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The European Union Clinical Trials Register allows you to search for protocol and results information on:

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The EU Clinical Trials Register currently displays 43865 clinical trials with a EudraCT protocol, of which 7286 are clinical trials conducted with subjects less than 18 years old. The register also displays information on 18700 older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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Clinical Trial Results:
A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy

Summary
EudraCT number	2009-012037-30
Trial protocol	BE DE FR NL SE AT ES IT
Global end of trial date	24 Apr 2014

Results information
Results version number	v2(current)
This version publication date	30 Oct 2019
First version publication date	07 Jun 2015
Other versions	v1 (removed from public view)
Version creation reason	Correction of full data set Reposting needed due to reassignement of results owner in EudrCT + updating of short name of study
Summary report(s)	Clinical Study Report Synopsis Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

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

Collapse all Expand all

Trial information

Top of page

Sponsor protocol code

SNT-III-003

ISRCTN number

-

US NCT number

NCT01027884

WHO universal trial number (UTN)

-

Sponsor organisation name

Santhera Pharmaceuticals (Switzerland) Ltd

Sponsor organisation address

Hammerstrasse 49, Liestal, Switzerland, CH-4410

Public contact

Dr. Gunnar Buyse, University Hospital Leuven-Children Hospital, +32 016343845,

Scientific contact

Dr. Gunnar Buyse, University Hospital Leuven-Children Hospital, +41 09068950,

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

No

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

No

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

No

Analysis stage

Final

Date of interim/final analysis

24 Apr 2014

Is this the analysis of the primary completion data?

Yes

Primary completion date

14 Jan 2014

Global end of trial reached?

Yes

Global end of trial date

24 Apr 2014

Was the trial ended prematurely?

No

Main objective of the trial

•To assess the efficacy of idebenone, compared to placebo, in improving respiratory function or delaying the loss of respiratory function in patients with DMD

Protection of trial subjects

This study was completed and archived according to the guidelines of Good Clinical Practice (GCP), ICH E3 (CPMP/ICH/135/95) and conducted in compliance with the World Medical Assembly Declaration of Helsinki and its most recent amendments.

Background therapy

-

Evidence for comparator

-

Actual start date of recruitment

05 Apr 2010

Long term follow-up planned

No

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Austria: 5

Country: Number of subjects enrolled

Belgium: 8

Country: Number of subjects enrolled

France: 15

Country: Number of subjects enrolled

Germany: 10

Country: Number of subjects enrolled

Italy: 9

Country: Number of subjects enrolled

United States: 4

Country: Number of subjects enrolled

Switzerland: 4

Country: Number of subjects enrolled

Netherlands: 7

Country: Number of subjects enrolled

Spain: 2

Country: Number of subjects enrolled

Sweden: 2

Worldwide total number of subjects

66

EEA total number of subjects

58

Number of subjects enrolled per age group

In utero

0

Preterm newborn - gestational age < 37 wk

0

Newborns (0-27 days)

0

Infants and toddlers (28 days-23 months)

0

Children (2-11 years)

16

Adolescents (12-17 years)

43

Adults (18-64 years)

7

From 65 to 84 years

0

85 years and over

0

Subject disposition

Top of page

Recruitment details

A total of 23 centers in 10 countries (Belgium, Germany, The Netherlands, Switzerland, France, Sweden, Austria, United States, Italy and Spain) participated in this study. Seventeen centers in 10 countries enrolled patients. Study Period: 27 July 2009 (first subject screened) to 14 January 2014 (last subject completed).

Screening details

96 patients were screened. Inclusion criteria required: - patients to be able to provide reliable and reproducible repeat PEF measurements (within 15% of the first assessment, i.e. Baseline vs. Screening) - no previous use of idebenone, CoQ10 or vitamin E - no glucocorticoid steroids use

Period 1 title

Overall trial (overall period)

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

Idebenone

Arm description

Treatment

Arm type

Experimental

Investigational medicinal product name

Idebenone

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Idebenone was formulated as film-coated 150 mg tablets. Patients took 2 x 150 mg tablets orally 3 times daily with meals (total dose 900 mg daily).

Placebo

Arm description

no Treatment

Arm type

Placebo

Investigational medicinal product name

matching placebo tablets

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Two matching placebo tablets were taken three times a day with meals.

