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    Summary
    EudraCT Number:2015-004405-16
    Sponsor's Protocol Code Number:SNT-IV-005
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-11-21
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBulgarian Drug Agency
    A.2EudraCT number2015-004405-16
    A.3Full title of the trial
    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)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study to assess the effectiveness and safety of Raxone during long time treatment in patients with LHON
    A.3.2Name or abbreviated title of the trial where available
    (LEROS) Open- Label Study to assess the Efficacy and Safety of Raxone in LHON Patients
    A.4.1Sponsor's protocol code numberSNT-IV-005
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSanthera Pharmaceuticals (Switzerland) Limited
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSanthera Pharmaceuticals
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanthera Pharmaceuticals (Switzerland) Ltd
    B.5.2Functional name of contact pointRegulatory Affairs Manager
    B.5.3 Address:
    B.5.3.1Street AddressHohenrainstrasse 24
    B.5.3.2Town/ cityPratteln
    B.5.3.3Post code4133
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number4161 906 8917
    B.5.5Fax number4161 906 8951
    B.5.6E-mailanna.carratu@santhera.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Raxone
    D.2.1.1.2Name of the Marketing Authorisation holderSanthera Pharmaceuticals (Deutschland) GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/434
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNidebenone
    D.3.9.1CAS number 58186-27-9
    D.3.9.3Other descriptive nameIDEBENONE
    D.3.9.4EV Substance CodeSUB08114MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Leber’s Hereditary Optic Neuropathy / LHON is a maternally inherited loss of vision due to atrophy of the optic nerve. It typically presents in young adults, mostly men, as painless acute or subacute visual failure of both eyes in quick succession. The estimated prevalence is approximately 2.2 per 100,000 (Mascialino et al., 2012) and LHON is thus an Orphan Disease according to EU and US criteria.
    E.1.1.1Medical condition in easily understood language
    Raxone is a medicine used to treat Leber’s hereditary optic neuropathy (LHON), a rare hereditary disease that can lead to blindness.
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10062951
    E.1.2Term Leber's hereditary optic atrophy neuropathy
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess efficacy of Raxone® in the promotion of recovery or stabilization of visual acuity in patients treated with Raxone® ≤1 year after the onset of symptoms, compared to an external natural history control group of idebenone naïve patients
    E.2.2Secondary objectives of the trial
    • To assess efficacy of Raxone® in the promotion of recovery or stabilization of vision in LHON patients treated with Raxone® >1 year after the onset of symptoms, compared to an external natural history control group of idebenone naïve patients
    • To compare the promotion of recovery or stabilization of visual acuity in LHON patients treated with Raxone® ≤1 and >1 year after the onset of symptoms
    • To assess the influence of mutation on the promotion of recovery or stabilization of visual acuity in LHON patients treated with Raxone®
    • To assess the influence of time since onset of symptoms prior to the initiation of treatment with Raxone® on the promotion of recovery or stabilization of visual acuity in LHON patients
    • To assess the influence of duration of treatment with Raxone® on changes in visual acuity in LHON patients
    • To assess safety of long-term treatment of LHON patients with Raxone®
    E.2.3Trial contains a sub-study No
    E.3Principal 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
    E.4Principal 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
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of eyes with clinically relevant recovery of 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
    Clinically Relevant Recovery (CRR) is defined as a change from “off-chart” visual acuity (VA) to at least 1.6 logMAR value or an improvement of at least 0.2 logMAR value within “on-chart”.
    E.5.1.1Timepoint(s) of evaluation of this end point
    see E.5.1 text
    E.5.2Secondary end point(s)
    • Components of the primary endpoint:
    - Proportion of eyes with CRR of VA from Baseline at Month 12 compared to matching external natural history control group
    - Proportion of eyes in which Baseline VA better than 1.0 logMAR was maintained at Month 12 compared to matching external natural history control group
    • Proportion of eyes in patients treated with Raxone® >1 year after the onset of symptoms with CRR of VA from Baseline or in which Baseline VA better than 1.0 logMAR was maintained at Month 12 compared to external natural history control group, in all patients and classified by mutation
    • Proportion of eyes and patients treated with Raxone® ≤1 year after the onset of symptoms with CRR of VA from Baseline or in which Baseline visual acuity better than 1.0 logMAR was maintained following 6, 18 and 24 months of treatment with Raxone® compared to matching external natural history control group, in all patients and classified by mutation
    • Proportion of eyes/patients treated with Raxone® ≤1 year or >1 year after the onset of symptoms with “Off-chart” VA at Baseline in whom VA improves to better than 1.60 logMAR by Month 6, 12, 18 and 24
    • Proportion of eyes/patients treated with Raxone® ≤1 year or >1 year after the onset of symptoms with VA in the categories of better than 1.0 logMAR, 1.0 to 1.68 logMAR and above 1.68 logMAR at each assessment time point up to Month 24
    • Safety as assessed by AE count and laboratory analyses during the study
    E.5.2.1Timepoint(s) of evaluation of this end point
    see E.5.2 text
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Austria
    Belgium
    Bulgaria
    Germany
    Italy
    Poland
    Portugal
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 30
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 123
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 7
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Paediatric population
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 130
    F.4.2.2In the whole clinical trial 160
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    At the end of the study, in countries where the drug is not commercially available, every effort will be made to assure continued access to Raxone® to the patients who the investigator believes derived and are still deriving a benefit from treatment administration and a continuous use is clinically indicated, according to the local specific regulation.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-12-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-01-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-09-23
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