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This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Idebenone | Experimental | 1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| idebenone | Drug | Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg |
|
| Measure | Description | Time Frame |
|---|---|---|
| Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs) | Global Overvbiew of accurance of AEs-Safety population. The Safety population included all subjects who received at least one dose of the study medication. | overall study, up to 24 months |
| Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS) | The International Cooperative Ataxia Rating Scale (ICARS) is a commonly used evaluation and is composed of four clinical sub-scores involving the following: posture and gait, limb coordination, speech and oculomotor function.The ICARS score is the total sum of the sub scores and ranges from 0 to 100, with 100 indicative of the most severely affected outcome. | Baseline, Month 12 and month 24 |
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| Measure | Description | Time Frame |
|---|---|---|
| Measures of Safety and Tolerability: Physical Examinations and Vital Signs | Assessment of the head, eyes, ears, nose, throat, heart, chest, lungs, abdomen, extremities, peripheral pulses, skin and any other physical conditions of note. | Month 1, 3, 6, 12, 18 and 24 |
| Measures of Safety and Tolerability: Electrocardiograms (ECGs) |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Nick Wood, Professor | Dept of Molecular Neuroscience, Institute of Neurology. The National Hospital, University College London | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Universitätsklinik Innsbruck | Innsbruck | Austria | ||||
| Hôpital Erasme - Univeristé Libre de Bruxelles |
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| ID | Title | Description |
|---|---|---|
| FG000 | Idebenone | 1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed. idebenone: Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Idebenone | 1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed. idebenone: Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs) | Global Overvbiew of accurance of AEs-Safety population. The Safety population included all subjects who received at least one dose of the study medication. | Posted | Number | participants | overall study, up to 24 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 | Idebenone | 1350 mg/day or 2250 mg/day for patients weighing ≤45 kg or >45 kg, respectively.In case of poor tolerability, dose reduction to 450 mg/day or 900 mg/day, respectively, were allowed. idebenone: Idebenone 1350 mg/d, patients < or equal 45 kg Idebenone 2250 mg/d, patients > 45 kg |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | MedDRA (Unspecified) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| abdominal pain upper | Gastrointestinal disorders | MedDRA (Unspecified) |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Thomas Meier, PhD | Santhera Pharmaceuticals | +41 61 906 89 50 | thomas.meier@santhera.com <Thomas.Meier@santhera.com> |
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| ID | Term |
|---|---|
| C036619 | idebenone |
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12-lead ECG recordings were performed at every visit. Each ECG was measured using 3 complexes: PR interval in lead II or V2, QRS and QT intervals and heart rate in lead II, corrected QT intervals QTcB and QTcF. |
| Month 1, 3, 6, 12, 18 and 24 |
| Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters | Safety haematological analysis were done at every visit. Analyses included red blood cell count, haemoglobin, haematocrit, red cell indices, white blood cell count including differential, platelet count Safety biochemistry were done at every visit. Analyses included sodium, potassium, chloride, bicarbonate, urea, creatinine, calcium, inorganic phosphate, glucose, total bilirubin, total protein, albumin, aspartate amiotransferase (AST), alanine aminotransferase (ALT), alkaline phosphotase, Gamma GT, creatine kinase (CK)^, cholesterol, triglycerides, uric acid. | Month 1, 3, 6, 12, 18 and 24 |
| Brussels |
| 1070 |
| Belgium |
| Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale | Paris | 75651 | France |
| HELIOS Klinikum Berlin | Berlin | 13125 | Germany |
| Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn | Bonn | 53105 | Germany |
| Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg | Freiburg im Breisgau | 79106 | Germany |
| Zentrum für Neurologische Medizin | Göttingen | 37073 | Germany |
| UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin | Hamburg | 20246 | Germany |
| Neurologische Klinik - Klinikum Grosshadern | München | 81377 | Germany |
| Neurologische Universitätsklinik und Poliklinik | Tübingen | 72076 | Germany |
| University medical Center Groningen | Groningen | 9700 RB | Netherlands |
| Withdrawal by Subject |
|
| Protocol Violation |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Participants |
|
|
| Primary | Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS) | The International Cooperative Ataxia Rating Scale (ICARS) is a commonly used evaluation and is composed of four clinical sub-scores involving the following: posture and gait, limb coordination, speech and oculomotor function.The ICARS score is the total sum of the sub scores and ranges from 0 to 100, with 100 indicative of the most severely affected outcome. | Changes in Total ICARS Score for all patients completing the study (CC Population) | Posted | Mean | Standard Deviation | units on a scale | Baseline, Month 12 and month 24 |
|
|
|
| Other Pre-specified | Measures of Safety and Tolerability: Physical Examinations and Vital Signs | Assessment of the head, eyes, ears, nose, throat, heart, chest, lungs, abdomen, extremities, peripheral pulses, skin and any other physical conditions of note. | Not Posted | Month 1, 3, 6, 12, 18 and 24 | Participants |
