Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy

NCT00747487 | Completed Phase 2 DMC

• 1 other identifier: SNT-II-003
• Study Type: interventional
• Target: 85
• Locations: 3 countries
• Sites: 3

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.

Conditions

Leber's Hereditary Optic Neuropathy

Keywords

Leber; LHON; Leber's Hereditary Optic Neuropathy; Idebenone

Outcome Measures

Primary Outcomes (1)

• Best recovery of logMAR visual acuity between baseline and Week 24 in either right or left eye

  24 weeks

Secondary Outcomes (12)

• 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

Study Arms (2)

1

EXPERIMENTAL

Idebenone

Drug: Idebenone

2

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

Idebenone DRUG

Idebenone 900 mg/day

1

Placebo DRUG

Placebo

2

Eligibility Criteria

• Age: 14 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (3)

Unité de recherche clinique Ophtalmologie- Hopital Notre-Dame

Montreal, Quebec, H2L 4M1, Canada

Location

Klinikum der Universität München - Grosshadern, Neurologische Klinik und Poliklinik

Munich, 81377, Germany

Location

Clinical Research Facility, 4th Floor Leazes Wing, Royal Victoria Infirmary

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

Related Publications (2)

• 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.

  PMID: 21788663 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.

  PMID: 23263355 RESULT

Related Links

• Website for LHON patients in Canada
• Website for LHON patients in Germany

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Interventions

idebenone

Condition Hierarchy (Ancestors)

Optic Atrophies, Hereditary; Optic Atrophy; Optic Nerve Diseases; Cranial Nerve Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Eye Diseases, Hereditary; Eye Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• 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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 4, 2008
• First Posted: September 5, 2008
• Study Start: November 1, 2007
• Primary Completion: February 1, 2010
• Study Completion: February 1, 2010
• Last Updated: May 27, 2013

Record last verified: 2013-05

Locations

CA (1); DE (1); GB (1)