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    EudraCT Number:2010-023111-34
    Sponsor's Protocol Code Number:TDU13583
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-09-28
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2010-023111-34
    A.3Full title of the trial
    A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients with Stargardt's Macular Degeneration.
    Studio di fase I/IIa volto a valutare la sicurezza dell’incremento della dose di SAR422459, somministrato per iniezione sottoretinica a pazienti affetti da degenerazione maculare di Stargardt
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/IIa Study of SAR422459 in Patients With Stargardt's Macular Degeneration

    Studio di fase I/IIa con SAR422459 in pazienti affetti da degenerazione maculare di Stargardt.
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberTDU13583
    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.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 supportSANOFI-AVENTIS RECHERCHE ET DEVELOPPEMENT
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSANOFI S.p.A.
    B.5.2Functional name of contact pointCONTACT POINT
    B.5.3 Address:
    B.5.3.1Street AddressVIALE BODIO, 37/B
    B.5.3.2Town/ cityMILANO
    B.5.3.3Post code20158
    B.5.4Telephone number800226343
    B.5.5Fax number00390239394168
    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 numberEMEA/OD/127/09
    D.3 Description of the IMP
    D.3.1Product nameSAR422459
    D.3.2Product code Vett Lentiviral con gene ABCA4
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSAR422459
    D.3.9.2Current sponsor codeSAR422459
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number180000 to 1800000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product Yes
    D. classification and reference numberCAT reference number : EMEA/CAT/801443/2009
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    Stargardt Macular Degeneration, also known as Stargardt Macular Dystrophy, fundus flavimaculatus or Stargardt disease.
    Degenerazione maculare di Stargard, conosciuta anche come Distrofia Maculare di Stargard, fundus flavimaculato o Malattia di Stargard
    E.1.1.1Medical condition in easily understood language
    Inherited juvenile macular degeneration, leading to deterioration of central vision, and legally-defined blindness in young adults.
    Degenerazione maculare giovanile ereditaria che porta al deterioramento della visione centrale e cecità legalmente definita nei giovani adulti.
    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 assess the safety and tolerability of ascending doses of SAR422459 in patients with Stargardt's Macular Degeneration.
    Valutare la sicurezza e la tollerabilità di dosi crescenti di SAR422459 in pazienti affetti da degenerazione maculare di Stargardt
    E.2.2Secondary objectives of the trial
    To evaluate for possible biological activity of SAR422459.
    Valutare la possibile attività biologica di SAR422459
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Signed and dated written informed consent obtained from the patient and/or the patient's legally acceptable representative.

    -Diagnosis of Stargardt's Macular Degeneration, with at least one pathogenic mutant ABCA4 allele on each chromosome.

    -Women of childbearing potential must have a negative pregnancy test at Day -1, and agree to use an effective form of contraception for at least three months, or be surgically sterile or postmenopausal, with the last menstrual period being over two years prior to enrollment .

    -Males must agree with their partner to use two forms of contraception for at least three months following SAR422459 administration.

    -Patients must agree to not donate blood, organs, tissues or cells for at least three months following SAR422459 administration

    -Patients enrolled in France must be affiliated to or benefit from a social security regimen.

    -Specific Inclusion Criteria Patient Group A:
    -Patients (18 years or older) with Advanced Stargardt's Macular degeneration.
    -Visual acuity ≤20/200 in the worst eye
    -Severe cone-rod dysfunction with no detectable or severely abnormal full-field
    electroretinogram responses.

    -Specific Inclusion Criteria Patient Group B:
    -Patients (18 years or older) with Stargardt’s Macular Degeneration.
    -Visual Acuity ≤20/200 in the worst eye.
    -Abnormal full-field electroretinogram responses.

    -Specific Inclusion Criteria Patient Group C:
    -Patients (18 years or older) with Stargardt’s Macular Degeneration.
    -Visual acuity ≤20/100 in the worst eye.
    -Abnormal full-field electroretinogram responses.

