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    Summary
    EudraCT Number:2016-001446-25
    Sponsor's Protocol Code Number:FHGT002
    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:2021-05-06
    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 number2016-001446-25
    A.3Full title of the trial
    AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH)
    Sostituzione del gene che codifica per il recettore delle lipoproteine a bassa densità AAV8-mediata in soggetti affetti da ipercolesterolemia familiare omozigote
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH).
    Questo è uno studio che mira a valutare se la terapia genica è sicura in persone con ipercolesterolemia familiare omozigote (anche chiamata HoFH)
    A.4.1Sponsor's protocol code numberFHGT002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02651675
    A.5.4Other Identifiers
    Name:IND #Number:16710
    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 SponsorREGENXBIO 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 supportDHHS/NHLB
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportREGENXBIO Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationREGENXBIO Inc.
    B.5.2Functional name of contact pointLisa Freeman
    B.5.3 Address:
    B.5.3.1Street Address9600 Blackwell Road, Suite 210
    B.5.3.2Town/ cityRockville
    B.5.3.3Post codeMD 20850
    B.5.3.4CountryUnited States
    B.5.4Telephone number+12405528181
    B.5.6E-maillfreeman@regenxbio.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 nameAAV8.TBG.hLDLR
    D.3.2Product code AAV8.TBG.hLDLR
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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) Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberProduct reference H0004905 Report: EMA/CAT/652909/2016
    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.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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameprednisone
    D.3.2Product code prednisone
    D.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Adults with homozygous familial hypercholesterolemia
    Adulti con ipercolesterolemia familiare omozigote
    E.1.1.1Medical condition in easily understood language
    High cholesterol levels in subjects with homozygous familial hypercholesterolemia due to mutation in LDL receptor
    livelli elevati di colesterolo in soggetti con ipercolesterolemia familiare omozigote dovute a mutazioni nel recettore LDL
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective will be to determine the safety of AAV8.TBG.hLDLR administration in this patient population.
    L’obiettivo primario è determinare il profilo di sicurezza di AAV8.TBG.hLDLR quando somministrato a questi pazienti
    E.2.2Secondary objectives of the trial
    The key secondary objective is to assess the efficacy of LDL-C reduction achieved with AAV8.TBG.hLDLR administration.
    Obiettivi secondari: L’obiettivo secondario principale è valutare l’efficacia nella riduzione delle LDL-C a seguito della somministrazione di AAV8.TBG.hLDLR
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH): Optional LDL Kinetics Substudy.

    Description: The purpose of this optional LDL Kinetics Substudy is to see if the study drug helps clear the LDL (the "bad" cholesterol) from circulation. To do this, LDL will be labeled with a "cold tracer" called deuterated leucine. LDL will be tagged with this tracer so we can use laboratory tests to look at how it behaves in your blood.
    Sostituzione del gene che codifica per il recettore delle lipoproteine a bassa densità AAV8-mediata in soggetti affetti da ipercolesterolemia familiare omozigote – sottostudio opzionale di cinetica.
    L’obiettivo di questo studio opzionale di cinetica delle LDL è verificare se il farmaco in studio aiuta a ridurre i livelli di LDL (il colesterolo “cattivo”) nel sangue. A tal fine, le LDL saranno marcate con un tracciante freddo chiamato leucina deuterata. Le LDL verranno quindi marcate con questo tracciante e questo permetterà, tramite test di laboratorio, di vedere come tali LDL si comportano nel sangue dei pazienti.
    E.3Principal inclusion criteria
    1. Male or female ≥ 18 years of age.
    2. Untreated and/or treated LDL-C levels and clinical presentation consistent with the diagnosis of homozygous FH
    3. Molecularly defined LDLR mutations at both LDLR alleles.
    4. Concurrent allowed lipid lowering medication must be stable for ≥ 4 weeks before the baseline visit and must remain stable until 18 weeks after vector administration. (or 4 weeks post steroid termination). These include but are not limited to: statins, ezetimibe, bile acid sequestrants, PCSK9 inhibitors, and LDL and/or plasma apheresis. Subjects on other lipid- lowering medications are eligible for the study but must wash out of these medications for the pre-specified time period.
    5. Females of childbearing potential must have a negative pregnancy test at screening and baseline visits and be willing to have additional pregnancy tests during the study.
    6. Sexually active subjects (both female and male) must be willing to use a medically accepted method of contraception from screening visit until 6 months after vector administration
    7. A baseline serum AAV8 NAb titer ≤ 1:10.
    8. Subjects must be able to comprehend and be willing to provide a signed institutional review board/ethics committee (IRB/EC) approved Informed Consent Form. (ICF).
    9. Subjects must be willing to comply with all study-related procedures and be available for the duration of the study.
    1. Uomini o donne di età >= 18 anni
    2. Livelli di LDL-C trattato e/o non trattato e quadro clinico compatibile con una diagnosi di HoFH
    3. mutazioni LDLR definite da un punto di vista molecolare in entrambi gli alleli
    4. Terapie ipolipidemizzanti concomitanti e consentite devono essere stabili per >= 4 settimane prima della visita basale e devono rimanere stabili fino a 18 settimane dopo la somministrazione del vettore. (o 4 settimane dal termine del trattamento con corticosteroidi) Questi includono, ma non si limitano a: -statine, ezetimibe, sequestranti degli acidi biliari, inibitori di PCSK9 e LDL e/o plasma aferesi. Pazienti con altre terapie ipolipidemizzanti sono eleggibili per lo studio ma devono interrompere l’assunzione di tali farmaci per un periodo di tempo predefinito.
    5.Donne potenzialmente fertili devono avere un test di gravidanza negativo allo screening ed alla visita basale ed acconsentire ad ulteriori test di gravidanza durante lo studio.
    6. Soggetti sessualmente attivi (uomini e donne) devono acconsentire ad utilizzare un metodo di contraccezione medicalmente accettato dalla visita di screening fino a 6 mesi successivi alla somministrazione del vettore.
    7. Titolo anticorpale basale neutralizzante AAV8 nel siero <= 1:10
    8. I soggetti devono essere in grado di recepire ed accettare di firmare il modulo di consenso informato approvato dall’IRB/EC
    9. I soggetti devono acconsentire ad attenersi a tutte le procedure di studio e ad essere disponibili per la durata dello studio.
    E.4Principal exclusion criteria
    1. Unwilling to wash out of the following lipid lowering therapies for the pre-specified time period:
    niacin > 250 mg/day: within 6 weeks of baseline
    fibrates: within 4 weeks of baseline
    lomitapide: within 8 weeks of baseline
    mipomersen: within 24 weeks of baseline
    2. Heart failure defined by the NYHA classification as functional Class III with history of hospitalization(s) within 12 weeks of the baseline visit or functional Class IV.
    3. History within 12 weeks of the baseline visit of a myocardial infarction (MI), unstable angina leading to hospitalization, coronary artery bypass graft surgery (CABG), percutaneous coronary intervention (PCI), uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, carotid revascularization, endovascular procedure or surgical intervention.
    4. Uncontrolled hypertension defined as: systolic blood pressure > 180 mmHg, diastolic blood pressure > 95 mmHg.
    5. Uncontrolled diabetes defined as HbA1c > 8.5% or an average fasting glucose ≥ 160 mg/dl..
    6. Known hypersensitivity to prednisone.
    7. History of cirrhosis or chronic liver disease based on documented histological evaluation or non- invasive imaging or testing.
    8. Documented diagnosis of any of the following liver diseases:
    Nonalcoholic steatohepatitis (biopsy-proven)
    Alcoholic liver disease
    Autoimmune hepatitis
    Liver cancer
    Primary biliary cirrhosis
    Primary sclerosing cholangitis
    Wilson’s disease
    Hemochromatosis
    α1 anti-trypsin deficiency
    9. Abnormal liver function tests (LFTs) at screening (AST or ALT > 2 x upper limit of normal (ULN) and/or Total Bilirubin of >1.5x ULN unless patient has unconjugated hyperbilirubinemia due to Gilbert’s syndrome).
    10. Hepatitis B as defined by positive for HepB SAg, or Hep B Core Ab, and/or viral DNA
    11. Chronic active Hepatitis C as defined by positive for HCV Ab and viral RNA.
    12. History of chronic alcohol abuse within 52 weeks of the screening visit.
    13. Certain prohibited medications known to be potentially hepatotoxic, especially those that can induce microvesicular or macrovesicular steatosis. These include but are not limited to: Accutane (isotretinoin), amiodarone, HAART medications, heavy acetaminophen use (2 g/day more than 3 times a week), isoniazid, methotrexate, tetracyclines, tamoxifen, valproate.
