Clinical trial NCT02774005: Study to Assess the Efficacy and Safety of Raxone in LHON Patients
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
Overall status: COMPLETED
Phase: PHASE4
Conditions: Leber's Hereditary Optic Neuropathy (LHON)
Lead sponsor: Santhera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2016-05-16
Enrollment: 199
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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 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

Primary outcomes:
measure: Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline
timeFrame: 12 months
Secondary outcomes:
measure: 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
timeFrame: 12 months
measure: 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
timeFrame: 12 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Retinal Consultants of Arizona
state: Arizona
city: Palo Alto
country: United States
facility: Palo Alto Medical Foundation
state: California
city: Stanford
country: United States
facility: Stanford Byers Eye Institute
state: California
city: Aurora
country: United States
facility: University of Colorado Health Eye Center
state: Colorado
city: Atlanta
country: United States
facility: Emory University Hospital
state: Georgia
city: Bethesda
country: United States
facility: Bethesda Neurology, LLC
state: Maryland
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: New York
country: United States
facility: New York Eye and Ear Infirmary
state: New York
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Wien
country: Austria
facility: AKH - Medizinische Universitaet Wien
city: Brussels
country: Belgium
facility: CHU Saint-Pierre
city: Brussels
country: Belgium
facility: Cliniques Universitaire Saint-Luc
city: Leuven
country: Belgium
facility: UZ Leuven - Campus Sint-Rafaël
city: Liège
country: Belgium
facility: C. H. U. Sart Tilman
city: Sofia
country: Bulgaria
facility: UMHAT "Alexandrovska" EAD
city: Muenchen
country: Germany
facility: Friedrich-Baur-Institut
city: Bologna
country: Italy
facility: Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
city: Krakow
country: Poland
facility: SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej
city: Poznań
country: Poland
facility: Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego
city: Szczecin
country: Poland
facility: Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie
city: Warszawa
country: Poland
facility: Samodzielny Publiczny Kliniczny Szpital Okulistyczny
city: Wrocław
country: Poland
facility: Uniwersytecki Szpital Kliniczny
city: Porto
country: Portugal
facility: Centro Hospitalar de São João, EPE
city: Barcelona
country: Spain
facility: Institut Catala de Retina
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
city: London
country: United Kingdom
facility: Moorfields Eye Hospital
city: Manchester
country: United Kingdom
facility: Manchester Royal Eye Hospital
city: Romford
country: United Kingdom
facility: Queen's Hospital
Study officials:
References: