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    Summary
    EudraCT Number:2018-001435-52
    Sponsor's Protocol Code Number:APL2-304
    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:2020-11-04
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-001435-52
    A.3Full title of the trial
    A Phase III, Multi-Center, Randomized, Double-Masked, Sham-Controlled Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy with Sham Injections in Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
    Studio multicentrico di Fase III, randomizzato, in doppia maschera, controllato con un trattamento fittizio (sham) per confrontare l'efficacia e la sicurezza della terapia con APL-2 intravitreale rispetto ad iniezioni sham in pazienti con atrofia geografica (AG) secondaria a degenerazione maculare legata all'età (DMLE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical research study involving an intravitreal injection in the eye with APL-2 or a sham injection for the treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration
    Studio clinico di ricerca che prevede un'iniezione intravitreale negli occhi con APL-2 o un'iniezione con un trattamento fittizio (sham) per il trattamento dell'atrofia geografica secondaria alla degenerazione maculare legata all'età
    A.3.2Name or abbreviated title of the trial where available
    Oaks
    Oaks
    A.4.1Sponsor's protocol code numberAPL2-304
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03525613
    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 SponsorAPELLIS PHARMACEUTCIALS, 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 supportApellis Pharmaceuticals
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationApellis Pharmaceuticals
    B.5.2Functional name of contact pointClin. Development and Med. Affairs
    B.5.3 Address:
    B.5.3.1Street Address6400 Westwind Way, Suite A
    B.5.3.2Town/ cityCrestwood
    B.5.3.3Post codeKY 40014
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016179775701
    B.5.5Fax number0016179775701
    B.5.6E-mailfederico@apellis.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 nameAPL-2
    D.3.2Product code [APL 2]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravitreal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor code-
    D.3.9.4EV Substance CodeSUB192794
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Geographic Atrophy Secondary to Age-Related Macular Degeneration
    Atrofia Geografica (AG) secondaria a degenerazione maculare legata all'età (DMLE)
    E.1.1.1Medical condition in easily understood language
    Patients with geographic atrophy generally develop a round area of cells called a lesion that can lead to vision loss.
    I pazienti affetti da atrofia geografica in genere sviluppano un'area rotonda di cellule chiamata lesione, che può portare alla perdita della vista.
    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 10063947
    E.1.2Term Geographic atrophy
    E.1.2System Organ Class 100000004853
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of APL-2 compared to sham injection in patients with GA secondary to AMD assessed by change in the total area of GA lesions from baseline as measured by FAF.
    Valutare l'efficacia di APL-2 rispetto all'iniezione sham in pazienti con AG secondaria a DMLE, valutata in base alla variazione dell'area totale lesionata da AG rispetto al basale, misurata mediante autofluorescenza del fondo oculare (fundus autofluorescence, FAF).
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of APL-2 compared to sham injection in patients with GA secondary to AMD with respect to:
    -Monocular reading speed, as assessed by Minnesota Reading or Radner Reading Charts
    -Functional Reading Independence Index composite score in the study eye
    -Normal luminance best-corrected visual acuity score (NL BCVA)
    To evaluate the efficacy of APL-2 compared to sham-injection in patients with GA secondary to AMD with respect to:
    -Low luminance best corrected visual acuity score (LL-BCVA) in the study eye
    -Low luminance deficit in the study eye
    -Total area of GA lesions in the study eye
    -Monocular critical print size, as assessed by MNRead or Radner Reading Charts
    -National Eye Institute Visual Functioning Questionnaire NEI VFQ-25 distance activity subscale score (in select countries)
    -Macular functional response as assessed by mesopic microperimetry for
    -To evaluate the PK of APL-2 as assessed by systemic plasma concentration of APL-2
