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    Summary
    EudraCT Number:2021-000497-28
    Sponsor's Protocol Code Number:PTC923-MD-004-PKU
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-09-15
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-000497-28
    A.3Full title of the trial
    Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria
    Fase 3 open-label uitbreidingsonderzoek van PTC923 bij fenylketonurie
    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
    Een fase 3 open-label uitbreidingsonderzoek van PTC923 bij patiënten met fenylketonurie
    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.3.2Product code PTC923
    D.3.4Pharmaceutical form Powder for oral suspension in sachet
    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.3.8INN - Proposed INNnot available
    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 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.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.3.2Product code PTC923
    D.3.4Pharmaceutical form Powder for oral suspension in sachet
    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.3.8INN - Proposed INNnot available
    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 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
    Metabolic Disorders - Phenylketonuria
    Stofwisselingsstoornissen - Fenylketonurie
    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)
    Fenylketonurie (PKU) is een autosomaal recessieve aangeboren stofwisselingsziekte die wordt gekenmerkt door een tekort aan enzym fenylalaninehydroxylase (PAH), dat fenylalanine (Phe) metaboliseert.
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10034873
    E.1.2Term Phenylketonuria (PKU)
    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 long-term safety of PTC923 in subjects with PKU
    • To evaluate changes from baseline in dietary Phe/protein consumption
    • Evaluaren van de veiligheid van PTC923 op de lange termijn bij proefpersoenen met PKU
    • Evaluaren van veranderingen in Phe/eiwitconsumptie ten opzichte van baseline, via de voeding
    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

    • Evalueren van het effect van PTC923 op de kwaliteit van leven (QOL) met behulp van de fenylketonurie-kwaliteit van leven (PKU-QOL) vragenlijst in de subset van proefpersonen die de PKU-QOL kunnen invullen (ie. proefpersonen waarvan de primaire taal Engels [Brits of Amerikaans], Turks, Nederlands, Duits, Spaans, Italiaans, Portugees of Frans is) (leeftijd 6 tot 8 jaar: Ouders PKU-QOL; leeftijd 9 tot 11 jaar: Kind PKU-QOL; leeftijd 12 tot 17 jaar: Adolescent PKU-QOL; leeftijd ≥18 jaar: Volwassen PKU-QOL)
    • Evalueren van het PTC923 effect van QOL met behulp van de European Quality of Life 5 Dimensions (EQ-5D) (EQ-5D voor jongeren [EQ-5D-Y] Proxy Version 1 [3 tot 7 jaar]; EQ-5D-Y [8 tot 15 jaar]; EQ-5D-5L ([≥16 jaar])
    • Evalueren van de farmacokinetiek (PK) van sepiapterine en tetrahydrobiopterine (BH4), na herhaalde doses 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. Geïnformeerde toestemming en instemming (indien nodig, naar goeddunken van de onderzoeker [dwz voor kinderen
    en/of personen met een verstandelijke beperking secundair aan ziekte]) met ouderlijke/juridische toestemming van de voogd.
    2. Een door PTC gesponsorde klinische fase 3 PKU-studie afgerond.
    3. Vrouwen die zwanger kunnen worden, zoals gedefinieerd in (CTFG 2020), moeten een negatieve zwangerschapstest hebben bij deelname aan het onderzoek en akkoord gaan met onthouding of het gebruik van ten minste één zeer effectieve vorm van anticonceptie (met een mislukkingspercentage van <1% per jaar bij consequent en correct gebruik):
    • Gecombineerde (oestrogeen- en progestageen bevattende) hormonale anticonceptie geassocieerd met remming van de ovulatie:
    - Oraal
    - Intravaginaal
    - Transdermaal
    • Hormonale anticonceptie met alleen progestageen geassocieerd met remming van de ovulatie:
    - Oraal
    - Injecteerbaar
    - Implanteerbaar
    • Spiraaltje
    • Intra-uterien hormoonafgevend systeem
    • Gevasectomiseerde partner met bevestigde azoöspermie
    Zeer effectieve anticonceptie of onthouding moet worden voortgezet voor de duur van de studie, en tot 90 dagen na de laatste dosis van het studiegeneesmiddel.
    Alle vrouwen worden beschouwd als vruchtbaar, tenzij ze postmenopauzaal zijn (minstens 12 maanden opeenvolgende amenorroe in de juiste leeftijdsgroep zonder andere bekende of vermoedelijke oorzaak) of operatief zijn gesteriliseerd (bijv. hysterectomie, bilateraal afbinden van de eileiders, bilaterale ovariëctomie).
    4. Mannen die seksueel actief zijn met vrouwen in de vruchtbare leeftijd die geen vasectomie hebben gehad, moet instemmen met het gebruik van een barrièremethode voor anticonceptie tijdens het onderzoek en voor maximaal tot 90 dagen na de laatste dosis van het onderzoeksgeneesmiddel. Mannen moeten zich ook onthouden van spermadonaties tijdens deze periode.
    Mannen die zich onthouden, hoeven geen anticonceptiemethode te gebruiken, tenzij ze seksueel actief worden. Mannen die een vasectomie hebben ondergaan, hoeven geen anticonceptiemethode te gebruiken indien ten minste 16 weken na de procedure.
    5. Bereid en in staat om het protocol en de studieprocedure na te leven.
    6. Bereid om het huidige dieet onveranderd voort te zetten tijdens deelname aan het onderzoek (tenzij specifiek geïnstrueerd om tijdens het onderzoek van dieet te veranderen door de onderzoeker).
    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. De betrokkene is naar het oordeel van de onderzoeker niet bereid of niet in staat zich te houden aan de eisen van de studie.
    2. Onvermogen om orale medicatie te verdragen.
    3. Een vrouw die zwanger is of borstvoeding geeft, of zwangerschap overweegt.
    4. Ernstige neuropsychiatrische ziekte (bijv. ernstige depressie) die momenteel niet onder medische behandeling valt, die naar de mening van de onderzoeker of sponsor zou interfereren met het vermogen van de proefpersoon om deel te nemen aan het onderzoek of het risico op deelname daarvoor te vergroten.
    5. Medische voorgeschiedenis en/of bewijs van nierinsufficiëntie en/of aandoening, waaronder matige/ernstige nierinsufficiëntie (glomerulaire filtratiesnelheid [GFR] <60 ml/min) zoals meest recent geschat tijdens kwalificerende deelname aan een feederonderzoek) en/of onder behandeling van een nefroloog.
    6. Elke andere voorwaarde die naar de mening van de onderzoeker of sponsor zou interfereren met het vermogen van de proefpersoon om aan het onderzoek deel te nemen of het risico op deelname voor de proefpersoon.
    7. Vereiste voor gelijktijdige behandeling met een geneesmiddel waarvan bekend is dat het de folaatsynthese remt (bijv. methotrexaat).
    8. Gelijktijdige behandeling met BH4-suppletie (bijv. sapropterinedihydrochloride, KUVAN) of 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.
    Veiligheid is een primair eindpunt: de veiligheid van langdurige behandeling met PTC923 zal worden gemeten door:
    - aantal op de behandeling optredende bijwerkingen (TEAE's), inclusief beoordeling van de ernst van TEAE's
    - klinische laboratoriumtests
    - vitale functies
    - lichamelijk onderzoek

