Study to Assess the Efficacy and Safety of Raxone in LHON Patients (LEROS)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT02774005 |
|
Recruitment Status :
Completed
First Posted : May 16, 2016
Last Update Posted : July 16, 2021
|
Sponsor:
Santhera Pharmaceuticals
Information provided by (Responsible Party):
Santhera Pharmaceuticals
- Study Details
- Tabular View
- Results Submitted
- Disclaimer
- How to Read a Study Record
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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Leber's Hereditary Optic Neuropathy (LHON) | Drug: Idebenone | Phase 4 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 199 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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 Start Date : | May 2016 |
| Actual Primary Completion Date : | March 29, 2021 |
| Actual Study Completion Date : | March 29, 2021 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Leber hereditary optic neuropathy
Autosomal dominant optic atrophy and cataract
MedlinePlus related topics:
Optic Nerve Disorders
| Arm | Intervention/treatment |
|---|---|
| Experimental: Raxone |
Drug: Idebenone
Other Name: Raxone |
Primary Outcome Measures :
- Proportion of eyes with clinically relevant recovery of visual acuity from Baseline [ Time Frame: 12 months ]Proportion 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
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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 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 AST, 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02774005
Locations
Show 29 study locations
Sponsors and Collaborators
Santhera Pharmaceuticals
| Responsible Party: | Santhera Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT02774005 |
| Other Study ID Numbers: |
SNT-IV-005 |
| First Posted: | May 16, 2016 Key Record Dates |
| Last Update Posted: | July 16, 2021 |
| Last Verified: | July 2021 |
Additional relevant MeSH terms:
|
Optic Nerve Diseases Optic Atrophy, Hereditary, Leber Cranial Nerve Diseases Nervous System Diseases Eye Diseases Optic Atrophies, Hereditary Optic Atrophy Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases |
Eye Diseases, Hereditary Genetic Diseases, Inborn Mitochondrial Diseases Metabolic Diseases Idebenone Antioxidants Molecular Mechanisms of Pharmacological Action Protective Agents Physiological Effects of Drugs |