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

Sponsor
Santhera Pharmaceuticals (Industry)
Overall Status
Completed
CT.gov ID
NCT02774005
Collaborator
(none)
199
Enrollment
29
Locations
1
Arm
58.9
Duration (Months)
6.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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
Phase 4

Study Design

Study Type:
Interventional
Actual Enrollment :
199 participants
Allocation:
N/A
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 1, 2016
Actual Primary Completion Date :
Mar 29, 2021
Actual Study Completion Date :
Mar 29, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: Raxone

Drug: Idebenone
Other Names:
  • Raxone

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Proportion of eyes with clinically relevant recovery of visual acuity from Baseline [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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Retinal Consultants of Arizona Phoenix Arizona United States
    2 Palo Alto Medical Foundation Palo Alto California United States 94040-2833
    3 Stanford Byers Eye Institute Stanford California United States 94303
    4 University of Colorado Health Eye Center Aurora Colorado United States
    5 Emory University Hospital Atlanta Georgia United States 30322
    6 Bethesda Neurology, LLC Bethesda Maryland United States
    7 Washington University Saint Louis Missouri United States 63110
    8 New York Eye and Ear Infirmary New York New York United States 10003
    9 University of Virginia Charlottesville Virginia United States 22903
    10 AKH - Medizinische Universitaet Wien Wien Austria
    11 CHU Saint-Pierre Brussels Belgium
    12 Cliniques Universitaire Saint-Luc Brussels Belgium
    13 UZ Leuven - Campus Sint-Rafaël Leuven Belgium
    14 C. H. U. Sart Tilman Liège Belgium
    15 UMHAT "Alexandrovska" EAD Sofia Bulgaria
    16 Friedrich-Baur-Institut Muenchen Germany
    17 Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche Bologna Italy
    18 SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej Krakow Poland 31-501
    19 Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego Poznań Poland
    20 Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie Szczecin Poland
    21 Samodzielny Publiczny Kliniczny Szpital Okulistyczny Warszawa Poland
    22 Uniwersytecki Szpital Kliniczny Wrocław Poland
    23 Centro Hospitalar de São João, EPE Porto Portugal
    24 Institut Catala de Retina Barcelona Spain
    25 Hospital Universitario Ramon y Cajal Madrid Spain
    26 University Hospital of Wales Cardiff United Kingdom
    27 Moorfields Eye Hospital London United Kingdom
    28 Manchester Royal Eye Hospital Manchester United Kingdom
    29 Queen's Hospital Romford United Kingdom

    Sponsors and Collaborators

    • Santhera Pharmaceuticals

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Santhera Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT02774005
    Other Study ID Numbers:
    • SNT-IV-005
    First Posted:
    May 16, 2016
    Last Update Posted:
    Jul 16, 2021
    Last Verified:
    Jul 1, 2021
    Additional relevant MeSH terms:
    Optic Nerve Diseases
    Optic Atrophy, Hereditary, Leber
    Idebenone

    Study Results

    No Results Posted as of Jul 16, 2021