Number of subjects in period 1	Idebenone	Placebo
Started	32	34
Completed	25	30
Not completed	7	4
Consent withdrawn by subject	1	-
Adverse event, non-fatal	1	2
spinal fixation surgery	3	-
Lost to follow-up	1	-
non-compliance	1	1
Protocol deviation	-	1

Baseline characteristics

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Reporting group title

Idebenone

Reporting group description

Treatment

Reporting group title

Placebo

Reporting group description

no Treatment

Reporting group values	Idebenone	Placebo	Total
Number of subjects	32	34	66
Age categorical
Units: Subjects
In utero			0
Preterm newborn infants (gestational age < 37 wks)			0
Newborns (0-27 days)			0
Infants and toddlers (28 days-23 months)			0
Children (2-11 years)			0
Adolescents (12-17 years)			0
Adults (18-64 years)			0
From 65-84 years			0
85 years and over			0
Age continuous
Idebenone arm: 13.5 (2.7) mean age Placebo arm: 15.0 (2.5) mean age
Units: years
arithmetic mean (standard deviation)	13.5 ( 2.7 )	15 ( 2.5 )	-
Gender categorical
Young males (10 to 18 years old)
Units: Subjects
Male	32	34	66

End points

Top of page

Reporting group title

Idebenone

Reporting group description

Treatment

Reporting group title

Placebo

Reporting group description

no Treatment

Primary: Peak Expiratory Flow (PEF)

Top of page

End point title

Peak Expiratory Flow (PEF)

End point description

End point type

Primary

End point timeframe

52 weeks

End point values	Idebenone	Placebo
Number of subjects analysed	30	27
Units: percentage
number (confidence interval 95%)	-3.05 (-7.08 to 0.97)	-9.01 (-13.18 to -4.84)

Mixed Model for Repeated Measurements (MMRM)

Comparison groups

Placebo v Idebenone

Number of subjects included in analysis

57

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.05

Method

Mixed models analysis

Parameter type

Mean difference (final values)

Point estimate

5.96

Confidence interval

level

95%

sides

2-sided

lower limit

0.16

upper limit

11.76

Secondary: Peak Expiratory Flow (PEF), Forced Vital Capacity (FVC), Peak Cough Flow (PCF)

Top of page

End point title

Peak Expiratory Flow (PEF), Forced Vital Capacity (FVC), Peak Cough Flow (PCF)

End point description

To assess the efficacy of idebenone, compared to placebo, in improving respiratory function or delaying the loss of respiratory function using measures other than those used for the primary endpoint.

End point type

Secondary

End point timeframe

Respiratory function measurements were performed periodically over the entire study duration.

Number of subjects analysed

Units: L/min

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Entire study duration

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

14

Reporting group title

Idebenone

Reporting group description

Treatment

Reporting group title

Placebo

Reporting group description

no Treatment

Serious adverse events	Idebenone	Placebo
Total subjects affected by serious adverse events
subjects affected / exposed	2 / 32 (6.25%)	5 / 34 (14.71%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0
Injury, poisoning and procedural complications
Femur fracture
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Gastrointestinal disorders
Vomiting
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Failure
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Pulmonary microemboli
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Respiratory failure
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Skin and subcutaneous tissue disorders
Urticaria
subjects affected / exposed	1 / 32 (3.13%)	0 / 34 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Psychiatric disorders
Sleep apnoea syndrome
subjects affected / exposed	1 / 32 (3.13%)	0 / 34 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Tendinous contracture
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 2
deaths causally related to treatment / all	0 / 0	0 / 0
Infections and infestations
Pneumonia
subjects affected / exposed	0 / 32 (0.00%)	2 / 34 (5.88%)
occurrences causally related to treatment / all	0 / 0	0 / 3
deaths causally related to treatment / all	0 / 0	0 / 0
Nasopharyngitis
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Metabolism and nutrition disorders
Dehydration
subjects affected / exposed	0 / 32 (0.00%)	1 / 34 (2.94%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 0%

