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    Summary
    EudraCT Number:2018-003498-82
    Sponsor's Protocol Code Number:4429-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-01-22
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2018-003498-82
    A.3Full title of the trial
    A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease
    Estudio en fase III, multicéntrico, aleatorizado y con doble ciego para comparar la eficacia y la seguridad de clorhidrato de emixustat con placebo para el tratamiento de la atrofia macular secundaria a la enfermedad de Stargardt
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of Emixustat for the Treatment of Stargardt Disease
    Evaluación de Emixustat para el tratamiento de la Enfermedad de Stargardt
    A.3.2Name or abbreviated title of the trial where available
    The SeaSTAR Study (Safety and efficacy of EmixustAt in STARgardt disease)
    Estudio SeaSTAR (Seguridad y eficacia de EmixustAt en la enfermedad de STArgardt)
    A.4.1Sponsor's protocol code number4429-301
    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 SponsorAcucela Inc.
    B.1.3.4CountryUnited States
    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 supportAcucela Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcucela
    B.5.2Functional name of contact pointClinical Trial Helpdesk
    B.5.3 Address:
    B.5.3.1Street Address818 Stewart St., Suite 1110
    B.5.3.2Town/ citySeattle, WA
    B.5.3.3Post code98101
    B.5.3.4CountryUnited States
    B.5.4Telephone number+12068058310
    B.5.6E-mailClinicalTrials@acucela.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameEmixustat hydrochloride
    D.3.2Product code ACU-4429
    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 INNEmixustat hydrochloride
    D.3.9.1CAS number 1141934-97-5
    D.3.9.2Current sponsor codeACU-4429
    D.3.9.3Other descriptive nameEMIXUSTAT HYDROCHLORIDE (ACU-4429)
    D.3.9.4EV Substance CodeSUB96210
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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.IMP: 2
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameEmixustat hydrochloride
    D.3.2Product code ACU-4429
    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 INNEmixustat hydrochloride
    D.3.9.1CAS number 1141934-97-5
    D.3.9.2Current sponsor codeACU-4429
    D.3.9.3Other descriptive nameEMIXUSTAT HYDROCHLORIDE (ACU-4429)
    D.3.9.4EV Substance CodeSUB96210
    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 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
    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
    Enfermedad de Stargardt
    E.1.1.1Medical condition in easily understood language
    Stargardt Disease
    Enfermedad de 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
    To determine if emixustat hydrochloride (emixustat) reduces the rate of progression of macular atrophy (MA) compared to placebo in subjects with Stargardt disease (STGD)
    Determinar si el clorhidrato de emixustat (emixustat) reduce la velocidad de progresión de la atrofia macular (AM) en comparación con placebo en pacientes con enfermedad de Stargardt (ESTG)
    E.2.2Secondary objectives of the trial
    1. To evaluate the safety and tolerability of emixustat compared to placebo
    2. To assess changes in retinal sensitivity as determined by photopic microperimetry with emixustat compared to placebo
    3. To assess changes in contrast sensitivity with emixustat compared to placebo
    4. To assess changes in reading speed with emixustat compared to placebo
    5. To assess changes in best-corrected visual acuity (BCVA) letter score with emixustat compared to placebo
    6. To assess changes in total area of decreased autofluorescence (definitely decreased plus questionably decreased) with emixustat compared to placebo
    7. To assess changes in total area of ellipsoid zone (EZ) loss with emixustat compared to placebo
    8. To assess changes in mean outer nuclear layer (ONL) thickness with emixustat compared to placebo
    9. To assess changes in quality-of-life instruments (QOLs) with emixustat compared to placebo
    Evaluar:
    1. la seguridad y la tolerabilidad de emixustat en comparación con el placebo (Vs placebo)
    2. los cambios en la sensibilidad de la retina según lo determinado por microperimetría fotópica con emixustat Vs placebo
    3. los cambios en la sensibilidad al contraste con emixustat Vs placebo
    4. los cambios en la velocidad de lectura con emixustat Vs placebo
    5. los cambios en la puntuación de letras de la agudeza visual mejor corregida (MAVC) con emixustat Vs placebo
    6. Evaluar los cambios en el área total de disminución de autofluorescencia (disminución clara más disminución cuestionable) con emixustat Vs placebo
    7. los cambios en el área total de pérdida de zona elipsoide (ZE) con emixustat Vs placebo
    8. los cambios en el grosor medio de la capa nuclear externa (CNE) con emixustat Vs placebo
    9. los cambios en los instrumentos de la calidad de vida (CdV) con emixustat Vs placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who meet all of the following criteria at Screening and Baseline (unless otherwise indicated) may be eligible for inclusion in the study:
