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    Summary
    EudraCT Number:2021-001628-16
    Sponsor's Protocol Code Number:VX20-445-126
    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:2021-07-20
    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 number2021-001628-16
    A.3Full title of the trial
    A Phase 3b Open-label Study Evaluating the Effects of Elexacaftor/Tezacaftor/Ivacaftor on Cough and Physical Activity in Cystic Fibrosis Subjects 12 Years of Age and Older Who Are Heterozygous for the F508delMutation and a Minimal Function Mutation (F/MF)
    Estudio en fase IIIb, abierto, para evaluar los efectos de elexacaftor/tezacaftor/ivacaftor en la tos y la actividad física en pacientes con fibrosis quística de 12 años de edad en adelante heterocigóticos para la mutación F508del y una mutación de la función mínima (F/MF)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Subjects with Cystic Fibrosis (CF)
    Estudio para evaluar ELX/TEZ/IVA en la tos y la actividad física en pacientes con fibrosis quística (FQ)
    A.4.1Sponsor's protocol code numberVX20-445-126
    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 SponsorVertex Pharmaceuticals Incorporated
    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 supportVertex Pharmaceuticals Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVertex Pharmaceuticals Incorporated
    B.5.2Functional name of contact pointClinical Trials and Medical Info
    B.5.3 Address:
    B.5.3.1Street Address50 Northern Avenue
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02210-1862
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1877 634 8789
    B.5.5Fax number+1510 595 8183
    B.5.6E-mailmedicalinfo@vrtx.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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/18/2116
    D.3 Description of the IMP
    D.3.1Product name100 mg ELX/50 mg TEZ/75 mg IVA fixed dose combination
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNTezacaftor
    D.3.9.1CAS number 1152311-62-0
    D.3.9.2Current sponsor codeVX-661
    D.3.9.3Other descriptive nameTEZ
    D.3.9.4EV Substance CodeSUB188271
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNElexacaftor
    D.3.9.1CAS number 2216712-66-0
    D.3.9.2Current sponsor codeVX-445
    D.3.9.3Other descriptive nameELX
    D.3.9.4EV Substance CodeSUB193216
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIVACAFTOR
    D.3.9.1CAS number 873054-44-5
    D.3.9.2Current sponsor codeVX-770
    D.3.9.3Other descriptive nameIVA
    D.3.9.4EV Substance CodeSUB33103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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 Yes
    D.2.1.1.1Trade name Kalydeco 150 mg film-coated tablets
    D.2.1.1.2Name of the Marketing Authorisation holderVertex Pharmaceuticals (Ireland) Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product name150mg IVACAFTOR
    D.3.2Product code VX-770
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNIVACAFTOR
    D.3.9.1CAS number 873054-44-5
    D.3.9.2Current sponsor codeVX-770
    D.3.9.3Other descriptive nameIVA
    D.3.9.4EV Substance CodeSUB33103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    Cystic Fibrosis
    Fibrosis Quística
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis
    Fibrosis Quística
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 ELX/TEZ/IVA on cough and physical activity using wearable technology
    Evaluar los efectos de elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) en la tos y la actividad física mediante tecnología portátil.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF), and, when appropriate, an assent form.
    2.Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
    3.Subjects (male and female) 12 years of age or older on the date of informed consent.
    4.Subjects heterozygous for F508del and an MF mutation (F/MF genotypes, see Protocol Appendix A. for definition and non-exhaustive list of eligible MF mutations).
    a. Genotype should be confirmed at the Screening Visit.
    b.If the screening CFTR genotype result is not received before the first dose of study drug, a previous CFTR genotype laboratory report may be used to establish eligibility.
    c. Subjects who have been enrolled and whose screening genotype does not confirm study eligibility must be discontinued from the study (Protocol Section9.9).
    5.Forced expiratory volume in 1 second (FEV1) value ≥30% and ≤90% of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI]) at the Screening Visit (spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria for acceptability and repeatability) and stable CF disease as judged by the investigator.
    6.Willing to remain on a stable CF treatment regimen (other than CFTR modulators) through completion of study participation.
    1.El paciente (o su representante legalmente designado y autorizado) firmará y fechará un formulario de consentimiento informado (FCI) y, cuando proceda, un formulario de asentimiento.
    2.Está dispuesto y es capaz de cumplir con las visitas programadas, el plan de tratamiento, las restricciones del estudio, las pruebas analíticas, las pautas anticonceptivas y otros procedimientos del estudio.
    3.Pacientes (hombres y mujeres) con 12 años o más en la fecha del consentimiento informado.
    4.Pacientes heterocigotos para la mutación F508del y una mutación de FM (genotipos F/FM, véase el Anexo A para consultar la definición y la lista no exhaustiva de mutaciones de FM aptas).
    a.El genotipo debe confirmarse en la visita de selección.
    b.Si el resultado del genotipo de CFTR en la selección no se recibe antes de la primera dosis del fármaco del estudio, se podrá usar un informe de laboratorio previo del genotipo de CFTR para determinar la elegibilidad.
    c.Los pacientes que hayan sido incluidos y cuyo genotipo en la selección no confirme la elegibilidad para el estudio deben ser retirados del mismo (Sección 9.9).
    5.El valor del volumen espiratorio forzado en 1 segundo (VEF1) es ≥30 % y ≤90 % de la media prevista para la edad, el sexo y la estatura (ecuaciones de la Iniciativa Mundial de la Función Pulmonar [Global Lung Function Initiative, GLI])9 en la visita de selección (las mediciones de la espirometría deben cumplir los criterios de la Sociedad Torácica Estadounidense/Sociedad Respiratoria Europea [American Thoracic Society/European Respiratory Society])10 para la aceptabilidad y la repetibilidad) y con enfermedad de FQ estable según el criterio del investigador.
    6.Dispuesto a seguir con una pauta de tratamiento estable para la FQ (distinta de moduladores de CFTR) hasta la finalización de la participación en el estudio.
    E.4Principal exclusion criteria
    1.History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This includes, but is not limited to, the following:
    •Clinically significant liver cirrhosis with or without portal hypertension
    •Solid organ or hematological transplantation (or on a transplant list)
    •Alcohol or drug abuse in the past year, including, but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator
    •Cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage0 cervical carcinoma in situ (all3 with no recurrence for the last 5years)
    •Non-ambulatory status
    2.Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (as deemed by the investigator).
    3.Any of the following abnormal laboratory values at screening:
    •Hemoglobin <10 g/dL
    •Total bilirubin ≥2 × upper limit of normal (ULN)
    •Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) ≥3×ULN
    •Abnormal renal function defined as glomerular filtration rate ≤50mL/min/1.73m2(calculated by the Modification of Diet in Renal Disease Study Equation) for subjects ≥18years of age and ≤45mL/min/1.73m2(calculated by the Counahan-Barratt equation) for subjects <18 years of age.
    4.An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug).
    5.Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For subjects who have had a history of a positive culture, the investigator will apply the following criteria to establish whether the subject is free of infection with such organisms:
    •The subject has not had a respiratory tract culture positive for these organisms within the 12months before the date of informed consent.
    •The subject has had at least 2respiratory tract cultures negative for such organisms within the 12months before the date of informed consent, with the first and last of these separated by at least 3months, and the most recent one within the 6months before the date of informed consent.
    6.An acute illness not related to CF (e.g., gastroenteritis) within 14 days before the first dose of study drug (Day 1).
    7.Ongoing or prior participation in an investigational drug study within 28days of the Screening Visit.
    •A washout period of 5terminal half-lives of the previous investigational study drug, or 28 days, whichever is longer, must elapse before the Screening Visit.
    •The duration of the elapsed time may be longer if required by local regulations.
    8.Use of restricted medication within specified duration before the first dose of study drug as defined in Protocol Table 9-1.
    9.Pregnant and breast-feeding females. All female subjects regardless of childbearing potential (Protocol Section11.4.6) must have a negative pregnancy test at the Screening Visit and the Day1 Visit.
    10.The subject or a close relative of the subject is the investigator or a sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. However, an adult (aged 18 years or older) who is a relative of a study staff member may be enrolled in the study provided that
    •the adult lives independently of and does not reside with the study staff member, and
    •the adult participates in the study at a site other than the site at which the family member is employed.
    1.Antecedentes de una enfermedad o afección que, en opinión del investigador, podrían confundir los resultados del estudio o suponer un riesgo adicional al administrar el fármaco o fármacos del estudio al paciente. Esto incluye, entre otras, las siguientes:
    •Cirrosis hepática clínicamente significativa con o sin hipertensión portal.
    •Trasplante hematológico o de órgano sólido (o está en la lista de trasplantes).
    •Abuso de alcohol o drogas en el último año, incluidas, entre otros, cannabis, cocaína y opiáceos, según el criterio del investigador.
    •Cáncer, excepto cáncer de piel epidermoide, cáncer de piel basocelular y carcinoma cervicouterino in situ en estadio 0 (los 3 sin recidiva durante los últimos 5 años).
    •Estado no ambulatorio.
    2.Cualquier anomalía en el análisis clínicamente significativa en la visita de selección que podría interferir en las evaluaciones del estudio o suponer un riesgo innecesario para el paciente (según lo determine el investigador).