    Valut efficacia APL-2 risp ad iniez sham in pz con AG second a DMLE in relazione a:
    -Vel di lettura monoculare (solo occhio in studio), misurata mediante tavole ottotipiche MNRead o di Radner (paesi selezionati)
    -Punt composito al test FRI, occhio in studio
    -Punt al test della BCVA a luminanza norm (NL-BCVA), occhio in studio
    Valut efficacia di APL-2 rispetto ad iniez sham in pz con AG second a DMLE in relazione a:
    -Punt al test della BCVA a bassa luminanza (LL-BCVA), occhio in studio
    -Deficit a bassa luminanza (LLD), occhio in studio
    -Area/e tot lesionata/e da AG, occhio in studio
    -Dimens critica di stampa nella lettura monoculare (solo occhio in studio), valut mediante tavole ottotipiche MNRead o di Radner (paesi selezionati)
    -Punt alla sottoscala della difficoltà in attività che richiedono una visione da lontano del NEI-VFQ 25 (paesi selezionati)
    -Risposta funz macula valut mediante microperimetria mesopica
    -Valut PK di APL-2 mediante determinaz concentraz plasmat sistemica di APL-2
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Pharmacogenetics
    Version: 1.0
    Date: 08/08/2018
    Title: A Phase III, Multi-Center, Randomized, Double-Masked, Sham-Controlled Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy with Sham Injections in Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
    Objectives: To evaluate the relationship between genetic polymorphisms with disease progression and response to APL-2

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: Clinical Repository Substudy:
    A Phase III, Multi-Center, Randomized, Double-Masked, Sham-Controlled Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy with Sham Injections in Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)_V.1.0_08Aug2018
    Objectives:
    • To study the association of biomarkers with efficacy, adverse events or disease progression
    • To increase knowledge and understanding of disease biology
    • To study drug response, including drug effects and the processes of drug absorption and disposition
    • To develop biomarker or diagnostic assays and establish the performance characteristics of these assays

    Farmacogenetica
    Versione: 1.0
    Data: 08/08/2018
    Titolo: Studio multicentrico di Fase III, randomizzato, in doppia maschera, controllato con un trattamento fittizio (sham) per confrontare l'efficacia e la sicurezza della terapia con APL-2 intravitreale rispetto ad iniezioni sham in pazienti con atrofia geografica (AG) secondaria a degenerazione maculare legata all'età (DMLE)
    Obiettivi: Valutare il rapporto tra i polimorfismi genetici con la progressione della malattia e la risposta ad APL-2

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Sottostudio per Biobanca:
    Studio multicentrico di Fase III, randomizzato, in doppia maschera, controllato con un trattamento fittizio (sham) per confrontare l'efficacia e la sicurezza della terapia con APL-2 intravitreale rispetto ad iniezioni sham in pazienti con atrofia geografica (AG) secondaria a degenerazione maculare legata all'età (DMLE)_V.1.0_08Aug2018
    Obiettivi:
    • Studiare il rapporto dei biomarcatori con l’efficacia, gli eventi avversi o la progressione della malattia
    • Ampliare la conoscenza e la comprensione della biologia della malattia
    • Studiare la risposta al farmaco, compresi gli effetti del farmaco e il suo processo di assorbimento ed escrezione
    • Sviluppare dosaggi dei biomarcatori o dosaggi diagnostici e stabilirne le rispettive caratteristiche prestazionali.
    E.3Principal inclusion criteria
    1.Age >= 60 years.
    2.NL-BCVA of 24 letters or better using ETDRS charts.
    3.Clinical diagnosis of GA of the macula secondary to AMD as determined by the Investigator and confirmed by the Reading Center.
    4.The GA lesion must meet the criteria as determined by the central reading center’s assessment of FAF imaging at screening.
    5.Adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the Investigator.
    6.Meets the criteria related to microperimetry.
    7.Female subjects must be women of non-child-bearing potential or women of child-bearing potential with a negative pregnancy test at screening and must agree to use protocol defined methods of contraception.
    8.Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception
    9.Willing and able to give informed consent and to comply with the study procedures and assessments.
