Leber's Hereditary Optic Neuropathy
Conditions
Keywords
Leber, LHON, Leber's Hereditary Optic Neuropathy, Idebenone
Brief summary
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.
Detailed description
The study involves 6 clinic visits.
Interventions
DRUGIdebenone
Idebenone 900 mg/day
DRUGPlacebo
Placebo
Sponsors
Santhera Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
14 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Age \> or = 14 years and \< 65 years * Impaired visual acuity in at least one eye due to LHON * Onset of visual loss due to LHON lies five years or less prior to Baseline * Confirmation of either G11778A, T14484C or G3460A LHON mtDNA mutations at \>60% in blood * No explanation for the visual failure besides LHON * Body weight ≥ 45 kg * Negative urine pregnancy test at Screening and at Baseline (women of childbearing potential).
Exclusion criteria
* Treatment with Coenzyme Q10 or idebenone within 1 month prior to Baseline * Pregnancy and/or breast-feeding * Weekly alcohol intake 35 units (men) or 24 units (women) * Current drug abuse * Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine * Participation in another clinical trial of any investigational drug within 3 months prior to Baseline * Other factor that, in the investigator's opinion, excludes the patient from entering the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye | 24 weeks |
Secondary
| Measure | 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 |
| 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 |
Countries
Canada, Germany, United Kingdom
Outcome results
None listed