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    Summary
    EudraCT Number:2018-004405-64
    Sponsor's Protocol Code Number:MT-2-03
    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:2019-01-24
    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 number2018-004405-64
    A.3Full title of the trial
    A Double-Blind, Placebo-Controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophysiological, and Clinical Markers in Patients with Friedreich’s Ataxia
    Estudio Doble Ciego y Controlado con Placebo sobre los Efectos de MIN-102 en Marcadores Bioquímicos, de Estudios de Imagen, Neurofisiológicos y Clínicos en Pacientes con Ataxia de Friedreich.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study comparing the effect of MIN-102 and an inactive substance to learn more about the effects of the MIN-102 on Biochemical, Imaging, Neurophysiological, and Clinical Markers in Patients with Friedreich’s Ataxia, in which neither investigators nor subjects know what treatment the subject is receiving.
    Estudio que compara el efecto del MIN-102 y una sustancia inactiva para saber más sobre los efectos del MIN-102 en Marcadores Bioquímicos, de Estudios de Imagen, Neurofisiológicos y Clínicos en Pacientes con Ataxia de Friedreich, en los que ni los investigadores ni los sujetos saben qué tratamiento estan recibiendo.
    A.4.1Sponsor's protocol code numberMT-2-03
    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 SponsorMinoryx Therapeutics BE, SA
    B.1.3.4CountryBelgium
    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 supportMinoryx Therapeutics S.L.
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMinoryx Therapeutics S.L.
    B.5.2Functional name of contact pointSílvia Pascual
    B.5.3 Address:
    B.5.3.1Street AddressAv. Ernest Lluch 32, TCM3
    B.5.3.2Town/ cityMataró (Barcelona)
    B.5.3.3Post code08302
    B.5.3.4CountrySpain
    B.5.4Telephone number0034935441466
    B.5.5Fax number0034930160119
    B.5.6E-mailspascual@minoryx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product name5-[[4-[2-[5-(1-Hydroxyethyl)-2-pyridinyl]ethoxy]phenyl]methyl]-2,4-thiazolidinedione hydrochloride
    D.3.2Product code MIN-102
    D.3.4Pharmaceutical form 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.3.8INN - Proposed INN5-[[4-[2-[5-(1-Hydroxyethyl)-2-pyridinyl]ethoxy]phenyl]methyl]-2,4-thiazolidinedione hydrochloride
    D.3.9.1CAS number 146062-46-6
    D.3.9.2Current sponsor codeMIN-102
    D.3.9.3Other descriptive nameHydroxypioglitazone hydrochloride
    D.3.9.4EV Substance CodeSUB16466MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral suspension
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Friedreich’s Ataxia
    Ataxia de Friedreich
    E.1.1.1Medical condition in easily understood language
    Friedreich’s Ataxia
    Ataxia de Friedreich
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10017374
    E.1.2Term Friedreich's ataxia
    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 effect of 48 weeks of treatment with MIN-102 compared to placebo on cervical spinal cord area as assessed by morphometric MRI measurements.
    El objetivo principal del estudio es evaluar el efecto de 48 semanas de tratamiento con MIN-102 en comparación con placebo en la zona de la médula espinal cervical evaluada mediante mediciones morfométricas por RM.
