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    Summary
    EudraCT Number:2019-001656-19
    Sponsor's Protocol Code Number:RP-L301-0119
    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:2019-06-11
    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 number2019-001656-19
    A.3Full title of the trial
    Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD.
    Terapia génica para la deficiencia de piruvato quinasa (PKD): un ensayo clínico de fase I para evaluar la seguridad de la infusión de células CD34 + autólogas transducidas con un vector lentiviral que contiene el gen de piruvato quinasa de glóbulos rojos (CoRPK) con optimización de codones en sujetos adultos y pediátricos con PKD
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the safety of a gene therapy product for the treatment of Pyruvate Kinase Deficiency in adults and children
    Estudio para evaluar la seguridad de un producto de terapia génica para el tratamiento de la deficiencia de piruvato quinasa en adultos y niños
    A.4.1Sponsor's protocol code numberRP-L301-0119
    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 SponsorRocket Pharmaceuticals, 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 supportRocket Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRocket Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointChief Medical Officer
    B.5.3 Address:
    B.5.3.1Street Address350 5th Avenue, Suite 7530
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10118
    B.5.3.4CountryUnited States
    B.5.4Telephone number1646440-9100
    B.5.6E-mailjs@rocketpharma.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/3/14/1330
    D.3 Description of the IMP
    D.3.1Product nameMERILEN
    D.3.2Product code RP-L301
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMERILEN
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeN/A
    D.3.9.3Other descriptive nameN/A
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number4000000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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
    Pyruvate kinase deficiency
    Deficiencia de piruvato quinasa
    E.1.1.1Medical condition in easily understood language
    Inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cells
    Trastorno metabólico hereditario de la enzima piruvato quinasa que afecta a la supervivencia de los glóbulos rojos
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10037682
    E.1.2Term Pyruvate kinase deficiency anaemia
    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
    The primary objective of trial Phase 1 is to characterize the safety and toxicity associated with infusion of the investigational product: autologous CD34+ cells transduced with the therapeutic LV, PGK-coRPK-WPRE.
    El objetivo de este estudio es evaluar la seguridad y la toxicidad asociada con la perfusión de un producto en investigación: células autólogas CD34+ enriquecidas, transducidas con un vector lentiviral (LV) que porta el gen de la piruvato quinasa de glóbulos rojos con optimización de codones (coRPK), en sujetos con deficiencia de piruvato quinasa (PKD).
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. PKD diagnosis with a confirmed PKLR mutation.
    2. Age ≥18 years old and <45 years for the initial 2 patients enrolled; ≥12–17 years for the next 2 patients; ≥8–11 years for the final 2 patients.
    3. History of severe, transfusion-dependent anemia, defined as:
    a. At least 6 RBC transfusion episodes over a prior 12-month period, or at least 3 RBC transfusion episodes per year over 2 prior years (in the absence of precipitating events such as infection or surgery) and
    b. Hb levels <9.5 g/dL in the previous 12 months despite prior splenectomy.
    4. Adequate cardiac, pulmonary, renal and hepatic function, as detailed in relevant exclusion criteria.
    5. Availability of detailed medical records, including transfusion requirements, for at least the prior 2 years.
    6. Willing and able to read and correctly understand the patient information sheet and provide consent (or informed assent for minors) regarding study participation.
    7. Negative serum pregnancy test for female patients of childbearing potential.
    1. Diagnóstico de PKD con mutación en el gen PKLR confirmada.
    2. Edad ≥ 18 años y <45 años para los 2 pacientes iniciales reclutados; ≥12–17 años para los siguientes 2 pacientes; ≥8–12 años para los 2 últimos pacientes.
    3. Antecedentes de anemia grave dependiente de transfusión, definida como:
    a. Al menos 6 episodios de transfusión de glóbulos rojos durante un período de 12 meses anterior, o al menos 3 episodios de transfusión de glóbulos rojos por año durante 2 años anteriores (en ausencia de eventos desencadenantes como infección o cirugía) y
    b. Niveles de Hb <9.5 g/dL en los 12 meses anteriores a pesar de una esplenectomía previa.
    4. Función cardíaca, pulmonar, renal y hepática adecuada, según se detalla en los criterios de exclusión pertinentes.
    5. Disponibilidad de registros médicos detallados, incluidos los requisitos de transfusión, por lo menos durante los 2 años anteriores a la participación en el ensayo.
    6. Estar dispuesto y ser capaz de leer y comprender correctamente la hoja de información al paciente y proporcionar su consentimiento (o asentimiento informado a los menores) con respecto a la participación en el estudio.
    7. Prueba de embarazo en suero negativa para mujeres en edad fértil.
    E.4Principal exclusion criteria
    1. Presence of other known causes of hemolysis (in addition to PKD). Patients with concurrent G6PD deficiency diagnosed during pre-study evaluation may be considered for eligibility if in the opinion of the Investigator, the hemolytic anemia is the result of PKD and the G6PD deficiency is considered an incidental finding.
    2. A venous thromboembolic event (VTE; i.e., pulmonary embolism or deep vein thrombosis) or arteriothromboembolic event (ATE; including unstable angina, myocardial infarction, stroke or transient ischemic attack) during the prior 12 months.
    3. Any evidence of severe iron overload that, per Investigator discretion, warrants exclusion.
    4. Evidence of bridging fibrosis, cirrhosis or active hepatitis on liver biopsy. Liver biopsy is required when liver iron concentration (LIC) is ≥15 mg/g on T2* magnetic resonance imaging (MRI) of liver. If a liver biopsy has been performed less than 6 months prior to enrollment, it does not need to be repeated.
    5. Significant medical conditions including documented HIV infection, active viral hepatitis, poorly-controlled hypertension, pulmonary hypertension, cardiac arrhythmia or congestive heart failure; or ATEs (including stroke or myocardial infarction) within the 6 prior months.
    6. Active hematologic or solid organ malignancy, not including non-melanoma skin cancer or another carcinoma in situ. Patients with previously resected solid organ malignancies or definitively treated hematologic malignancies may be eligible if there has been no evidence of active malignancy during the prior 3 years.
    7. Uncontrolled seizure disorder.
    8. Cardiac T2*<10 ms by magnetic resonance imaging (MRI) or left ventricular ejection fraction (LVEF) <45% by echocardiogram or multiple gated acquisition scan (MUGA).
    9. Hepatic dysfunction as defined by:
    • ALT or AST >2.5× the ULN
    10. Renal dysfunction defined as serum creatinine >ULN. Patients with creatinine above ULN may be eligible pending documentation of a glomerular filtration rate (GFR) ≥60 mL/min/1.73m2 as calculated by Modification of Diet in Renal Disease equation, the revised Schwartz formula (for patients under 18 years old), or 24-hour urine collection.
    11. Pulmonary dysfunction as defined by either:
    • Need for supplemental oxygen during the prior 2 weeks (in absence of acute infection) or
    • Oxygen saturation (by pulse oximetry) <90%.
    12. Any medical or other contraindication for both leukapheresis and BM harvest procedure as determined by the treating Investigator.
    13. Any medical or psychiatric condition that in the opinion of the Investigator renders the subject unfit for trial participation or at higher than acceptable risk for participation.
    14. Poor functional status, evidenced by Karnofsky Index <70 in adults or Lansky <70 in children.
    15. Participation in another clinical trial with an investigational drug within 14 days before the informed consent signature. Participation in observational studies is allowed.
    16. Pregnant women or women with a positive serum pregnancy test at screening or breast feeding or planning to become pregnant within the next 24 months. Women not willing to use highly effective contraceptive methods during the complete study period.
    1. Presencia de otras causas conocidas de hemólisis (además de PKD). Los pacientes con deficiencia de G6PD concurrente diagnosticada durante la evaluación previa al estudio pueden considerarse elegibles si, en opinión del investigador, la anemia hemolítica es el resultado de la PKD y la deficiencia de G6PD se considera un hallazgo accesorio.
