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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02774005
Recruitment Status : Active, not recruiting
First Posted : May 16, 2016
Last Update Posted : June 17, 2019
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Brief Summary:
LEROS is an open-label interventional Phase IV study, designed to further assess the efficacy and safety of Raxone® in the long-term treatment of LHON patients.

Condition or disease Intervention/treatment Phase
Leber's Hereditary Optic Neuropathy (LHON) Drug: Idebenone Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: External Natural History Controlled, Open-Label Intervention Study to Assess the Efficacy and Safety of Long-Term Treatment With Raxone® in Leber's Hereditary Optic Neuropathy (LHON)
Study Start Date : May 2016
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2021


Arm Intervention/treatment
Experimental: Raxone Drug: Idebenone
Other Name: Raxone




Primary Outcome Measures :
  1. Proportion of eyes with clinically relevant recovery of visual acuity from Baseline [ Time Frame: 12 months ]
    Proportion of eyes with clinically relevant recovery of visual acuity (VA) from Baseline or in which Baseline VA better than 1.0 logMAR was maintained at Month 12 in patients treated with Raxone® ≤1 year after the onset of symptoms, compared to matching external natural history control group



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Impaired visual acuity in affected eyes due to LHON
  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 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:

  1. Patient has provided natural history data to the Case Record Survey (SNT-CRS-002)
  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 AST, 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02774005


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Locations
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United States, Arizona
Retinal Consultants of Arizona
Phoenix, Arizona, United States
United States, California
Palo Alto Medical Foundation
Palo Alto, California, United States, 94040-2833
Stanford Byers Eye Institute
Stanford, California, United States, 94303
United States, Colorado
University of Colorado Health Eye Center
Aurora, Colorado, United States
United States, Georgia
Emory University Hospital
Atlanta, Georgia, United States, 30322
United States, Maryland
Bethesda Neurology, LLC
Bethesda, Maryland, United States
United States, Missouri
Washington University
Saint Louis, Missouri, United States, 63110
United States, New York
New York Eye and Ear Infirmary
New York, New York, United States, 10003
United States, Virginia
University of Virginia
Charlottesville, Virginia, United States, 22903
Austria
AKH - Medizinische Universitaet Wien
Wien, Austria
Belgium
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
Bulgaria
UMHAT "Alexandrovska" EAD
Sofia, Bulgaria
Germany
Friedrich-Baur-Institut
Muenchen, Germany
Italy
Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
Bologna, Italy
Poland
SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej
Krakow, Poland, 31-501
Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego
Poznań, Poland
Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie
Szczecin, Poland
Samodzielny Publiczny Kliniczny Szpital Okulistyczny
Warszawa, Poland
Uniwersytecki Szpital Kliniczny
Wrocław, Poland
Portugal
Centro Hospitalar de São João, EPE
Porto, Portugal
Spain
Institut Catala de Retina
Barcelona, Spain
Hospital Universitario Ramon y Cajal
Madrid, Spain
United Kingdom
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
Sponsors and Collaborators
Santhera Pharmaceuticals

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Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02774005     History of Changes
Other Study ID Numbers: SNT-IV-005
First Posted: May 16, 2016    Key Record Dates
Last Update Posted: June 17, 2019
Last Verified: June 2019
Additional relevant MeSH terms:
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Optic Nerve Diseases
Optic Neuritis
Optic Atrophy, Hereditary, Leber
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs