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    Summary
    EudraCT Number:2018-001496-20
    Sponsor's Protocol Code Number:SMR3438
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-17
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-001496-20
    A.3Full title of the trial
    A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt disease
    Uno studio pilota, multinazionale, multicentrico, in doppio-cieco, controllato con placebo, per valutare la sicurezza, l’efficacia di soraprazan per via orale nella malattia Stargardt.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase II study of soraprazan in patients with Stargardt disease
    Uno studio di fase II con Soraprazan in pazienti con malattia di Stargardt
    A.3.2Name or abbreviated title of the trial where available
    SMR3438
    SMR3438
    A.4.1Sponsor's protocol code numberSMR3438
    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 SponsorKatairo GmbH
    B.1.3.4CountryGermany
    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 supportEuropean Union’s Horizon 2020 research and innovation
    B.4.2CountryEuropean Union
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSMERUD Medical Research
    B.5.2Functional name of contact pointProject management
    B.5.3 Address:
    B.5.3.1Street AddressO7,1
    B.5.3.2Town/ cityMannheim
    B.5.3.3Post code68161
    B.5.3.4CountryGermany
    B.5.4Telephone number004962117028490
    B.5.5Fax number0049621170284928
    B.5.6E-mailoliver.jungmann@smerud.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 No
    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/3/13/1208
    D.3 Description of the IMP
    D.3.1Product namesoraprazan
    D.3.2Product code [EU/3/13/1208 orphan]
    D.3.4Pharmaceutical form 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 INN(7R,8R,9R)-2.3-Dimethyl-8-hydroxy-7(2-methoxyethoxy)- 9-phenyl-7.8.9.10-tetrahydro-imidazo-[1.2-h].[1.7].- naphthyridine
    D.3.9.2Current sponsor codeSoraprazan
    D.3.9.4EV Substance CodeSUB25228
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Stargardt Disease
    Malattia di Stargardt
    E.1.1.1Medical condition in easily understood language
    Stargardt Disease
    Malattia di Stargardt
    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 PT
    E.1.2Classification code 10062766
    E.1.2Term Stargardt's disease
    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
    Evaluate the efficacy of soraprazan in reducing the amount of lipofuscin in RPE cells of subjects with Stargardt disease by assessing the change in quantitative autofluorescence (qAF8) from baseline to after treatment with soraprazan compared to placebo for up to 12 months.
    Valutare l'efficacia di soraprazan nel ridurre la quantità di lipofuscina nelle cellule dell’epitelio retinico pigmentoso (RPE) di soggetti con malattia Stargardt, valutando la variazione di auto-fluorescenza quantitativa (qAF8) dal basale a dopo il trattamento con soraprazan, rispetto al placebo, per un periodo di trattamento massimo di 12 mesi.
    E.2.2Secondary objectives of the trial
    Assess the safety and efficacy of soraprazan based on safety parameters and secondary efficacy parameters such as change in visual function and structural changes after treatment with soraprazan compared to placebo for up to 12 months.
    Valutare la sicurezza e l'efficacia di soraprazan sulla base di parametri di sicurezza e parametri di efficacia secondaria come il cambiamento nella funzione visiva e i cambiamenti strutturali dopo il trattamento con soraprazan rispetto al placebo per un periodo di trattamento massimo di 12 mesi
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Elevated qAF in at least one eye at screening (value =300 Units).
    • Male or female of any ethnicity and = 18 years old.
    • Onset of STGD disease before the age of 45 years
    • Visual acuity of the study eye: BCVA 0.2-0.8 (decimal unit).
    • Clinical diagnosis of typical autosomal recessive Stargardt’s macular dystrophy (STGD1).
    • Genetic report indicating at least two ABCA4 mutations (one with confirmed pathogenesis by a certified lab, one reported previously).
    • Study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eye drops and with sufficient fixation to permit good quality retinal imaging.
    • Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 12-months study.
    • Signed and dated informed consent form.
    • Female subjects of childbearing potential and male subjects participating in the study who are sexually active must use acceptable contraception. Male and female subjects documented as being of non-child bearing potential (e.g. infertile, surgically sterile, postmenopausal) are exempt from the contraceptive requirements.
