 3-36

Primary: Forced Vital Capacity (FVC)

Primary: Forced Vital Capacity (FVC)

Primary: TSQM Global Satisfaction Subscale Score

Primary: TSQM Global Satisfaction Subscale Score

Secondary: Forced Vital Capacity (FVC) %

Secondary: Forced Vital Capacity (FVC) %

Secondary: 10 meter walk/run velocity

Secondary: 10 meter walk/run velocity

Secondary: NSAA Total Score

Secondary: NSAA Total Score

Secondary: Six-minute Walk Test distance

Secondary: Six-minute Walk Test distance

Secondary: TSQM Effectiveness Subscale

Secondary: TSQM Effectiveness Subscale

Secondary: TSQM Side effects Subscale

Secondary: TSQM Side effects Subscale

Secondary: Participant weight

Secondary: Participant weight

Secondary: Participant height

Secondary: Participant height

Secondary: Body Mass Index (BMI)

Secondary: Body Mass Index (BMI)

Secondary: IOWA Conners Total Score

Secondary: IOWA Conners Total Score

Secondary: PARS-III Total Score

Secondary: PARS-III Total Score

Secondary: SDQ Total Difficulties Score

Secondary: SDQ Total Difficulties Score

Secondary: Revised Rutter Scale Score

Secondary: Revised Rutter Scale Score

Secondary: Systolic Blood Pressure

Secondary: Systolic Blood Pressure

Secondary: Diastolic Blood Pressure

Secondary: Diastolic Blood Pressure

Secondary: Pulse

Secondary: Pulse

Secondary: PedsQL Physical functioning (Parent)

Secondary: PedsQL Physical functioning (Parent)

Secondary: PedsQL Emotional functioning (Parent)

Secondary: PedsQL Emotional functioning (Parent)

Secondary: PedsQL Social functioning (Parent)

Secondary: PedsQL Social functioning (Parent)

Secondary: PedsQL School functioning (Parent)

Secondary: PedsQL School functioning (Parent)

Secondary: PedsQL Psychological health (Parent)

Secondary: PedsQL Psychological health (Parent)

Secondary: PedsQL Total Score (Parent)

Secondary: PedsQL Total Score (Parent)

Secondary: PedsQL Physical functioning (Child)

Secondary: PedsQL Physical functioning (Child)

Secondary: PedsQL Emotional functioning (Child)

Secondary: PedsQL Emotional functioning (Child)

Secondary: PedsQL Social functioning (Child)

Secondary: PedsQL Social functioning (Child)

Secondary: PedsQL School functioning (Child)

Secondary: PedsQL School functioning (Child)

Secondary: PedsQL Psychological health (Child)

Secondary: PedsQL Psychological health (Child)

Secondary: PedsQL Total Score (Child)

Secondary: PedsQL Total Score (Child)

Secondary: PedsQL Neuromuscular Disease (NMD) (Parent)

Secondary: PedsQL Neuromuscular Disease (NMD) (Parent)

Secondary: PedsQL Communication (Parent)

Secondary: PedsQL Communication (Parent)

Secondary: PedsQL Family resources (Parent)

Secondary: PedsQL Family resources (Parent)

Secondary: PedsQL Neuromuscular Module Total (Parent)

Secondary: PedsQL Neuromuscular Module Total (Parent)

Clinical Trial Results:
Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen