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    Summary
    EudraCT Number:2019-001493-28
    Sponsor's Protocol Code Number:OPA1
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-09-09
    Trial 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 ConcernedAustria - BASG
    A.2EudraCT number2019-001493-28
    A.3Full title of the trial
    Raxone® treatment for patients with dominant optic atrophy due to OPA1 gene mutation
    Raxone® Behandlung bei OPA1 bedingter dominanter Optikusatrophie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Raxone® treatment for patients with autosomal dominant optic atrophy (inherited optic nerve disorder)
    Raxone® Behandlung bei Patienten mit autosomal dominanter Optikusatrophie (erblich bedingte Erkrankung des Sehnerves).
    A.3.2Name or abbreviated title of the trial where available
    OPA1
    OPA1
    A.4.1Sponsor's protocol code numberOPA1
    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 SponsorMedizinische Universität Graz, Univ.-Augenklinik
    B.1.3.4CountryAustria
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportChiesi Pharmaceuticals GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedizinische Universität Graz, Univ.-Augenklinik
    B.5.2Functional name of contact pointClinical trials information
    B.5.3 Address:
    B.5.3.1Street AddressAuenbruggerplatz 4
    B.5.3.2Town/ cityGraz
    B.5.3.3Post code8036
    B.5.3.4CountryAustria
    B.5.4Telephone number00430316385 82899
    B.5.6E-mailkatharina.valentin@medunigraz.at
    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 150 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderChiesi Pharmaceuticals 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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRaxone 150 mg film-coated tablets
    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
    Autosomal dominant optic atrophy (ADOA)
    Autosomal dominante Optikusatrophie
    E.1.1.1Medical condition in easily understood language
    inherited disorder of the optic nerve
    erblich bedingte Erkrankung des Sehnervs
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10019895
    E.1.2Term Hereditary optic atrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluation of the therapeutic effect of 900mg Raxone® per day regarding visual acuity in ADOA patients with OPA1 mutation within a 12 month period.
    Evaluierung der langfristigen Wirksamkeit von 900mg Raxone® pro Tag auf den Visus bei ADOA PatientInnen mit OPA1 Mutation im Zeitraum von 12 Monaten
    E.2.2Secondary objectives of the trial
    Evaluation of the therapeutic effect of 900mg Raxone® per day in ADOA patients with OPA1 gene mutation regarding visual field, colour vision, contrast vision, change of the retinal nerve fibre layer (RNFL) and change of the visual performance- related quality of life (NEI-VFQ)
    Evaluation der Wirksamkeit von 900mg Raxone® pro Tag auf das Gesichtsfeld, Farbsehen und Kontrastsehen bei ADOA PatientInnen mit OPA1 Mutation. Es soll ebenfalls die Veränderung der retinalen Nervenfaserschicht (RNFL) und die Veränderung der sehleistungsbezogenen Lebensqualität (NEI-VFQ) beobachtet werden.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ADOA patients with confirmed OPA1 mutation
    Age of 12 years or more
    Willingness and ability to comply with study related procedures
    Informed consent
    Bestätigte OPA1 Mutation bei PatientInnen mit diagnostizierter genetischer ADOA
    Lebensalter von 12 oder mehr Jahren
    Absicht und Möglichkeit der PatientInnen an der Studie und den Kontrollen teilzunehmen
    Vorliegende Einwilligungserklärung
    E.4Principal exclusion criteria
    - patients in the state of pregnancy or lactation at the time of the examination or a planned pregnancy within the next 12 months
    - patients with allergies or hypersensitivity to the active substance or to any of the excipients contained in Raxone®
    - patients with hereditary diseases like galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption
    - patients with a high-grade hepatic or renal impairment
    - previous treatment with idebenone
    - patients with other diseases which would limit the compliance required for trial participation
    Before the prescription of the trial medication, the clinical investigator will conduct a detailed medication anamnesis. If there are possible drug interactions, the patient can be excluded from the trial
    - patients who have participated in other pharmaceutical product or medicine related trials in the last 3 months
    - Patients with a glaucoma or with any optic neuropathy other than ADOA
    - Patients with a baseline best- corrected- visual acuity less than counting fingers
    PatientInnen, die sich zum Untersuchungszeitpunkt in einer Schwangerschaft oder in der Stillzeit befinden bzw. eine Schwangerschaft innerhalb der nächsten 12 Monate planen

    PatientInnen mit Allergien oder Unverträglichkeiten auf einen in Raxone® enthaltenen Bestandteil,

    PatientInnen mit hereditären Erkrankungen wie Galactoseintoleranz, Lapp-Lactase-Mangel oder Glucose-Galactose-Malabsorption

    PatientInnen mit hochgradiger Leber- und Nierenfunktionsstörung

    Vorangehende Behandlung mit Idebenon

    PatientInnen mit Krankheiten, welche die Compliance einschränken könnten

    PatientInnen, die innerhalb von 3 Monaten an anderen klinischen Studien teilgenommen haben

    PatientInnen mit einem Glaukom oder mit einer anderen Optikusneuropathie als ADOA

    PatientInnen mit einem bestkorrigierten Visus zum Zeitpunkt der Basisuntersuchung von weniger als Fingerzählen
    E.5 End points
    E.5.1Primary end point(s)
    Best recovery/ least deterioration of visual acuity from baseline to 12 months measured with ETDRS charts (converted to logMAR).
    Der größte Visusanstieg bzw. der geringste Visusverlust von der Basisuntersuchung bis zur Kontrolle nach 12 Monaten gemessen mit ETDRS Tafel (in logMAR).
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    12 Monate
    E.5.2Secondary end point(s)
    - change of visual acuity on the better eye at the time of baseline examination measured with ETDRS charts (in logMAR)
    - change of visual acuity of both eyes measured with ETDRS charts (in logMAR)
    - change of visual field measured with Goldmann and/or Octopus perimetry
    - change of colour vision measured with Ishihara plates
    - change of contrast vision measured with Pelli Robson charts
    - change of retinal nerve fibre layer (OCT)
    - change of visual performance- related quality of life measured with National Eye Institute Visual Function Questionnaire (NEI-VFQ)
    - change of all primary and secondary outcome measures
    Veränderung des Visus am zur Basisuntersuchung besten Auges gemessen mit ETDRS Tafeln (in logMAR)
    Veränderung des Visus beider Augen gemessen mit ETDRS Tafeln (in logMAR)
    Veränderung des Gesichtsfeldes gemessen durch Octopus und/oder Goldmann Perimetrie
    Veränderung des Farbsehens gemessen durch Ishihara Tafeln
    Veränderung des Kontrastsehens gemessen durch Pelli Robson Tafeln
    Veränderung der retinalen Nervenfaserschicht
    Veränderung der sehleistungsbezogenen Lebensqualität gemessen durch NEI-VFQ
    Veränderungen aller primärer und sekundärer Zielgrößen
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    12 Monate
    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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    Letzter Studienbesuch des letzten Patienten
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    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: 3
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 13
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    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)
    According to local practice
    Gemäß lokaler Standards
    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 Decision2020-09-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-04-25
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