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

March 31, 2023 updated by: Santhera Pharmaceuticals

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

199

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Wien, Austria
        • AKH - Medizinische Universitaet Wien
  • Belgium
      • Brussels, Belgium
        • CHU Saint-Pierre
      • Brussels, Belgium
        • Cliniques Universitaire Saint-Luc
      • Leuven, Belgium
        • UZ Leuven - Campus Sint-Rafaël
      • Liège, Belgium
        • C. H. U. Sart Tilman
  • Bulgaria
      • Sofia, Bulgaria
        • UMHAT "Alexandrovska" EAD
  • Germany
      • Muenchen, Germany
        • Friedrich-Baur-Institut
  • Italy
      • Bologna, Italy
        • Universita di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
  • Poland
      • Krakow, Poland, 31-501
        • SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej
      • Poznań, Poland
        • Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego
      • Szczecin, Poland
        • Samodzielny Publiczny Szpital Kliniczny nr 2 PUM w Szczecinie
      • Warszawa, Poland
        • Samodzielny Publiczny Kliniczny Szpital Okulistyczny
      • Wrocław, Poland
        • Uniwersytecki Szpital Kliniczny
  • Portugal
      • Porto, Portugal
        • Centro Hospitalar de Sao Joao, EPE
  • Spain
      • Barcelona, Spain
        • Institut Catala de Retina
      • Madrid, Spain
        • Hospital Universitario Ramon y Cajal
  • United Kingdom
      • Cardiff, United Kingdom
        • University Hospital of Wales
      • London, United Kingdom
        • Moorfields Eye Hospital
      • Manchester, United Kingdom
        • Manchester Royal Eye Hospital
      • Romford, United Kingdom
        • Queen's Hospital
  • United States
    • Arizona
      • Phoenix, Arizona, United States
        • Retinal Consultants of Arizona
    • California
      • Palo Alto, California, United States, 94040-2833
        • Palo Alto Medical Foundation
      • Stanford, California, United States, 94303
        • Stanford Byers Eye Institute
    • Colorado
      • Aurora, Colorado, United States
        • University of Colorado Health Eye Center
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University Hospital
    • Maryland
      • Bethesda, Maryland, United States
        • Bethesda Neurology, LLC
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • New York
      • New York, New York, United States, 10003
        • New York Eye and Ear Infirmary
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 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:

  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 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Raxone
Drug: Idebenone
Other Names:
  • Raxone

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline
Time Frame: 12 months
Proportion (number) 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
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Components of the Primary Endpoint: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA From Baseline at Month 12 Compared to Matching External National History (NH) Control Group
Time Frame: 12 months

CRR is defined as:

  • Improvement from "off-chart" (the equivalent of Counting fingers, Hand motion, Light perception or No light perception) Visual Acuity to at least 1.6 logMAR value, OR
  • Improvement of at least 0.2 logMAR value within "on-chart" Visual Acuity A patient had a CRR if at least one eye had CRR.

logMAR = Logarithm of the minimum angle of resolution
12 months
Components of the Primary Endpoint: Proportion of Eyes in Which Baseline Visual Acuity (VA) Better Than 1.0 logMAR Was Maintained at Month 12 (Clinically Relevant Stabilization) Compared to Matching External NH Control Group
Time Frame: 12 months

For proportion of eyes in which baseline VA better than 1.0 logMAR was maintained at Month 12 (CRS) compared to matching external NH control group only patients having VA < 1.0 at baseline are taking into account.

Clinically relevant stabilization (CRS) was defined as maintenance of VA <1.0 logMAR in eyes with VA <1.0 logMAR at Baseline.

A patient had a CRS if at least one eye had CRS.

logMAR = Logarithm of the minimum angle of resolution
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2016

Primary Completion (Actual)

March 29, 2021

Study Completion (Actual)

March 29, 2021

Study Registration Dates

First Submitted

May 12, 2016

First Submitted That Met QC Criteria

May 13, 2016

First Posted (Estimate)

May 16, 2016

Study Record Updates

Last Update Posted (Actual)

April 21, 2023

Last Update Submitted That Met QC Criteria

March 31, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-IV-005

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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