Phase 4 N=199 Treatment

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

Leber's Hereditary Optic Neuropathy (LHON)

Bottom Line

View on ClinicalTrials.gov: NCT02774005 ↗

Enrolled (actual)

199

Serious AEs

13.6%

Results posted

Apr 2023

Primary outcome: Primary: Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline — 60 eyes — p=0.0021

Study Design & Population

Study type: Interventional
Phase: Phase 4
Interventions: Idebenone (Drug)
Age: Pediatric, Adult, Older Adult · 12+ yrs
Sex: All
Sponsor: Santhera Pharmaceuticals
Primary completion: Mar 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline	60	0.0021 sig
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	47	0.0873
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	20	0.0005 sig

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

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02774005). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.