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

• ClinicalTrials.gov Identifier: NCT00654784
• Recruitment Status: Completed
• First Posted: April 9, 2008
• Results First Posted: July 29, 2011
• Last Update Posted: August 1, 2011
• Sponsor: Santhera Pharmaceuticals
• Information provided by: Santhera Pharmaceuticals

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy (DMD)
• Interventions: Drug: idebenone; Drug: placebo
• Enrollment: 21

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Idebenone 450 mg/ Day	Placebo
Arm/Group Description	idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo tablet: One tablet 3 times a day (Tid) with meals
Period Title: Overall Study
Started	13	8
Completed	13	8
Not Completed	0	0

Baseline Characteristics

Arm/Group Title		Idebenone 450 mg/ Day	Placebo	Total
Arm/Group Description		idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo tablet: One tablet 3 times a day (Tid) with meals	Total of all reporting groups
Overall Number of Baseline Participants		13	8	21
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	13 participants	8 participants	21 participants
	<=18 years	13 100.0%	8 100.0%	21 100.0%
	Between 18 and 65 years	0 0.0%	0 0.0%	0 0.0%
	>=65 years	0 0.0%	0 0.0%	0 0.0%
Age Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	13 participants	8 participants	21 participants
		13.4 (2.1)	10.8 (1.9)	12.4 (2.0)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	13 participants	8 participants	21 participants
	Female	0 0.0%	0 0.0%	0 0.0%
	Male	13 100.0%	8 100.0%	21 100.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
Belgium	Number Analyzed	13 participants	8 participants	21 participants
		13	8	21

Outcome Measures

1. Primary Outcome

Title	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).
Description	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.
Time Frame	baseline and Week 52

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Idebenone 450 mg/ Day	Placebo
Arm/Group Description:	idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals	Placebo tablet: One tablet 3 times a day (Tid) with meals
Overall Number of Participants Analyzed	11	7
Mean (Standard Deviation); Unit of Measure: % change in peak systolic
	104.4 (92.0)	28.9 (40.7)

2. Secondary Outcome

Title	Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF)
Description	[Not Specified]
Time Frame	1 year

Outcome Measure Data Not Reported

3. Secondary Outcome

Title	Skeletal Muscle Strength (Upper Limb, Right and Left): Hand Grip, Elbow Flexors and Elbow Extensors (Upper Limb Score) Timed Walking Test (10 Metres) (Ambulant Patients Only)
Description	[Not Specified]
Time Frame	1 year

Outcome Measure Data Not Reported

4. Secondary Outcome

Title	Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG.
Description	[Not Specified]
Time Frame	1 year

Outcome Measure Data Not Reported

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Idebenone 450 mg/ Day		Placebo
Arm/Group Description	idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals		Placebo tablet: One tablet 3 times a day (Tid) with meals
All-Cause Mortality
	Idebenone 450 mg/ Day		Placebo
	Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--
Serious Adverse Events
	Idebenone 450 mg/ Day		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/13 (7.69%)		1/8 (12.50%)
Injury, poisoning and procedural complications
Ankle fracture *	1/13 (7.69%)	1	0/8 (0.00%)	0
Femur fracture *	0/13 (0.00%)	0	1/8 (12.50%)	1
* Indicates events were collected by non-systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	1%
	Idebenone 450 mg/ Day		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	12/13 (92.31%)		8/8 (100.00%)
Gastrointestinal disorders
Gastrointestinal disorders *	4/13 (30.77%)	5	5/8 (62.50%)	6
General disorders
General disorders *	2/13 (15.38%)	4	1/8 (12.50%)	1
Infections and infestations
Infections *	8/13 (61.54%)	19	5/8 (62.50%)	12
* Indicates events were collected by non-systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

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

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Thomas Meier
• Organization: Santhera
• Phone: +4161 906 8964
• EMail: thomas.meier@santhera.com

Publications of Results:

Buyse GM, Goemans N, van den Hauwe M, Thijs D, de Groot IJ, Schara U, Ceulemans B, Meier T, Mertens L. Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial. Neuromuscul Disord. 2011 Jun;21(6):396-405. doi: 10.1016/j.nmd.2011.02.016. Epub 2011 Mar 23.

• ClinicalTrials.gov Identifier: NCT00654784
• Other Study ID Numbers: SNT-II-001
• First Submitted: April 3, 2008
• First Posted: April 9, 2008
• Results First Submitted: June 7, 2011
• Results First Posted: July 29, 2011
• Last Update Posted: August 1, 2011