    1. Males or females, age ≥ 16 years.
    2. Clinical diagnosis of MA secondary to STGD in one or both eyes as determined by the Investigator.
    3. The subject must have 1 or more pathogenic mutation(s) of the ABCA4 (ATP binding cassette subfamily A member 4) gene. If only one ABCA4 pathogenic mutation is identified or if two ABCA4 pathogenic mutations that typically occur on the same allele (ie, “in cis”) are identified, the subject must have a typical STGD phenotype (at least one eye has flecks at the level of the retinal pigmented epithelium [RPE] typically seen in STGD) and be approved by the Sponsor. If 2 or more pathogenic mutations that do not typically occur on the same allele are identified, a typical STGD phenotype and separate Sponsor approval are not required. Segregation analysis is not required. The pathogenicity of all mutations will be determined by the Sponsor working with experts in ophthalmic genetics.
    4. The study eye must meet the following criteria as determined by the central reading center’s assessment of FAF imaging at Screening:
    a. Total area of DDAF
    i. If the lesion is unifocal: ≥ 3.0 – 22.0 mm2 (~1.2 – 8.7 disc areas) in size.
    ii. If the lesion is multifocal: ≥ 1.0 – 22.0 mm2 (~0.4 – 8.7 disc areas) in size.
    b. The entire lesion must be completely visualized on the macula-centered image (Field 2 – Macula Image). The DDAF lesion must be able to be imaged in its entirety, and all lesion borders must be ≥ 300 microns from all image edges.
    5. Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA letter score of ≥ 25 letters (approximately ≥ 20/320 Snellen) in the study eye at Screening.
    6. Adequate clarity of ocular media and adequate pupillary dilation to permit good quality imaging of macular atrophy in the study eye as determined by the Investigator.
    7. Able to reliably administer oral medication by self or with available assistance.
    8. Able and willing to provide written informed consent/assent
    a. For subjects ≥ 18 years of age: able and willing to provide written informed consent before undergoing any study-related procedures.
    b. For subjects ≥ 16 and < 18 years of age: able and willing to provide written informed assent, and has a parent or legal guardian able and willing to provide written informed consent for the minor before the subject undergoes any study-related procedures.
    Los sujetos que cumplan todos los criterios siguientes en la selección y el inicio (a menos que se indique lo contrario) pueden ser aptos para su inclusión en el estudio:
    1. Hombres o mujeres, edad ≥16 años.
    2. Diagnóstico clínico de AM secundaria a ESTG en uno o ambos ojos, según lo determinado por el investigador.
    3. El sujeto debe tener 1 o más mutaciones patógenas en el gen ABCA4 (miembro 4 de la subfamilia de transportadores dependientes de ATP). Si solo se identifica una mutación patógena en ABCA4 o dos mutaciones patógenas en ABCA4 que normalmente se producen en el mismo alelo (es decir, “en cis”), el sujeto debe tener un fenotipo de ESTG característico (al menos un ojo presenta manchas a nivel del epitelio pigmentado de la retina [EPR] observadas normalmente en la ESTG) y ser aprobado por el promotor. Si se identifican 2 o más mutaciones patógenas que no suelen aparecer en el mismo alelo, no se precisa un fenotipo de ESTG característico ni la aprobación aparte del promotor. No se precisa análisis de separación. El promotor determinará la capacidad patógena de todas las mutaciones en colaboración con expertos en genética oftálmica.
    4. El ojo del estudio debe cumplir los siguientes criterios según lo determinado mediante evaluación de la central de lectura para estudios de imagen de autofluorescencia del fondo de ojo (FAF) en la selección:
    a. El área total de autofluorescencia claramente reducida (DDAF)
    i. Si la lesión es unifocal: ≥3,0 – 22,0 mm2 (~1,2 – 8,7 áreas de disco) en tamaño.
    ii. Si la lesión es multifocal: ≥1,0 – 22,0 mm2 (~0,4 – 8,7 áreas de disco) en tamaño.
    b. La totalidad de la lesión debe visualizarse por completo en la imagen centrada en la mácula (campo 2 – Imagen de la mácula). La lesión de DDAF debe poder capturarse en su totalidad y todos los bordes de la lesión debe ser ≥300 micras desde todos los bordes de la imagen.