    E.2.2Secondary objectives of the trial
    To evaluate the effect of 48 wks of treatment with MIN-102 compared to placebo on the following:
    -Change from baseline in the total score of the SARA
    -Cervical spinal cord mean, axial and radial diffusivity as assessed by MRI DTI
    -Cervical spinal cord tNAA/mIns ratio as assessed by MRS
    -Brain fiber density, fiber cross-section and fiber density and cross-section as assessed by fixel-based analyses
    -Cerebellar Composite Functional Scale, composed of 2 functional tests: 9 hole peg test and clicking
    -EQ-5D-5L
    -Fatigue Severity Scale
    -Activities of daily living (FARS)
    -Clinician and patient global impressions of improvement
    -Change in the pre-treatment versus post-treatment slope of the SARA (exploratory objective)
    -Neurophysiological parameters as assessed by motor evoked potentials (MEP). Optional assessment (exploratory objective)
    -Various biochemical parameters in plasma, and CSF. Optional assessment. (exploratory objective)
    -Safety and tolerability of MIN-102
    Los objetivos secundarios del estudio son evaluar el efecto de 48 semanas de tratamiento con MIN-102 comparado con placebo en los siguientes valores:
    • Variación respecto al periodo basal en la puntuación total de SARA
    • Difusividad media axial y radial de la médula espinal cervical evaluadas por RM con tensor de difusión
    • Relación tNAA/mIns de la médula espinal cervical evaluada mediante ERM
    • Densidad de las fibras cerebrales, sección transversal de las fibras y densidad de las fibras y sección transversal evaluadas mediante análisis basados en píxeles (FBA)
    • Escala funcional compuesta cerebelosa (Cerebellar Composite Functional Scale, CCFS), formada por 2 pruebas funcionales: Prueba de clavijas con 9 orificios y de hacer clic
    • Cuestionario europeo de calidad de vida con cinco dimensiones (EQ-5D-5L).
    • Escala de intensidad de la fatiga (Fatigue Severity Scale, FSS)
    • Actividades de la vida cotidiana (FARS])
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed Informed Consent by the subject or parent/legal guardian, and/or consent or assent for minors as required by national laws.
    2. Male and female subjects aged ≥12 and ≤60 years, inclusive, with Friedreich’s Ataxia confirmed by genetic testing.
    3. Total score on SARA of <25.
    4. Score on SARA item 1 (gait) ≥2 and ≤6.
    5. Male with a potentially fertile partner must be willing to use an acceptable method of contraception for the duration of the study and for 3 months after study drug discontinuation (acceptable methods are: use of a condom with spermicide or use of oral, implantable or injectable contraceptives, or intrauterine devices, or a diaphragm with spermicide or diaphragm with condom) or have had a vasectomy.
    6. Female with childbearing potential must be willing to use highly effective contraceptive methods during screening, during the period of drug administration and for 30 days after stopping study drug administration. Highly effective contraception methods include the following:
    ▪ Total abstinence (defined as refraining from heterosexual intercourse during the entire period outlined above).
    ▪ Male or female sterilization. (bilateral tubal occlusion or vasectomized partner)
    ▪ Use of at least one of the following:
    a. Use of oral, injectable, transdermal, intravaginal, or implantable hormonal methods of contraception
    ▪ combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: Oral, intravaginal or transdermal.
    ▪ progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable or implantable
    b. Placement of an intrauterine device (IUD) or intrauterine system (IUS)
    7. Female patients of childbearing potential must have a negative serum pregnancy test result within 7 days before first administration of study drug.
    1. Consentimiento informado firmado por el sujeto o su progenitor o tutor legal, o consentimiento o asentimiento en menores, según las leyes nacionales.
    2. Sujetos de ambos sexos, con edades comprendidas entre los 12 y los 60 años (ambas edades inclusive) con ataxia de Friedreich confirmada mediante pruebas genéticas.
    3. Puntuación total de SARA de <25.
    4. Puntuación en el ítem 1 (marcha) de SARA ≥2 y ≤6.
    5. El varón con una pareja potencialmente fértil debe haberse sometido a vasectomía o estar dispuesto a utilizar un método anticonceptivo aceptable durante todo el estudio y los 3 meses posteriores a la suspensión del fármaco del estudio (los métodos aceptables son: el uso de un preservativo con espermicida o el uso de anticonceptivos orales, implantables o inyectables, dispositivos intrauterinos, o un diafragma con espermicida o diafragma con preservativo) o ha tenido una vasectomía.
