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    Summary
    EudraCT Number:2021-000497-28
    Sponsor's Protocol Code Number:PTC923-MD-004-PKU
    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:2022-11-16
    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 number2021-000497-28
    A.3Full title of the trial
    Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria
    Studio di fase III di estensione in aperto su PTC923 nel trattamento della fenilchetonuria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Extension Study of PTC923 in Subjects with Phenylketonuria
    Studio di fase III di estensione in aperto su PTC923 nel trattamento della fenilchetonuria
    A.3.2Name or abbreviated title of the trial where available
    PTC923-MD-004-PKU
    PTC923-MD-004-PKU
    A.4.1Sponsor's protocol code numberPTC923-MD-004-PKU
    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 SponsorPTC THERAPEUTICS 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 supportPTC Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPTC Therapeutics Inc.
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address100 Corporate Court
    B.5.3.2Town/ citySouth Plainfield
    B.5.3.3Post codeNJ07080
    B.5.3.4CountryUnited States
    B.5.6E-mailMedinfo@ptcbio.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/3/21/2435
    D.3 Description of the IMP
    D.3.1Product namePTC923
    D.3.2Product code [ PTC923]
    D.3.4Pharmaceutical form Powder for oral solution
    D.3.4.1Specific paediatric formulation No
    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 17094-01-8
    D.3.9.2Current sponsor codePTC923
    D.3.9.3Other descriptive name(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
    D.3.9.4EV Substance CodeSUB193410
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/21/2435
    D.3 Description of the IMP
    D.3.1Product namePTC923
    D.3.2Product code [ PTC923]
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation No
    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 17094-01-8
    D.3.9.2Current sponsor codePTC923
    D.3.9.3Other descriptive name(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one
    D.3.9.4EV Substance CodeSUB193410
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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
    Metabolic Disorders - Phenylketonuria
    Fenilchetonuria
    E.1.1.1Medical condition in easily understood language
    Phenylketonuria (PKU) is an autosomal-recessive inborn error of metabolism characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH), which metabolizes phenylalanine (Phe)
    La fenilchetonuria (PKU) è una malattia congenita autosomica recessiva del metabolismo caratterizzato dal deficit dell'enzima fenilalanina idrossilasi (PAH), che metabolizza la fenilalanina (Phe).
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    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
    • To evaluate the long-term safety of PTC923 in subjects with PKU
    • To evaluate changes from baseline in dietary Phe/protein consumption
    • Valutare la sicurezza a lungo termine di PTC923 in soggetti affetti da fenilchetonuria (Phenylketonuria, PKU)
    • Valutare le variazioni rispetto al basale del consumo alimentare di fenilalanina (Phenylalanine, Phe)/proteine
    E.2.2Secondary objectives of the trial
    • To evaluate PTC923 effect on quality of life (QOL) using the Phenylketonuria-quality of life (PKU-QOL) questionnaire in the subset of subjects that are able to complete the PKU-QOL (ie, subjects whose primary language is English [British or American], Turkish, Dutch, German, Spanish, Italian, Portuguese, or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to 11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL; ages =18 years Adult PKU-QOL)
    • To evaluate PTC923 effect of QOL using the European Quality of Life 5 Dimensions (EQ-5D) (EQ-5D for Youth [EQ-5D-Y] Proxy Version 1 [3 to 7 years]; EQ-5D-Y [8 to 15 years]; EQ-5D-5L ([=16 years])
    •To evaluate the pharmacokinetics (PK) of sepiapterin and tetrahydrobiopterin (BH4) following repeated doses of PTC923
    • Valutare l'effetto di PTC923 sulla qualità della vita (QOL) mediante il questionario sulla qualità della vita specifico per la fenilchetonuria (PKU-QOL) nel sottogruppo di soggetti in grado di completare il PKU-QOL (soggetti la cui lingua primaria è inglese, turco, olandese, tedesco, spagnolo, italiano, portoghese o francese) (Versioni: Parent PKU-QOL per bambini 6-8 anni; Child PKU-QOL per bambini 9-11 anni; Adolescent PKU-QOL per soggetti 12-17 anni e Adult PKU-QOL per i soggetti di età=18 anni)
    • Valutare l'effetto di PTC923 sulla QOL mediante il Questionario sulla qualità della vita europeo a 5 dimensioni (Versioni: European Quality of Life-5 Dimensions, EQ-5D) (EQ-5D per i giovani [EQ-5D-Y] versione 1 per la compilazione da parte di un delegato [per bambini 3-7 anni]; EQ-5D-Y [per soggetti 8-15 anni]; EQ-5D a 5 livelli [EQ-5D-5L] [per soggetti con =16 anni])
    • Valutare la farmacocinetica della sepiapterina e della tetraidrobiopterina dopo dosi ripetute di PTC923
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children and/or subjects that are mentally impaired secondary to disease]) with parental/legal guardian consent
    2. Completed a PTC-sponsored Phase 3 PKU clinical study
    3. Women of childbearing potential, as defined in (CTFG 2020), must have a negative pregnancy test at study entry and agree to abstinence or the use of at least one highly effective form of contraception (with a failure rate of <1% per year when used consistently and correctly):
    • Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
    - Oral
    - Intravaginal
    - Transdermal
    • Progestogen-only hormonal contraception associated with inhibition of ovulation:
    - Oral
    - Injectable
    - Implantable
    • Intrauterine device
    • Intrauterine hormone-releasing system
    • Vasectomized partner with confirmed azoospermia
    Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 90 days after the last dose of the study drug. All females will be considered of childbearing potential unless they are postmenopausal (at least 12 months consecutive amenorrhea in the appropriate age group without other known or suspected cause) or have been sterilized surgically (eg, hysterectomy, bilateral tubal ligation, bilateral oophorectomy).
    4. Males who are sexually active with women of childbearing potential who have not had a vasectomy must agree to use a barrier method of birth control during the study and for up
    to 90 days after the last dose of study drug. Males must also refrain from sperm donations during this time period. Males who are abstinent will not be required to use a contraceptive method unless they become sexually active. Males who have undergone a vasectomy are not required to use a contraceptive method if at least 16 weeks post procedure.
    5. Willing and able to comply with the protocol and study procedure
    6. Willing to continue current diet unchanged while participating in the study (unless specifically instructed to change diet during the study by the investigator)
    1. Consenso informato e assenso (se necessario, a discrezione dello sperimentatore [vale a dire, per bambini e/o soggetti con ridotte facoltà mentali a seguito della malattia]) associato al consenso dei genitori/tutori legali.
    2. Partecipazione completata a uno studio clinico di fase III sulla PKU sponsorizzato da PTC.
    3. Le donne potenzialmente fertili, secondo la definizione del CTFG 2020, devono presentare un test di gravidanza negativo all’ingresso nello studio e accettare di praticare l'astinenza o utilizzare almeno un metodo contraccettivo altamente efficace (con un tasso di fallimento <1% all'anno, se utilizzato in modo costante e corretto):
    • Contraccezione ormonale combinata (a base di estrogeni e progesterone) associata a inibizione dell'ovulazione:
    - Orale
    - Intravaginale
    - Transdermica
    • Contraccezione ormonale a base di solo progesterone associata a inibizione dell'ovulazione:
    - Orale
    - Iniettabile
    - Impiantabile
    • Dispositivo intrauterino
    • Sistema intrauterino a rilascio ormonale
    • Partner vasectomizzato con azoospermia confermata
    È necessario seguire il regime di contraccezione altamente efficace o l’astinenza per tutta la durata dello studio e fino a 90 giorni dopo l’assunzione dell’ultima dose del farmaco in studio.
