Study to Assess the Efficacy and Safety of Raxone in LHON Patients
NCT ID: NCT02774005
Last Updated: 2023-04-21
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE4
199 participants
INTERVENTIONAL
2016-05-31
2021-03-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Raxone
Idebenone
Interventions
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Idebenone
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. No explanation for visual loss besides LHON
3. Age more or equal 12 years
4. Onset of symptoms ≤5 years of Baseline
5. Confirmation of either G11778A, G3460A or T14484C LHON mtDNA (for the Intent-to Treat (ITT) population, not required for enrolment)
6. Written informed consent obtained from the patient
7. Ability and willingness to comply with study procedures and visits
8. Women of Childbearing Potential (WCBP) who have a negative urine or serum pregnancy test at Baseline visit and who are willing to use a highly effective contraceptive measure and maintain it until treatment discontinuation.
Exclusion Criteria
2. Any previous use of idebenone
3. Any other cause of visual impairment (e.g. glaucoma, diabetic retinopathy, AIDS related visual impairment, cataract, macular degeneration, etc.) or any active ocular disorder (uveitis, infections, inflammatory retinal disease, thyroid eye disease, etc.)
4. Known history of clinically significant elevations (greater than 3 times the upper limit of normal) of aspartate aminotransferase (AST), alanine transaminase (ALT) or creatinine
5. Patient has a condition or is in a situation which, in an investigator's opinion may put the patient at significant risk, may confound study results or may interfere significantly with the patient's participation in the study
6. Participation in another clinical trial of any investigational drug within 3 months prior to Baseline
7. Hypersensitivity to the active substance or to any of the following excipients (as listed in section 6.1 of Raxone SmPC): Lactose monohydrate, Microcrystalline cellulose, Croscarmellose sodium, Povidone K25, Magnesium stearate, Colloidal silica, Macrogol 3350, Poly(vinyl alcohol), Talc, Titanium dioxide, Sunset yellow FCF (E110).
8. Women who are pregnant or have a positive pregnancy test at Baseline visit
9. Women who are breastfeeding
12 Years
ALL
No
Sponsors
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Santhera Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Retinal Consultants of Arizona
Phoenix, Arizona, United States
Palo Alto Medical Foundation
Palo Alto, California, United States
Stanford Byers Eye Institute
Stanford, California, United States
University of Colorado Health Eye Center
Aurora, Colorado, United States
Emory University Hospital
Atlanta, Georgia, United States
Bethesda Neurology, LLC
Bethesda, Maryland, United States
Washington University
St Louis, Missouri, United States
New York Eye and Ear Infirmary
New York, New York, United States
University of Virginia
Charlottesville, Virginia, United States
AKH - Medizinische Universitaet Wien
Vienna, , Austria
CHU Saint-Pierre
Brussels, , Belgium
Cliniques Universitaire Saint-Luc
Brussels, , Belgium
UZ Leuven - Campus Sint-Rafaël
Leuven, , Belgium
C. H. U. Sart Tilman
Liège, , Belgium
UMHAT "Alexandrovska" EAD
Sofia, , Bulgaria
Friedrich-Baur-Institut
München, , Germany
Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
Bologna, , Italy
SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej
Krakow, , Poland
Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego
Poznan, , Poland
Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie
Szczecin, , Poland
Samodzielny Publiczny Kliniczny Szpital Okulistyczny
Warsaw, , Poland
Uniwersytecki Szpital Kliniczny
Wroclaw, , Poland
Centro Hospitalar de São João, EPE
Porto, , Portugal
Institut Catala de Retina
Barcelona, , Spain
Hospital Universitario Ramon y Cajal
Madrid, , Spain
University Hospital of Wales
Cardiff, , United Kingdom
Moorfields Eye Hospital
London, , United Kingdom
Manchester Royal Eye Hospital
Manchester, , United Kingdom
Queen's Hospital
Romford, , United Kingdom
Countries
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References
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Yu-Wai-Man P, Carelli V, Newman NJ, Silva MJ, Linden A, Van Stavern G, Szaflik JP, Banik R, Lubinski W, Pemp B, Liao YJ, Subramanian PS, Misiuk-Hojlo M, Newman S, Castillo L, Kociecki J, Levin MH, Munoz-Negrete FJ, Yagan A, Cherninkova S, Katz D, Meunier A, Votruba M, Korwin M, Dziedziak J, Jurkute N, Harvey JP, La Morgia C, Priglinger C, Lloria X, Tomasso L, Klopstock T; LEROS Study Group. Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. Cell Rep Med. 2024 Mar 19;5(3):101437. doi: 10.1016/j.xcrm.2024.101437. Epub 2024 Feb 29.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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SNT-IV-005
Identifier Type: -
Identifier Source: org_study_id
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