    14. Active tuberculosis, systemic fungal disease, or other chronic infection.
    15. History of immunodeficiency diseases, including a positive HIV test result.
    16. Chronic renal insufficiency defined as estimated GFR < 30 mL/min/1.73m2.
    17. History of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer.
    18. Previous organ transplantation.
    19. Administration of an investigational drug within 12 weeks or 5 half-lives of the drug (whichever is longer) prior to the screening visit and until 52 weeks after receiving AAV8.TBG.hLDLR. Subjects are not prohibited from receiving investigational drugs after 52 weeks.
    20. Any major surgical procedure occurring less than 3 months prior to the screening visit, or any planned future surgical procedure within 3 months of baseline.
    21. Serious or unstable medical or psychological conditions that, in the opinion of the investigator, would compromise the subject’s safety or successful participation in the study.
    22. Any other medical condition or finding that would make it not in the subject’s best interest to participate in the study
    23. Study staff member or any direct family member.
    1. Rifiuto di interrompere i seguenti agenti ipolipidemizzanti per il periodo specificato:
    -niacina > 250 mg/gg entro 6 settimane dalla prima visita
    -fibrati, 4 settimane dalla prima visita
    -lomitapide, 8 settimane dalla prima visita
    -mipomersen, 24 settimane dalla prima visita
    2. Problemi cardiaci definiti dalla classificazione NYHA come Classe III funzionali con storia pregressa di ospedalizzazioni entro le 12 settimane dalla prima visita o funzionale di classe IV
    3. Infarto miocardico entro le 12 settimane dalla prima visita, angina instabile che richiede ospedalizzazione, bypass aorto-coronarico, angioplastica coronarica, aritmia cardiaca incontrollata, intervento alla carotide o applicazione stent, infarto, attacco ischemico transitorio, rivascolarizzazione della carotide, procedure endovascolari o interventi chirurgici.
    4. Ipertensione non controllata definita come: pressione sanguigna sistolica > 180 mmHg, pressione sanguigna diastolica > 95 mmHg.
    5. Diabete non controllato definito come HbA1c > 8.5% o come glucosio medio a digiuno >= 160 mg/dL
    6. Nota ipersensibilità al prednisone
    7. Pregressa cirrosi o cirrosi epatica cronica basata su valutazioni istologiche, imaging non invasivo o test.
    8. Diagnosi documentata di una qualsiasi delle seguenti malattie:
    a. steatoepatite non alcolica (confermata da biopsia)
    b. epatopatia alcolica
    c. epatite autoimmune
    d. tumore al fegato
    e. cirrosi delle vie biliari primarie
    f. colangite sclerosante primitiva
    g. malattia di Wilson
    h. emocromatosi
    i. deficit di Alfa-1-antitripsina
    9. LFTs anomali allo screening (AST o ALT >2X oltre il limite normale) e/o bilirubina totale > 1.5x UNL a meno che il paziente non abbia iperbilirubinemia non coniugata a seguito di sindrome di Gilbert
    10. Epatite b definite come test positivo per HepB SAg, o Hep B core Ab e/o DNA virale
    11. Epatite c cronica attiva definita come test positivo per anticorpi contro HCV e RNA virale
    12. Storia di abuso di alcool cronico entro 52 settimane dalla visita basale
    13. Farmaci non consentiti con riconosciuto potenziale epatotossico, in particolare quelli in grado di indurre steatosi micro e macrovescicolare. Questi includono ma non sono limitati a. Accutane (Isotretinoina), amiodarone, farmaci HAART, utilizzo consistente di acetaminofene (2g/gg più di 3 volte a settimana), isoniazide, metotrexato, tetracicline, tamoxifene, acido valproico.
    14. Tubercolosi attiva, malattia fungina sistemica o altre infezioni croniche.
    15. Storia di malattie da immunodeficienza, incluso un risultato positivo al test HIV
    16. insufficienza renale cronica definita come GFR < 30 ml/min/1.73m2
    17. Cancro entro i precedenti 5 anni, ad eccezione di cancro della pelle a cellule basali adeguatamente trattato, cancro alla pelle a cellule squamose, o tumore in situ cervicale.