    1.Età >= 60 anni
    2.Massima acuità visiva corretta a luminanza normale di 24 lettere o migliore mediante le tavole ETDRS (studio Trattamento precoce della retinopatia diabetica, Early Treatment Diabetic Retinopathy Study) (equivalente a un valore di circa 20/320 misurato utilizzando le tabelle ottotipiche di Snellen)
    3.Diagnosi clinica di AG della macula secondaria a DMLE, formulata dallo sperimentatore e confermata dall'unità di lettura
    4.L'area lesionata da AG deve rispondere ai seguenti criteri, determinati dall'unità di lettura centrale mediante autofluorescenza del fondo oculare (fundus autofluorescence, FAF) allo screening
    5.Adeguata trasparenza dei mezzi oculari, adeguata midriasi e fissazione tali da permettere l'acquisizione di immagini di buona qualità in base al giudizio dello sperimentatore
    6.Soddisfa i criteri relativi alla microperimetria
    7.Soggetti di sesso femminile non in età fertile (WONCBP), oppure soggetti di sesso femminile in età fertile con test di gravidanza su siero negativo allo screening, che acconsentono a utilizzare i metodi di contraccezione previsti dal protocollo
    8.I soggetti di sesso maschile con partner di sesso femminile in età fertile devono acconsentire a utilizzare i metodi di contraccezione previsti dal protocollo
    9.Disposti e in grado di fornire il consenso informato scritto e di rispettare le procedure e le valutazioni dello studio.
    E.4Principal exclusion criteria
    1.GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye.
    2.Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm.
    3.Any history or active CNV, associated with AMD or any other cause, including any evidence of retinal pigment epithelium rips or evidence of neovascularization anywhere based on SD-OCT imaging and/or fluorescein angiography as assessed by the Reading Center.
    4.Presence of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function, including but not limited to, uveitis, other macular diseases (e.g. clinically significant epiretinal membrane [ERM], full thickness macular hole or uncontrolled glaucoma/ocular hypertension). Benign conditions in the opinion of the investigator such as peripheral retina dystrophy are not exclusionary.
    5.Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.
    6.History of laser therapy in the macular region.
    7.Aphakia or absence of the posterior capsule. Note: YAG laser posterior capsulotomy for posterior capsule opacification done at least 60 days prior to screening is not exclusionary.
    8.Any ocular condition other than GA secondary to AMD that may require surgery or medical intervention during the study period or, in the opinion of the Investigator, could compromise visual function during the study period.
    9.Any contraindication to IVT injection including current ocular or periocular infection.
    10.History of prior intravitreal injection.
    11.Unable to perform microperimetry reliably in the opinion of the investigator.
    12. Prior participation in another interventional clinical study for intravitreal therapies in either eye (including subjects receiving sham).
    13. Prior participation in another interventional clinical study for geographic atrophy in either eye including investigational oral medication and placebo.
    14. Participation in any systemic experimental treatment or any other systemic investigational new drug within 6 weeks or 5 half-lives of the active ingredient (whichever is longer) prior to the start of study treatment.
    15. Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the 24-month treatment period unlikely, or would make the subject an unsafe study candidate.
    16. Any screening laboratory value (hematology, serum chemistry or urinalysis) that in the opinion of the Investigator is clinically significant and not suitable for study participation.
    17. Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to APL-2 or any of the excipients in APL-2 solution.
    1.AG secondaria a patologia diversa dalla DMLE, ad esempio malattia di Stargardt, distrofia dei coni e dei bastoncelli o maculopatie tossiche come la maculopatia da idrossiclorochina (Plaquenil) a carico dell'uno o dell'altro occhio.
    2.Equivalente sferico dell'errore refrattivo > 6 diottrie di miopia o lunghezza assiale >26 mm.
    3.Storia di neovascolarizzazione coroidale (CNV) o CNV attiva, associata a DMLE o a qualsiasi altra causa, inclusa qualsiasi evidenza di lacerazione dell'epitelio pigmentato retinico o evidenza di neovascolarizzazione in qualsiasi sede, determinata mediante tomografia ottica ad alta definizione (Spectral Domain OCT) e/o angiografia a fluorescenza con fluoresceina da parte dell'unità di lettura.
    4.Presenza di una patologia oftalmica attiva che, a giudizio dello sperimentatore, compromette o confonde la funzione visiva, inclusi, senza a essi limitarsi: uveite, altre malattie della macula (ad es. membrana epiretinica [epiretinal membrane, ERM] clinicamente significativa, foro maculare a pieno spessore o glaucoma/ipertensione oculare non controllati). Le condizioni valutate come benigne dallo sperimentatore, quali ad esempio le distrofie della retina periferica non comportano l'esclusione del soggetto dallo studio.
    5.Intervento di chirurgia intraoculare (inclusa la sostituzione del cristallino) nei 3 mesi precedenti la randomizzazione.
    6.Precedente trattamento con laserterapia nella regione maculare.
    7.Afachia o assenza della capsula posteriore. Nota: un eventuale intervento di capsulotomia posteriore YAG laser per opacizzazione della capsula posteriore effettuato almeno 60 giorni prima dello screening non comporta l'esclusione del soggetto dallo studio.
    8.Qualsiasi patologia oftalmica diversa dalla AG secondaria a DMLE che potrebbe richiedere un intervento chirurgico o medico nel periodo dello studio o che, a giudizio dello sperimentatore, potrebbe compromettere la funzione visiva nel periodo dello studio.
    9.Qualsiasi controindicazione alle iniezioni IVT inclusa un'eventuale infezione oculare o perioculare in corso.
    10.Esecuzione di iniezioni intravitreali precedenti.
    11.Incapacità di collaborare in maniera attendibile allo svolgimento della microperimetria secondo l'opinione dello sperimentatore.
    12.Partecipazione precedente ad altri studi clinici interventisti che abbiano previsto terapie intravitreali relative all'uno o all'altro occhio (inclusi i soggetti trattati con sham).
    13.Partecipazione precedente ad altri studi clinici interventistici per atrofia geografica in entrambi gli occhi, incluso il farmaco sperimentale per via orale e il placebo.
    14.Partecipazione a trattamento sperimentale sistemico o a qualsiasi altra sperimentazione clinica relativa a un nuovo farmaco sistemico entro 6 settimane o 5 emivite del principio attivo (a seconda di quale periodo sia maggiore) prima dell'inizio del trattamento dello studio.
    15.Condizioni mediche o psichiatriche che, a giudizio dello sperimentatore, renderebbero improbabile il regolare svolgimento delle attività di follow-up nei 24 mesi di trattamento, o comunque comporterebbero incertezze relative all'idoneità del soggetto a partecipare allo studio.
    16.Qualsiasi valore di laboratorio allo screening (analisi ematologiche, chimico-cliniche sul siero o analisi delle urine) che, a giudizio dello sperimentatore, sia clinicamente significativo e non idoneo per la partecipazione allo studio.
    17.Ipersensibilità nota a fluoresceina sodica iniettabile o ipersensibilità ad APL-2 o a uno qualsiasi degli eccipienti della soluzione di APL-2.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to Month 12 in total area of GA lesion(s) in the study eye (in mm2) based on Fundus Autofluorescence (FAF).
    Variazione dal basale al Mese 12 dell'area o delle aree totali lesionate da AG nell'occhio in studio (in mm2), misurata mediante autofluorescenza del fondo oculare (FAF).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 12
    Mese 12
    E.5.2Secondary end point(s)
    -Change from baseline in monocular reading speed (study eye), as assessed by Minnesota Reading (MNRead) or Radner Reading Charts
    -Change from baseline in Functional Reading Independence Index (FRI) composite score
    -Change from baseline in normal luminance best-corrected visual acuity score (NL-BCVA) as assessed by ETDRS chart
    -Change from baseline in low luminance best corrected visual acuity score (LL-BCVA) as assessed by ETDRS chart
    -Change from baseline in low luminance deficit (LLD)
    -Change from baseline at each planned assessment in the total area of GA lesion(s) in the study eye (in mm2) as assessed by FAF
    -Change from baseline in monocular critical print size (study eye), as assessed by MNRead or Radner Reading Charts, (in selected countries)
    -Change from baseline in the National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) distance activity subscale score (in select sites)
    -Number of scotomatous points assessed by mesopic microperimetry for the evaluation of the macular functional response
    -Change in macular sensitivity as assessed by mesopic microperimetry for the evaluation of the macular functional response
    -Systemic Plasma concentration of APL-2
    -Variazione dal basale della velocità di lettura monoculare (del solo occhio in studio), valutata mediante tavole ottotipiche MNRead o di Radner
    -Variazione dal basale del punteggio composito al test FRI
    -Variazione dal basale del punteggio al test della BCVA a luminanza normale (NL-BCVA), determinata mediante la tavola ETDRS
