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    Summary
    EudraCT Number:2017-001730-26
    Sponsor's Protocol Code Number:205756
    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:2019-04-08
    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 number2017-001730-26
    A.3Full title of the trial
    A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA OTL-200, for the treatment of early onset Metachromatic Leukodystrophy (MLD).
    Studio clinico a singolo braccio, in aperto su cellule autologhe CD34+ trasdotte con vettore lentivirale contenente cDNA ARSA OTL-200 umano crioconservate, per il trattamento della leucodistrofia metacromatica (MLD_Metachromatic Leukodystrophy) a esordio precoce.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Gene therapy study using a frozen formulation of OTL-200 in patients with Metachromatic Leukodystrophy (MLD).
    Studio di terapia genica che utilizza una formulazione congelata di OTL-200 in pazienti con leucodistrofia metacromatica (MLD).
    A.3.2Name or abbreviated title of the trial where available
    A clinical study using cryopreserved OTL-200 for treatment of MLD.
    Studio clinico che utilizza OTL-200 in formulazione crioconservato per il trattamento di MLD.
    A.4.1Sponsor's protocol code number205756
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN12345678
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03392987
    A.5.3WHO Universal Trial Reference Number (UTRN)U1234-1234-1234
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/212/2018
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOrchard Therapeutics (Europe) Ltd
    B.1.3.4CountryUnited Kingdom
    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 supportOrchard Therapeutics (Europe) Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOrchard Therapeutics (Europe) Limited
    B.5.2Functional name of contact pointClinical
    B.5.3 Address:
    B.5.3.1Street Address108 Cannon Street
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeEC4N 6EU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+4402033846700
    B.5.5Fax number+4402037270797
    B.5.6E-mailclinical@orchard-tx.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/3/07/446
    D.3 Description of the IMP
    D.3.1Product nameOTL-200 Dispersion for Infusion
    D.3.2Product code [OTL-200]
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeOTL-200
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number2000000
    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.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 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
    Metachromatic Leukodystrophy
    leucodistrofia metacromatica
    E.1.1.1Medical condition in easily understood language
    Metachromatic Leukodystrophy, is an inherited genetic disease affecting mainly babies and young children, and more rarely also affecting adolescents and adults.
    Leucodistrofia metacromatica, è una malattia genetica ereditaria che colpisce principalmente lattanti e bambini, e più raramente colpisce anche gli adolescenti e gli adulti.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10067609
    E.1.2Term Metachromatic leukodystrophy
    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 evaluate the clinical efficacy of the cryopreserved formulation of OTL-200 (specific endpoint)
    Valutare l’efficacia clinica della formulazione crioconservata di OTL-200 (endopoint specifico)
    E.2.2Secondary objectives of the trial
    1. To evaluate the clinical efficacy of the cryopreserved formulation of OTL-200 (Other endpoints)
    2. To evaluate engraftment of the cryopreserved formulation of OTL-200
    3. To evaluate the pharmacodynamic effect of the cryopreserved formulation of OTL-200
    4. To evaluate the safety and tolerability of the cryopreserved formulation of OTL-200
    1.Valutare l’efficacia clinica della formulazione crioconservata di OTL-200 (altri end-points)
    2.Valutare l’attecchimento della formulazione crioconservata di OTL-200
    3.Valutare l’effetto farmacodinamico della formulazione crioconservata di OTL-200
    4.Valutare la sicurezza e la tollerabilità della formulazione crioconservata di OTL-200
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Documented biochemical and molecular diagnosis of MLD, based on ARSA activity below the normal range and identification of two diseasecausing ARSA alleles, either known or novel mutations. Novel mutations will be analyzed with in silico prediction tools and excluded from being known common polymorphisms. In the case of a novel mutation(s), a 24-hour urine collection must show elevated sulfatide levels.
    2) Eligible participants must have EITHER:
    a) an older sibling affected by MLD (index case), whose age of symptom onset was = 6 years of age (i.e. had not celebrated 7th birthday).
    Participants will be classified as Late Infantile, Early Juvenile or Intermediate LI/EJ based on age of symptom onset in the index case and their ARSA genotype:
    i. Late Infantile (LI): symptom onset in index case = 30 months of age; genotype typically 0/0
    ii. Early Juvenile (EJ): symptom onset in index case >30 months and = 6 years of age; genotype typically 0/R
    iii. Intermediate LI/EJ: symptom onset in index case = 6 years of age but unable to unambiguously characterize index case as LI or EJ
    OR
    b) If MLD is diagnosed in a pre-symptomatic child without an older affected sibling, (e.g, incidentally or via newborn screening) and the totality of the data available to the investigator strongly suggest that the patient has an early onset variant of MLD likely to benefit from gene therapy, and the patient is = 6 years of age (i.e. has not celebrated 7th birthday), the patient may be considered eligible after discussion and approval by the Orchard Therapeutics (Europe) medical monitor (Orchard- MM)
    3) Parental/guardian signed and dated informed consent
    1)Diagnosi biochimica e molecolare documentata di MLD, basata su attività di ARSA inferiore al range di normalità e identificazione di due alleli patogeni di ARSA, con mutazioni note o nuove. Le nuove mutazioni saranno analizzate con strumenti predittivi in silico e saranno esclusi i polimorfismi comuni noti.
    Nel caso di una o più nuove mutazioni, la raccolta di urine delle 24 ore deve mostrare livelli elevati di sulfatidi.
    2)I soggetti eleggibili devono presentare UNA delle seguenti caratteristiche:
    a)un/a fratello/sorella maggiore affetto/a da MLD (caso indice), con età di esordio dei sintomi = 6 anni (ossia, non aveva ancora compiuto 7 anni). I partecipanti saranno classificati in Tardo infantile, Giovanile precoce o Intermedio in base all’età di esordio dei sintomi nel caso indice e al loro genotipo ARSA:
    i.Infantile tardiva: esordio dei sintomi nel caso indice = 30 mesi di età; genotipo solitamente 0/0
    ii.Giovanile precoce: esordio dei sintomi nel caso indice >30 mesi e = 6 anni di età; genotipo solitamente 0/R
    iii.Intermedio: esordio dei sintomi nel caso indice = 6 anni di età ma non in grado di classificare in modo non ambiguo il caso indice come tardo infantile o giovanile precoce
    OPPURE
    b)Se la MLD è diagnosticata in un bambino pre-sintomatico che non ha fratelli o sorelle maggiori affetti/e (ad es. casualmente o mediante screening neonatale), e la totalità dei dati disponibili per lo sperimentatore suggerisce fortemente che il paziente presenti una variante di MLD esordio precoce che può trarre beneficio dalla terapia genica, e il paziente ha un’età = 6 anni (ossia, non ha ancora compiuto 7 anni), il paziente può essere considerato eleggibile dopo la discussione e l’approvazione da parte del MM (Orchard-MM) di Orchard Therapeutics (Europe).
    3)I genitori/tutore legale hanno firmato e datato il consenso informato
    E.4Principal exclusion criteria
    a) If LI MLD variant, clinical manifestations of the disease defined as EITHER one of the following:
    i. Delay in the achievement of independent standing or walking, together with abnormal signs at neurological evaluation
    OR
    ii. Documented neurological signs and symptoms of MLD associated with cognitive, motor or behavioral functional impairment or regression (substantiated by neurological exam and/or neuropsychological tests appropriate for age)
    b) If EJ MLD variant, symptoms of MLD resulting in the loss of capacity of walking independently as defined by a GMFC>2 or symptoms consistent with cognitive impairment as defined by IQ<85 using age-appropriate
    neurocognitive instruments
    The following will not be exclusionary if present alone:
    i.Seizures
    ii.Signs of the disease revealed at instrumental evaluations.
    2)Documented HIV infection
    3)Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome.Subjs with a prior successfully treated malignancy and a sufficient f-up to exclude recurrence can be included after discussion and approval by the MM.
    4)Myelodysplasia, cytogenetic alterations characteristic of MDS and AML or other serious haematological disorders.
    5)Pats currently enrolled in other interventional trials.
    6)Has previously undergone allogeneic hematopoietic stem cell transplantation and has evidence of residual cells of donor origin.
    7)Previous gene therapy.
    8)Has symptomatic herpes zoster, not responsive to specific treatment. Subjs with a recent history of herpes zoster may be included in the study. In such cases, inclusion, additional monitoring and treatment of the condition must be discussed and approved by the OrchMM.
    9)Evidence of TB based upon medical examination, chest imaging and TB testing i.e. QuantiFERON-TB Gold test and microbiological evidence. Subjs with latent TB, as documented by medical history and/or TB testing may be included in the study if receiving antibiotic prophylaxis (e.g. isoniazid). Inclusion, monitoring and treatment of TB in such subjects must be
    discussed and approved by the OrchMM.
    10)Acute or chronic stable Hepat B as evidenced by positive HBsAg test result at screening or within 3 months prior to onset of conditioning and/or posit HBV DNA.
    Subjs with posit Hepat B core antibody due to prior resolved disease may be enrolled, only if a confirmatory negat Hepat B surface antigen and negat Hepat B DNA test are obtained.
    Inclusion, monitoring and treatment of hepat in such subjs must be discussed and approved by the OrchMM.
    11)Presence of posit HepatC RNA test result at screening; Pats who have previously tested posit for antibodies against hepat C can be treated, provided they demonstrate absence of ongoing infection using a nucleic acid test with a limit of quantification of =15 UI/ml. Negat test results are required on at least 3 sequential occasions over a period of at least 4 weeks, after completion of treatment for hepatC, with the final test conducted no more than 3 days prior to cell harvest. Inclusion, monitoring and treatment of hepatitis in such subs must be discussed and approved by the OrchMM.
    12)End-organ dysfunction, severe active infection not responsive to treatment, or other severe disease or clinical condition which, in the judgment of the investigator, would make the subject inappropriate for entry into this study. In addition to the potential infections tested per protocol, the PI should consider testing for other transmissible infectious agents listed in the EU Cell and Tissue Directive as clinically appropriate and results discussed with the OrchMM prior to cell harvest. Subjects with ALT>2x upper limit of normal (ULN) or total bilirubin >1.5xULN may be included only after discussed and agreed with the MM and considered in the context of the criterion for excluding subjects with other severe disease.
    Isolated elevation of total bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35% of total.
    a) In caso di variante LI della LMD, manifestazioni cliniche della malattia definite come UNA delle seguenti:
    i.Ritardo nel raggiungimento autonomia posizione eretta o camminare, insieme a segni anomali in sede di valutz neurolog
    O
    ii)Segni e sintomi neurolog documentati di MLD associati a compromis o regres funzionale cognitiva, motoria o comportam (conf da esame neurologico e/o test neuropsicolog per età).
    b) In caso di variante EJ della LMD, sintom della LMD che risultano in perdita capacità di camminare in modo indip, come definito da un livello =2 della GMFC o sintomi coerenti con l’insuff cognitiva, definita da un punteggio IQ<85 valutato mediante strumenti neurocognitivi appropriati per età
    Le seguenti condiz non saranno causa di esclusio se presenti singolarmente:
    i.Crisi convulsive
    ii.Segni della patologia rivelati con valutazioni strumentali
    2)Infezione da HIV
    3)Neoplasia maligna (ad eccezione di cancro cutaneo a livello locale) o anamnesi docu di sindrome cancerosa ereditaria. I sog con neoplasia maligna precedente trattata con successo e f-up sufficiente a escludere una recidiva possono essere inclusi dopo la discussione e l'approvazione da parte del MM.
    4)Mielodisplasia, alterazioni citogenetiche caratteristiche della SMD e della LMA o altri disturbi ematologici gravi.
    5)Paz attualmente arruolati in altri studi clinici interventistici.
    6)Paz sottoposti in precedenza a trapianto allogenico di cellule staminali ematopoietiche e che presentano evidenza di cell residue di origine del donatore.
    7)Terapia genica precedente.
    8)Herpes zoster sintomatico che non risponde al trattamento specifico.
    9)Evidenza di TB attiva basata su visita medica,imaging del torace e test per la TB, ossia test QuantiFERON-TB Gold, ed evidenza microbiologica.
    I sog con TB latente, come documentata da anamnesi medica e/o test per la TB, possono essere inclusi nello studio se ricevono una profilassi antibiotica.L’inclusione, il monitorag e il trattamento della TB in tali sog devono essere discussi e approvati dal MM.
    10)Epati B acuta o cronica stabile come evidenziata dal risultato positivo del test per HBsAg allo screening o nei 3 mesi precedenti l’inizio del condizionamento e/o positività per il DNA di HBV.
    I sog con anticorpi pos per l’antigene core del virus dell’epat B a causa di una precedente patologia risolta possono essere arruolati soltanto se si ottiene un risultato negativo di conferma al test per l’antigene di superficie dell’epatite B o al test del DNA per il virus dell’epat B.Inclusione, monitoraggio e trattamento dell’epatite in tali sog devono essere discussi e approvati da OrchMM.
    11)Risultato positivo al test per la ricerca dell’RNA dell’epat C al momento dello screening;I paz che in precedenza sono risultati positivi agli anticorpi contro l’epat C possono essere trattati, purché dimostrino l’assenza di un’infezione in corso tramite un test dell'acido nucleico con un limite di quantificazione di =15 UI/ml.L’inclusione, il monitoraggio e il trattamento dell’epati in tali sog devono essere discussi e approvati dal MMOrch.
    12)Disfunzioni d’organo, infezione attiva grave che non risponde al trattamento, o altre patologie o condizioni cliniche gravi che, secondo il giudizio dello sperimentatore, renderebbero il sogg non idoneo all’ingresso in questo studio.
    Oltre alle potenziali infezioni testate secondo protocollo, il PI deve prendere in considerazione i test per altri agenti infettivi trasmissibili elencati nella Direttiva UE sulla donazione di tessuti e cellule umani, in modo adeguato dal punto di vista clinico, e i risultati devono essere discussi con il MMOrch prima della raccolta delle cellule.
    Sog con ALT >2x LSN o la bilirub tot >1,5x LSN possono essere inclusi soltanto previa discuss e approvaz del MMOrch e devono essere valutati nel contesto del criterio per l’esclusione di sog con altre pat gravi.
    L’aumento isolato della bilirub tot >1,5x LSN è accettabile se la bilirub è frazionata e la bilirub diretta è <35% del tot
    E.5 End points
    E.5.1Primary end point(s)
    GMFM score at 24 months post gene-therapy
    Punteggio GMFM (Gross Motor Function Measure) 24 mesi dopo la terapia genica
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months
    24 mesi
    E.5.2Secondary end point(s)
    GMFM score post gene-therapy at multiple visits over time
    •Clinical efficacy at 24 months post gene-therapy and multiple visits over time, as measured by:
    a. GMFC-MLD score
    b. Neurological Examinations
    c. Assessment of Nerve Conduction Velocity (NCV)
    d. Evaluation of Brain Magnetic Resonance (MR) imaging assessments / parameters (e.g. Modified Loes Score)
    e. Neurocognitive assessments %LV positive clonogenic progenitors in BM at Day 30 post-gene therapy and at multiple visits over time
    · VCN (in BM mononuclear cells) at Day 30 post-gene therapy and at multiple visits over time
    · VCN (in PB mononuclear cells) at Day 60 post-gene therapy and at multiple visits over time
    The following at Day 90 post-gene therapy and at multiple visits over time:
    · (i) ARSA activity in Total PBMCs
    · (ii) ARSA activity in PB CD15+ cells
    · (iii) ARSA activity in PB CD14+ cells
    · (iv) ARSA activity in CSF
    • Safety and tolerability as measured by adverse events (AEs) reporting;
    • Haematological recovery, defined as reconstitution of absolute neutrophil count (ANC) > 500 neutrophils/ µL, associated with evidence of BM recovery (i.e. no hypocellular marrow) by day +60
    • Incidence and titers of antibodies against ARSA
    • Absence of malignancy or abnormal clonal proliferation due to insertional oncogenesis
    • Absence of Replication Competent Lentivirus (RCL)
    •Punteggio GMFM dopo la terapia genica, in diverse visite nel corso del tempo;
    •Efficacia clinica 24 mesi dopo la terapia genica e in diverse visite nel corso dello studio, misurata mediante:
    a)Punteggio GMFC-MLD (Gross Motor Function Classification in MLD)
    b)Esami neurologici
    c)Valutazione della velocità di conduzione nervosa (NCV_Nerve conduction velocity)
    d)Valutazione degli immagini/ parametri di Risonanza magnetica (RM) cerebrali (ad es. punteggio di Loes modificato)
    e)Valutazioni neurocognitive
    •%LV (lentiviral vector) di progenitori clonogenici positivi nel midollo osseo al Giorno 30 successivo alla terapia genica e in diverse visite nel corso del tempo
    •VCN (vector copy number) (in cellule mononucleate del midollo osseo) al Giorno 30 successivo alla terapia genica e in diverse visite nel corso del tempo
    •VCN (in cellule mononucleate del sangue periferico) al Giorno 60 successivo alla terapia genica e in diverse visite nel corso del tempo
    •I fattori seguenti saranno valutati al Giorno 60 successivo alla terapia genica e in diverse visite nel corso dello studio:
    i)Attività di ARSA (Arilsulfatasi A) nelle PBMC (pheripheral blood mononuclear cell) totali
    ii)Attività di ARSA nelle cellule CD15+ del sangue periferico
    iii)Attività di ARSA nelle cellule CD14+ del sangue periferico
    iv)Attività di ARSA nel liquido cerebrospinale
    •Sicurezza e tollerabilità misurate tramite la segnalazione di eventi avversi (EA);
    •Recupero ematologico, definito come ricostituzione della conta assoluta dei neutrofili (CAN) > 500 neutrofili/L, associato a evidenza di recupero del midollo osseo (ossia assenza di ipocellularità midollare) entro il Giorno +60
    •Incidenza e titoli degli anticorpi contro ARSA
    •Assenza di neoplasia maligna o proliferazione clonale anomala dovuta a oncogenesi inserzionale
    •Assenza di lentivirus competente per la replicazione (RCL)
    E.5.2.1Timepoint(s) of evaluation of this end point
    various
    vario
    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 No
    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 No
    E.6.11Pharmacogenomic Yes
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    singolo braccio, in aperto
    A single arm, open label
    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.8.2.4Number of treatment arms in the trial1
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    Ultima visita dell'ultimo paziente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    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.1.1.1In 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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 4
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    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 No
    F.3.3.7Others No
    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 10
    F.4.2.2In the whole clinical trial 10
    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)
    Due to the inherent nature of the gene therapy being a once-only treatment, there is no plan to offer further treatment / doses of OTL-200 following the end of the study.
    A causa della natura intrinseca della terapia genica che consiste in una monosomministrazione unica, non vi è alcun piano per offrire ulteriori trattamenti / dosi di OTL-200 a seguito della fine dello studio.
    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 Decision2017-11-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-09-14
    P. End of Trial
    P.End of Trial StatusOngoing
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