Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43974   clinical trials with a EudraCT protocol, of which   7312   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2021-000497-28
    Sponsor's Protocol Code Number:PTC923-MD-004-PKU
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-04-04
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2021-000497-28
    A.3Full title of the trial
    Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria
    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
    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
    Enfermedades Metabólicas - fenilcetunuria
    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 fenilcetonuria (PKU) es un error congénito autosómico-recesivo del metabolismo caracterizado por la deficiencia de la enzima fenilalanina hidroxilasa (HAP), que metaboliza la fenilalanina (Phe
    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
    • Evaluar la seguridad a largo plazo de PTC923 en pacientes con fenilcetonuria (FCU).
    • Evaluar las variaciones con respecto al valor inicial en el consumo de fenilalanina (FA)/proteínas en la dieta.
    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
    •Evaluar el efecto de PTC923 en la calidad de vida(CdV)mediante el cuestionario sobre la calidad de vida en la fenilcetonuria(PKU-QOL)en el subgrupo de sujetos que pueden completar el cuestionario PKUQOL(sujetos cuyo idioma principal es inglés [británico o americano],turco,neerlandés,alemán,español,italiano,portugués o francés)(pacientes con edades de 6 a 8 años,cuestionario PKU-QOL para padres;pacientes con edades de 9 a 11 años,cuestionario PKU-QOL para niños;pacientes con edades de 12 a 17 años,cuestionario PKU-QOL para adolescentes;pacientes con ≥18 años de edad,cuestionario PKU-QOL para adultos).
    •Evaluar el efecto de PTC923 en la CdV utilizando el cuestionario europeo de calidad de vida de 5 dimensiones (EQ-5D)(EQ-5D para jóvenes [EQ-5D for Youth, EQ-5D-Y] respondido por sus cuidadores, V. 1 [de 3 a 7 años];EQ-5D-Y [de 8 a 15 años];EQ-5D-5L [≥16 años]).
    •Evaluar la farmacocinética de la sepiapterina y la tetrahidrobiopterina tras la administración de dosis repetidas de 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. Consentimiento y asentimiento informado (si es necesario, a criterio del investigador [p. ej., para niños y/o pacientes con deterioro mental secundario a la enfermedad]) con el consentimiento de los padres/tutores legales.
    2. Pacientes que hayan completado un estudio clínico de fase III sobre la FCU patrocinado por PTC.
    3. Las mujeres con capacidad de procrear, según se define en (CTFG 2020), deben tener un resultado negativo en una prueba de embarazo en el momento de entrada en el estudio y aceptar la abstinencia sexual o el uso de al menos un método anticonceptivo de granPTC923-MD-004-PKU Protocolo clínico V4.0 07-02-2022
    V4.0 07-02-2022 PTC Therapeutics. - Confidencial Página 7 de 9 eficacia (con una tasa de fracaso de <1 % al año cuando se usan de forma sistemática y correcta):
    • Anticonceptivos hormonales combinados (con estrógenos y gestágenos) asociados a la inhibición de la ovulación:
    − Orales
    − Intravaginales
    − Transdérmicos
    • Anticonceptivos hormonales únicamente con gestágenos asociados a la inhibición de la ovulación:
    − Orales
    − Inyectables
    − Implantables
    • Dispositivo intrauterino
    • Sistema intrauterino liberador de hormonas
    • Pareja vasectomizada con azoospermia confirmada
    Los métodos anticonceptivos de gran eficacia o la abstinencia deben mantenerse durante todo el estudio y hasta 90 días después de la última dosis del fármaco del estudio.
    Todas las mujeres se considerarán fértiles a menos que sean posmenopáusicas (al menos 12 meses consecutivos de amenorrea en el grupo de edad correspondiente sin otra causa conocida o sospechada) o hayan sido esterilizadas quirúrgicamente (por ejemplo, histerectomía, ligadura de trompas bilateral, ovariectomía bilateral).
    4. Los varones sexualmente activos con parejas femeninas con capacidad de procrear que no se hayan sometido a una vasectomía deben aceptar el uso de un método anticonceptivo de barrera durante el estudio y hasta 90 días después de la última dosis del medicamento del estudio. Los varones también deben abstenerse de donar semen durante este periodo de tiempo.
    Los varones que practiquen la abstinencia no estarán obligados a utilizar un método
    anticonceptivo a menos que sean sexualmente activos. Los varones que se hayan
    sometido a una vasectomía no están obligados a utilizar un método anticonceptivo si han pasado al menos 16 semanas desde la intervención.