    -Specific Inclusion Criteria Patient Group D:
    -Symptomatic patients (between 12 years and 26 years old in Italy) with early or childhood-onset SMD (age at disease onset < 18 years) with at least one pathogenic mutant ABCA4 allele on each chromosome confirmed by direct sequencing and co-segregation analysis within the patient’s family.
    -Visual acuity of ≥20/200 in both eyes at the time of the screening visit.
    -Patients are anticipated to experience rapid deterioration in visual function
    and/or retinal structure as determined by at least one of the following parameters occurring within the span of a year:
    -Loss of ≥1 line of Snellen visual acuity (equivalent to 5 ETDRS letters).
    -Reduction in macular mean sensitivity of ≥1.2 dB as assessed by microperimetry.
    -Reduction in macular mean sensitivity of ≥5 dB or reduction in hill of vision by >14 dB-sr as assessed by static perimetry.
    -Enlargement in the area of macular RPE atrophy by fundus autofluorescence at a rate of ≥0.5 mm^2.
    -Enlargement in the area of central macular retinal thinning/photoreceptor loss by ocular coherence tomography at a rate of ≥0.5 mm^2.
    -All eligible patients must demonstrate an ability to understand, willingness to cooperate and ability to reliably perform required study procedures as judged and confirmed by the study investigator.

    Note: Recruitment in Group D will be staggered starting with patients 16 year or older. Enrolment of younger patients will then be guided by collected data, following review of the independent DSMB.

    - Consenso informato scritto firmato e datato, ottenuto dal paziente e/o dal rappresentante legalmente riconosciuto del paziente
    - Diagnosi di degenrazione maculare di Stargardt, con presenza di almeno un allele ABCA4 mutato patogeno su ciascun cromosoma
    - Le donne in età fertile devono presentare un test di gravidanza negativo al Giorno -1 e acconsentire all’utilizzo di una forma di contraccezione efficace per almeno tre mesi, oppure devono essere chirurgicamente sterili o in postmenopausa, con l’ultimo ciclo mestruale risalente a più di due anni prima dell’arruolamento.
    - I soggetti di sesso maschile devono accettare, insieme alla propria partner, l’utilizzo di due forme di contraccezione per almeno tre mesi dopo la somministrazione di SAR422459
    - I pazienti devono acconsentire a non donare sangue, organi, tessuti o cellule per almeno tre mesi in seguito alla somministrazione di SAR422459.
    -I pazienti arruolati in Francia devono essere affiliati o comunque coperti da un regime di sicurezza sociale.

    Criteri di inclusione specifici per i pazienti del gruppo A
    - Pazienti (di età pari o superiore a 18 anni) con degenerazione maculare di Stargardt avanzata.
    - VA ≤ 20/200 nell’occhio peggiore.
    - Grave disfunzione dei coni e dei bastoncelli con risposte all’ERG a campo pieno non rilevabili o gravemente anomale.

    Criteri di inclusione specifici per i pazienti del gruppo B:
    - Pazienti (di età pari o superiore a 18 anni) con SMD.
    - VA ≤ 20/200 nell’occhio peggiore.
    - Risposte anomale all’ERG a campo pieno.

    Criteri di inclusione specifici per i pazienti del gruppo C
    - Pazienti (di età pari o superiore a 18 anni) con SMD.
    - VA ≤ 20/100 nell’occhio peggiore.
    - Risposte anomale all’ERG a campo pieno.