    -Variazione dal basale del punteggio al test della BCVA a bassa luminanza (LL-BCVA) determinata mediante la tavola ETDRS
    -Variazione dal basale del deficit a bassa luminanza
    -Variazione dal basale a ogni valutazione pianificata dell'area o delle aree totali lesionate da AG nell'occhio in studio (in mm2), misurata mediante FAF
    -Variazione dal basale della dimensione critica di stampa nella lettura monoculare (del solo occhio in studio), valutata mediante tavole ottotipiche MNRead o di Radner (in paesi selezionati)
    -Variazione del punteggio alla sottoscala della difficoltà nelle attività che richiedono una visione da lontano del 25-item National Eye Institute Visual Function Questionnaire (NEI-VFQ 25) (in centri selezionati)
    -Numero di aree scotomatose valutate mediante microperimetria mesopica per la determinazione della risposta funzionale della regione maculare
    -Variazione della sensibilità maculare valutata mediante microperimetria mesopica per la determinazione della risposta funzionale della regione maculare
    -Concentrazione plasmatica sistemica di APL-2
    E.5.2.1Timepoint(s) of evaluation of this end point
    -Month 24-Change from baseline in monocular reading speed MNRead or Radner Reading Charts
    -Month 24-Change from baseline in FRI composite score
    -Month 24-Change from baseline in NL-BCVA
    -Month 12, Month 24-Change from baseline in LL-BCVA
    -Month 12, Month 24-Change from baseline in LLD
    -Month 12, Month 24-Change from baseline in monocular critical print size
    -Month 12, Month 24-Change from baseline in NEI VFQ-25 distance activity subscale score
    -Months 6, 12, 18, 24-Number of scotomatous points and change in macular sensitivity, as assessed by mesopic microperimetry for the evaluation of the macular functional response.
    -Over time - Systemic Plasma concentration of APL-2
    -Mese 24-Variazione dal basale della velocità di lettura monoculare MNRead o di Radner
    -Mese 24-Variazione dal basale del punteggio composito al test FRI
    -Mese 24-Variazione dal basale in NL-BCVA
    -Mese 12, Mese 24-Variazione rispetto al basale in LL-BCVA
    -Mese 12, Mese 24-Variazione rispetto al basale in LLD
    -Mese 12, Mese 24-Variazione dal basale della dimensione critica di stampa nella lettura monoculare
    -Mese 12, Mese 24-Variazione rispetto al basale nel punteggio di sottoscala dell'attività a distanza NEI VFQ-25
    -Mese 6, 12, 18, 24-Numero di aree scotomatose e variazione della sensibilità maculare valutata mediante microperimetria mesopica per la valutazione della risposta funzionale maculare.
    -Over time-Concentrazione plasmatica sistemica di APL-2
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Trattamento fittizio (Sham)
    Sham procedure
    E.8.2.4Number of treatment arms in the trial4
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA45
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Czechia
    France
    Germany
    Hungary
    Israel
    Italy
    Netherlands
    New Zealand
    Poland
    Russian Federation
    Spain
    Ukraine
    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
    LVLS
    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 months6
    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 months6
    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 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: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 550
    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 state24
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 270
    F.4.2.2In the whole clinical trial 600
    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)
    Subjects will be offered entry into an open-label extension study at the end of the 24 month treatment period; those who enter the open label extension study will not require the 6 month follow-up period (Months 27 and 30).
    Alla fine del periodo di trattamento di 24 mesi, ai soggetti verrà proposto di partecipare a uno studio di estensione in aperto; per coloro che accetteranno di partecipare a tale estensione in aperto non sarà richiesto il follow-up di 6 mesi (valutazioni ai Mesi 27 e 30).
    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 Decision2019-03-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-20
    P. End of Trial
    P.End of Trial StatusOngoing
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