    Tutti i soggetti di sesso femminile saranno considerati potenzialmente fertili a meno che non siano in post-menopausa (almeno 12 mesi consecutivi di amenorrea nella relativa fascia di età senza nessun’altra causa nota o sospetta) o sterilizzati chirurgicamente (ad es. tramite isterectomia, legatura tubarica bilaterale, ooforectomia bilaterale).
    4. I pazienti di sesso maschile sessualmente attivi con donne potenzialmente fertili non sottoposti a vasectomia devono accettare di utilizzare un metodo contraccettivo di barriera durante lo studio e fino a 90 giorni dopo la somministrazione dell’ultima dose del farmaco in studio. Gli uomini devono inoltre astenersi dalle donazioni di sperma durante questo periodo di tempo. I soggetti maschili che praticano l’astinenza non saranno obbligati ad utilizzare un metodo contraccettivo, a meno che non diventino sessualmente attivi. I soggetti maschili che si sono sottoposti a vasectomia non sono obbligati ad utilizzare un metodo contraccettivo se sono passate almeno 16 settimane dall’intervento.
    5. Volontà e capacità di rispettare il protocollo e le procedure dello studio.
    6. Volontà di portare avanti la dieta attuale senza modificarla durante la partecipazione allo studio (salvo nel caso in cui lo sperimentatore non indichi specificamente di modificare la dieta durante lo studio).
    E.4Principal exclusion criteria
    1. The individual, in the opinion of the investigator, is unwilling or unable to adhere to the requirements of the study
    2. Inability to tolerate oral medication
    3. A female who is pregnant or breastfeeding, or considering pregnancy
    4. Serious neuropsychiatric illness (eg, major depression) not currently under medical control, that in the opinion of the investigator or sponsor, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject
    5. Past medical history and/or evidence of renal impairment and/or condition including moderate/severe renal insufficiency (glomerular filtration rate [GFR] <60 mL/min) as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist
    6. Any other condition that in the opinion of the investigator or sponsor, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject
    7. Requirement for concomitant treatment with any drug known to inhibit folate synthesis (eg, methotrexate)
    8. Concomitant treatment with BH4 supplementation (eg, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ)
    1. Soggetto che, a giudizio dello sperimentatore, non è disposto o non è in grado di attenersi ai requisiti dello studio.
    2. Incapacità di tollerare il farmaco per via orale.
    3. Donne in gravidanza o allattamento o che stanno pianificando una gravidanza.
    4. Grave malattia neuropsichiatrica (ad es. depressione maggiore) non attualmente sotto controllo medico, che, secondo il parere dello sperimentatore o dello sponsor, interferirebbe con la capacità del soggetto di partecipare allo studio o aumenterebbe il rischio associato alla partecipazione per tale soggetto.
    5. Anamnesi medica pregressa e/o evidenza di patologia e/o compromissione renale, compresa un’insufficienza renale moderata/grave (tasso di filtrazione glomerulare [GFR] <60 ml/min secondo l’ultima stima stabilita durante la qualificazione per la partecipazione in uno studio precedente) e/o in cura da un nefrologo.
    6. Qualsiasi altra condizione che, secondo il parere dello sperimentatore o dello sponsor, interferirebbe con la capacità del soggetto di partecipare allo studio o aumenterebbe il rischio associato alla partecipazione per tale soggetto.
    7. Necessità di trattamento concomitante con qualsiasi farmaco noto per inibire la sintesi del folato (ad es. metotrexato).
    8. Trattamento concomitante con integrazione di BH4 (ad es. sapropterina dicloridato, KUVAN) o pegvaliase-pqpz (PALYNZIQ).
    E.5 End points
    E.5.1Primary end point(s)
    Safety is a primary endpoint: Safety of long-term treatment with PTC923 will be measured by number of treatment-emergent adverse events (TEAEs), including assessment of severity of
    TEAEs, clinical laboratory tests, vital signs, and physical examinations.

    The primary efficacy endpoint is the change from baseline in dietary Phe/protein consumption measured during Phe Tolerance Assessment period.
