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    Summary
    EudraCT Number:2021-003791-13
    Sponsor's Protocol Code Number:BP42992
    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:2021-10-06
    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 number2021-003791-13
    A.3Full title of the trial
    A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF 52 WEEKS OF TREATMENT WITH BASMISANIL IN CHILDREN WITH DUP15Q SYNDROME.
    STUDIO DI FASE II, RANDOMIZZATO, IN DOPPIO CIECO, CONTROLLATO CON PLACEBO, A GRUPPI PARALLELI PER VALUTARE LA SICUREZZA, L’EFFICACIA E LA FARMACODINAMICA DI 52 SETTIMANE DI TRATTAMENTO CON BASMISANIL IN BAMBINI CON SINDROME DUP15Q
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of 52 weeks of Treatment with Basmisanil in Children with Dup15q Syndrome.
    Uno studio per valutare la sicurezza, l'efficacia e la farmacodinamica di 52 settimane di trattamento con basmisanil in bambini con sindrome Dup15q.
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberBP42992
    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-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number0000000
    B.5.5Fax number000000
    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 namebasmisanil
    D.3.2Product code [Ro 518-6582/F13-01]
    D.3.4Pharmaceutical form Pillules
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1159600-41-5
    D.3.9.2Current sponsor codeRO5186582
    D.3.9.3Other descriptive nameGABAA a5 negative allosteric modulator, previously referred to as GABAA a5 inverse agonist
    D.3.9.4EV Substance CodeSUB31464
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPillules
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Dup15q Syndrome
    Sindrome Dup15q
    E.1.1.1Medical condition in easily understood language
    Dup15q Syndrome is a neurodevelopmental disorder, caused by the partial duplication of Chromosome 15, that confers a strong risk for autism spectrum disorder, epilepsy, and intellectual disability.
    La S. Dup15q è un disturbo dello sviluppo neurol., causato dalla parziale duplicazione del crom.15, che conferisce un forte rischio di disturbi dello spettro autistico, epilessia e disabilità intel.
    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 23.0
    E.1.2Level LLT
    E.1.2Classification code 10083952
    E.1.2Term Dup15q syndrome
    E.1.2System Organ Class 100000004850
    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 effects of basmisanil treatment on core symptom domains of Dup15q syndrome (language and social skills) and daily functioning.
    Valutare gli effetti del trattamento con basmisanil sui domini principali dei sintomi della sindrome Dup15q (linguaggio e abilità sociali) e sul funzionamento quotidiano.
    E.2.2Secondary objectives of the trial
    • To evaluate the effects of basmisanil treatment on Motor function, Cognition, Language, Social skills, Clinical impression of global functioning, Challenging behaviors
    • To evaluate the tolerability and safety of 52 weeks treatment of basmisanil
    • To characterize the PK of basmisanil and its metabolite M1 and determine the dose that will deliver target exposure in participants aged 2-11 years
    • To evaluate potential relationships between selected covariates and exposure to basmisanil
    • To evaluate the effects of basmisanil treatment on the characteristic Dup15q EEG phenotype acutely and at steady state.
    - Valutare gli effetti del trattamento con basmisanil su Funzione motoria, Cognizione, Linguaggio, Abilità sociali, Impressione clinica del funzionamento globale, Comportamenti di sfida
    • Valutare la tollerabilità e la sicurezza di 52 settimane di trattamento con basmisanil
    • Per caratterizzare la PK del basmisanil e del suo metabolita M1 e determinare la dose che fornirà l'esposizione target nei partecipanti di età compresa tra 2 e 11 anni
    • Valutare le potenziali correlazioni tra covariate selezionate ed esposizione a basmisanil
    • Valutare gli effetti del trattamento con basmisanil sul fenotipo EEG di Dup15q caratteristico in fase acuta e allo stato stazionario.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Participants aged 2 to 11 years inclusive at the time the caregiver signs the informed consent (for Stage 1, participants aged 6 to 11 years inclusive; for Stage 2, participants aged 2 to 5 years inclusive; and for Stage 3, participants aged 2 to 11 years inclusive)
    • Documented maternal duplication (3 copies) or triplication (4 copies) of the chromosome 15q11.2-q13.1 region that includes the Prader-Willi/Angelman critical region defined as [BP2-BP3] segment
    • Dup15q syndrome Clinical Global Impression Severity scale (Dup15q CGI-S) overall severity score >_ 4 (at least moderately ill)
    • Stages 1 and 2: Participants with epilepsy only
    • Body weight equal to or above the third percentile for age
    • Male and female participants: Some of the provisions that follow may have limited applicability based on the age range of study participants (i.e., up to the age of 11) and the nature of the disease under study
    • Female Participants: A female participant is eligible to participate if she is not pregnant, not breastfeeding
    • Male Participants: Male contraception is not required in this study because of the minimal seminal dose transmitted through sexual intercourse
    • The participant has a parent, caregiver, or legally authorized representative (hereinafter “caregiver”) of at least 18 years of age, who is fluent in the local language at the site, and capable and willing to provide written informed consent for the participant according to International Council on Harmonisation and local regulations
    • The participant’s caregiver must be living with the participant and, in the opinion of the Investigator, able and willing to reliably assess the participant’s ongoing condition, to accompany the participant to all clinic visits, and ensure compliance to study treatment throughout the study. The same caregiver is able and willing to complete the caregiver assessments and is available to the Investigational Site by telephone or email if needed
    • The participant’s caregiver is able and willing to use electronic devices to record information on the participant’s condition and to complete assessments at home and agrees to home nursing visits, if local regulations allow for it and if home nursing service is available in the country/region.
    - Partecipanti di età compresa tra 2 e 11 anni inclusi al momento della firma del consenso informato da parte del caregiver (per la Parte 1, partecipanti di età compresa tra 6 e 11 anni inclusi; per la Parte 2, partecipanti di età compresa tra 2 e 5 anni inclusi; e per la Parte 3, partecipanti di età compresa tra 2 e 11 anni inclusi).
    - Duplicazione (3 copie) o triplicazione (4 copie) materna documentata della regione del cromosoma 15q11.2-q13.1 che include la regione critica di Prader-Willi/Angelman definita come segmento [BP2-BP3].
    - Punteggio complessivo relativo alla gravità della scala Clinical Global Impression of Severity per la sindrome Dup15q (Dup15q CGI-S) >_ 4 (malattia almeno moderata).
    - Parti 1 e 2: solo partecipanti con epilessia
    - Peso corporeo pari o superiore al terzo percentile per l’età
    - Partecipanti di entrambi i sessi: Alcune delle disposizioni che seguono possono avere un’applicabilità limitata in base alla fascia d’età dei partecipanti allo studio (cioè, fino all’età di 11 anni) e alla natura della malattia in studio
    - Partecipanti di sesso femminile: Sono idonee alla partecipazione se non sono in stato di gravidanza o in fase di allattamento
    - Partecipanti di sesso maschile: questo studio non richiede la contraccezione maschile vista la minima quantità di liquido seminale trasmessa durante un rapporto sessuale
    - Il partecipante ha un genitore, un tutore o un rappresentante legale (di seguito “caregiver”) di almeno 18 anni di età, che parla correntemente la lingua locale del centro e che è in grado e disposto a fornire il consenso informato scritto per il partecipante, ai sensi della Conferenza internazionale per l’armonizzazione e delle normative locali
    - Il partecipante ha un genitore, un tutore o un rappresentante legale (di seguito “caregiver”) di almeno 18 anni di età, che parla correntemente la lingua locale del centro e che è in grado e disposto a fornire il consenso informato scritto per il partecipante, ai sensi della Conferenza internazionale per l’armonizzazione e delle normative locali
    - Il caregiver del partecipante deve essere in grado e disposto a utilizzare dispositivi elettronici per registrare informazioni sulle condizioni del partecipante e per completare le valutazioni a casa e deve acconsentire alle visite infermieristiche domiciliari, se le normative locali lo consentono e se il servizio di assistenza infermieristica domiciliare è disponibile nel Paese/regione
    E.4Principal exclusion criteria
    • Individuals with known pathogenic mutations or copy number variants, of established neurodevelopmental significance, other than duplication or triplication of the chromosome 15q11.2-q13.1
    • Uncontrolled epilepsy at Screening as indicated by: Use of rescue medication more than once per month on average in the past 6 months or concomitant use of more than 4 anti-epileptic medications or status epilepticus within the past 6 months requiring urgent hospitalization or any implanted devices to treat drug-resistant epilepsy
    • Lymphoma, leukemia, or any malignancy within the past 5 years, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years
    • Clinically significant ECG abnormalities at Screening, including an average triplicate QTcF > 450 ms for participants > 10 years or QTcB > 450 ms for children up to and including age 10 years
    • Clinically significant abnormalities in laboratory test results at screening (including positive results for HIV, hepatitis B and/or hepatitis C). ALT values > 1.5 × the upper limit of normal
    • Allowed prior existing medication should be on a stable regimen (or frequency of intervention) for at least 6 weeks, and at least 8 weeks for anti-epileptic treatment, prior to Screening
    • Non-pharmacological / behavioral therapies should not be stopped or newly started at least 6 weeks prior to Screening and are expected to remain stable for the entire study duration (excluding changes related to standard age and educational interventional programs and minor interruptions such as illness or vacation
    • Concomitant use of prohibited medications
    • Participation in an investigational drug study within one month or within 6 × the elimination half-life, whichever is longer, prior to dosing in the study
    • Significant risk for suicidal behavior, in the opinion of the Investigator as assessed by suicidality questions adapted from the Columbia Classification Algorithm for Suicide (C-CASA)
    • Known sensitivity to any of the study treatments or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates the participation in the study, including severe lactose intolerance
    • Concomitant clinically relevant disease or condition or any clinically significant finding at screening that could interfere with, or for which, the treatment might interfere with, the conduct of the study or that would pose an unacceptable risk to the participants in this study
    • Known active or uncontrolled bacterial, viral, or other infection or any major clinically significant episode of infection or hospitalization (relating to the completion of the course of antibiotics) within 6 weeks prior to the start of drug administration.
    - Individui con mutazioni patogene note o varianti del numero di copie, di rilevanza accertata per il neurosviluppo, diverse dalla duplicazione o triplicazione del cromosoma 15q11.2-q13.1
    - Epilessia non controllata allo screening, indicata da: Uso di medicinali di salvataggio più di una volta al mese in media negli ultimi 6 mesi o Uso concomitante di più di 4 medicinali antiepilettici o stato epilettico che negli ultimi 6 mesi ha richiesto un ricovero urgente o eventuali dispositivi impiantati per trattare l’epilessia farmaco-resistente
    - Linfoma, leucemia o qualsiasi neoplasia maligna negli ultimi 5 anni, ad eccezione dei carcinomi epiteliali basocellulari o squamosi che sono stati asportati e non mostrano evidenza di malattia metastatica da 3 anni