    5. Puntuación de letras de MAVC según el estudio de la retinopatía diabética con tratamiento temprano (ETDRS) ≥25 letras (aproximadamente ≥20/320 de Snellen) en el ojo del estudio en la selección.
    6. Claridad suficiente del medio ocular y dilatación suficiente de las pupilas para permitir la obtención de buena calidad de la imagen de la atrofia macular en el ojo del estudio, según lo determinado por el investigador.
    7. Capacidad para administrarse de forma fiable la medicación oral por sí mismo o con la ayuda disponible.
    8. Capacidad y disponibilidad para proporcionar consentimiento/asentimiento informado por escrito
    a. Para los sujetos ≥18 años de edad: capacidad y disponibilidad para proporcionar el consentimiento informado por escrito antes de someterse a cualquier procedimiento relacionado con el estudio.
    b. Para los sujetos ≥16 y <18 años de edad: capacidad y disponibilidad para proporcionar el asentimiento informado por escrito y tener un progenitor o tutor legal capaz y dispuesto a proporcionar el consentimiento informado por escrito para el menor antes de que el sujeto se someta a cualquier procedimiento relacionado con el estudio.
    E.4Principal exclusion criteria
    Subjects will be excluded from participation in the study if they meet any of the following criteria at Screening or Baseline (unless otherwise indicated):
    1. Macular atrophy associated with a condition other than STGD in either eye.
    2. DDAF with contiguous area of peripapillary atrophy in the study eye, as determined by the reading center.
    3. Mutation(s) in any of the following genes – elongation of very long chain fatty acids-like 4 (ELOVL4), prominin 1 (PROM1), or peripherin 2 (PRPH2)/retinal degeneration slow (RDS) – determined by the Sponsor working with experts in ophthalmic genetics to likely be disease-causing.
    4. If tested, any mutation(s):
    a. In a gene(s) encoding a visual cycle protein [e.g., retinal pigment epithelium 65 (RPE65), lecithin:retinol acyltransferase (LRAT), retinol dehydrogenase 12 (RDH12), RDH5, and retinaldehyde binding protein 1 (RLBP1)], confirmed by the Sponsor working with experts in ophthalmic genetics to likely be disease-causing. Testing for these mutations is not required.
    b. Associated with a non-STGD retinal dystrophy/degeneration, confirmed by the Sponsor working with experts in ophthalmic genetics to likely be disease-causing. Testing is not required.
    5. Presence in either eye of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function, including, but not limited to, choroidal neovascularization, diabetic retinopathy, uveitis, other macular diseases, or uncontrolled glaucoma/ocular hypertension.
    6. History of any intraocular or ocular surface surgery in either eye ≤ 3 months prior to Screening.
    7. Current or previous participation in an interventional study to treat STGD using gene therapy or stem cell therapy.
    8. Current or previous participation in a study to treat STGD using a vitamin A derivative ≤ 6 months prior to Screening.
    9. Current or previous participation in a study to treat STGD using a complement inhibitor ≤ 6 months prior to Screening.
    10. Known serious hypersensitivity to emixustat or any of the excipients in emixustat tablets (ie, silicified microcrystalline cellulose, pregelatinized starch, colloidal silicon dioxide, and stearic acid).
    11. Prohibited medications: Please refer to the protocol.
    12. Any of the following laboratory abnormalities at Screening: Please refer to the protocol
    13. Participation in any study using an investigational drug within 30 days of Screening.
    14. Participation in any study of an interventional, investigational device within 60 days of Screening.
    15. Anticipated participation during the study period in any study using an investigational drug or an interventional, investigational device.
    16. Presence of other medical or ophthalmic disease, physical examination finding, or clinical laboratory finding that in the opinion of the Investigator contraindicates the use of an investigational drug, places the subject at risk by participating in the study, might interfere with the evaluation of the efficacy or safety of emixustat, negatively impacts subject compliance with the protocol, confounds the ability to interpret data from the study, or jeopardizes the subject’s ability to complete the protocol.