    5. Disposición y capacidad para cumplir el protocolo y los procedimientos del estudio.
    6. Disposición para seguir con la dieta actual sin cambios durante la participación en el estudio (a menos que el investigador indique específicamente un cambio en la dieta durante el estudio)
    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. El sujeto, en opinión del investigador, no está dispuesto a cumplir los requisitos del estudio o es incapaz de hacerlo.
    2. Incapacidad para tolerar la medicación oral.
    3. Mujeres embarazadas o en periodo de lactancia, o que estén pensando en quedarse embarazadas.
    4. Enfermedad neuropsiquiátrica grave (p. ej., depresión mayor) que actualmente no está bajo control médico, que, en opinión del investigador o del promotor, podría interferir en la capacidad del paciente para participar en el estudio o aumentar el riesgo de la participación en ese paciente.
    5. Antecedentes médicos y/o indicios de alteración y/o afección renal, incluida
    insuficiencia renal moderada/grave (filtración glomerular [FG] <60 ml/min según lo
    calculado más recientemente durante la cualificación para la participación en un estudio previo) y/o atendidos por un nefrólogo.
    6. Cualquier otra afección que, en opinión del investigador o del promotor, pudiera
    interferir en la capacidad del paciente para participar en el estudio o aumentar el riesgo de la participación en ese paciente.
    7. Necesidad de tratamiento concomitante con cualquier fármaco conocido por inhibir la síntesis del folato (p. ej., metotrexato).
    8. Tratamiento concomitante con un suplemento de BH4 (p. ej., dihidrocloruro de
    sapropterina, KUVAN) o pegvaliasa-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 seguridad es un criterio principal de valoración: la seguridad del tratamiento a largo plazo con PTC923 se determinará mediante el número de acontecimientos adversos aparecidos durante el tratamiento (AAT), incluida la evaluación de la intensidad de los AAT, los análisis clínicos, las constantes vitales y las exploraciones físicas.
    El criterio principal de valoración de la eficacia es la variación con respecto al inicio en el consumo de FA/proteínas en la dieta, medido durante el periodo de evaluación de la tolerancia a la FA
    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 seguridad es un criterio principal de valoración: la seguridad del tratamiento a largo plazo con PTC923 se determinará mediante el número de acontecimientos adversos aparecidos durante el tratamiento (AAT), incluida la evaluación de la intensidad de los AAT, los análisis clínicos, las constantes vitales y las exploraciones físicas.
    El criterio principal de valoración de la eficacia es la variación con respecto al inicio en el consumo de FA/proteínas en la dieta, medido durante el periodo de evaluación de la tolerancia a la FA
    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
    • Variaciones con respecto al inicio en la calidad de vida determinada mediante el
    cuestionario PKU-QOL en el subgrupo de sujetos que pueden completar el cuestionario PKU-QOL (es decir, sujetos cuyo idioma principal es inglés [británico o americano], turco, neerlandés, alemán, español, italiano, portugués o francés) (en pacientes con edades de 6 a 8 años, cuestionario PKU-QOL para padres; en pacientes con edades de 9 a 11 años, cuestionario PKU-QOL para niños; en pacientes con edades de 12 a 17 años, cuestionario PKU-QOL paraPTC923-MD-004-PKU
    Protocolo clínico V4.0 07-02-2022 V4.0 07-02-2022 PTC Therapeutics. - Confidencial Página 9 de 9 adolescentes; en pacientes con ≥18 años de edad, cuestionario PKU-QOL para adultos).
    • Variaciones respecto al inicio en la CdV determinada mediante el cuestionario
    EQ-5D (EQ-5D-Y respondido por sus cuidadores, versión 1 [de 3 a 7 años]; EQ-5D-Y [de 8 a 15 años]; EQ-5D-5L [≥16 años]).
    • Evaluación de la FC de las concentraciones de sepiapterina y BH4 en plasma
    mediante muestreo disperso.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary measures will be evaluated at 6-month intervals starting at M8D1.
    Los criterios secundarios de valoración se evaluarán en intervalos de 6 meses a partir del D1M8
    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 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 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
    Georgia
    Mexico
    Turkey
    United States
    Denmark
    France
    Germany
    Italy
    Portugal
    United Kingdom
    Netherlands
    Spain
    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
    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 days
    E.8.9.2In all countries concerned by the trial years3
    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
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    0-18 years
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    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
    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 Decision2022-06-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-19
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA