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    Summary
    EudraCT Number:2015-005761-23
    Sponsor's Protocol Code Number:CTD-TCNPC-201
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-09-07
    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 number2015-005761-23
    A.3Full title of the trial
    A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes
    Studio di fase I/II per valutare la sicurezza e la farmacocinetica di Trappsol¿ Cyclo ¿ (HP-ß-CD) per via endovenosa in pazienti con malattia di Niemann-Pick di tipo C (NPC-1) e gli effetti farmacodinamici del trattamento sui marcatori del metabolismo del colesterolo e sugli esiti clinici
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Niemann-Pick disease type C (NPC-1) to assess what the drug does to the body, and what the body does to the drug, and the side effects and benefits experienced by patients
    Studio di fase I/II per la valutazione di Trappsol¿ Cyclo ¿ (hydroxypropyl-ß-cyclodextrin) in pazienti con malattia di Niemann-Pick di tipo C (NPC-1) per verificare l'azione del farmaco e i suoi effetti collaterali e i relativi benefici sui pazienti
    A.3.2Name or abbreviated title of the trial where available
    A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Nieman
    Studio di fase I/II per la valutazione di Trappsol¿ Cyclo ¿ (hydroxypropyl-ß-cyclodextrin) in pazie
    A.4.1Sponsor's protocol code numberCTD-TCNPC-201
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN12345678
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT12345678
    A.5.3WHO Universal Trial Reference Number (UTRN)U1234-1234-1234
    A.5.4Other Identifiers
    Name:CTD-TCNPC-201Number:CTD-TCNPC-201
    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 SponsorCTD HOLDINGS, 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 supportCTD HOLDINGS, INC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCTD Holdings, Inc
    B.5.2Functional name of contact pointSenior VP for Medical Affairs
    B.5.3 Address:
    B.5.3.1Street Address14120 NW 126th Terrace
    B.5.3.2Town/ cityAlachua
    B.5.3.3Post codeFL32615
    B.5.3.4CountryUnited States
    B.5.4Telephone number0
    B.5.5Fax number0
    B.5.6E-mailsharon.hrynkow@cyclodex.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/3/11/895
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Solution for 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 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
    Niemann-Pick disease type C
    Malattia di Niemann-Pick di tipo C
    E.1.1.1Medical condition in easily understood language
    A rare, genetically inherited and neurologically progressive disease in which fatty substances accumulate in the brain and other major organs
    Una malattia rara di tipo genetico
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10029403
    E.1.2Term Niemann-Pick 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
    Stage 1
    • To compare the plasma pharmacokinetics of hydroxypropyl-ß-cyclodextrin following 3 different single doses of intravenous Trappsol Cyclo in patients with NPC-1

    Stage 2
    • To evaluate the efficacy and tolerability of 3 different doses of Trappsol Cyclo in the management of clinical manifestations of NPC-1
    Stadio 1
    • Confrontare la farmacocinetica plasmatica di HP-ß-CD dopo 3 diverse dosi singole di Trappsol¿ per via endovenosa in pazienti affetti da NPC-1

    Stadio 2
    • Valutare l'efficacia e la tollerabilità di 3 diverse dosi di Trappsol¿ nel trattamento delle manifestazioni cliniche della NPC-1
    E.2.2Secondary objectives of the trial
    Stage 1
    • To investigate the effect of 3 different doses of intravenous Trappsol Cyclo in patients upon serum and lymphocytic markers of cholesterol metabolism in patients with NPC-1
    • To evaluate hydroxypropyl-ß-cyclodextrin concentrations in cerebrospinal fluid following intravenous administration of Trappsol Cyclo in patients with NPC-1

    Stage 2
    • To investigate the effect of 3 different doses of intravenous Trappsol Cyclo upon serum and lymphocytic markers of cholesterol metabolism in patients with NPC-1
    • To evaluate the impact of treatment upon measures of neurological function including ataxia, cognitive impairment, fine motor skills and saccadic eye movements in patients with NPC-1
    • To evaluate the impact of treatment upon behavioural aspects of NPC-1 disease
    Stadio 1
    Secondari
    •Investigare l'effetto di 3 diverse dosi di Trappsol¿ per via endovenosa sui marcatori sierici e linfocitici del metabolismo del colesterolo in pazienti affetti da NPC-1
    • Valutare le concentrazioni di HP-ß-CD nel liquido cerebrospinale (CSF) dopo la somministrazione endovenosa di Trappsol¿ in pazienti affetti da NPC-1

    Stadio 2
    • Investigare l'effetto di 3 diverse dosi di Trappsol¿ per via endovenosa sui marcatori sierici e linfocitici del metabolismo del colesterolo in pazienti affetti da NPC-1
    • Valutare l'impatto del trattamento sulle misurazioni della funzione neurologica comprese atassia, compromissione cognitiva, abilità motorie fini e movimenti oculari saccadici nei pazienti con NPC-1
    • Valutare l'impatto del trattamento sugli aspetti comportamentali della malattia NPC-1
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Confirmed diagnosis of NPC-1 defined as one of the following:
    a) Two NPC-1 mutations on genotyping
    b) One NPC-1 mutation and positive filipin staining (current or prior)
    c) Vertical supranuclear gaze palsy [VSNGP] plus either = one NPC-1 mutation or positive filipin staining and no NPC-2 mutations
    2. NIH NPC Severity Score <30 and with no more than 4 individual domains with a score = 3.
    3. Age range: 2 years upwards
    a) Inclusion of the first six patients will be restricted to individuals aged = 5 years. In the event that three patients aged >18 (i.e. adults) are recruited before the total recruitment has reached n=6, no more adults will be randomised to treatment until the first cohort of 6 patients is complete. Once the first six are recruited and randomised, study entry will be open to all ages =2 years as per the protocol.
    4. Negative pregnancy test for females of child bearing potential
    5. Written, informed consent
    1.Diagnosi confermata di NPC-1 definita come una delle seguenti condizioni
    a.Due mutazioni NPC-1 alla genotipizzazione
    b.Una mutazione NPC-1 e colorazione filipin positiva (attuale o precedente)
    c.Paralisi sopranucleare verticale dello sguardo [VSGP] oltre ad una o più mutazioni NPC-1, oppure colorazione filipin positiva e nessuna mutazione NPC-2
    2.Punteggio NIH di gravità della NPC <30 e con non più di 4 domini individuali con un punteggio = 3.
    3.Intervallo di età: a partire da 2 anni
    a.L'inclusione dei primi sei pazienti sarà ristretta ad individui con età = 5 anni. Nel caso in cui siano arruolati tre pazienti con età >18 anni (cioè adulti) prima che l'arruolamento totale abbia raggiunto n=6, non saranno randomizzati al trattamento altri adulti fino a quando la prima coorte di sei pazienti sia completa. Quando i primi sei hanno completato in modo sicuro lo stadio 1, l'ingresso nello studio sarà aperto a tutte le età =2 anni, come da protocollo.
    4.Test di gravidanza negativo per tutte le pazienti di sesso femminile in età fertile.
    5.Consenso informato scritto
    E.4Principal exclusion criteria
    1. The presence of NPC-2 mutations on genotyping
    2. Previous receipt of cyclodextrin therapy
    3. Lanksy score < 50 if aged =16 or Karnofsky score < 40 if aged > 16.
    4. Inability to comply with the proposed protocol assessments
    5. Concurrent treatment with any type of cholesterol lowering agents such as statins, fibrates, ezetimibe
    6. Concurrent medical conditions representing a contraindication to any of the study medications
    7. Stage 3 chronic kidney disease (CKD) or worse as indicated by an eGFR < 60 mL/min/1.73 m2. In patients aged = 18 years, eGFR is calculated according to the Schwartz equation (ref: Schwartz GJ & Work DF) and in patients aged > 18 years eGFR is calculated using the MDRD equation
    8. Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalised ratio (INR) >1. 8
    9. Involvement in another interventional clinical trial within the previous 6 months from screening
    10. Weight >100 kg
    11. Females of childbearing potential who are not willing to use a method of highly effective contraception (hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner, or sexual abstinence) during the study
    1.Presenza di mutazioni NPC-2 alla genotipizzazione
    2.Aver ricevuto una precedente terapia con ciclodestrina
    3.Punteggio Lanksy < 50 per età = 16 anni o punteggio Karnofsky < 40 per età > 16.
    4.Incapacità di aderire alle valutazioni proposte nel protocollo.