    18. Precedente trapianto d’organo
    19. Somministrazione di un farmaco sperimentale entro 12 settimane o 5 emivite del farmaco (a seconda di quale periodo sia più lungo) prima dello screening e fino a 52 settimane dopo aver ricevuto AAV8.TBG.hLDLR. I soggetti possono ricevere farmaci sperimentali 52 settimane dopo aver ricevuto AAV8.TBG.hLDLR.
    20. Nessun intervento chirurgico maggiore entro 3 mesi dalla visita di screening, o nessun intervento chirurgico pianificato entro 3 mesi dalla visita basale.
    21. Condizioni mediche o psicologiche serie o instabili che, a giudizio dello sperimentatore, potrebbero compromettere la sicurezza del paziente o la sua corretta partecipazione allo studio.
    22. Qualsiasi altra condizione medica o problema che comporterebbe non essere nel miglior interesse del paziente partecipare allo studio
    23. Membro o familiare diretto di un membro dello staff di studio
    E.5 End points
    E.5.1Primary end point(s)
    Safety assessments up to 24 weeks post vector administration
    Determinare la sicurezza del vettore fino a 24 settimane successive alla somministrazione del vettore
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety assessments up to the primary endpoint:
    Days 7, 14, 21, 28, 35, 42, 49, 56, 63, 70, 77, 84
    Week 14, 16, 18, 20, 22, 24
    Le visite per la sicurezza relative all’endpoint primario avverrano ai giorni 7, 14,21, 28, 35, 42, 49, 56, 63, 70, 77, 84, e alla settimana 14, 16, 18, 20 22, 24.
    E.5.2Secondary end point(s)
    - To assess the LDL-C reduction achieved with AAV8.TBG.hLDLR administration as defined by percent change in LDL-C at Week 12 (Cohort 1 only) or Week 18.
    - To assess changes in other lipid parameters at Week 12 (Cohort 1 only) or Week 18 compared to baseline values, specifically percent change in total cholesterol (TC), non-high density lipoprotein cholesterol (non-HDL-C), HDL-C, fasting triglycerides (TG), very low density lipoprotein cholesterol (VLDL-C), lipoprotein(a) (Lp(a)), apolipoprotein B (apoB), and apolipoprotein A-I (apoA-I).
    - To assess safety up to 104 weeks.
    - To assess vector shedding in plasma and urine.
    - Determinare la riduzione delle LDL-C ottenuta con la somministrazione di AAV8.TBG.hLDLR definita come cambio percentuale nelle LDL-C a 12 settimane rispetto al basale (solo per la coorte 1) o alla settimana 18.
    - Determinare i cambiamenti di altri parametri lipidici a 12 settimane comparati ai valori al basale (solo per la coorte 1) o alla settimana 18, specialmente in riferimento alla percentuale di colesterolo totale, colesterolo legato alle lipoproteine non ad alta densità (non HDL-C), HDL-C, trigliceridi a digiuno (TG), colesterolo legato alle lipoproteine a densità molto bassa (VLDL-C), lipoproteina a (Lp(a)), apolipoproteina B (apoB), apolipoproteina A.I (apoA-I)
    - Determinare la sicurezza a lungo termine (fino a 104 settimane)
    - Determinare il rilascio del vettore nel plasma o nelle urine
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety endpoints after primary endpoint:
    Week 30, 36, 52, 78, 104

    Efficacy timepoints:
    Day 14, 21, 28, 35, 42, 49, 56, 63, 70, 77, 84
    Week 14, 16, 18, 20, 22, 24, 30, 36, 52, 78, 104
    Endpoint di sicurezza: settimana 30, 36, 52, 78, 104
    Endpoint di efficacia. Giorno 14,21, 28, 35, 42, 49, 56, 63, 70, 77, 84 e alla settimana 14, 16, 18, 20 22, 24, 30, 36, 52, 78, 104.
    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 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.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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 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.8.2.4Number of treatment arms in the trial3
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Canada
    Italy
    Netherlands
    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
    September 2022
    Settembre 2022
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months9
    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: 12
    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 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 12
    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
    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 Decision2017-10-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-25
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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