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Idebenone

Generic Name
Idebenone
Brand Names
Raxone
Drug Type
Small Molecule
Chemical Formula
C19H30O5
CAS Number
58186-27-9
Unique Ingredient Identifier
HB6PN45W4J
Background

Idebenone is a synthetic analogue of ubiquinone (also known as Coenzyme Q10), a vital cell antioxidant and essential component of the Electron Transport Chain (ETC). It has been proposed that by interacting with the ETC, idebenone increases ATP production required for mitochondrial function, reduces free radicals, inhibits lipid peroxidation, and consequently protects the lipid membrane and mitochondria from oxidative damage . More specifically, idebenone is thought to transfer electrons directly to complex III of the mitochondrial ETC, thereby circumventing complex I and restoring cellular energy (ATP) generation .

Due to its ability to reduce oxidative damage and improve ATP production, idebenone was originally investigated for its potential use in Alzheimer's Disease and other cognitivie disorders . Lack of improvement in cognitive function halted its production for these conditions, however it continues to be investigated for use in other conditions associated with mitochondrial damage.

Idebenone is currently only indicated for use by the European Medicines Agency (EMA) for the treatment of visual impairment in adolescent and adult patients with

Leber’s Hereditary Optic Neuropathy (LHON). LHON is a mitochondrially inherited degeneration of retinal ganglion cells, resulting in acute central vision loss. Due to its biochemical mode of action, it's thought that idebenone may re-activate viable-but-inactive retinal ganglion cells (RGCs) in LHON patients . It is not currently approved for use by either the Food and Drug Administration (USA) or Health Canada.

Indication

Idebenone is indicated for use by the European Medicines Agency (EMA) for the treatment of visual impairment in adolescent and adult patients with

Leber’s Hereditary Optic Neuropathy (LHON). It is not currently approved for use by either the Food and Drug Administration (USA) or Health Canada .

Associated Conditions
Alzheimer's Disease (AD), Leber’s hereditary optic neuropathy

A Study to Assess the Potential for Pre-systemic Inhibition of CYP3A by Idebenone Using Midazolam as a Substrate

Phase 1
Completed
Conditions
Drug-Drug Interaction
First Posted Date
2016-09-02
Last Posted Date
2017-10-05
Lead Sponsor
Santhera Pharmaceuticals
Target Recruit Count
32
Registration Number
NCT02887443
Locations
🇫🇷

Eurofins Optimed, Gières, France

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

Phase 3
Terminated
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
First Posted Date
2016-06-27
Last Posted Date
2021-12-03
Lead Sponsor
Santhera Pharmaceuticals
Target Recruit Count
255
Registration Number
NCT02814019
Locations
🇺🇸

Johns Hopkins University, Baltimore, Maryland, United States

🇺🇸

Rare Disease Research, Atlanta, Georgia, United States

🇺🇸

Childrens Hospital of Los Angeles, Los Angeles, California, United States

and more 60 locations

Study to Assess the Efficacy and Safety of Raxone in LHON Patients

Phase 4
Completed
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
First Posted Date
2016-05-16
Last Posted Date
2023-04-21
Lead Sponsor
Santhera Pharmaceuticals
Target Recruit Count
199
Registration Number
NCT02774005
Locations
🇬🇧

University Hospital of Wales, Cardiff, United Kingdom

🇪🇸

Institut Catala de Retina, Barcelona, Spain

🇪🇸

Hospital Universitario Ramon y Cajal, Madrid, Spain

and more 26 locations

Post Authorisation Safety Study With Raxone in LHON Patients

Completed
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
First Posted Date
2016-05-13
Last Posted Date
2021-07-16
Lead Sponsor
Santhera Pharmaceuticals
Target Recruit Count
229
Registration Number
NCT02771379
Locations
🇮🇹

Ospedale San Raffaele, Milano, Italy

🇩🇪

Universitaetsklinikum Essen, Essen, Germany

🇫🇷

CHU Amiens - Centre Saint Victor, Amiens, Somme, France

and more 23 locations

Idebenone for Primary Progressive Multiple Sclerosis

Phase 1
Completed
Conditions
Multiple Sclerosis
Primary Progressive Multiple Sclerosis
Interventions
First Posted Date
2013-05-15
Last Posted Date
2021-05-19
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Target Recruit Count
61
Registration Number
NCT01854359
Locations
🇺🇸

National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States

Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)

Phase 3
Withdrawn
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
Drug: Placebo
First Posted Date
2011-12-20
Last Posted Date
2014-08-22
Lead Sponsor
Santhera Pharmaceuticals
Registration Number
NCT01495715

Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)

Phase 3
Completed
Conditions
Friedreich's Ataxia
Interventions
Drug: Placebo
First Posted Date
2011-02-24
Last Posted Date
2016-03-09
Lead Sponsor
Santhera Pharmaceuticals
Target Recruit Count
29
Registration Number
NCT01303406
Locations
🇬🇧

The National Hospital, University College London, London, United Kingdom

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

Phase 3
Completed
Conditions
Muscular Dystrophy, Duchenne
Ambulatory Care
Interventions
Drug: Placebo
First Posted Date
2009-12-09
Last Posted Date
2015-10-19
Lead Sponsor
Santhera Pharmaceuticals
Target Recruit Count
65
Registration Number
NCT01027884
Locations
🇺🇸

University of California Davis Medical Center, Sacramento, California, United States

🇺🇸

Children's Hospital Colorado, Aurora, Colorado, United States

🇺🇸

Carolinas Medical Center, Neurosciences and Spine Institute, Charlotte, North Carolina, United States

and more 20 locations

Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

Phase 3
Completed
Conditions
Freidreich's Ataxia
Interventions
First Posted Date
2009-10-14
Last Posted Date
2018-03-05
Lead Sponsor
Santhera Pharmaceuticals
Target Recruit Count
200
Registration Number
NCT00993967
Locations
🇫🇷

Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale, Paris, France

🇩🇪

Zentrum für Neurologische Medizin, Göttingen, Germany

🇩🇪

HELIOS Klinikum Berlin, Berlin, Germany

and more 8 locations

Clinical Trial of Idebenone in Primary Progressive Multiple Sclerosis (IPPoMS)

Phase 1
Completed
Conditions
Primary Progressive Multiple Sclerosis
Interventions
Other: placebo
First Posted Date
2009-07-31
Last Posted Date
2019-03-19
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Target Recruit Count
85
Registration Number
NCT00950248
Locations
🇺🇸

National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States

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