    2. Un evento tromboembólico venoso (VTE; es decir, embolia pulmonar o trombosis venosa profunda) o evento arteriotromboembólico (ATE; incluyendo angina inestable, infarto de miocardio, accidente cerebrovascular o ataque isquémico transitorio) durante los 12 meses anteriores.
    3. Cualquier evidencia de sobrecarga de hierro severa que, a criterio del Investigador, justifique la exclusión.
    4. Evidencia de fibrosis hepática, cirrosis o hepatitis activa en la biopsia de hígado. La biopsia hepática es necesaria cuando la concentración de hierro en el hígado (LIC) sea ≥15 mg/g en imágenes de resonancia magnética (IRM) T2* del hígado. Si se ha realizado una biopsia de hígado menos de 6 meses antes del reclutamiento, no será necesario repetirla.
    5. Afecciones médicas significativas que incluyen infección por VIH documentada, hepatitis viral activa, hipertensión mal controlada, hipertensión pulmonar, arritmia cardíaca o insuficiencia cardíaca congestiva; o ATEs (incluyendo apoplejía o infarto de miocardio) dentro de los 6 meses anteriores.
    6. Malignidad hematológica o de órgano sólido activa, sin incluir el cáncer de piel no melanoma u otro carcinoma in situ. Los pacientes con tumores malignos de órganos sólidos resecados previamente o tumores malignos hematológicos tratados definitivamente pueden ser elegibles si no ha habido evidencia de cáncer activo durante los 3 años anteriores.
    7. Trastorno convulsivo no controlado.
    8. T2* cardíaco <10 ms mediante imágenes de resonancia magnética (IRM) o fracción de eyección del ventrículo izquierdo (FEVI) <45% mediante ecocardiograma o MUGA.
    9. Disfunción hepática definida por:
    • ALT o AST> 2.5 × ULN
    10. Disfunción renal definida como creatinina sérica>ULN. Los pacientes con creatinina superior a ULN pueden ser elegibles a la espera de demostrar una tasa de filtración glomerular (TFG) ≥60 ml/min/1.73m2, calculada según ecuación de la Modificación de la Dieta en la Enfermedad Renal (Stevens 2006), la fórmula de Schwartz revisada (para pacientes menores de 18 años) (Schwartz 2009), o recolección de orina de 24 horas.
    11. Disfunción pulmonar definida por:
    a. Necesidad de oxígeno suplementario durante las 2 semanas anteriores (en ausencia de infección aguda) o
    b. Saturación de oxígeno (por oximetría de pulso) <90%.
    12. Cualquier contraindicación médica o de otro tipo para la leucaféresis y el procedimiento de extracción de BM según lo determine el investigador tratante.
    13. Cualquier afección médica o psiquiátrica que, en opinión del investigador, hace que el sujeto no sea apto para participar en el ensayo o que tenga un riesgo de participación mayor al aceptable.
    14. Estado funcional deficiente evidenciado por el Índice de Karnofsky <70 en adultos o Lansky <70 en niños.
    15. Participación en otro ensayo clínico con un fármaco en investigación dentro de los 14 días anteriores a la firma del consentimiento informado. Se permite la participación en estudios observacionales.
    16. Mujeres embarazadas o mujeres con una prueba de embarazo en suero positiva en el momento del cribado, en lactancia o planeando quedar embarazada dentro de los próximos 24 meses. Mujeres que no estén dispuestas a usar métodos anticonceptivos altamente efectivos durante el período completo del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Safety assessments specifically relevant to gene therapy are as follows:
    1. Insertional mutagenesis: Evaluation of gene-modified clonal repertoire and lentiviral ISA in blood and, if feasible, BM cells via modified gene sequencing (MGS)-PCR.
    2. RCL (as required and in settings where there is clinical suspicion of unexplained viral illness) in blood.
    3. Immunogenicity: Evidence of antibodies against coRPK (or other LV components) in blood (serum) (if necessary in settings where there is clinical suspicion of immunogenic response or evidence of decreasing coRPK expression).

    Other assessments include:
    1. Achievement of stem cell engraftment ≤45 days after infusion. Engraftment is defined as first day with neutrophil count >500/μL and platelets >20,000/μL on 3 consecutive blood counts.
    2. Time to stem cell engraftment.
    3. Incidence of respiratory complications (including, but not limited to, pneumonitis).
    4. Incidence of hepatic complications (including, but not limited to, veno-occlusive disease (VOD)).
    Las evaluaciones de seguridad específicamente relevantes para la terapia génica son las siguientes:
    1. Mutagénesis insercional: evaluación del repertorio clonal de las células modificadas genéticamente y análisis de sitios de inserción lentiviral (ISA, insertion site analysis, por sus siglas en inglés) en sangre y, si es posible, en células de médula ósea mediante PCR y secuenciación de genes modificada (MGS, modified gene sequencing, por sus siglas en inglés).
    2. Lentivirus competentes para la replicación (replication competent lentivirus, RCL, por sus siglas en inglés) (según sea necesario y en situaciones donde exista una sospecha clínica de enfermedad viral de causa no identificada) en la sangre.
    3. Inmunogenicidad: evidencia de anticuerpos contra coRPK (u otros componentes del vector lentiviral, LV) en sangre (suero) (si es necesario en situaciones donde exista una sospecha clínica de respuesta inmunogénica o evidencia de disminución de la expresión de coRPK).
    Otras evaluaciones incluyen:
    1. Alcance del injerto de células madre ≤45 días después de la infusión. Se define el injerto como el primer día con recuento de neutrófilos > 500/μL y plaquetas >20,000/μL en 3 recuentos de sangre consecutivos.
    2. Tiempo para el injerto de células madre.
    3. Incidencia de complicaciones respiratorias (incluida, pero no limitadas a, neumonitis).
    4. Incidencia de complicaciones hepáticas (incluida, pero no limitadas a, enfermedad veno-oclusiva (veno-oclusive disease, VOD, por sus siglas en inglés).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the duration of the study
    A lo largo del transcurso del estudio
    E.5.2Secondary end point(s)
    The secondary endpoints of this study are as follows:
    1. PB and BM genetic correction, as demonstrated by VCN subsequent to investigational product infusion and evidence of multi-lineage gene correction in PB and BM cells.
    2. Transfusion independence at 12 months defined as need for ≤1 transfusion in the previous 6 months.
    3. Achievement of 50% reduction in transfusion requirements at 12 months (assessed in the previous 6 months for the 12-month assessment) relative to the 1-year period prior to enrollment (not including transfusions administered to support/facilitate HSC
    collection).
    4. Clinically significant reduction of anemia defined as either:
    - An increase in pre-transfusion Hb levels of 1.5 g/dL (determined by 2 assessments separated at least three months over the first and second year of follow up) relative to the average of a patient’s Hb levels before therapeutic blood transfusion administrations over the year prior to enrollment OR
    - An increase of at least two-fold in the time to pre-transfusion Hb nadir relative to the average transfusion interval over the year prior to enrollment, where pretransfusion Hb nadir is defined as the average Hb value (during the year prior to enrollment) prior to RBC transfusions.
    5. Reduction of hemolysis. The following endpoints will be assessed in accordance:
    - Clinically significant reduction of reticulocytosis, defined as number of patients with a reduction of 50% from the average of a patient’s absolute reticulocyte counts from therapeutic blood transfusion administrations over the year prior to enrolment at 12 months subsequent to investigational therapy.

    Exploratory endpoints will include:
    1. Assessment of normalization of additional circulating parameters characteristic of anemia and/or hemolysis, including reductions in serum bilirubin (specifically indirect bilirubin), lactate dehydrogenase (LDH) or erythropoietin, and increases in haptoglobin or hepcidin levels.
    2. Assessment of changes in parameters of iron overload, including serum ferritin, iron, iron binding capacity and transferrin saturation.
    3. Assessment of changes in the ratio of pyruvate kinase to hexokinase in erythroid cells (including erythrocytes, reticulocytes, and erythroid precursors in BM when feasible; this assessment will not be feasible for patients with co-existing G6PD deficiency).
    4. Evaluation of Quality-of-life/Patient Reported Outcomes (QOL/PRO) over time, as determined by the SF-36, EQ5D, FACT-an, and PEDSQL (pediatric patients) questionnaires.
    Los objetivos secundarios de este estudio son los siguientes:
    1. Corrección genética de PB y BM, demostrada por VCN posterior a la infusión del producto en investigación y la evidencia de la corrección genética multilinaje en células de PB y BM.
    2. Independencia de transfusiones a los 12 meses, definida como necesidad de ≤1 transfusión en los 6 meses anteriores.
    3. Logro de una reducción del 50% en los requisitos transfusionales a los 12 meses (evaluados en los 6 meses anteriores) en relación con el período de 1 año anterior al reclutamiento (sin incluir las transfusiones administradas para respaldar/facilitar la recolección de HSC).
    4. Reducción clínicamente significativa de la anemia, definida como:
    - un aumento en los niveles de Hb antes de la transfusión de 1.5 g / dL (determinado por 2 evaluaciones separadas al menos tres meses durante el primer año de seguimiento), en relación con el promedio de los niveles de Hb de un paciente antes de las administraciones terapéuticas de transfusión de sangre durante el año anterior a la inscripción, O
    - un aumento de al menos dos veces del tiempo hasta el nadir de Hb antes de la transfusión en relación con el intervalo de transfusión promedio durante el año anterior al reclutamiento, donde el nadir de Hb antes de la transfusión se define como el valor de Hb promedio (durante el año anterior al reclutamiento) previo a las transfusiones de glóbulos rojos.
    5. Reducción de la hemólisis. Los siguientes puntos finales serán evaluados de acuerdo con:
    - Reducción clínicamente significativa de la reticulocitosis, definida como el número de pacientes con una reducción del 50% del promedio de los recuentos absolutos de reticulocitos de un paciente provenientes de administraciones terapéuticas de transfusión de sangre durante el año anterior a la inscripción a los 12 meses posteriores a la terapia de investigación.
    Los puntos finales exploratorios incluirán:
    1. Evaluación de la normalización de parámetros en sangre, característicos de la anemia y/o la hemólisis, incluidas las reducciones en bilirrubina sérica (específicamente bilirrubina indirecta), lactato deshidrogenasa (LDH) o eritropoyetina, y el aumento de los niveles de haptoglobina o hepcidina.
    2. Evaluación de los cambios en los parámetros de la sobrecarga de hierro, incluida ferritina sérica, hierro, capacidad de unión a hierro y saturación de transferrina.
    3. Evaluación de los cambios en el ratio de piruvato quinasa con hexoquinasa en células eritroides (incluidos eritrocitos, reticulocitos y precursores eritroides en médula ósea cuando sea posible; esta evaluación no será factible para pacientes con deficiencia de G6PD co-existente).
    4. Evaluación de la calidad de vida/resultados informados por el paciente (QOL/PRO) a lo largo del tiempo, según sea determinado por los cuestionarios SF-36, EQ5D, FACT-an y PEDSQL (pacientes pediátricos).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the duration of the study
    A lo largo del transcurso del estudio
    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) Yes
    E.7.1.1First administration to humans Yes
    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) No
    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.8.3 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 No
    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
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 4
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Some patients may be below the age for giving consent and a legally authorized representative will provide informed consent and/or assent as mandated by local regulations
    Algunos pacientes pueden tener estar por debajo de la edad para dar su consentimiento informado por lo que un representante legalmente autorizado proporcionará el consentimiento informado y/o asentirá como bajo mandato según la normativa local
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 6
    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)
    Normal treatment
    Tratamiento normal
    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 Decision2019-09-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-23
    P. End of Trial
    P.End of Trial StatusOngoing
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