    Criteri di inclusione specifici per i pazienti del gruppo D
    - Pazienti sintomatici (di età compresa tra i 12 e i 26 anni in Italia) con SMD ad esordio precoce o infantile (età all’esordio della malattia < 18 anni) con presenza di almeno un allele ABCA4 mutato patogeno su ciascun cromosoma confermata mediante sequenziamento diretto e analisi di co-segregazione all’interno della famiglia del paziente.
    - Acuità visiva ≥ 20/200 in entrambi gli occhi al momento della visita di screening.
    - Pazienti per i quali si prevede un rapido deterioramento della funzione visiva e/o della struttura retinica dei pazienti, determinato dal verificarsi di almeno uno dei seguenti parametri entro l’intervallo di un anno:
    - Perdita di ≥ 1 riga sulla tabella di Snellen dell’acuità visiva (equivalente a 5 lettere ETDRS)
    - Riduzione della sensibilità maculare media di ≥ 1,2 dB valutata mediante microperimetria
    - Riduzione della sensibilità maculare media di ≥ 5 dB o riduzione della hill of vision di >14 dB-sr valutata mediante perimetria statica
    - Allargamento dell’area di atrofia dell’epitelio pigmentato retinico (RPE) maculare mediante autofluorescenza del fondo a una frequenza ≥ 0,5 mm2
    - Allargamento dell’area di assottigliamento retinico/perdita di fotorecettori nella zona centrale della macula mediante tomografia a coerenza ottica a una frequenza ≥ 0,5 mm2
    - Tutti i pazienti idonei devono dimostrare capacità di comprensione, volontà di collaborare e capacità di eseguire in modo affidabile le procedure richieste dallo studio, secondo il giudizio e come confermato dallo sperimentatore dello studio.
    Nota: Il reclutamento nel gruppo D sarà graduale a partire dai pazienti di 16 anni o più. L'arruolamento di pazienti più giovani sarà poi guidata dai dati raccolti, in seguito alla revisione del DSMB indipendente.
    E.4Principal exclusion criteria
    -Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints.

    -Cataract surgery with intraocular lens implantation within 6 months of enrolment.

    -Aphakia or prior vitrectomy in the study eye.

    -Concomitant systemic diseases including those in which the disease itself, or the treatment for the disease, can alter ocular function.

    -Any intraocular surgery or laser in either eye planned within 6 months of Day 0.

    -Any contraindication to pupil dilation in either eye.

    -Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study, or medications planned for use in the peri-operative period, particularly topical, injected or systemic corticosteroids.

    -Any injectable intravitreal treatment to the treated eye or intravitreal device in the treated eye within 6 months prior to screening.

    -Any periocular injections of corticosteroids to the treated eye within 4 months prior to screening.

    -Laboratory test abnormalities or abnormalities in electrocardiogram, chest X rays that in the opinion of the principal investigator, would make the patient unsuitable for participation in the study.

    -Significant intercurrent illness or infection during the 28 days prior to enrolment.

    -Pre-menopausal or non-surgically sterile women who are unwilling to use an effective form of contraception such as the contraceptive pill or intrauterine device.

    -Alcohol or other substance abuse.

    -Contraindications to use of anaesthesia (local or general, as appropriate).

    -Concurrent anti-retroviral therapy that would inactivate the investigational agent.

    -History of any investigational agent within 28 days prior to SAR422459 administration.

    -Participation in a prior ocular gene transfer therapy study.

    -Enrollment in any other clinical treatment study throughout the duration of the SAR422459 study.

    -Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy within the four weeks prior to surgery.

    -A past medical history of human immunodeficiency virus (HIV) or hepatitis A, B or C infection.

    -Women who are pregnant or are breastfeeding.

