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Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level. It can protect mitochondria from oxidative damage and boost their impaired function. It is thought that this mechanism will slow decline in heart function that is part of the disease process of Duchenne Muscular Dystrophy (DMD). It is possible that patients may benefit in terms of muscle strength and respiratory function. This pilot trial is designed to investigate this.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental |
| |
| 2 | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| idebenone | Drug | idebenone 450 mg/day (150 mg three times a day) |
| |
| placebo |
| Measure | Description | Time Frame |
|---|---|---|
| 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). |
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. | baseline and Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF) | 1 year | |
| 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) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gunnar Buyse, MD PhD | Universitaire Ziekenhuizen KU Leuven | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital, University Hospital | Leuven | Belgium |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21435876 | Result | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Idebenone 450 mg/ Day | idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals |
| FG001 | Placebo | Placebo tablet: One tablet 3 times a day (Tid) with meals |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Idebenone 450 mg/ Day | idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals |
| BG001 | Placebo | Placebo tablet: One tablet 3 times a day (Tid) with meals |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). |
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. | 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. | Posted | Mean | Standard Deviation | % change in peak systolic | baseline and Week 52 |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Idebenone 450 mg/ Day | idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ankle fracture | Injury, poisoning and procedural complications | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastrointestinal disorders | Gastrointestinal disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Thomas Meier | Santhera | +4161 906 8964 | thomas.meier@santhera.com |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| C036619 | idebenone |
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| Drug |
|
| 1 year |
| Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG. | 1 year |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Description |
|---|
| OG000 | Idebenone 450 mg/ Day | idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals |
| OG001 | Placebo | Placebo tablet: One tablet 3 times a day (Tid) with meals |
|
|
| Secondary | Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF) | Not Posted | 1 year |
| Secondary | 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) | Not Posted | 1 year |
| Secondary | Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG. | Not Posted | 1 year |
| 1 |
| 13 |
| 12 |
| 13 |
| EG001 | Placebo | Placebo tablet: One tablet 3 times a day (Tid) with meals | 1 | 8 | 8 | 8 |
| Femur fracture | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Infections | Infections and infestations | Non-systematic Assessment |
|
| General disorders | General disorders | Non-systematic Assessment |
|
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |