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This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm 1 - 900 mg/day Idebenone | Experimental | mid dose Idebenone |
|
| Arm 2 - up to 2250 mg/day Idebenone | Experimental | high dose Idebenone |
|
| Arm 3 - Placebo | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Idebenone | Drug | Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | 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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline | baseline and 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS) 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 exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. Calculation details FARS score at week 24 minus FARS score baseline |
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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. | |
| 21392622 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Mid Dose Idebenone | 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 450 mg/day (1 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 900 mg/day (2 x 150 mg tablet, t.i.d.) |
| FG001 | High Dose Idebenone | 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 1350 mg/day (2 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 2250 mg/day (2 x 150 mg tablet, t.i.d.) |
| FG002 | Placebo | Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Mid Dose Idebenone | 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 450 mg/day (1 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 900 mg/day (2 x 150 mg tablet, t.i.d.) |
| BG001 | High Dose Idebenone |
| 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 the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | 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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline | Posted | Mean | Standard Deviation | ICARS points | baseline and 6 months |
|
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"0" means none.
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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 | Mid Dose Idebenone | 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 450 mg/day (1 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 900 mg/day (2 x 150 mg tablet, t.i.d.) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| idiopathic thrombocytopenic purpura | Blood and lymphatic system disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infections | Infections and infestations | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr Thomas Meier | Santhera Pharmaceuticals | +41619068964 | 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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| Idebenone | Drug | Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day |
|
| Placebo | Drug | Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets. |
|
| baseline and 6 months |
| Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS) score 0 to 36, higher score indicates greater impairment | baseline and 6 months |
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 | To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 12 minus ICARS score baseline | baseline and 12 weeks |
| Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 6 months |
| Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 6 months |
| Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI) | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 6 months |
| Percent Change From Baseline to Week 24 in Left Ventricular Mass Index | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 6 months |
| Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 6 months |
| Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 6 months |
| Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 6 months |
| Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | baseline and 24 weeks |
| Philadelphia |
| Pennsylvania |
| 19104 |
| United States |
| Lagedrost SJ, Sutton MS, Cohen MS, Satou GM, Kaufman BD, Perlman SL, Rummey C, Meier T, Lynch DR. Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J. 2011 Mar;161(3):639-645.e1. doi: 10.1016/j.ahj.2010.10.038. Epub 2011 Jan 31. |
| 20697044 | Derived | Lynch DR, Perlman SL, Meier T. A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia. Arch Neurol. 2010 Aug;67(8):941-7. doi: 10.1001/archneurol.2010.168. |
25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 1350 mg/day (2 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 2250 mg/day (2 x 150 mg tablet, t.i.d.)
| BG002 | Placebo | Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets. |
| BG003 | 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 |
|
| OG001 |
| High Dose Idebenone |
25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 1350 mg/day (2 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 2250 mg/day (2 x 150 mg tablet, t.i.d.) |
| OG002 | Placebo | Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets. |
|
|
| Secondary | Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS) 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 exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. Calculation details FARS score at week 24 minus FARS score baseline | Posted | Mean | Standard Deviation | FARS score | baseline and 6 months |
|
|
|
| Secondary | Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS) score 0 to 36, higher score indicates greater impairment | Posted | Mean | Standard Deviation | ADL points | baseline and 6 months |
|
|
|
| Secondary | Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 | To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 12 minus ICARS score baseline | Posted | Mean | Standard Deviation | ICARS points | baseline and 12 weeks |
|
|
|
| Secondary | Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | cm | baseline and 6 months |
|
|
|
| Secondary | Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | % change | baseline and 6 months |
|
|
|
| Secondary | Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI) | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | g/m2 | baseline and 6 months |
|
|
|
| Secondary | Percent Change From Baseline to Week 24 in Left Ventricular Mass Index | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | % change | baseline and 6 months |
|
|
|
| Secondary | Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | cm | baseline and 6 months |
|
|
|
| Secondary | Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | % change | baseline and 6 months |
|
|
|
| Secondary | Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | cm | baseline and 6 months |
|
|
|
| Secondary | Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) | To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography | Posted | Mean | Standard Deviation | % change | baseline and 24 weeks |
|
|
|
| 0 |
| 22 |
| 18 |
| 22 |
| EG001 | High Dose Idebenone | 25 kg/55 lbs to ≤45 kg/99 lbs: idebenone 1350 mg/day (2 x 150 mg tablet, t.i.d.) >45 kg/99 lbs: idebenone 2250 mg/day (2 x 150 mg tablet, t.i.d.) | 2 | 24 | 23 | 24 |
| EG002 | Placebo | Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets. | 0 | 24 | 21 | 24 |
| chest pain | General disorders |
|
| Nervous system disorders | Nervous system disorders |
|
| Gastrointestinal disorders | Gastrointestinal disorders |
|
| General disorders | General disorders |
|
| Musculoskeletal & connective tissue disorders | Musculoskeletal and connective tissue disorders |
|
Not provided
Not provided
| 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 |