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This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
The study involves 6 clinic visits.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | high dose Idebenone |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Idebenone | Drug | Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in ICARS | International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability. | baseline and 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| FARS (Friedreich's Ataxia Rating Scale) | FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. |
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Inclusion criteria:
Exclusion criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Susan Perlman, MD | University of California, Los Angeles | Principal Investigator |
| David Lynch, MD | Children's Hospital of Philadelphia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| David Geffen School of Medicine, UCLA | Los Angeles | California | 90095-1769 | United States | ||
| The Children's Hopsital of Philadelphia |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21779958 | Result | Meier T, Perlman SL, Rummey C, Coppard NJ, Lynch DR. Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study. J Neurol. 2012 Feb;259(2):284-91. doi: 10.1007/s00415-011-6174-y. Epub 2011 Jul 22. |
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A total of 59 subjects completed the study. Nine subjects did not complete the study. However, neurological efficacy data (ICARS, FARS) are available for Week 52 from 61 patients.
All 68 subjects enrolled in this study were included in the Safety population.
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| ID | Title | Description |
|---|---|---|
| FG000 | High Dose Idebenone | Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.) Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.) |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | High Dose Idebenone | Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.) Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.) |
| 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 | Change in ICARS | International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability. | A total of 59 subjects completed the study. Nine subjects did not complete the study. However, neurological efficacy data (ICARS, FARS) are available for Week 52 from 61 patients. | Posted | Mean | Standard Deviation | units on a scale | baseline and 12 months |
|
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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 | High Dose Idebenone | Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.) Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| cardiac failure | Cardiac disorders |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| headache | General disorders |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr Thomas Meier | Santhera | +41 61 906 8964 | thomas.meier@santhera.com |
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| ID | Term |
|---|---|
| D005621 | Friedreich Ataxia |
| ID | Term |
|---|---|
| D013132 | Spinocerebellar Degenerations |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C036619 | idebenone |
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| baseline and 12 Months |
| Nature of Adverse Events | 12 Months |
| Frequency/Number of Mild, Moderate, and Severe Adverse Events | 12 months |
| Philadelphia |
| Pennsylvania |
| 19104 |
| United States |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day (3 x 150 mg tablets, t.i.d.)
Patients > 45 kg/99 lbs: idebenone 2250 mg/day (5 x 150 mg tablets, t.i.d.)
|
|
| Secondary | FARS (Friedreich's Ataxia Rating Scale) | FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. | A total of 59 subjects completed the study. Nine subjects did not complete the study. However, neurological efficacy data (ICARS, FARS) are available for Week 52 from 61 patients. | Posted | Mean | Standard Deviation | units on a scale | baseline and 12 Months |
|
|
|
| Secondary | Nature of Adverse Events | All 68 subjects enrolled in this study were included in the Safety population. | Posted | Number | events | 12 Months |
|
|
|
| Secondary | Frequency/Number of Mild, Moderate, and Severe Adverse Events | All 68 subjects enrolled in this study were included in the Safety population. | Posted | Number | events | 12 months |
|
|
|
| 4 |
| 68 |
| 66 |
| 68 |
| tachycardia | Cardiac disorders |
|
| Hyperthyroidism | Endocrine disorders |
|
| Hypotension | General disorders |
|
| Deep vein thrombosis | General disorders |
|
| Dehydratation | General disorders |
|
| Pregnancy | Pregnancy, puerperium and perinatal conditions |
|
| intra uterine death | Pregnancy, puerperium and perinatal conditions |
|
| abortion spontaneous | Pregnancy, puerperium and perinatal conditions |
|
| Pharyngitis streptococcal | Infections and infestations |
|
| Pneumonia | Infections and infestations |
|
| upper respiratory tract infection | Infections and infestations |
|
| nausea | Gastrointestinal disorders |
|
| pyrexia | General disorders |
|
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| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
|