Freidreich's Ataxia Clinical Trial
Official title:
A Phase III Open-Label, Single-Group, Extension Study to Obtain Long-Term Safety and Tolerability Data of Idebenone in the Treatment of Friedreich's Ataxia Patients.
| Verified date | August 2017 |
| Source | Santhera Pharmaceuticals |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 200 |
| Est. completion date | June 2012 |
| Est. primary completion date | June 2012 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 9 Years and older |
| Eligibility |
Inclusion Criteria: - Completion of 52 weeks in study SNT-III-001 - Body weight = 25 kg - Negative urine pregnancy test - Eligibility to participate in the present extension study as confirmed by investigator Exclusion Criteria: - Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone - Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine - Parallel participation in another clinical drug trial - Pregnancy or breast-feeding - Abuse of drugs or alcohol |
| Country | Name | City | State |
|---|---|---|---|
| Austria | Universitätsklinik Innsbruck | Innsbruck | |
| Belgium | Hôpital Erasme - Univeristé Libre de Bruxelles | Bruxelles | |
| France | Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale | Paris | |
| Germany | HELIOS Klinikum Berlin | Berlin | |
| Germany | Neurologische Universitätsklinik und Ploklinik - Universitätsklimikum Bonn | Bonn | |
| Germany | Klinik II, Neuropädiatrie und Muskelerkrankungen - Universitätsklinik Freiburg | Freiburg | |
| Germany | Zentrum für Neurologische Medizin | Göttingen | |
| Germany | UKE Hamburg Neuropädiatrie - Zentrum für Frauen, Kinder und Jugendmedizin | Hamburg | |
| Germany | Neurologische Klinik - Klinikum Grosshadern | München | |
| Germany | Neurologische Universitätsklinik und Poliklinik | Tübingen | |
| Netherlands | University medical Center Groningen | Groningen |
| Lead Sponsor | Collaborator |
|---|---|
| Santhera Pharmaceuticals |
Austria, Belgium, France, Germany, Netherlands,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | 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 | |
| Other | 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. | Month 1, 3, 6, 12, 18 and 24 | |
| Other | 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 | |
| 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. | overall study, up to 24 months | |
| 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. | Baseline, Month 12 and month 24 |