    - Anomalie clinicamente significative dell’ECG allo screening, incluso un intervallo QTcF medio in triplicato ¿ 450 ms per i partecipanti di¿ 10 anni o un intervallo QTcB ¿ 450 ms per i bambini fino a 10 anni inclusi
    - Anomalie clinicamente significative dei risultati degli esami di laboratorio allo screening (inclusi risultati positivi per HIV, epatite B e/o epatite C). Valori ALT ¿ 1,5 ¿ il limite superiore della norma
    - Le terapie farmacologiche precedenti consentite devono essere assunte a un regime stabile (o con una frequenza di intervento stabile) da almeno 6 settimane, e da almeno 8 settimane per il trattamento antiepilettico, prima dello screening
    - Le terapie non farmacologiche/comportamentali non devono essere interrotte o iniziate nuovamente almeno 6 settimane prima dello screening e si deve prevedere che rimarranno stabili per l’intera durata dello studio (escluse le modifiche standard relative all’età e i programmi interventistici educazionali e le interruzioni minori come in caso di malattia o ferie)
    - Uso concomitante di medicinali proibiti
    - Partecipazione a uno studio su un farmaco sperimentale entro un mese o entro 6 volte l’emivita di eliminazione, adottando il periodo più lungo, prima della somministrazione nell’ambito dello studio
    - Rischio significativo di comportamento suicidario, secondo il giudizio dello sperimentatore basato sulle domande relative alla suicidalità adattate dal Columbia Classification Algorithm for Suicide (C-CASA)
    - Sensibilità nota a uno qualsiasi dei trattamenti dello studio o ai suoi componenti o a un farmaco o altra allergia che, secondo il parere dello sperimentatore, rende controindicata la partecipazione allo studio, inclusa una grave intolleranza al lattosio
    - Malattia o affezione clinicamente rilevante concomitante o qualsiasi risultato clinicamente significativo allo screening che potrebbe interferire o essere influenzato dalla conduzione dello studio o che rappresenterebbe un rischio inaccettabile per i partecipanti allo studio
    - Infezioni batteriche, virali o di altro tipo clinicamente significative, in atto o non controllate, o qualsiasi episodio maggiore clinicamente significativo di infezione o ospedalizzazione (relativo al completamento del ciclo di antibiotici) nelle 6 settimane precedenti l’inizio della somministrazione del farmaco
    E.5 End points
    E.5.1Primary end point(s)
    1. Vineland Adaptive Behavior Scale–Third Edition (Vineland-3): Adaptive Behavior Composite.
    1. Vineland Adaptive Behavior Scale–terza edizione (Vineland-3): Adaptive Behavior Composite.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Baseline (Day -7 to Day -1), Day 183, Day 365.
    1. Basale (dal giorno -7 al giorno -1), giorno 183, giorno 365.
    E.5.2Secondary end point(s)
    1. Vineland-3: gross and fine motor, expressive and receptive language, play and leisure time and interpersonal relationships subdomains
    2. Mullen Scales of Early Learning (MSEL): gross and fine motor domains, visual reception domain, expressive and receptive language subdomains domains
    3. Dup15q syndrome Clinical Global Impression Severity scale (Dup15q CGI-S)
    4. Dup15q syndrome Clinical Global Impression Change scale (Dup15q CGI-C)
    5. Aberrant Behavior Checklist - Second Edition – Community Version (ABC-2-C) domain scores
    6. Incidence, nature, and severity of AEs and SAEs
    7. Incidence of treatment discontinuations due to AEs
    8. Incidence of laboratory abnormalities based on hematology, clinical chemistry, and urinalysis test results
    9. ECG changes from baseline; incidence of abnormal ECG assessments
    10. Change from baseline in all seizure frequency, duration, and type of seizure as reported in a seizure diary by caregivers
    11. Abnormal changes in EEG recordings compared to baseline with a focus on treatment-emergent epileptiform abnormalities
    12. Systolic and diastolic blood pressure and heart rate measurements
    13. Suicidality as assessed through questions from selected items adapted from the Columbia Classification Algorithm for Suicide (C-CASA) in children aged 6-11 years
    14. Height, weight, head circumference
    15. Tanner staging over time (in children aged 9-11 years)
    16. Plasma concentration of basmisanil and its major metabolite M1 at specified timepoints
    17. Plasma concentration ratio of M1 to basmisanil at trough
    18. qEEG beta-band power.