| Other Pre-specified | Measures of Safety and Tolerability: Electrocardiograms (ECGs) | 12-lead ECG recordings were performed at every visit. Each ECG was measured using 3 complexes: PR interval in lead II or V2, QRS and QT intervals and heart rate in lead II, corrected QT intervals QTcB and QTcF. | Not Posted | Month 1, 3, 6, 12, 18 and 24 | Participants |
| Other Pre-specified | Measures of Safety and Tolerability: Haematological and Biochemical Laboratory Parameters | Safety haematological analysis were done at every visit. Analyses included red blood cell count, haemoglobin, haematocrit, red cell indices, white blood cell count including differential, platelet count Safety biochemistry were done at every visit. Analyses included sodium, potassium, chloride, bicarbonate, urea, creatinine, calcium, inorganic phosphate, glucose, total bilirubin, total protein, albumin, aspartate amiotransferase (AST), alanine aminotransferase (ALT), alkaline phosphotase, Gamma GT, creatine kinase (CK)^, cholesterol, triglycerides, uric acid. | Not Posted | Month 1, 3, 6, 12, 18 and 24 | Participants |
| 49 |
| 200 |
| 188 |
| 200 |
| Atrial flutter | Cardiac disorders | MedDRA (Unspecified) |
|
| cardiac failure | Cardiac disorders | MedDRA (Unspecified) |
|
| talipes | Congenital, familial and genetic disorders | MedDRA (Unspecified) |
|
| abdominal pain lower | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| diarrhoea | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
| streptococcal infection | Infections and infestations | MedDRA (Unspecified) |
|
| dental caries | Gastrointestinal disorders | MedDRA (Unspecified) |
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| tooth disorder | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (Unspecified) |
|
| gastroenteritis | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| spinal disorder | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| deep vein thrombosis | Vascular disorders | MedDRA (Unspecified) |
|
| pulmonary embolism | Vascular disorders | MedDRA (Unspecified) |
|
| ankle fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| Cerebrovascular accident | Nervous system disorders | MedDRA (Unspecified) |
|
| Humerus fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| lower respiratory tract infection | Infections and infestations | MedDRA (Unspecified) |
|
| Thermal burn | Injury, poisoning and procedural complications | MedDRA (Unspecified) |
|
| Atrial tachycardia | Cardiac disorders | MedDRA (Unspecified) |
|
| Reflux oesophagitis | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| Pneumonia | Infections and infestations | MedDRA (Unspecified) |
|
| Haemorrhage | Vascular disorders | MedDRA (Unspecified) |
|
| Pelvic fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (Unspecified) |
|
| Diabetes mellitus inadequate control | Metabolism and nutrition disorders | MedDRA (Unspecified) |
|
| influenza | Infections and infestations | MedDRA (Unspecified) |
|
| fibula fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| Tibia fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA (Unspecified) |
|
| proctitis | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| Tachycardia | Cardiac disorders | MedDRA (Unspecified) |
|
| femur fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| myocardial infarction | Cardiac disorders | MedDRA (Unspecified) |
|
| abdominal pain upper | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| calculus ureteric | Renal and urinary disorders | MedDRA (Unspecified) |
|
| dehydration | Metabolism and nutrition disorders | MedDRA (Unspecified) |
|
| epilepsy | Nervous system disorders | MedDRA (Unspecified) |
|
| torticollis | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| salmonella sepsis | Infections and infestations | MedDRA (Unspecified) |
|
| scoliosis | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| prostatitis | Reproductive system and breast disorders | MedDRA (Unspecified) |
|
| respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
| back pain | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| radiculopathy | Nervous system disorders | MedDRA (Unspecified) |
|
| thyroid cancer | Endocrine disorders | MedDRA (Unspecified) |
|
| paraplegia | Nervous system disorders | MedDRA (Unspecified) |
|
| hand fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| wrist fracture | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| foot deformity | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| corneal erosion | Eye disorders | MedDRA (Unspecified) |
|
| ventricular tachycardia | Cardiac disorders | MedDRA (Unspecified) |
|
| hypoglycemia | Metabolism and nutrition disorders | MedDRA (Unspecified) |
|
| pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
| liver injury | Injury, poisoning and procedural complications | MedDRA (Unspecified) |
|
| fall | Injury, poisoning and procedural complications | MedDRA (Unspecified) |
|
| constipation | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| diarrhoea | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| nausea | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| vomiting | Gastrointestinal disorders | MedDRA (Unspecified) |
|
| pyrexia | General disorders | MedDRA (Unspecified) |
|
| bronchitis | Infections and infestations | MedDRA (Unspecified) |
|
| cystitis | Infections and infestations | MedDRA (Unspecified) |
|
| influenza | Infections and infestations | MedDRA (Unspecified) |
|
| nasopharyngitis | Infections and infestations | MedDRA (Unspecified) |
|
| fall | Injury, poisoning and procedural complications | MedDRA (Unspecified) |
|
| back pain | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| dizziness | Nervous system disorders | MedDRA (Unspecified) |
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| headache | Nervous system disorders | MedDRA (Unspecified) |
|
| depression | Psychiatric disorders | MedDRA (Unspecified) |
|
| cough | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
| oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) |
|
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