    -History or signs consistent with unilateral amblyopia (strabismic, anisometropic or stimulus deprivation).
    - Condizioni oculari preesistenti che precludano l’intervento chirurgico previsto o interferiscano con l’interpretazione degli endpoint dello studio
    - Chirurgia della cataratta con impianto di lente intraoculare nei 6 mesi precedenti l’arruolamento.
    - Afachia o precedente vitrectomia nell’occhio di studio.
    - Malattie sistemiche concomitanti, incluse quelle in cui la malattia stessa, o il trattamento per la malattia, possa alterare la funzione oculare.
    - Qualsiasi procedura intraoculare chirurgica o laser in qualunque occhio prevista nei 6 mesi precedenti il Giorno 0.
    - Qualsiasi controindicazione alla dilatazione della pupilla in qualunque occhio.
    - Qualsiasi allergia nota a qualsiasi componente del veicolo di somministrazione o agli agenti diagnostici utilizzati durante lo studio oppure ai farmaci con uso previsto nel periodo perioperatorio, in particolare
    corticosteroidi topici, iniettabili o sistemici.
    - Qualsiasi trattamento intravitreale iniettabile nell’occhio trattato o dispositivo intravitreale nell’occhio trattato nei 6 mesi precedenti lo screening.
    - Qualsiasi iniezione perioculare di corticosteroidi a livello dell’occhio trattato nei 4 mesi precedenti lo screening.
    - Anomalie agli esami di laboratorio o anomalie all’elettrocardiogramma o alle radiografie toraciche che, a giudizio dello sperimentatore principale , potrebbero rendere il paziente inidoneo alla partecipazione allo studio.
    -Significativa malattia intercorrente o infezione durante i 28 giorni precedenti l’arruolamento.
    - Donne in premenopausa o non chirurgicamente sterili che non sono disposte a utilizzare una forma di contraccezione efficace, quali la pillola anticoncezionale o un dispositivo intrauterino.
    - Abuso di alcol o di altre sostanze.
    - Controindicazioni all’uso dell’anestesia (locale o generale, come appropriato).
    - Terapia antiretrovirale concomitante che inattiverebbe l’agente sperimentale.
    - Anamnesi di qualsiasi agente sperimentale nei 28 giorni precedenti la somministrazione di SAR422459.
    - Partecipazione a un precedente studio di terapia oculare di trasferimento genico.
    - Arruolamento in qualsiasi altro studio di trattamento clinico per tutta la durata della partecipazione allo studio con SAR422459.
    - Trattamento attuale o previsto con terapia anticoagulante o uso di terapia anticoagulante nelle 4 settimane precedenti l’intervento chirurgico.
    - Anamnesi medica remota di infezione da HIV o epatite A, B o C.
    - Donne in stato di gravidanza o in allattamento.
    - Anamnesi o segni coerenti con ambliopia monolaterale (strabismo, anisometropia o deprivazione sensoriale).
    E.5 End points
    E.5.1Primary end point(s)
    • Number of patients with treatment emergent adverse events
    • Change from baseline in ocular safety assessments
    • Measured as change from baseline in Best Corrected Visual Acuity, Slit-lamp, Ophthalmoscopy, Fundus Photography, Intraocular Pressure, Microperimetry, full-field static and kinetic perimetry, OCT and ERG
    • Numero di pazienti con eventi avversi emergenti dal trattamento.
    • Variazioni rispetto al basale nella valutazioni della sicurezza oculare
    • Misurazione della variazione rispetto al basale nella Miglior acuità visiva corretta, Esame con lampada a fessura, Oftalmoscopia, Fotografia del fondo oculare, Pressione intraoculare, Microperimetria, Perimetria statica e cinetica a campo pieno, Tomografia a coerenza ottica (OCT), Elettroretinogramma (ERG)
    E.5.1.1Timepoint(s) of evaluation of this end point
    at 48 weeks
    A 48 settimane
    E.5.2Secondary end point(s)
    • Delay in retinal degeneration
    • Measured as change from baseline in function relative to untreated
    contralateral eye on: BCVA, microperimetry, full-field static/kinetic
    perimetry, OCT and FAF
    • Rallentamento nella degenerazione della retina
    • Misurazione delle variazioni rispetto al basale nella funzione visiva e nella struttura retinica rispetto
    all’occhio controlaterale non trattato, utilizzando le seguenti tecniche di analisi retinica:
    - BCVA
    - Microperimetria
    - Perimetria statica a campo pieno
    - Perimetria cinetica a campo pieno
    - OCT
    - Autofluorescenza del fondo oculare (FAF)
    E.5.2.1Timepoint(s) of evaluation of this end point
    at 48 weeks
    A 48 settimane
    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 Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. 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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    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.8.4.1Number 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 EEA3
    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
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last visit last subject in the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years8
    E.8.9.2In all countries concerned by the trial months0
    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: 1
    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) Yes
    F. of subjects for this age range: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 28
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. of subjects incapable of giving consent
    Pediatric patients, needing parental consent
    Pazienti in età pediatrica per i quali è necessario il consenso dei genitori.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 46
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    At the end of the study, the patient will be invited to enter in an open-label safety study and long-term follow-up visits including ophthalmological examinations and recording of adverse events for up to 15 years.
    Al termine dello studio, il paziente verrà invitato ad entrare in uno studio di estensione a lungo termine al fine di effettuare le visite di follow-up inclusi gli accertamenti oftalmologici e al fine di raccogliere gli eventi avversi fino a 15 anni.
    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 Decision2018-03-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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