    3.Cualquiera de los siguientes valores analíticos anómalos en la selección:
    •Hemoglobina <10 g/dl.
    •Bilirrubina total ≥2 × límite superior de la normalidad (LSN).
    •Aspartato aminotransferasa (AST), alanina aminotransferasa (ALT), gamma glutamil transferasa (GGT) o fosfatasa alcalina (FA) ≥3 × LSN.
    •Función renal anómala definida como un índice de filtración glomerular ≤50 ml/min/1,73 m2 (calculado por la ecuación del estudio de modificación de la dieta en la enfermedad renal)11, 12 para pacientes ≥18 años de edad y ≤45 ml/min/1,73 m2 (calculado por la ecuación de Counahan-Barratt)13 para pacientes <18 años de edad.
    4.Una infección aguda de las vías respiratorias superiores o inferiores, exacerbación pulmonar o cambios en el tratamiento (incluidos antibióticos) para la enfermedad pulmonar en los 28 días previos al día 1 (primera dosis del fármaco del estudio).
    5.Infección pulmonar por microorganismos asociados a un deterioro más rápido del estado pulmonar (incluidos, entre otros, Burkholderia cenocepacia, Burkholderia dolosa y Mycobacterium abscessus). En el caso de pacientes con antecedentes de cultivo positivo, el investigador aplicará los siguientes criterios para determinar si el paciente está libre de infección por dichos microorganismos:
    •El paciente no ha tenido un cultivo positivo de las vías respiratorias para estos microorganismos en los 12 meses anteriores a la fecha del consentimiento informado.
    •El paciente ha tenido al menos 2 cultivos negativos de las vías respiratorias para dichos microorganismos en los 12 meses anteriores a la fecha del consentimiento informado, con el primero y el último de ellos separados por al menos 3 meses, y el más reciente en los 6 meses anteriores a la fecha del consentimiento informado.
    6.Una enfermedad aguda no relacionada con la FQ (p. ej., gastroenteritis) en los 14 días anteriores a la primera dosis del fármaco del estudio (día 1).
    7.Participación previa o en curso en un estudio con un fármaco en investigación en los 28 días previos a la visita de selección.
    •Debe transcurrir un periodo de reposo farmacológico de 5 semividas terminales del fármaco en investigación previo o 28 días, lo que sea más largo, antes de la visita de selección.
    •El tiempo transcurrido puede ser mayor si así lo requiere la normativa local.
    8.Uso de medicamentos restringidos dentro de la duración especificada antes de la primera dosis del fármaco del estudio, tal como se define en la Tabla 9 1.
    9.Mujeres embarazadas y en periodo de lactancia. Todas las pacientes independientemente de su capacidad de concebir (Sección 11.4.6) deben tener una prueba de embarazo negativa en la visita de selección y en la visita del día 1.
    10.El paciente o un pariente cercano del paciente es el investigador o un subinvestigador, asistente de investigación, farmacéutico, coordinador del estudio u otro miembro del personal implicado directamente en la realización del estudio en ese centro. Sin embargo, un adulto (con 18 años o más) que sea pariente de un miembro del personal del estudio puede participar en el estudio siempre que
    •el adulto viva independiente del miembro del personal del estudio y no resida con él, y
    •el adulto participe en el estudio en un centro que no sea el centro en el que trabaja su pariente.
    E.5 End points
    E.5.1Primary end point(s)
    Percent reduction from baseline in cough frequency (cough events per day) to the average of Week 8 through Week 12
    Porcentaje de reducción de la frecuencia de la tos (episodios de tos por día) con respecto a la media de la semana 8 a la semana 12
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to week 12
    Desde el inicio hasta la semana 12
    E.5.2Secondary end point(s)
    Absolute change from baseline in total step count per day to the average of Week 8 through Week 12
    Cambio absoluto desde la referencia en el recuento total de pasos por día hasta la media de la semana 8 a la semana 12
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline to Week 12
    Desde el inicio hasta la semana 12
    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 No
    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) 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    Belgium
    Canada
    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
    last scheduled visit (or scheduled contact) of the last subject / Protocol Section 13.2.9
    última visita programada (o contacto programado) del último paciente / sección 13.2.9 del protocolo
    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 months6
    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 months6
    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: 24
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 24
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 75
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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
    Subjects under the age of being capable of giving consent personally
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 100
    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)
    Subjects who are eligible will be offered the opportunity to enroll in an open-label extension (OLE) safety study.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation ECFS Clinical Trial Network
    G.4.3.4Network Country Belgium
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-07-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-28
    P. End of Trial
    P.End of Trial StatusOngoing
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