    17. Current or history of cancer (except for adequately treated basal cell or squamous cell carcinoma of the skin) within 1 year of Screening.
    18. History of myocardial infarction, stroke, unstable ischemic heart disease, uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure within 6 months of Screening.
    19. Abnormal electrocardiogram (ECG) results that are considered by the Investigator to be clinically significant at Screening.
    20. Female subjects who are pregnant or lactating.
    21. Female subjects of childbearing potential (ie, not postmenopausal for at least two years and not surgically sterile) who are not willing to practice a medically accepted method of birth control with their non-surgically sterile male sexual partner from Screening through 30 days following the completion of the study. Medically accepted methods of birth control include abstinence, hormonal contraceptives, nonhormonal intrauterine contraceptive device with spermicide, condom with spermicide, contraceptive sponge with spermicide, diaphragm with spermicide, or cervical cap with spermicide.
    22. Male subjects who are not surgically sterile and are not willing to practice a medically accepted method of birth control with their female partner of childbearing potential (as listed above) from Screening through 30 days after completion of the study.
    Los sujetos quedarán excluidos de la participación en el estudio si cumplen cualquiera de los siguientes criterios en la selección o al inicio (a menos que se indique lo contrario):
    1.AM asociada con una afección distinta de la ESTG en cualquiera de los ojos.
    2.DDAF con zona contigua de la atrofia peripapilar en el ojo del estudio, según lo determinado por el centro de lectura.
    3.Mutación/es en cualquiera de los siguientes genes: elongación de ácidos grasos de cadena muy larga de tipo 4 (ELOVL4), prominina 1 (PROM1) o periferina 2 (PRPH2)/degeneración retiniana lenta (DRL), determinados por el promotor en colaboración con expertos en genética oftálmica como posibles causantes de enfermedad.
    4.Si se analizan, cualquier mutación:
    a.En un gen/es que codifica(n) una proteína del ciclo visual (p. ej., epitelio pigmentario retiniano 65 [EPR65], lecitin:retinol aciltransferasa [LRAT], retinol deshidrogenasa 12 [RDH12], RDH5 y proteína de unión a retinaldehído 1 [RLBP1]), confirmada por el promotor en colaboración con expertos en genética oftálmica como posible causante de enfermedad. No se precisa análisis para estas mutaciones.
    b.Se asocia con distrofia/degeneración retiniana distinta de ESTG, confirmada por el promotor en colaboración con expertos en genética oftálmica como posible causante de enfermedad. No se precisa análisis.
    5.Presencia en cualquiera de los ojos de una enfermedad ocular activa que, en opinión del investigador, ponga en peligro o confunda la función visual, incluidos, entre otros, neovascularización coroidea, retinopatía diabética, uveítis, otras enfermedades maculares o glaucoma no controlado/hipertensión ocular.
    6.Antecedentes de cirugía intraocular o de la superficie ocular en cualquier ojo ≤3 meses anteriores a la selección.
    7.Participación actual/previa en un estudio de intervención para tratar la ESTG utilizando terapia génica o tratamientos con células madre.
    8.Participación actual/previa en un estudio para tratar la ESTG utilizando un derivado de la vitamina A ≤6 meses antes de la selección.
    9.Participación actual/previa en un estudio para tratar la ESTG utilizando un inhibidor del complemento ≤6 meses antes de la selección.
    10.Hipersensibilidad grave conocida a emixustat o cualquiera de los excipientes de los comprimidos de emixustat (celulosa microcristalina silicificada, almidón pregelatinizado, dióxido de silicio coloidal y ácido esteárico).
    11.Medicamentos prohibidos: Consulte el protocolo
    12.Cualquiera de las siguientes anomalías analíticas en la selección: Consulte el protocolo
    13.Participación en cualquier estudio con un fármaco en investigación en los 30 días previos a la selección.
    14.Participación en cualquier estudio con un dispositivo en investigación intervencionista en los 60 días previos a la selección.
    15.Participación prevista durante el período del estudio en cualquier estudio con un fármaco en investigación o un dispositivo en investigación intervencionista.
    16.Presencia de otra enfermedad general u oftálmica, hallazgo en la exploración física o hallazgo en pruebas analíticas que, según el investigador, contraindique el uso de un fármaco en investigación, ponga al paciente en riesgo por su participación en el estudio, podría interferir en la evaluación de la eficacia o la seguridad de emixustat, afecte negativamente al cumplimiento del sujeto con el protocolo, confunda la interpretación de los datos del estudio, o pueda poner en peligro la capacidad del sujeto para completar el protocolo.
    17.Cáncer actual o antecedentes (excepto carcinoma basocelular tratado adecuadamente o carcinoma de células escamosas de la piel) en el año previo a la selección.
    18.Antecedentes de infarto de miocardio, accidente cerebrovascular, cardiopatía isquémica inestable, arritmia cardíaca no controlada u hospitalización por insuficiencia cardíaca congestiva en los 6 meses previos a la selección.
    19.Resultados anómalos en el ECG que el invest. considere clínicamente significativos en la selección.
    20.Mujeres embarazadas o en período de lactancia.
    21.Las pacientes en edad fértil (no son posmenopáusicas durante al menos dos años y no son estériles quirúrgicamente) que no estén dispuestas a utilizar un método anticonceptivo aceptado médicamente con su pareja sexual masculina no esterilizada quirúrgicamente desde la selección hasta 30 días después de la finalización del estudio. Los métodos anticoncept. médicamente aceptados son la abstinencia, anticoncept. hormonales, dispositivo anticoncept. intrauterino no hormonal con espermicida, preservativo con espermicida, esponja anticoncept. con espermicida, diafragma con espermicida o capuchón cervical con espermicida.
    22.Los sujetos varones que no estén esterilizados quirúrgicamente y que no estén dispuestos a utilizar un método anticonceptivo aceptado médicamente con su pareja de sexo femenino en edad fértil (como se indica anteriormente) desde la selección hasta 30 días después de la finalización del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the mean rate of change from baseline in the total area of the MA lesion(s) in the study eye (in mm2 per year), defined as the area of definitely decreased autofluorescence as imaged by reduced-illuminance FAF.
    El criterio de valoración principal de la eficacia será la tasa media del cambio desde el inicio en el área total de las lesiones de AM en el ojo del estudio (en mm2 al año), definida como el área con una disminución clara de autofluorescencia capturada por FAF de iluminancia reducida.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months
    24 meses
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints will include:
    1. Mean change from baseline in retinal sensitivity as assessed by photopic microperimetry
    2. Mean change from baseline in contrast sensitivity.
    3. Mean change from baseline in reading speed.
    4. Mean change from baseline in ETDRS BCVA letter score.
    5. Mean rate of change from baseline in the total area of decreased autofluorescence (definitely decreased plus questionably decreased), as imaged by reduced-illuminance FAF.
    6. Mean rate of change from baseline in the total area of EZ loss, as imaged on SD-OCT
    7. Mean change from baseline in mean ONL thickness, as imaged on SD-OCT
    8. Mean change from baseline in QOL instruments.
    Los criterios secundarios de valoración de la eficacia incluyen:
    1. Cambio medio desde el inicio en la sensibilidad retiniana según la evaluación mediante microperimetría fotópica
    2. Cambio medio desde el inicio en la sensibilidad al contraste.
    3. Cambio medio desde el inicio en la velocidad de lectura.
    4. Cambio medio desde el inicio en la puntuación de letras de MAVC del ETDRS.
    5. Tasa media de cambio desde el inicio en el área total de la disminución de autofluorescencia (disminución clara más disminución cuestionable), según lo determinado mediante FAF con iluminancia reducida.
    6. Tasa media de cambio desde el inicio en el área total de pérdida de ZE, según TCO-DE
    7. Cambio medio desde el inicio en el grosor medio de CNE, según TCO-DE
    8. Cambio medio desde el inicio en los instrumentos de CdV.
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 months
    24 meses
    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 Yes
    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) Yes
    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 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 EEA14
    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
    Brazil
    Canada
    Denmark
    France
    Germany
    Italy
    Netherlands
    South Africa
    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
    LVLS
    Última visita del último paciente
    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 months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial 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: 7
    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: 7
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 150
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Minor 16 to <18
    Menores de edad entre los 16 y 18 años
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 84
    F.4.2.2In the whole clinical trial 162
    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)
    None
    Ninguno
    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 Decision2019-04-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-09
    P. End of Trial
    P.End of Trial StatusOngoing
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