    1. Vineland-3: sottodomini abilità motorie grossolane e fini, espressione e linguaggio recettivo, gioco e relazioni interpersonali
    2. MSEL: domini abilità motorie grossolane e fini, dominio ricezione visiva, sottodomini espressione e linguaggio recettivo
    3. Dup15q CGI-S
    4. Dup15q CGI-C
    5. Punteggi del dominio ABC-2-C
    6. Incidenza, natura e gravità degli eventi avversi (AE) e degli eventi avversi gravi (SAE)
    7. Incidenza di interruzioni del trattamento per AE
    8. Incidenza di anomalie di laboratorio relative ai parametri ematologici, di chimica clinica e ai risultati delle analisi delle urine
    9. Variazioni dell’ECG rispetto al basale; incidenza di valutazioni ECG anomale
    10. Variazione rispetto al basale della frequenza di crisi convulsive di tutti i tipi, della durata e del tipo di crisi, secondo quanto riportato dai caregiver in un diario delle crisi
    11. Variazioni anomale nelle registrazioni EEG rispetto al basale, con particolare attenzione alle anomalie epilettiformi emergenti dal trattamento
    12. Misurazione della pressione arteriosa sistolica e diastolica e della frequenza cardiaca
    13. Suicidalità valutata attraverso domande attinte da item selezionati adattati della scala C-CASA in bambini di età compresa tra 6 e 11 anni
    14. Altezza, peso, circonferenza cranica
    15. Stadiazione di Tanner nel tempo (nei bambini di età 9-11 anni)
    16. Concentrazione plasmatica di basmisanil e del suo principale metabolita M1 in determinati punti temporali
    17. Rapporto tra la concentrazione plasmatica di M1 e di basmisanil a valle
    18. Potenza della banda beta del qEEG
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-2. Baseline, Day 183, Day 365
    3. Screening, Baseline, Day 28, Day 92, Day 183, Day 274, Day 365, Follow-up
    4. Day 28, Day 92, Day 183, Day 274, Day 365, Follow-up
    5. Baseline, Day 28, Day 92, Day 183, Day 274, Day 365
    6-8. Up to Day 395
    9-11. Baseline to Day 395
    12-15. Up to Day 395
    16-17. Day 1, Day 2, Day 14, Day 92, Day 183, Day 274, Day 365
    18. Baseline, Day 1, Day 2, Day 14, Day 183, Day 365.
    1-2. Basale, giorno 183, giorno 365
    3. Screening, basale, giorno 28, giorno 92, giorno 183, giorno 274, giorno 365, follow-up
    4. Giorno 28, Giorno 92, Giorno 183, Giorno 274, Giorno 365, Follow-up
    5. Basale, giorno 28, giorno 92, giorno 183, giorno 274, giorno 365
    6-8. Fino al giorno 395
    9-11. Dal Basale al giorno 395
    12-15. Fino al giorno 395
    16-17. Giorno 1, Giorno 2, Giorno 14, Giorno 92, Giorno 183, Giorno 274, Giorno 365
    18. Basale, giorno 1, giorno 2, giorno 14, giorno 183, giorno 365.
    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) 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Australia
    China
    Russian Federation
    Italy
    Poland
    Portugal
    Spain
    United Kingdom
    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
    A participant is considered to have completed the study if he/she has completed all phases of the study, including the last scheduled procedure per SoA or withdrawal of consent.
    The end of the study is defined as the date when the last participant’s last observation occurs.
    All participants who complete the 52 weeks of study treatment will be offered the option to rollover into an open-label extension study for continued drug access (separate protocol).
    Si riterrà che un partecipante abbia completato lo studio se ha completato tutte le fasi dello studio, inclusa l’ultima procedura pianificata secondo il SoA o se avrà revocato il consenso.
    La data di fine studio coinciderà con la data dell’ultima osservazione dell’ultimo pt.
    A tutti i partecipanti che completeranno le 52 settimane di tratt. dello studio sarà offerta la possibilità di passare a 1 studio di estensione in aperto per continuare ad avere accesso al farmaco (protocollo separato).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months55
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months55
    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: 90
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Participants aged 2 to 11 years inclusive at the time the caregiver signs the informed consent.
    Partecipanti di età compresa tra 2 e 11 anni inclusi nel momento in cui il caregiver firma il consenso informato
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 90
    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)
    All participants who complete the 52 weeks of study treatment will be offered the option to roll over into an open-label extension study for continued drug access under a separate study protocol.
    A participant will not be eligible to receive study treatment after the end of the study if any of the conditions outlined in the study protocol are not met.
    A tutti i partecipanti che completeranno le 52 settimane di trattamento in studio verrà offerta la possibilità di passare a uno studio di estensione in aperto per continuare ad avere accesso al farmaco in un protocollo di studio separato.
    Un partecipante non sarà idoneo a ricevere il trattamento in studio dopo la fine dello studio se una delle condizioni delineate nel protocollo di studio non è soddisfatta.
    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 Decision2022-01-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-05
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2024-01-12
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