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

NCT02774005 | Completed Phase 4 Results

• 1 other identifier: SNT-IV-005
• Study Type: interventional
• Target: 199
• Locations: 10 countries
• Sites: 29

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.

Conditions

Leber's Hereditary Optic Neuropathy (LHON)

Outcome Measures

Primary Outcomes (1)

• 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

  12 months

Secondary Outcomes (2)

• 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

  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

  12 months

Study Arms (1)

Raxone

EXPERIMENTAL

Drug: Idebenone

Interventions

Idebenone DRUG

Also known as: Raxone

Raxone

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (29)

Retinal Consultants of Arizona

Phoenix, Arizona, United States

Location

Palo Alto Medical Foundation

Palo Alto, California, 94040-2833, United States

Location

Stanford Byers Eye Institute

Stanford, California, 94303, United States

Location

University of Colorado Health Eye Center

Aurora, Colorado, United States

Location

Emory University Hospital

Atlanta, Georgia, 30322, United States

Location

Bethesda Neurology, LLC

Bethesda, Maryland, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

New York Eye and Ear Infirmary

New York, New York, 10003, United States

Location

University of Virginia

Charlottesville, Virginia, 22903, United States

Location

AKH - Medizinische Universitaet Wien

Vienna, Austria

Location

CHU Saint-Pierre

Brussels, Belgium

Location

Cliniques Universitaire Saint-Luc

Brussels, Belgium

Location

UZ Leuven - Campus Sint-Rafaël

Leuven, Belgium

Location

C. H. U. Sart Tilman

Liège, Belgium

Location

UMHAT "Alexandrovska" EAD

Sofia, Bulgaria

Location

Friedrich-Baur-Institut

München, Germany

Location

Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche

Bologna, Italy

Location

SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej

Krakow, 31-501, Poland

Location

Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego

Poznan, Poland

Location

Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie

Szczecin, Poland

Location

Samodzielny Publiczny Kliniczny Szpital Okulistyczny

Warsaw, Poland

Location

Uniwersytecki Szpital Kliniczny

Wroclaw, Poland

Location

Centro Hospitalar de São João, EPE

Porto, Portugal

Location

Institut Catala de Retina

Barcelona, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, Spain

Location

University Hospital of Wales

Cardiff, United Kingdom

Location

Moorfields Eye Hospital

London, United Kingdom

Location

Manchester Royal Eye Hospital

Manchester, United Kingdom

Location

Queen's Hospital

Romford, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 38428428 DERIVED

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Interventions

idebenone

Condition Hierarchy (Ancestors)

Optic Atrophies, Hereditary; Optic Atrophy; Optic Nerve Diseases; Cranial Nerve Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Eye Diseases, Hereditary; Eye Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Head of Regulatory Affairs EU
• Organization: Santhera Pharmaceuticals (Switzerland) Ltd

julien.gaudias@santhera.com

+41 6190689

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2016
• First Posted: May 16, 2016
• Study Start: May 1, 2016
• Primary Completion: March 29, 2021
• Study Completion: March 29, 2021
• Last Updated: April 21, 2023
• Results First Posted: April 21, 2023

Record last verified: 2023-03

Locations

PL (5); GB (4); BE (4); PT (1); ES (2); BU (1); DE (1); IT (1); US (9); AU (1)