    • QAF8 elevata in almeno un occhio allo screening (valore = 300 unità).
    • Maschio o femmina di qualsiasi etnia e = 18 anni.
    • Inizio della malattia di STGD prima dell'età di 45 anni · Acuità visiva dell'occhio di studio: BCVA 0.2-0.8 (unità decimale).
    • Diagnosi clinica della distrofia maculare di Stargardt autosomica recessiva tipica (STGD1).
    • Rapporto genetico che indica almeno due mutazioni ABCA4 (una con patogenesi confermata da un laboratorio certificato, una segnalata precedentemente).
    • L'occhio di studio deve avere medium oculari liberi e la dilatazione pupillare sufficiente, nessun'allergia alle gocce per la dilatazione dell'occhio, e con fissazione sufficiente per permettere la formazione immagine retinica di buona qualità
    • Capacità e disposizione a conformarsi ai requisiti di studio, alle limitazioni e alle istruzioni, e buona probabilità di completare lo studio di 12 mesi.
    • Rilascio di firma e data sul modulo di consenso informato.
    • Soggetti femminili fertili e soggetti maschi che sono sessualmente attivi devono usare contraccezione accettabile. I soggetti maschili e femminili che documentano la sterilità (ad esempio sterilità congenita, chirurgica, postmenopausale) sono esenti dai requisiti contraccettivi.
    E.4Principal exclusion criteria
    • Intolerance to acid pump antagonists (APAs).
    • Intake of prohibited medications/supplements (supplements containing vitamin A or beta-carotene, medications to treat any liver disease, or oral retinoid medications) within 28 days prior to screening and throughout the study.
    • Intake of other drugs with a pH dependent absorption, e.g. ketoconazole.
    • Breastfeeding, pregnant, or positive urine pregnancy test at screening or visit 2 (first intake of Investigational Medicinal Product (IMP)).
    • At screening, clinically significant abnormal haematology or biochemistry findings.
    • Acute or unstable severe disease or history of disease which in the opinion of the investigator would preclude participation in the study.
    • Active or history of an additional ocular disorder in the primary study eye that, in the opinion of the investigator, may confound the study results. These include, but are not limited to, any reason that might interfere with the imaging techniques used in the study (such as optic media opacity or poor pupil dilatation), inflammatory eye disease, other retinal disorders besides STGD, confirmed glaucoma or baseline intraocular pressure of =25mmHg, optic neuritis, high myopia (>8D spherical equivalent), amblyopia.
    • Intraocular surgery or injections in the primary study eye within 180 days of the screening visit.
    • Clinically significant abnormal electrocardiogram (ECG), or a corrected QT interval (QTc) of =450 ms in males or =470 ms in females.
    • Participation in any other investigational clinical trials within 28 days of the screening visit.
    • Intolleranza agli antagonisti delle pompe protoniche (APAs).
    • Assunzione di farmaci/integratori vietati (integratori contenenti vitamina A o beta-carotene, farmaci per il trattamento di qualsiasi malattia epatica, o retinoidi per bocca), entro 28 giorni prima dello screening e durante lo studio.
    • Assunzione di altri farmaci con assorbimento pH dipendente, ad es. ketoconazolo.
    • Allattamento al seno, gravidanza, o test in urina positivo di gravidanza a screening o alla Visita 2 (prima assunzione di medicinale sperimentale).
    • Allo screening, Ematologia o risultati di biochimica anormali clinicamente significativi.
    • Allo screening, malattia grave acuta o instabile o storia clinica che, a giudizio dello sperimentatore, precluderebbe la partecipazione allo studio.
    • Pregressa o presente malattia oculare supplementare nell'occhio primario di studio che, secondo il parere dello sperimentatore, può confondere i risultati di studio. Sono inclusi, a titolo di esempio, qualsiasi motivo che potrebbe interferire con le tecniche di imaging utilizzate nello studio (come l'opacità ottica del medium o la scarsa dilatazione delle pupille), una malattia infiammatoria degli occhi, altri disturbi retinici oltre a STGD, glaucoma confermato o pressione intraoculare di base = 25mmHg, neurite ottica, alta miopia (equivalente sferico di > 8D), ambliopia.