    La sicurezza è un endpoint primario: la sicurezza del trattamento a lungo termine con PTC923 sarà misurata secondo il numero di eventi avversi emergenti con il trattamento (Treatment-Emergent Adverse Events, TEAEs), ivi inclusa la valutazione della loro gravità, test clinici di laboratorio, segni vitali ed esami obiettivi.

    L'endpoint primario di efficacia è la variazione dal basale nel consumo alimentare di Phe/proteine misurato nel corso del periodo di valutazione della tolleranza alla Phe.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety of long-term treatment with PTC923 will be measured by number of treatment-emergent adverse events (TEAEs), including assessment of severity of TEAEs, clinical laboratory tests, vital signs, and physical examinations.

    Phe tolerance evaluated at Baseline and every 2 weeks over the course of 26 weeks assessment period
    La sicurezza del trattamento a lungo termine con PTC923 sarà misurata secondo il numero di eventi avversi emergenti con il trattamento (Treatment-Emergent Adverse Events, TEAEs), ivi inclusa la valutazione della loro gravità, test clinici di laboratorio, segni vitali ed esami obiettivi.

    Tolleranza alla Phe valutata al basale e ogni 2 settimane nel corso del periodo di valutazione di 26 settimane.
    E.5.2Secondary end point(s)
    • Changes from baseline in QOL using PKU-QOL questionnaire in the subset of subjects that are able to complete the PKU-QOL (ie, subjects whose primary language is English [British or American], Turkish, Dutch, German, Spanish, Italian, Portuguese, or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to 11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL; ages =18 years Adult PKU-QOL)
    • Changes from baseline in QOL using the EQ-5D (EQ-5D-Y Proxy Version 1 [3 to 7 years]; EQ-5D-Y [8 to 15 years]; EQ-5D-5L ([=16 years])
    • PK assessment of sepiapterin and BH4 concentrations in plasma using sparse sampling
    • Variazioni rispetto al basale nella qualità della vita misurata mediante il questionario PKU-QOL nel sottogruppo di soggetti in grado di completare il PKU-QOL (ovvero per i soggetti la cui lingua primaria è inglese [britannico o americano], turco, olandese, tedesco, spagnolo, italiano, portoghese o francese) (PKU-QOL per i genitori per i bambini di età compresa tra 6 e 8 anni; PKU-QOL per bambini per i soggetti di età compresa tra 9 e 11 anni; PKU-QOL per adolescenti per i soggetti di età compresa tra 12 e 17 anni e PKU-QOL adulti per i soggetti di età =18 anni).
    • Variazioni rispetto al basale nella QOL mediante il questionario EQ-5D (EQ-5D-Y versione 1 per la compilazione da parte di un delegato [per bambini da 3 a 7 anni]; EQ-5D-Y [8-15 anni]; EQ-5D-5L ([=16 anni]).
    • Valutazione di PK delle concentrazioni di sepiapterina e BH4 nel plasma mediante campionamento ridotto.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary measures will be evaluated at 6-month intervals starting at M8D1.
    Le misure secondarie saranno valutate ad intervalli di 6 mesi partendo dal M8G1
    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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other 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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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
    Brazil
    Canada
    Mexico
    United States
    France
    Netherlands
    Spain
    Germany
    Italy
    Denmark
    Georgia
    Portugal
    Turkey
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 10
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 20
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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
    Informed consent and assent (if necessary, at the investigator’s discretion [ie, for children and/or subjects who are mentally impaired secondary to disease]) with parental/legal guardian consent
    Consenso informato e assenso (se necessario, a discrezione dello sperimentatore [cioè, per bambini e/o soggetti con disabilità mentale secondaria a malattia]) con il consenso del genitore/tutore legale
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    0-18 years
    0-18 anni
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 98
    F.4.2.2In the whole clinical trial 200
    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
    nessuno
    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 Decision2022-11-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-12
    P. End of Trial
    P.End of Trial StatusOngoing
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