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

Leber's Hereditary Optic Neuropathy (LHON) Clinical Trial

— 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02774005
• Other study ID #: SNT-IV-005
• Status: Completed
• Phase: Phase 4
• Start date: May 2016
• Est. completion date: March 29, 2021

Study information

• Verified date: March 2023
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 199
• Est. completion date: March 29, 2021
• Est. primary completion date: March 29, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Leber's Hereditary Optic Neuropathy (LHON)
• Optic Atrophy, Hereditary, Leber
• Optic Nerve Diseases

Intervention

Drug:
• Idebenone

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Bulgaria,  Germany,  Italy,  Poland,  Portugal,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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	12 months
Secondary	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	12 months