Trial Outcomes & Findings for Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy (NCT NCT00654784)

Results Overview

* Assessing the peak systolic radial strain of the left ventricle inferolateral wall is used to characterize the cardiac involvement in the DMD patients. * Color Doppler Myocardial Imaging technique is used to quantify regional myocardial function. The cardiac involvement in DMD is characterized by degeneration, atrophy and fibrosis of the myocardium, leading to dilated cardiomyopathy. The process begins in the posterolateral wall of the left ventricle, with septal involvement appearing at later stages.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

baseline and Week 52

Results posted on

2011-08-01

Participant Flow

Participant milestones

Participant milestones
Measure	Idebenone 450 mg/ Day idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo Placebo tablet: One tablet 3 times a day (Tid) with meals
Overall Study STARTED	13	8
Overall Study COMPLETED	13	8
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Idebenone 450 mg/ Day n=13 Participants idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo n=8 Participants Placebo tablet: One tablet 3 times a day (Tid) with meals	Total n=21 Participants Total of all reporting groups
Age, Categorical <=18 years	13 Participants n=5 Participants	8 Participants n=7 Participants	21 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age Continuous	13.4 years STANDARD_DEVIATION 2.1 • n=5 Participants	10.8 years STANDARD_DEVIATION 1.9 • n=7 Participants	12.4 years STANDARD_DEVIATION 2.0 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants	8 Participants n=7 Participants	21 Participants n=5 Participants
Region of Enrollment Belgium	13 participants n=5 Participants	8 participants n=7 Participants	21 participants n=5 Participants

PRIMARY outcome

Timeframe: baseline and Week 52

Population: At Week 52, data from only 18 patients were available for the primary endpoint analysis due to missing data from 2 patients and the inability to acquire data from a 3rd patient. The efficacy comparison for the primary endpoint was conducted using the LOCF method in the ITT population. In addition, the analysis was repeated using the OC dataset.

* Assessing the peak systolic radial strain of the left ventricle inferolateral wall is used to characterize the cardiac involvement in the DMD patients. * Color Doppler Myocardial Imaging technique is used to quantify regional myocardial function. The cardiac involvement in DMD is characterized by degeneration, atrophy and fibrosis of the myocardium, leading to dilated cardiomyopathy. The process begins in the posterolateral wall of the left ventricle, with septal involvement appearing at later stages.

Outcome measures

Outcome measures
Measure	Idebenone 450 mg/ Day n=11 Participants idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo n=7 Participants Placebo tablet: One tablet 3 times a day (Tid) with meals
The Relative Change in Peak Systolic Radial Strain of the Left Ventricle (LV) Inferolateral Wall From Baseline (at Screening) to Week 52, Assessed by Color Doppler Myocardial Imaging (CDMI).	104.4 % change in peak systolic Standard Deviation 92.0	28.9 % change in peak systolic Standard Deviation 40.7

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

Adverse Events

Idebenone 450 mg/ Day

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Idebenone 450 mg/ Day n=13 participants at risk idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo n=8 participants at risk Placebo tablet: One tablet 3 times a day (Tid) with meals
Injury, poisoning and procedural complications Ankle fracture	7.7% 1/13 • Number of events 1	0.00% 0/8
Injury, poisoning and procedural complications Femur fracture	0.00% 0/13	12.5% 1/8 • Number of events 1

Other adverse events

Other adverse events
Measure	Idebenone 450 mg/ Day n=13 participants at risk idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo n=8 participants at risk Placebo tablet: One tablet 3 times a day (Tid) with meals
Gastrointestinal disorders Gastrointestinal disorders	30.8% 4/13 • Number of events 5	62.5% 5/8 • Number of events 6
Infections and infestations Infections	61.5% 8/13 • Number of events 19	62.5% 5/8 • Number of events 12
General disorders General disorders	15.4% 2/13 • Number of events 4	12.5% 1/8 • Number of events 1

Additional Information

Thomas Meier

Santhera

Phone: +4161 906 8964

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place