EUCTR2015-004405-16-ITActive, not recruitingPhase 1
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) - (LEROS) Open- Label Study to assess the Efficacy and Safety of Raxone in LHON Patients
Conditions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- SANTHERA PHARMACEUTICALS
- Enrollment
- 199
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •The following criteria should be assessed during Baseline Visit before randomization. If any does not apply, the patient must not be included in the study:
- •1. Impaired visual acuity in affected eyes due to LHON
- •2. No explanation for visual loss besides LHON
- •3. Age=12 years
- •4. Onset of symptoms =5 years prior to 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
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 188
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 12
Exclusion Criteria
- •The following criteria should be checked during Baseline Visit before randomization. If any applies, the patient must not be included in the study:
- •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
Investigators
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