A double blind, randomised, placebo-controlled study of the efficacy, safety and tolerability of idebenone in the treatment of patients with Leber's Hereditary Optic Neuropathy

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-002679-42-GB

Enrollment

Registered

2006-09-18

Start date

2006-11-22

Completion date

Unknown

Last updated

2019-11-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leber's Hereditary Optic Neuropathy

Interventions

Product Name: Idebenone Product Code: SNT-MC17 Pharmaceutical Form: Tablet INN or Proposed INN: Idebenone CAS Number: 58186-27-9

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Age >/= 14 years and 60% in blood 5. No explanation for the visual failure besides LHON 6. Body weight = 45 kg 7. Negative urine pregnancy test at Screening (Visit 1) and at Baseline (Visit 2) (women of childbearing potential) Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Treatment with Coenzyme Q10 or idebenone within 1 month prior to Baseline 2. Pregnancy and/or breast-feeding 3. Weekly alcohol intake >35 units (men) or > 24 units (women) 4. Current drug abuse 5. 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 6. Participation in another clinical trial of any investigational drug within 3 months prior to Baseline 7. Other factor that, in the investigator’s opinion, excludes the patient from entering the study

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine whether administration of idebenone can improve visual function in Leber's Hereditary Optic Neuropathy (LHON) patients ; Secondary Objective: In LHON patients entering the trial with an eye still less affected than 0.5 logMAR, to determine whether administration of idebenone can mitigate further visual loss in that eye To assess changes in Clinical Global Impression of Change (CGIC) and in Health-Related Quality of Life (HRQOL) To assess safety and tolerability following 24 weeks' treatment with idebenone To explore any relationship between optic nerve fibre layer thickness and LHON and its treatment with placebo and idebenone in both eyes To explore any relationship between colour contrast sensitivity and LHON and its treatment with placebo and idebenone in both eyes (in a subset of patients) To explore the relationship between plasma levels of idebenone and measures of efficacy and safety ;Primary end point(s): Best recovery of logMAR visual acuity between Baseline and week 24 in either right or left eye	—

Measure

Time frame

Main Objective: To determine whether administration of idebenone can improve visual function in Leber's Hereditary Optic Neuropathy (LHON) patients ; Secondary Objective: In LHON patients entering the trial with an eye still less affected than 0.5 logMAR, to determine whether administration of idebenone can mitigate further visual loss in that eye To assess changes in Clinical Global Impression of Change (CGIC) and in Health-Related Quality of Life (HRQOL) To assess safety and tolerability following 24 weeks' treatment with idebenone To explore any relationship between optic nerve fibre layer thickness and LHON and its treatment with placebo and idebenone in both eyes To explore any relationship between colour contrast sensitivity and LHON and its treatment with placebo and idebenone in both eyes (in a subset of patients) To explore the relationship between plasma levels of idebenone and measures of efficacy and safety ;Primary end point(s): Best recovery of logMAR visual acuity between Baseline and week 24 in either right or left eye

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Countries

Germany, United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026