Trial · NCT01027884

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

NCT01027884, Phase 3, Completed, Santhera Pharmaceuticals; results first posted 2015-10-19

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD). Trials, record NCT01027884. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01027884

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
NCT number: NCT01027884
Status: Completed
Phases: Phase 3
Enrollment: 65 participants, actual
Lead sponsor: Santhera Pharmaceuticals
Conditions: Muscular Dystrophy, Duchenne, Ambulatory Care
Interventions: Placebo, Idebenone
First posted: 2009-12-09
Last updated: 2015-10-19
Results first posted: 2015-10-19

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52	Baseline and Week 52	Change from Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52
Secondary	Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52	Baseline and Week 52	Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Secondary	Change From Baseline to Week 52 in Muscle Strength	Baseline and Week 52	The change from Baseline to Week 52 in muscle strength as measured by Hand-Held Myometry (HHM) was performed following standardized procedures. As almost all patients were non-ambulatory, only analyses of upper limb muscle strength were performed. Results for elbow flexors and for elbow extensors are reported below.The highest value of 3 consecutive measurements with an interval of at least 10 seconds were recorded. The HHM was measured using MicroFET2, a digital hand held muscle tester. The selected unit of measure was Newtons (N).
Secondary	Change From Baseline to Week 52 in Quality of Life Assessed by PedsQL™ Paediatric Quality of Life Inventory	Baseline and Week 52	PedsQL Quality of Life Inventory contains paediatric HRQOL measurements: Physical, Emotional,Social and School Functioning. Item Scaling: 5-point Likert scale from 0 (Never) to 4 (Almost always). 3-point scale: 0 (Not at all), 2 (Sometimes) and 4 (A lot) for the Young Child (ages 5-7). Scores are transformed on a scale from 0 to 100 ( 0=100, 1=75, 2=50, 3=25, 4=0) Total Score: Sum of all the items over the number of items answered on all the Scales. The values reported below are overall scores on Paediatric Quality of Life Inventory in Child/Teen Report. These scores were obtained by averaging scores for all the described subscales. The overall scores range between 0-100 with 0 = worst outcome and 100= best outcome
Secondary	Percentage of Patients Reporting Adverse Events	52 Weeks

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52	Baseline and Week 52	This analysis was performed on the ITT population(n=64). This population included all randomized patients who received at least one dose of the study medication and provided at least one post-Baseline assessment. It excluded siblings who had been allocated to the same study treatment as a randomized sibling.	percentage	GEOMETRIC_MEAN
SECONDARY	Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52	Baseline and Week 52	This analysis was performed on the ITT population(n=64). This population included all randomized patients who received at least one dose of the study medication and provided at least one post-Baseline assessment. It excluded siblings who had been allocated to the same study treatment as a randomized sibling.	percentage of Predicted FVC	MEAN
SECONDARY	Change From Baseline to Week 52 in Muscle Strength	Baseline and Week 52	The number of patients (N) in each treatment group is the number of patients with baseline assessments	Newtons	MEAN
SECONDARY	Change From Baseline to Week 52 in Quality of Life Assessed by PedsQL™ Paediatric Quality of Life Inventory	Baseline and Week 52	The number of patients (N) in each treatment group is the number of patients with Baseline assessments.	units on a scale	MEAN
SECONDARY	Percentage of Patients Reporting Adverse Events	52 Weeks		percentage of patients reporting AEs	NUMBER

Source ClinicalTrials.gov

Results by group12

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52	Placebo		-8.84	95% Confidence Interval -12.73 to -4.95	percentage
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52	Idebenone		-2.57	95% Confidence Interval -6.68 to 1.54	percentage
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52	Placebo		-8.95	95% Confidence Interval -11.47 to -6.42	percentage of Predicted FVC
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52	Idebenone		-5.67	95% Confidence Interval -8.36 to -2.99	percentage of Predicted FVC
Change From Baseline to Week 52 in Muscle Strength	Placebo	Elbow Flexors	0.13	95% Confidence Interval -4.16 to 4.41	Newtons
Change From Baseline to Week 52 in Muscle Strength	Idebenone	Elbow Flexors	-2.32	95% Confidence Interval -6.73 to 2.09	Newtons
Change From Baseline to Week 52 in Muscle Strength	Placebo	Elbow Extensors	1.32	95% Confidence Interval -2.53 to 5.18	Newtons
Change From Baseline to Week 52 in Muscle Strength	Idebenone	Elbow Extensors	0.26	95% Confidence Interval -3.95 to 4.46	Newtons
Change From Baseline to Week 52 in Quality of Life Assessed by PedsQL™ Paediatric Quality of Life Inventory	Placebo		2.46	95% Confidence Interval -1.77 to 6.69	units on a scale
Change From Baseline to Week 52 in Quality of Life Assessed by PedsQL™ Paediatric Quality of Life Inventory	Idebenone		-1.34	95% Confidence Interval -5.99 to 3.31	units on a scale

1 to 10 of 12Source ClinicalTrials.gov

See all 12 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo	34
Overall Study	STARTED	Idebenone	32
Overall Study	COMPLETED	Placebo	30
Overall Study	COMPLETED	Idebenone	25
Overall Study	NOT COMPLETED	Placebo	4
Overall Study	NOT COMPLETED	Idebenone	7

Source ClinicalTrials.gov

Adverse events72

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Left ventricular failure	Cardiac disorders	Placebo	1	34	1
other	Left ventricular failure	Cardiac disorders	Idebenone	3	32	3
other	Electrocardiogram abnormal	Cardiac disorders	Placebo	1	34	1
other	Electrocardiogram abnormal	Cardiac disorders	Idebenone	2	32	3
other	Blood phosporus increased	Blood and lymphatic system disorders	Placebo	3	34	4
other	Blood phosporus increased	Blood and lymphatic system disorders	Idebenone	1	32	1
other	Bronchitis	Respiratory, thoracic and mediastinal disorders	Placebo	6	34	7
other	Bronchitis	Respiratory, thoracic and mediastinal disorders	Idebenone	4	32	5
other	Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	Placebo	2	34	2
other	Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	Idebenone	3	32	3

1 to 10 of 72Source ClinicalTrials.gov

See all 72 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo
Total, all-cause mortality	Idebenone
Total, other adverse events	Placebo	32	34
Total, other adverse events	Idebenone	30	32
Total, serious adverse events	Placebo	5	34
Total, serious adverse events	Idebenone	2	32

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
25907158	RESULT	PubMed
28189481	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
Idebenone vs Placebo: serious adverse events

Source Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01027884

GET https://conductscience.org/api/trials/v1/NCT01027884/events?page=2

API reference For AI assistants