    6. Las mujeres con capacidad de procrear deben estar dispuestas a utilizar métodos anticonceptivos muy eficaces durante la selección, durante el período de administración del fármaco y durante los 30 días siguientes a que deje de administrarse el fármaco del estudio. Son métodos anticonceptivos muy eficaces:
    - Abstinencia total (definida como no mantener relaciones heterosexuales durante todo el período descrito anteriormente)
    - Esterilización masculina o femenina, (ligadura de trompas bilateral, pareja vasectomizada)
    Uso de al menos uno de los siguientes:
    a. Uso de métodos anticonceptivos orales, inyectables, transdérmicos, intravaginales o implantables
    - anticonceptivos hormonales combinados (que contengan estrógenos y gestágenos) asociados a la inhibición de la ovulación: orales, intravaginales o transdérmicos
    - anticonceptivos hormonales de solo gestágenos asociados a la inhibición de la ovulación: orales, inyectables o implantables
    b. Colocación de un dispositivo intrauterino (DIU) o de un sistema intrauterino (SIU)
    7. Las pacientes de sexo femenino con capacidad de procrear deben presentar un resultado negativo en una prueba de embarazo en suero realizada en un plazo de 7 días antes de la primera administración del fármaco del estudio
    E.4Principal exclusion criteria
    1. Age of onset of disease ≥25 years
    2. Left ventricular ejection fraction (LVEF) <55%, on echocardiogram.
    3. Subjects with a history of heart failure, ventricular arrhythmia, supraventricular tachycardia, or having a QTcF time of >480 msec in 3 consecutive ECG recordings taken at least 5 minutes apart.
    4. Known intolerance to pioglitazone or other thiazolidinediones.
    5. Subjects who are taking or have taken pioglitazone, or other thiazolidinediones, within the past 6 months prior to Screening.
    6. Currently participating in or having participated in another interventional clinical study within 2 months prior to Screening.
    7. Requiring other prohibited concomitant medication (see Table 4). Note: use of idebenone, coenzyme Q10, antioxidants, riboflavin, thiamine, vitamins C and E is permitted provided the dose has been constant for ≥3 months before Screening and is kept constant during the entire study.
    8. Previous or current history of vesical polyps, bladder cell hyperplasia, or bladder cancer.
    9. Previous or current history of other cancer, unless surgically resected and without evidence of recurrence for a minimum of 5 years.
    10. Subjects with clinically significant anemia (i.e. hemoglobin below 110 g/L).
    11. Glycated hemoglobin (HbA1c) levels >6.4% and fasting blood glucose levels ≤ 0.9 times the lower limit of normal and ≥ 1.1 times the upper limit of normal.
    12. NT-proB-type natriuretic peptide level (BNP) >125 pg/mL
    13. Abnormal liver enzyme tests for aspartate aminotransferase or alanine aminotransferase of >2 times the upper limit of normal or total bilirubin >1.5 times the upper limit of normal (unless
    due to Gilbert’s syndrome).
    14. A positive result on laboratory tests for hepatitis B surface antigen, hepatitis C antibody and human immunodeficiency virus antibody at screening.
    15. Moderate or severe hepatic impairment (groups B and C according to the revised Child-Pugh classification; (Pugh, Murray-Lyon et al. 1973).
    16. Chronic kidney disease (CKD) stages 3a or higher (according to CKD staging by Renal Association with an estimated glomerular filtration rate of <60 ml/min/1.73m2).
    17. Contraindications for MRS/MRI procedure such as subjects with paramagnetic materials in the body, such as dental braces, spinal rods, aneurysm clips, pacemakers, intraocular metal or cochlear implants.
    18. Drug or alcohol abuse in the past 2 years by subject history and/or investigator assessment.
    19. Conditions which could modify the absorption of the study drug, such as inflammatory bowel diseases, or stomach or intestinal resection.
    20. Inability or unwillingness of the subject or subjects’ parents/caregivers to comply with the study procedures.
    21. Other medical, neurological, psychiatric, or social conditions that in the investigator’s opinion are likely to confound the assessment of safety or efficacy, interfere with study conduct, or unfavorably alter the risk-benefit of subject participation.
    22. Pregnant women as confirmed by a positive blood pregnancy test.
    23. Female patients intending to breast feed a child while taking study drug or have taken study drug within 30 days after administration of the last dose.
    1.Edad de inicio de la enfermedad ≥25 años
    2.Fracción de eyección del ventrículo izquierdo (FEVI) <55 %, en ecocardiograma.
    3.Antecedentes de insuficiencia cardíaca, arritmia ventricular, taquicardia supraventricular o tener un intervalo QTcF >480 ms en 3 registros consecutivos del ECG obtenidos con al menos 5 minutos de diferencia.
