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    Summary
    EudraCT Number:2016-004184-39
    Sponsor's Protocol Code Number:BP39054
    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:2018-02-06
    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 number2016-004184-39
    A.3Full title of the trial
    AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY
    STUDIO IN APERTO PER LA VALUTAZIONE DELLA SICUREZZA, DELLA TOLLERABILITÀ E DELLA FARMACOCINETICA/FARMACODINAMICA DI RO7034067 IN PAZIENTI ADULTI E PEDIATRICI AFFETTI DA ATROFIA MUSCOLARE SPINALE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy.
    Uno studio per valutare la sicurezza, la tollerabilità e la farmacocinetica/farmacodinamica di RO7034067 in pazienti adulti e pediatrici con atrofia muscolare spinale.
    A.3.2Name or abbreviated title of the trial where available
    A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067
    Uno studio per valutare la sicurezza, la tollerabilità e la farmacocinetica/farmacodinamica di RO703
    A.4.1Sponsor's protocol code numberBP39054
    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 SponsorF. HOFFMANN - LA ROCHE LTD.
    B.1.3.4CountrySwitzerland
    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 supportF. Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF.Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasilea
    B.5.3.3Post codeCH-4070
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number000000000000000
    B.5.5Fax number000000000000000
    B.5.6E-mailglobal.rochegenentechtrials@roche.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 nameRO7034067
    D.3.2Product code RO7034067/F06 + solvente (RO
    D.3.4Pharmaceutical form Powder and solvent for oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeRO7034067/F06
    D.3.9.3Other descriptive nameRO7034067
    D.3.9.4EV Substance CodeSUB179686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.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 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 nameRO7034067
    D.3.2Product code RO7034067/F07 + solvente (RO
    D.3.4Pharmaceutical form Powder and solvent for oral solution
    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.3.9.2Current sponsor codeRO7034067/F07
    D.3.9.3Other descriptive nameRO7034067
    D.3.9.4EV Substance CodeSUB179686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Spinal Muscular Atrophy (SMA)
    Atrofia Muscolare Spinale (AMS)
    E.1.1.1Medical condition in easily understood language
    Spinal muscular atrophy, a genetic progressive neuromuscular disease characterized by profound weakness and muscle atrophy that is the leading genetic cause of death in babies and toddlers
    Atrofia Muscolare Spinale,una malattia genetica
    neuromuscolareprogressiva caratterizzata daunaprofondadebolezzaedatrofiamuscolare,cheèunadellecausegenetiche
    principalididecessoinbambini eadolescenti
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10041582
    E.1.2Term Spinal muscular atrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To evaluate the safety and tolerability of RO7034067;
    •To investigate the pharmacokinetics (PK) of RO7034067 and metabolites as appropriate.
    •Valutare la sicurezza e la tollerabilità di RO7034067;
    •Determinare la farmacocinetica di RO7034067 e dei suoi metaboliti, se appropriato.
    E.2.2Secondary objectives of the trial
    •To investigate the PK/ pharmacodynamics (PD) relationship of RO7034067. The PD investigations will include analyses of Survival of motor neuron (SMN) mRNA splice forms and SMN protein.
    •Determinare la correlazione farmacocinetica/farmacodinamica di RO7034067. Le determinazioni farmacodinamiche comprenderanno le analisi delle isoforme di mRNA di SMN e della proteina SMN.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Males and females 12 to 60 years of age inclusive (at screening).

    - Confirmed diagnosis of 5q-autosomal recessive SMA, including:
    •Genetic confirmation of homozygous deletion or heterozygosity predictive of loss of function of the SMN1 gene,
    •Clinical symptoms attributable to Type 2 or Type 3 SMA.

    - Previous participation in a study with an SMN2-targeting antisense oligonucleotide or SMN2 splicing modifier other than RO7034067.

    - Negative blood pregnancy test at screening (all women of childbearing potential, including those who have had a tubal ligation), and agreement to comply with measures to prevent pregnancy and restrictions on sperm donation.
    For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures and agreement to refrain from donating sperm.
    - Soggetti di sesso maschile e femminile di età da 12 a 60 anni compresi (al momento dello screening).

    - Diagnosi confermata di SMA autosomica recessiva 5q, tra cui:
    • Conferma genetica di delezione omozigote o eterozigosità predittiva di perdita di funzione del gene SMN1,
    • Sintomi clinici attribuibili alla SMA di tipo 2 o di tipo 3.

    - Partecipazione precedente a uno studio con un oligonucleotide antisenso mirato a SMN2 oppure con un modificatore dello splicing di SMN2 diverso da RO7034067.

    - Test di gravidanza sul sangue negativo al momento dello screening (tutte le donne in età fertile, comprese quelle sottoposte a legatura delle tube) e accettazione del rispetto delle misure di prevenzione della gravidanza e delle limitazioni della donazione di sperma.
    Per gli uomini: accettazione a praticare l'astinenza (astenersi dai rapporti eterosessuali) o a utilizzare metodi di contraccezione e accettazione ad astenersi dalla donazione di sperma.
    E.4Principal exclusion criteria
    - Inability to meet study requirements

    - Concomitant participation in any investigational drug or device study

    - Previous participation in an SMN2-targeting antisense oligonucleotide or SMN2 splicing modifier study other than RO7034067 within 90 days prior to screening

    - Previous participation in any investigational drug or device study, other than SMN2 targeting antisense oligonucleotide or SMN2 splicing modifier study, within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer

    - Any history of gene or cell therapy

    - Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator

    - Lactating women

    - Suspicion of regular consumption of drugs of abuse

    - Positive urine test for drugs of abuse or alcohol at the screening or Day − 1 visit

    - Presence of clinically significant ECG abnormalities before study drug administration

    - Presence of clinically significant cardiovascular, blood pressure, and heart rate measures

    - History of malignancy if not considered cured

    - Significant risk for suicidal behavior, in the opinion of the Investigator as assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS)

    - Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration

    - Use of any OCT-2 and MATE substrates within 2-weeks before dosing

    - Use of the following medications within 90 days prior to enrollment: riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, agents anticipated to increase or decrease muscle strength, agents with known or presumed histone deacetylase (HDAC) inhibitory effect, and medications with known phototoxicity liabilities (e.g., oral or topical retinoids including over-the-counter formulations, amiodarone. Use of inhaled corticosteroids is allowed.

    - Recently initiated treatment (within < 6 months prior to enrollment) with oral salbutamol or another β2-adrenergic agonist taken orally is not allowed. Patients who have been on oral salbutamol (or another β2-adrenergic agonist) for >= 6 months before enrollment and have shown good tolerance are allowed. The dose of β2-adrenergic agonist should remain stable as much as possible for the duration of the study. Use of inhaled β2-adrenergic agonists (e.g., for the treatment of asthma) is allowed.

    - Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine is not allowed. Use of other medications known to or suspected of causing retinal toxicity within one year prior to enrollment is not allowed

    - Clinically significant abnormalities in laboratory test results

    - Donation or loss of blood >= 10% of blood volume within three months prior to screening

    - Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7034067 or to the constituents of its formulation

    - Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the patient in this study

    - Recent history (less than one year) of ophthalmological diseases
    - Incapacità di soddisfare i requisiti dello studio.

    - Partecipazione concomitante a qualsiasi studio con un farmaco o un dispositivo sperimentale.

    - Partecipazione precedente a uno studio con un oligonucleotide antisenso mirato a SMN2 oppure con un modificatore dello splicing di SMN2 diverso da RO7034067 nei 90 giorni precedenti allo screening.

    - Partecipazione precedente a qualsiasi studio con un farmaco o un dispositivo sperimentale, diverso da uno studio con un oligonucleotide antisenso mirato a SMN2 oppure con un modificatore dello splicing di SMN2, nei 90 giorni o nelle 5 emivite del farmaco precedenti allo screening, a seconda di quale dei due sia il periodo più lungo.

    - Anamnesi di terapia genica o cellulare.

    - Malattie gastrointestinali, renali, epatiche, endocrine o cardiovascolari instabili, considerate clinicamente significative dallo sperimentatore.

    - Donne che allattano al seno.

    - Sospetto di consumo regolare di droghe d'abuso.

    - Test delle urine positivo a droghe d'abuso o alcool al momento dello screening o alla visita del Giorno -1.

    - Presenza di anomalie clinicamente significative sull'ECG prima della somministrazione del farmaco dello studio.

    - Presenza di anomalie clinicamente significative di tipo cardiovascolare, relative alla pressione sanguigna ed alla frequenza cardiaca.

    - Anamnesi di neoplasia se non considerata curata.

    - Rischio significativo di comportamento suicida secondo il parere dello sperimentatore, valutato utilizzando la scala della Columbia University per la classificazione della gravità del rischio di suicidio (C-SSRS, Columbia-Suicide Severity Rating Scale).

    - Qualsiasi malattia importante nel mese precedente all'esame di screening o qualsiasi malattia febbrile nella settimana precedente allo screening e fino alla somministrazione della prima dose

    - Uso di qualsiasi substrato di OCT-2 e MATE nelle 2 settimane precedenti alla somministrazione.

    - Uso dei seguenti farmaci nei 90 giorni precedenti l'arruolamento: riluzolo, acido valproico, idrossiurea, fenilbutirrato di sodio, derivati del butirrato, creatina, carnitina, ormone della crescita, steroidi anabolizzanti, probenecid, agenti che si prevede aumentino o riducano la forza muscolare, agenti con effetto inibitorio noto o presunto dell'istone deacetilasi (HDAC) e farmaci con effetti noti di fototossicità (per es. retinoidi per uso orale o topico, comprese le formulazioni da banco, amiodarone. E’ consentito l’uso di corticosteroidi per via inalatoria.

    - Non è ammesso il trattamento iniziato recentemente (entro  6 mesi prima dell'arruolamento) con salbutamolo orale o un altro agonista 2-adrenergico assunto per via orale. Sono ammessi i pazienti che sono stati in trattamento con salbutamolo orale (o un altro agonista 2-adrenergico) per  6 mesi prima dell'arruolamento e hanno mostrato una buona tollerabilità. La dose di agonista 2-adrenergico deve rimanere stabile il più possibile per l'intera durata dello studio. È ammesso l'uso di agonisti 2 adrenergici per via inalatoria (per es. per il trattamento dell'asma).

    - Non è ammesso qualsiasi uso precedente di clorochina, idrossiclorochina, retigabin, vigabatrin o tioridazina. Non è ammesso l'uso nell'anno precedente all'arruolamento di altri farmaci noti o sospetti di causare tossicità retinica.

    - Anomalie clinicamente significative nei risultati delle analisi di laboratorio.

    - Donazione o perdita di sangue  10% del volume ematico nei tre mesi precedenti allo screening.

    - Ipersensibilità accertata o presunta (per es. reazione anafilattica) a RO7034067 o ai componenti della sua formulazione.

    - Malattia o condizione concomitante che potrebbe interferire, o il cui trattamento potrebbe interferire, con la conduzione dello studio oppure che, secondo il parere dello sperimentatore, rappresenterebbe un rischio inaccettabile per il paziente nello studio.

    - Anamnesi recente (meno di un anno) di malattie oftalmologiche.
    E.5 End points
    E.5.1Primary end point(s)
    1.Incidence and severity of adverse events and serious adverse events.
    2.Incidence of treatment discontinuations due to adverse events.
    3.Incidence of clinically significant abnormal vital sign, laboratory, and ECG abnormalities.
    4.Incidence of clinically significant findings on ophthalmological examination.
    5.Incidence of suicidal ideation or behavior (C-SSRS).
    6.Mean plasma concentrations of RO7034067 and metabolites.
    7.Peak plasma concentration (Cmax).
    8.Area under the curve (AUC).
    9.Concentration at the end of a dosing interval (Ctrough) to assess steady-state.
    1. Incidenza e gravità degli eventi avversi seri.
    2. Incidenza di interruzioni del trattamento dovute a eventi avversi.
    3. Incidenza di segni vitali anormali, valori di laboratorio e valori ECG.
    4.Incidenza di esiti clinicamente significativi all’esame oftalmologico.
    5.Incidenza di idea di suicidio o comportamento suicida (C-SSRS).
    6.Concentrazione plasmatica media di RO7034067 e metaboliti.
    7.Picco di concentrazoine plasmatica (Cmax).
    8.Area sotto la curva (AUC).
    9. Concentrazione alla fine dell'intervallo di somministrazione (Cvalle) per valutare lo stato stazionario.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-4. Up to 160 weeks.
    5. Screening (Day-30 to -2), Day-1, Day 119, 182, 364, 546, 728, at follow-up visit 1 (Week 112).
    6-9. Days 1, 7, 14, 28, 56, 119, 183, 245, 301, 365, 490, 609, 729, at follow-up visit 1.
    1-4. Fino a 160 settimane.
    5. Screening (Giorno-30 to -2), Giorno -1, Giorno 119, 182, 364, 546, 728, alla visita 1 di follow-up (Settimana 112).
    6-9. Giorni 1, 7, 14, 28, 56, 119, 183, 245, 301, 365, 490, 609, 729, alla visita 1 di follow-up.
    E.5.2Secondary end point(s)
    1.SMN mRNA in blood.
    2.SMN protein levels in blood.
    1. mRNA di SMN nel sangue.
    2.Livelli di proteina SMN nel sangue.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.Days -1, 1, 7, 14, 28, 183, 365, 729, at follow-up visit 1.
    2.Day -1, Day 7, 14, 28, 183, 365, 729, at follow-up visit 1.
    1.Giorni -1, 1, 7, 14, 28, 183, 365, 729, alla visita 1 di follow-up.
    2.Giorno -1, Day 7, 14, 28, 183, 365, 729, alla visita 1 di follow-up.
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tollerabilità
    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) 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.8.3 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
    Italy
    Switzerland
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last Patient Last Observation.
    Ultima osservazione dell'ultimo paziente.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 18
    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
    Pediatric patients may provide according to local regulation, informed assent; informed consent for study participation will be obtained from parents or legal guardian.
    I pazienti pediatrici possono fornire un assenso informato, in accord alla normative locale; il consenso informato per la partecipazione allo studio sarà raccolto dai gentori o dai tutori legali.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 24
    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)
    The Sponsor will offer post-trial access to the study drug free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product.

    The Roche Global Policy on Continued Access to Investigational Medicinal Product is available at the following Web site:
    http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf
    Lo Sponsor offrirà un accesso post-sperimentazione allo studio con farmaco fornito gratuitamente ai pazienti eleggibili in accord con la Policy Globale di Roche sull’Accesso Continuo al Prodotto Medico Speriementale. La Policy Globale di Roche sull’Accesso Continuo al Prodotto Medico Speriementale è disponibile al seguente sito web: http://www.roche.com/policy_continued_access_to_investigational_ medicines.pdf
    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 Decision2017-05-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-03-16
    P. End of Trial
    P.End of Trial StatusOngoing
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