    5.Trattamento corrente con qualsiasi terapia indicata per abbassare il colesterolo come statine, fibrati, ezetimibe (Sezione 4.4.2)
    6.Condizioni mediche concomitanti che rappresentano una controindicazione per uno qualsiasi dei medicinali in studio
    7.Malattia renale cronica di stadio 3 o peggiore come indicato da un eGFR< 60 mL/min/1,73 m2. In pazienti di età = 18 anni l’eGFR è calcolato secondo l’equazione di Schwartz (rif: Schwartz GJ & Work DF) e in pazienti di età > 18 anni l’eGFR è calcolato usando l'equazione MDRD
    8.Evidenza clinica di malattia epatica acuta compresi sintomi di ittero o dolore nel quadrante destro superiore o INR > 1,8.
    9.Coinvolgimento in un altro studio clinico interventistico nei 6 mesi precedenti allo screening
    10.Peso > 100 kg
    11.Donne in età fertile che non vogliono usare durante lo studio un metodo contraccettivo altamente efficace (contraccezione ormonale, dispositivo intrauterino, sistema intrauterino a rilascio ormonale, occlusione bilaterale delle tube, partner vasectomizzato o astinenza reale) nel periodo dello studio e del follow-up. L’astinenza sessuale può solo essere in linea con lo stile di vita preferito e abituale del soggetto. L’astinenza periodica (ad es. metodi del calendario, ovulazione, temperatura basale, post-ovulazione), la dichiarazione di astinenza per la durata della sperimentazione e il coitus interruptus non sono metodi contraccettivi accettabili.
    12.Donne che allattano con latte materno
    E.5 End points
    E.5.1Primary end point(s)
    Stage 1
    • Plasma concentrations of HP-ß-CD during and following infusion to
    evaluate Tmax, Cmax, volume of distribution and elimination half-life (at
    0, 2, 4, 6 and 8 hours after the start of infusion and 30 minutes, 1, 2, 4, 8
    and 12 hours after the end of the infusion)
    Stage 2
    • Change from baseline in global impression of disease severity at 48 weeks
    • The proportion of patients at 48 weeks with a reduction from baseline of at least one point in two or more domains of the NIH NPC severity scale
    Stadio 1
    Endpoint primario
    • Concentrazione plasmatica di HP-ß-CD durante e dopo l'infusione di Trappsol¿ per valutare la Tmax, Cmax, il Volume di distribuzione e l'emivita di eliminazione.
    - I campioni saranno raccolti a 0, 2, 4, 6 e 8 ore dopo l'inizio dell'inizio dell'infusione. Ulteriori campioni saranno prelevati alla fine dell'infusione e quindi 0,5, 1, 2, 4, 8 e 12 ore dopo
    Stadio 2
    Endpoint co-primari di efficacia
    • Cambiamento dal basale nell’impressione globale della gravità della malattia a 48 settimane
    • La proporzione di pazienti a 48 settimane con una riduzione dal basale di almeno un punto in due o più domini della scala HIH di gravità della NCP
    E.5.1.1Timepoint(s) of evaluation of this end point
    See Section E.5.1 of the protocol
    Sezione E.5.1 del protocollo
    E.5.2Secondary end point(s)
    Stage 1
    • Change from baseline in serum cholesterol precursors (lanosterol,
    lathosterol, desmosterol) and cholesterol metabolites/bile acid precursors (4b-, 24S-, 25-, 27- hydroxycholesterol) (days 2, 3, 5, 8, and
    15 post-dose in Stage 1)
    • Change from baseline in markers of lipid trafficking in peripheral blood mononuclear cells (PBMCs) (at day 15 post-dose in Stage 1)
    • CSF concentrations of HP-ß-CD at 4, 8 and 12 hours following the start of the intravenous administration (Stage 1)
    • AEs, laboratory abnormalities, time to withdrawal due to AE (every
    visit)
    Stage 2
    • Change from baseline in serum cholesterol precursors (lanosterol, lathosterol, desmosterol) and cholesterol metabolites/bile acid precursors (4b-, 24S-, 25-, 27- hydroxycholesterol) (4, 6, 8, 10, 12, 16, 20, 24, 28, 32, 36, 40, 44 and 48 weeks)
    • Change from baseline in markers of lipid trafficking in peripheral blood mononuclear cells (PBMCs) (12, 24, 36 and 48 weeks)
    • Change from baseline in NIH NPC severity scale at 12, 24, 36 and 48 weeks
    • Change from baseline in individual NIH NPC scale domains at 12, 24, 36 and 48 weeks
    • Change from baseline in neurologic symptoms at 12, 24, 36 and 48 weeks
    - ataxia (using the Scale for the assessment and rating of ataxia [SARA] scale)
    - cognitive impairment (using the Mini-Mental State Evaluation score [MMSE])
    - saccadic eye movements
    - fine motor skills (using the bead threading test)