    4.Intolerancia conocida a la pioglitazona o a cualquier otra tiazolidinadiona.
    5.Estar tomando o haber tomado pioglitazona, u otras tiazolidinadionas, en los 6 meses anteriores a la selección.
    6.Estar participando actualmente o haber participado en otro estudio clínico intervencionista en los 2 meses anteriores a la selección.
    7.Precisar otros medicamentos concomitantes prohibidos (véase la Tabla 4). Nota: El uso de idebenona, coenzima Q10, antioxidantes, riboflavina, tiamina y vitaminas C y E está permitido siempre que la dosis haya sido constante durante ≥3 meses antes de la selección y se mantenga constante durante todo el estudio.
    8.Presencia actual o antecedentes de pólipos vesicales, hiperplasia de las células vesicales o cáncer vesical.
    9.Presencia o antecedentes de cualquier otro tipo de cáncer, a menos que se extirpado quirúrgicamente y no haya indicios de recaída durante un mínimo de 5 años.
    10.Anemia clínicamente significativa (es decir, hemoglobina por debajo de 110 g/l).
    11.Niveles de hemoglobina glucosilada (HbA1c) >6,4 % y glucemia sanguínea en ayunas ≤0,9 veces el límite inferior de la normalidad y ≥1,1 veces el límite superior de la normalidad.
    12.Nivel de propéptido natriurético de tipo B (NT-BNP) >125 pg/ml.
    13.Valores anómalos de las enzimas hepáticas aspartato-aminotransferasa o alanina-aminotransferasa >2 veces el límite superior de la normalidad o bilirrubina total >1,5 veces el límite superior de la normalidad (a menos que se deba a síndrome de Gilbert).
    14.Resultado positivo en las pruebas analíticas de detección del antígeno de superficie del virus de la hepatitis B, anticuerpos contra el virus de la hepatitis C y anticuerpos contra el virus de la inmunodeficiencia humana en la selección.
    15.Disfunción hepática moderada o grave (grupos B y C según la clasificación revisada de Child-Pugh; véase el Anexo 6.2) (Pugh, Murray-Lyon y cols., 1973).
    16.Nefropatía crónica (NC) en estadio 3a o superior (según la estadificación de la NC de la Asociación renal con una filtración glomerular estimada <60 ml/min/1,73 m2).
    17.Contraindicaciones para el procedimiento de ERM/RM; por ejemplo, si los sujetos tienen materiales paramagnéticos en el cuerpo, como aparatos de ortodoncia, varillas vertebrales, clips de aneurisma, marcapasos, implantes cocleares o intraoculares de metal.
    18.Alcoholismo o drogadicción en los últimos 2 años según los antecedentes del sujeto o la evaluación del investigador.
    19.Afecciones que podrían modificar la absorción del fármaco del estudio, como enfermedades inflamatorias intestinales o resección estomacal o intestinal.
    20.Incapacidad o falta de voluntad del sujeto o de los progenitores o cuidadores para cumplir con los procedimientos del estudio.
    21.Otros trastornos físicos, psiquiátricos o sociales que, en opinión del investigador, puedan influir en la interpretación de la evaluación de la seguridad o la eficacia, interferir en la realización del estudio o alterar de forma desfavorable la relación beneficio-riesgo de la participación del sujeto.
    22.Mujeres embarazadas según confirmación mediante un resultado positivo en un análisis hormonal.