    • Chirurgia intraoculare o iniezioni nell'occhio primario di studio entro 180 giorni dalla visita di screening.
    • Elettrocardiogramma anormale clinicamente significativo (ECG), o un intervallo di QT corretto (QTc) = 450 ms nei maschi o = 470 ms nelle femmine.
    • Partecipazione a qualsiasi altro studio clinico sperimentale entro 28 giorni dalla visita di screening.
    E.5 End points
    E.5.1Primary end point(s)
    Changes in qAF8 score from baseline to end of treatment for soraprazan compared to placebo treated subjects.
    Variazione del -punteggio qAF8 dal basale alla fine del trattamento (EoT) per soraprazan rispetto ai soggetti trattati con placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and until end of treatment
    Baseline e fino alla fine del trattamento
    E.5.2Secondary end point(s)
    Functional:
    • Best corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) charts
    • BCVA as measured by ETDRS charts under low luminance conditions (i.e. Low Luminance Visual Acuity, LLVA)
    • Low Luminance Deficit (LLD) calculated on basis of BCVA and LLVA
    • Reading speed as assessed by Radner Charts
    • Critical print size as assessed by Radner Charts
    • Macular functional response as assessed by mesopic microperimetry
    • Subject reported visual function as assessed by the National Eye Institute Visual Functioning Questionnaire 25 (NEIVFQ-25) and Functional Reading Independence (FRI) Index

    Structural:
    • Changes in central subfield retinal thickness (CSRT) as assessed by spectral-domain optical coherence tomography (SD-OCT)
    • Changes in macular volume as assessed by SD-OCT
    • Changes in outer nuclear layer (ONL) thickness as assessed by SD-OCT
    • Changes in Ellipsoid Zones (EZ) as assessed by SD-OCT
    • Presence and changes of macular atrophy by fundus autofluorescence
    • Lesion progression

    Exploratory:
    • Pupillographic Campimetry
    • Adaptive Optics
    Funzionali
    1. Best-corrected Visual Acuity (BCVA) come misurata dalle tabelle dello studio ETDRS Early Treatment Diabetic Retinopathy Study
    2. BCVA come misurata da grafico ETDRS in condizioni di bassa luminanza (LLVA)
    3. Low Luminance Deficit (LLD) calcolato come differenza tra BCVA e LLVA
    4. Variazione dal basale per la massima velocità di lettura nella lettura binoculare e monoculare come valutato dal Test di Radner
    5. Variazione dal basale per la dimensione di stampa critica in visione binoculare e monoculare come valutato dal test di Radner.
    6. Risposta funzionale maculare come valutato da microperimetria mesopica
    7. Funzione visiva riferita dal soggetto come valutata dai questionari National Eye Institute Visual Functioning Questionnaire NEI VFQ-25 e Functional Reading Independence FRI.
    Strutturali
    8. Cambiamenti in Central Subfield Retinal Thickness (CSRT) come valutato da SD-OCT (Spectral Domain Optical Coherence Tomography
    9. Cambiamenti nel volume maculare come valutato da SD-OCT
    10. Cambiamenti nello spessore esterno dello strato nucleare (ONL) come valutato da SD-OCT
    11. Cambiamenti nelle zone ellissoidali (EZ) come valutato da SD-OCT
    12. Presenza e cambiamenti di atrofia maculare per autofluorescenza del fundus
    13. Progressione della lesione
    Esploratorie
    14. Campimetria pupillografica (solo al Centro di Tübingen)
    15 Adaptive Optics (solo al Centro di Tübingen)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline and until end of treatment
    Baseline e fino alla fine del trattamento
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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.8.4.1Number 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 EEA5
    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 Information not present in EudraCT
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 45
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 45
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 90
    F.4.2.2In the whole clinical trial 90
    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)
    Patients will be treated after the trial has been ended with the medical standard therapy according to §7 Abs. 2 Ziffer 13 GCP-V.
    I pazienti saranno trattati dopo che il processo è stato terminato con la terapia medica standard secondo § 7 ABS. 2 Ziffer 13 GCP-V.
    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 Decision2019-06-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-18
    P. End of Trial
    P.End of Trial StatusOngoing
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