Leber's Hereditary Optic Neuropathy (LHON)Phase 4LEROSNCT02774005
Study to Assess the Efficacy and Safety of Raxone in LHON Patients
A Phase 4 study testing Idebenone in people with Leber's Hereditary Optic Neuropathy (LHON), run by Santhera Pharmaceuticals at 29 sites in 10 countries.
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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Leber's Hereditary Optic Neuropathy (LHON)
- What is being tested?
- Idebenone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 199 participants
- Where
- 29 sitesin 10 countries
- Study dates
- May 2016to 29 Mar 2021
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
29 sites in 10 countries.
29 sites in 10 countries
United States9 sites
Emory University Hospital
Atlanta, Georgia, 30322
University of Colorado Health Eye Center
Aurora, Colorado
Bethesda Neurology, LLC
Bethesda, Maryland
University of Virginia
Charlottesville, Virginia, 22903
New York Eye and Ear Infirmary
New York, New York, 10003
Palo Alto Medical Foundation
Palo Alto, California, 94040-2833
Retinal Consultants of Arizona
Phoenix, Arizona
Washington University
St Louis, Missouri, 63110
Stanford Byers Eye Institute
Stanford, California, 94303
Poland5 sites
SPZOZ Spital Uniwersytecki w Krakowie, Oddzial Kliniczny Okulistyki i Onkologii Okulistycznej
Krakow, 31-501
Szpital Kliniczny Przemienienia Panskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego
Poznan
Samodzielny Publiczny Szpital Kliniczny Nr 2 PUM w Szczecinie
Szczecin
Samodzielny Publiczny Kliniczny Szpital Okulistyczny
Warsaw
Uniwersytecki Szpital Kliniczny
Wroclaw
Belgium4 sites
CHU Saint-Pierre
Brussels
Cliniques Universitaire Saint-Luc
Brussels
UZ Leuven - Campus Sint-Rafaël
Leuven
C. H. U. Sart Tilman
Liège
United Kingdom4 sites
University Hospital of Wales
Cardiff
Moorfields Eye Hospital
London
Manchester Royal Eye Hospital
Manchester
Queen's Hospital
Romford
Spain2 sites
Institut Catala de Retina
Barcelona
Hospital Universitario Ramon y Cajal
Madrid
Italy1 site
Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
Bologna
About this study
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)
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.
- Being tested
- Idebenone
- Medical subject headings
- Optic Atrophy, Hereditary, Leber
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdebenoneDrug
Also called Raxone
| Study group | Role | What they receive |
|---|---|---|
| Raxone | Experimental | Not described Receives: Drug: Idebenone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
Secondary outcomes (2)
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
Measured: 12 months
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
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2016 (estimated)
- Study startMay 2016
- Primary completion29 Mar 2021
- Study completion29 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Leber's Hereditary Optic Neuropathy (LHON)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 199 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02774005
- Sponsor's protocol ID
- SNT-IV-005
- Acronym
- LEROS
- Status last verified
- Mar 2023
- First submitted
- 12 May 2016
- Last updated
- 21 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Yu-Wai-Man P, Carelli V, Newman NJ, Silva MJ, Linden A, Van Stavern G, Szaflik JP, Banik R, Lubinski W, Pemp B, Liao YJ, Subramanian PS, Misiuk-Hojlo M, Newman S, Castillo L, Kociecki J, Levin MH, Munoz-Negrete FJ, Yagan A, Cherninkova S, Katz D, Meunier A, Votruba M, Korwin M, Dziedziak J, Jurkute N, Harvey JP, La Morgia C, Priglinger C, Lloria X, Tomasso L, Klopstock T; LEROS Study Group. Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. Cell Rep Med. 2024 Mar 19;5(3):101437. doi: 10.1016/j.xcrm.2024.101437. Epub 2024 Feb 29.
PubMed 38428428
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02774005
- Acronym / sponsor ID
- LEROS / SNT-IV-005
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 199 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- May 2016
- Primary completion
- 29 Mar 2021 (actual)
- Study completion
- 29 Mar 2021 (actual)
- First posted
- 16 May 2016 (estimated)
- Results
- Posted 21 Apr 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Raxone | Experimental | Drug: Idebenone |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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)
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IdebenoneAlso: Raxone | Drug | Raxone |
Primary outcome
Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline
Time frame: 12 months
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
2 secondary outcomes
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
Time frame: 12 months
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
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38428428Yu-Wai-Man P, Carelli V, Newman NJ, Silva MJ, Linden A, Van Stavern G, Szaflik JP, Banik R, Lubinski W, Pemp B, Liao YJ, Subramanian PS, Misiuk-Hojlo M, Newman S, Castillo L, Kociecki J, Levin MH, Munoz-Negrete FJ, Yagan A, Cherninkova S, Katz D, Meunier A, Votruba M, Korwin M, Dziedziak J, Jurkute N, Harvey JP, La Morgia C, Priglinger C, Lloria X, Tomasso L, Klopstock T; LEROS Study Group. Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. Cell Rep Med. 2024 Mar 19;5(3):101437. doi: 10.1016/j.xcrm.2024.101437. Epub 2024 Feb 29.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
29 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Idebenone in people with Leber's Hereditary Optic Neuropathy (LHON), run by Santhera Pharmaceuticals at 29 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, Poland, Belgium, United Kingdom, Spain and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Apr 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02774005, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Apr 2023.