    23.Mujeres que tengan previsto amamantar mientras reciben el fármaco del estudio o en los 30 días siguientes a la administración de la última dosis.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in spinal cord area cervical segment C2-C3 [mm2]
    Cambio desde el inicio del area del segmento cervical C2-C3 [mm2] en la medula espinal
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline, week 24 and week 48
    Periodo Basal, semana 24 y semana 48
    E.5.2Secondary end point(s)
    Efficacy endpoints include the change from baseline in:
    •Scale for Assessment and Rating of Ataxia:
    -Change from baseline in total score of the SARA
    •Diffusion tensor imaging fractional anisotropy: mean, radial and axial diffusivity cervical segments C2-C7 [10-3 mm2/s]
    •Magnetic resonance spectroscopy tNAA/mIns ratio in spinal cord segments C4-C5
    •Fixel-based analyses of the brain including fiber density, fiber cross-section and FDC
    •Cerebellar Composite Functional Scale
    •EQ-5D-5L
    •Fatigue Severity Scale (FSS)
    •Activities of Daily Living subscale of FARS
    •Clinical Global Impressions – Severity (CGI-S)
    •Clinical Global Impressions – Improvement (CGI-I)
    •Patient Global Impressions – Improvement (PGI-I)
    Exploratory;
    •Clinical; Change in slope from baseline of the SARA compared from pre-treatment to post-treatment
    •Neurophysiological ( Motor evoked potentials with central conduction time and amplitude as an optional assessment)
    •Biochemical
    - Adiponectin in plasma, and CSF (optional)
    - Fatty acid binding protein 4 in CSF (optional)
    - Gene expression of frataxin, PGC-1α, NRF1, TFAM in peripheral blood mononuclear cells
    - Neurofilament light chain in plasma, and CSF (optional)
    Safety;
    •AEs, SAEs & SUSARs
    •vital signs
    •12-lead ECG
    •echocardiogram
    •clinical laboratory tests (included Pregnancy Screen)
    •Physical examination
    •palatability
    • Escala de evaluación y clasificación de Ataxia
    - Variación respecto al periodo basal en la puntuación total de SARA
    • Anisotropía fraccional con imágenes tensoriales de difusión: difusividad media, radial y axial, segmentos cervicales C2-C7 [10-3 mm2/s]
    • Espectroscopia de Resonancia Magnetica tNAA/mlns relación en segmentos de la médula espinal C4-C5
    • Análisis basados en píxeles del cerebro incluido la densidad de las fibras, sección transversal de las fibras y FDC
    • Escala funcional compuesta cerebelosa
    • EQ-5D-5L
    • Escala de intensidad de la fatiga
    • Actividades de la vida cotidiana (subescala de la escala de valoración de la ataxia de Friedreich)
    • Impresiones clínicas globales - Gravedad (CGI-S)
    • Impresiones clínicas globales - Mejora (CGI-I)
    • Impresiones globales del paciente - Mejora (PGI-I)
    Objetivos Exploratorios:
    • Variación en la pendiente de SARA previa al tratamiento frente a la pendiente posterior al tratamiento
    • Neurofisiológicos (potenciales evocados motores con tiempo de conducción central y amplitud como una evaluación opcional)
    Bioquímicos:
    - Adiponectina en plasma y LCR (opcional)
    - Proteína de unión a ácidos grasos 4 en LCR (opcional)
    - Expresión génica de frataxina, PGC-1α, NRF1, TFAM en células mononucleares de sangre periférica
    - Cadena ligera de neurofilamento en plasma y LCR (opcional)
    Seguridad
    •AEs, SAEs & SUSARs
    • Constantes vitales
    • ECG de 12 derivaciones
    • Ecocardiograma
    • Pruebas de laboratorio clínico (incluido embarazo visita de selección)
    • Exploración física
    • Evaluación de la palatabilidad
    E.5.2.1Timepoint(s) of evaluation of this end point
    - All; Baseline, week 24 and week 48
    - CGI-I; Week 24 and week 48
    - PGI-I; week 24 and week 48
    - Safety: From screening, during the 48 weeks of treatment and 4 weeks after discontinuation
    - Palatability: Baseline, week 4, week 12, week 24 and week 36
    - Todos; visita de selección, semana 24 y semana 48.
    - CGI-I; semana 24 y semana 48
    - PGI-I; semana 24 y semana 48
    - Seguridad: desde visita de selección, durante las 48 semanas de tratamiento y 4 semanas despues de la interrupción
    - • Evaluación de la palatabilidad:
    Palatability: Periodo Basal, semana 4, semana 12, semana 24 y semana 36
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA4
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    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 years1
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months1
    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: 18
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 18
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 18
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 36
    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)
    Expected normal treatment of that condition
    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 Decision
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-06
    P. End of Trial
    P.End of Trial StatusOngoing
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