    • Change from baseline in hepatic, splenic and renal morphology (abdominal ultrasound) at 24 and 48 weeks
    • Change from baseline in quality of life using the PedsQL (Pediatric Quality of Life Inventory) at 12, 24, 36 and 48 weeks
    • AEs, laboratory abnormalities, time to withdrawal due to AE (every visit)
    • Auditory acuity assessment (weeks 12, 24, 36, 48 and follow-up)
    Stadio 1
    •Cambiamento dal basale nei precursori sierici del colesterolo (lanosterolo, latosterolo, desmosterolo) e dei metaboliti del colesterolo/precursori dell'acido biliare (4b-, 24S-, 25-, 27- idrossicolesterolo) nei giorni 2, 3, 5, 8 e 15 post-dose
    •Cambiamento dal basale nei marcatori del traffico lipidico nelle cellule mononucleate del sangue periferico (PBMC) il giorno 15 dopo la dose (W2)
    •Concentrazioni nel CSF di HP-ß-CD a 4, 8 e 12 ore dopo l'inizio della somministrazione endovenosa
    Stadio 2
    •Cambiamento dal basale nei precursori sierici del colesterolo (lanosterolo, latosterolo, desmosterolo) e dei metaboliti del colesterolo/precursori dell'acido biliare (4b-, 24S-, 25-, 27- idrossicolesterolo) a 4, 6, 8, 10, 12, 16, 20, 24, 28, 32, 36, 40, 44 e 48 settimane
    • Cambiamento dal basale nei marcatori del traffico lipidico nelle cellule mononucleate del sangue periferico (PBMC) a 12, 24, 36 e 48 settimane
    • Cambiamento dal basale nella scala NIH di gravità della NPC a 12, 24, 36 e 48 settimane
    • Cambiamento dal basale nella scala individuale NIH della NPC a 12, 24, 36 e 48 settimane
    • Cambiamento dal basale dei sintomi neurologici a 12, 24, 36 e 48 settimane
    -atassia (usando la scala SARA)
    -compromissione cognitiva (usando il punteggio di valutazione dello stato mentale Mini-Mental State Evaluation)
    -movimenti oculari saccadici
    -capacità motorie fini (usando il test di infilare perline)
    • Cambiamento dal basale della morfologia epatica, splenica e renale (ecografia addominale) a 24 e 48 settimane
    • Cambiamento dal basale nella qualità della vita usando il questionario PedsQL (Pediatric Quality of Life Inventory) a 12, 24, 36 e 48 settimane
    Sicurezza (Stadi 1 e 2)
    • AE, anomalie di laboratorio, tempo al ritiro a causa di AE
    • Valutazione di acutezza uditiva
    E.5.2.1Timepoint(s) of evaluation of this end point
    See Section E.5.2
    Sezione E.5.2 del protocollo
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Trappsol Cyclo has previously been administered in a compassionate use/named patient programme.
    Trappsol Cyclo è stato precedentemente somministrato in un programma compassionevole use/named patie
    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 Yes
    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 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 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 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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 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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    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.3.3.6.1Details of subjects incapable of giving consent
    The nature of the disease under study means that some potential adult participants may have impaired capacity to make informed consent decisions or the participants may be too young to provide informed assent.
    La natura della malattia oggetto dello studio significa che alcuni potenziali partecipanti adulti possono aver alterate capacità di assumere decisioni o che i partecipanti possono essere troppo giovani per fornire il consenso informato.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    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)
    Patients who complete the study treatment will be offered the opportunity to continue receiving IMP either through a follow on protocol or on an individual treatment basis as appropriate.
    Ai pazienti che completano il trattamento in studio verrà offerta l'opportunità di continuare a ricevere il farmaco in studio mediante un protocollo di follow up oppure attraverso un trattamento individuale come meglio appropriato.
    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 Decision2017-05-04
    N.Ethics Committee Opinion of the trial applicationWithdrawn
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-01
    P. End of Trial
    P.End of Trial Status
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