    Het primaire werkzaamheidseindpunt is de verandering ten opzichte van baseline in Phe/eiwitconsumptie via de voeding gemeten tijdens de Phe Tolerance Assessment periode.
    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
    Veiligheid is een primair eindpunt: de veiligheid van langdurige behandeling met PTC923 zal worden gemeten door:
    - aantal op de behandeling optredende bijwerkingen (TEAE's), inclusief beoordeling van de ernst van TEAE's
    - klinische laboratoriumtests
    - vitale functies
    - lichamelijk onderzoek

    Phe-tolerantie geëvalueerd bij baseline en elke 2 weken gedurende een beoordelingsperiode van 26 weken.
    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
    • Veranderingen ten opzichte van baseline in QOL met behulp van PKU-QOL-vragenlijst in de subset van proefpersonen die de PKU-QOL kunnen voltooien (dwz proefpersonen waarvan de primaire taal Engels [Brits of Amerikaans], Turks, Nederlands, Duits, Spaans, Italiaans, Portugees of Frans) (leeftijd 6 tot 8 jaar: Ouder PKU-QOL; leeftijd 9 tot 11 jaar: Kind PKU-QOL; 12 tot 17 jaar: Adolescent PKU-QOL; leeftijden ≥18 jaar: volwassen PKU-QOL)
    • Veranderingen ten opzichte van baseline in QOL met behulp van de EQ-5D (EQ-5D-Y Proxy Version 1, [3 tot 7 jaar]; EQ-5D-Y [8 tot 15 jaar]; EQ-5D-5L ([≥16 jaar])
    •PK-beoordeling van sepiapterine- en BH4-concentraties in plasma met behulp van steekproeven
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary measures will be evaluated at 6-month intervals starting at M8D1.
    Secundaire metingen worden geëvalueerd met tussenpozen van 6 maanden vanaf M8D1.
    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 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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.8.5The 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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    LBLP (laatste bezoek laatste patiënt)
    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 months
    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 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: 80
    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 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
    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
    Geïnformeerde toestemming en instemming (indien nodig, naar goeddunken van de onderzoeker [dwz voor kinderen en/of personen met een verstandelijke beperking als gevolg van ziekte]) met toestemming van de ouder/wettelijke voogd
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    0-18 years
    0-18 jaar
    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 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
    geen
    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 Decision2022-09-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-04-26
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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