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

Primary Purpose

Leber's Hereditary Optic Neuropathy (LHON)

Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Idebenone
Sponsored by
Santhera Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leber's Hereditary Optic Neuropathy (LHON)

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Retinal Consultants of Arizona
  • Palo Alto Medical Foundation
  • Stanford Byers Eye Institute
  • University of Colorado Health Eye Center
  • Emory University Hospital
  • Bethesda Neurology, LLC
  • Washington University
  • New York Eye and Ear Infirmary
  • University of Virginia
  • AKH - Medizinische Universitaet Wien
  • CHU Saint-Pierre
  • Cliniques Universitaire Saint-Luc
  • UZ Leuven - Campus Sint-Rafaël
  • C. H. U. Sart Tilman
  • UMHAT "Alexandrovska" EAD
  • Friedrich-Baur-Institut
  • Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
  • SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej
  • Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego
  • Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie
  • Samodzielny Publiczny Kliniczny Szpital Okulistyczny
  • Uniwersytecki Szpital Kliniczny
  • Centro Hospitalar de São João, EPE
  • Institut Catala de Retina
  • Hospital Universitario Ramon y Cajal
  • University Hospital of Wales
  • Moorfields Eye Hospital
  • Manchester Royal Eye Hospital
  • Queen's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Raxone

Arm Description

Outcomes

Primary Outcome Measures

Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline
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

Secondary Outcome Measures

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
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
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
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

Full Information

First Posted
May 12, 2016
Last Updated
March 31, 2023
Sponsor
Santhera Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02774005
Brief Title
Study to Assess the Efficacy and Safety of Raxone in LHON Patients
Acronym
LEROS
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 Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Completed
Study Start Date
May 2016 (undefined)
Primary Completion Date
March 29, 2021 (Actual)
Study Completion Date
March 29, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Santhera Pharmaceuticals

4. Oversight

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leber's Hereditary Optic Neuropathy (LHON)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
199 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Raxone
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Idebenone
Other Intervention Name(s)
Raxone
Primary Outcome Measure Information:
Title
Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline
Description
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
Time Frame
12 months
Secondary Outcome Measure Information:
Title
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
Description
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
Time Frame
12 months
Title
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
Description
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
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Impaired visual acuity in affected eyes due to LHON No explanation for visual loss besides LHON Age more or equal 12 years Onset of symptoms ≤5 years of Baseline Confirmation of either G11778A, G3460A or T14484C LHON mtDNA (for the Intent-to Treat (ITT) population, not required for enrolment) Written informed consent obtained from the patient Ability and willingness to comply with study procedures and visits 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: Patient has provided natural history data to the Case Record Survey (SNT-CRS-002) Any previous use of idebenone 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.) Known history of clinically significant elevations (greater than 3 times the upper limit of normal) of aspartate aminotransferase (AST), alanine transaminase (ALT) or creatinine 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 Participation in another clinical trial of any investigational drug within 3 months prior to Baseline 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). Women who are pregnant or have a positive pregnancy test at Baseline visit Women who are breastfeeding
Facility Information:
Facility Name
Retinal Consultants of Arizona
City
Phoenix
State/Province
Arizona
Country
United States
Facility Name
Palo Alto Medical Foundation
City
Palo Alto
State/Province
California
ZIP/Postal Code
94040-2833
Country
United States
Facility Name
Stanford Byers Eye Institute
City
Stanford
State/Province
California
ZIP/Postal Code
94303
Country
United States
Facility Name
University of Colorado Health Eye Center
City
Aurora
State/Province
Colorado
Country
United States
Facility Name
Emory University Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Bethesda Neurology, LLC
City
Bethesda
State/Province
Maryland
Country
United States
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
New York Eye and Ear Infirmary
City
New York
State/Province
New York
ZIP/Postal Code
10003
Country
United States
Facility Name
University of Virginia
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Facility Name
AKH - Medizinische Universitaet Wien
City
Wien
Country
Austria
Facility Name
CHU Saint-Pierre
City
Brussels
Country
Belgium
Facility Name
Cliniques Universitaire Saint-Luc
City
Brussels
Country
Belgium
Facility Name
UZ Leuven - Campus Sint-Rafaël
City
Leuven
Country
Belgium
Facility Name
C. H. U. Sart Tilman
City
Liège
Country
Belgium
Facility Name
UMHAT "Alexandrovska" EAD
City
Sofia
Country
Bulgaria
Facility Name
Friedrich-Baur-Institut
City
Muenchen
Country
Germany
Facility Name
Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
City
Bologna
Country
Italy
Facility Name
SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej
City
Krakow
ZIP/Postal Code
31-501
Country
Poland
Facility Name
Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego
City
Poznań
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie
City
Szczecin
Country
Poland
Facility Name
Samodzielny Publiczny Kliniczny Szpital Okulistyczny
City
Warszawa
Country
Poland
Facility Name
Uniwersytecki Szpital Kliniczny
City
Wrocław
Country
Poland
Facility Name
Centro Hospitalar de São João, EPE
City
Porto
Country
Portugal
Facility Name
Institut Catala de Retina
City
Barcelona
Country
Spain
Facility Name
Hospital Universitario Ramon y Cajal
City
Madrid
Country
Spain
Facility Name
University Hospital of Wales
City
Cardiff
Country
United Kingdom
Facility Name
Moorfields Eye Hospital
City
London
Country
United Kingdom
Facility Name
Manchester Royal Eye Hospital
City
Manchester
Country
United Kingdom
Facility Name
Queen's Hospital
City
Romford
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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