Non-serious adverse events	Idebenone	Placebo
Total subjects affected by non serious adverse events
subjects affected / exposed	30 / 32 (93.75%)	32 / 34 (94.12%)
Cardiac disorders
Left ventricular failure
subjects affected / exposed	3 / 32 (9.38%)	1 / 34 (2.94%)
occurrences all number	3	1
Electrocardiogram abnormal
subjects affected / exposed	2 / 32 (6.25%)	1 / 34 (2.94%)
occurrences all number	3	1
Nervous system disorders
Headache
subjects affected / exposed	6 / 32 (18.75%)	7 / 34 (20.59%)
occurrences all number	13	15
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	5 / 32 (15.63%)	3 / 34 (8.82%)
occurrences all number	6	4
Influenza like illness
subjects affected / exposed	2 / 32 (6.25%)	1 / 34 (2.94%)
occurrences all number	2	2
Blood and lymphatic system disorders
Blood phosphorus increased
subjects affected / exposed	1 / 32 (3.13%)	3 / 34 (8.82%)
occurrences all number	1	4
Gastrointestinal disorders
Diarrhoea
subjects affected / exposed	8 / 32 (25.00%)	4 / 34 (11.76%)
occurrences all number	10	6
Constipation
subjects affected / exposed	3 / 32 (9.38%)	6 / 34 (17.65%)
occurrences all number	4	6
Abdominal pain
subjects affected / exposed	3 / 32 (9.38%)	3 / 34 (8.82%)
occurrences all number	4	5
Nausea
subjects affected / exposed	1 / 32 (3.13%)	2 / 34 (5.88%)
occurrences all number	2	2
Vomiting
subjects affected / exposed	1 / 32 (3.13%)	2 / 34 (5.88%)
occurrences all number	1	3
Respiratory, thoracic and mediastinal disorders
Bronchitis
subjects affected / exposed	4 / 32 (12.50%)	6 / 34 (17.65%)
occurrences all number	5	7
Rhinorrhoea
subjects affected / exposed	3 / 32 (9.38%)	2 / 34 (5.88%)
occurrences all number	3	2
Nasal congestion
subjects affected / exposed	2 / 32 (6.25%)	1 / 34 (2.94%)
occurrences all number	2	1
Oropharyngeal pain
subjects affected / exposed	2 / 32 (6.25%)	1 / 34 (2.94%)
occurrences all number	2	1
Skin and subcutaneous tissue disorders
Seborrhoeic dermatitis
subjects affected / exposed	1 / 32 (3.13%)	2 / 34 (5.88%)
occurrences all number	1	2
Renal and urinary disorders
Chromaturia
subjects affected / exposed	3 / 32 (9.38%)	0 / 34 (0.00%)
occurrences all number	3	0
Musculoskeletal and connective tissue disorders
Back pain
subjects affected / exposed	2 / 32 (6.25%)	4 / 34 (11.76%)
occurrences all number	2	6
Scoliosis
subjects affected / exposed	2 / 32 (6.25%)	1 / 34 (2.94%)
occurrences all number	2	1
Infections and infestations
Nasopharyngitis
subjects affected / exposed	8 / 32 (25.00%)	9 / 34 (26.47%)
occurrences all number	12	11
Upper respiratory tract infection
subjects affected / exposed	2 / 32 (6.25%)	6 / 34 (17.65%)
occurrences all number	2	10
Gastroenteritis
subjects affected / exposed	5 / 32 (15.63%)	1 / 34 (2.94%)
occurrences all number	6	1
Rhinitis
subjects affected / exposed	1 / 32 (3.13%)	6 / 34 (17.65%)
occurrences all number	1	8
Otitis media
subjects affected / exposed	3 / 32 (9.38%)	0 / 34 (0.00%)
occurrences all number	3	0

More information

Top of page

Were there any global substantial amendments to the protocol? Yes

24 Sep 2009

Introduction of Group Sequential Design (of already pre-specified subgroups; cohort 1: glucocorticoid non-users; cohort 2: glucocorticoid users) Introduction of definitions for “Glucocorticoid non-users” Introduction of regular (weekly) assessment of PEF by the patient at home (in addition to assessment s during study site visits) Discontinuation of handgrip strength assessment (handheld myometry upper limb unchanged) Removal of cough frequency assessment as study endpoint Introduction of muscle strength and motor function testing at the Screening Visit

22 Feb 2010

Allow enrolment of siblings of randomized patients

18 Aug 2010

Introduction of second PEF assessment at every study visit

05 Jul 2011

Increase sample size of glucocorticoid non users

05 Dec 2012

Amendment of sample size required for prespecified futility analysis

19 Jun 2013

Amendment of time point for starting recruitment of glucocorticoid using patients

24 Apr 2014

Termination of the study following planned analysis of glucocorticoid non-user subgroup. Amendment of Type I error rate. Introduction of secondary endpoint: annual rate of change in PEF measured by ASMA-1 device.

Were there any global interruptions to the trial? No

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

None reported

http://www.ncbi.nlm.nih.gov/pubmed/25907158

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