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This study is meant to assess the effectiveness of idebenone on visual function measures in patients with Leber's Hereditary Optic Neuropathy over a 6 months period.
The study involves 6 clinic visits.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | Idebenone |
|
| 2 | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Idebenone | Drug | Idebenone 900 mg/day |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change in the patient's best logMAR visual acuity between baseline and week 24 | 24 weeks | |
| Change in scotoma area in both eyes | Day -1, Week 4, Week 12, Week 24 | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Prof Patrick F Chinnery, MD | Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary | Principal Investigator |
| Prof Thomas Klopstock, MD | Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame | Montreal | Quebec | H2L 4M1 | Canada | ||
| Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21788663 | Result | Klopstock T, Yu-Wai-Man P, Dimitriadis K, Rouleau J, Heck S, Bailie M, Atawan A, Chattopadhyay S, Schubert M, Garip A, Kernt M, Petraki D, Rummey C, Leinonen M, Metz G, Griffiths PG, Meier T, Chinnery PF. A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):2677-86. doi: 10.1093/brain/awr170. Epub 2011 Jul 25. | |
| 23263355 |
| Label | URL |
|---|---|
| Website for LHON patients in Canada | View source |
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| ID | Term |
|---|---|
| D029242 | Optic Atrophy, Hereditary, Leber |
| ID | Term |
|---|---|
| D015418 | Optic Atrophies, Hereditary |
| D009896 | Optic Atrophy |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
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| ID | Term |
|---|---|
| C036619 | idebenone |
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| Drug |
Placebo |
|
| Change in optic nerve fibre layer thickness in both eyes |
| Day -1, Week 4, Week 12, Week 24 |
| Colour contrast sensitivity in both eyes (in a subset of patients) | Day -1, Week 4, Week 12, Week 24 |
| logMAR visual acuity as a continuous variable in both eyes | Screening, Day -1, Week 4, Week 12, Week 24, Week 28 |
| Clinical Global Impression of Change | Week 4, Week 12 and Week 24 |
| Change in Health-Related Quality of Life (HRQOL) | Day -1, Week 4, Week 12, Week 24 |
| Change in self-reported general energy levels | Day -1, Week 4, Week 12, Week 24, Week 28 |
| Proportion of patients in which visual acuity in the initially least affected eye does not deteriorate to 1.0 log MAR or more ( in LHON patients with eye still less affected than 0.5 logMAR at trial entry) | 24 weeks |
| Plasma levels of idebenone matched to measures of efficacy and safety | 24 weeks |
| • Best visual acuity at Week 24 (best eye at Week 24) compared to best visual acuity at Baseline (best eye at Baseline) | 24 weeks |
| • Count of eyes/ patients for which the visual acuity improves between baseline and week 24 | 24 weeks |
| Munich |
| 81377 |
| Germany |
| Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| Result |
| Rudolph G, Dimitriadis K, Buchner B, Heck S, Al-Tamami J, Seidensticker F, Rummey C, Leinonen M, Meier T, Klopstock T. Effects of idebenone on color vision in patients with leber hereditary optic neuropathy. J Neuroophthalmol. 2013 Mar;33(1):30-6. doi: 10.1097/WNO.0b013e318272c643. |
| Website for LHON patients in Germany | View source |
| D009422 |
| Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |