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    Summary
    EudraCT Number:2021-000211-23
    Sponsor's Protocol Code Number:AG348-C-017
    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:2021-06-11
    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 number2021-000211-23
    A.3Full title of the trial
    A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)
    Estudio multicéntrico en fase 3, doble ciego, aleatorizado y controlado con placebo para evaluar la eficacia y seguridad de mitapivat en sujetos con Alfa- o Beta-talasemia no dependiente de transfusiones (ENERGIZE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Non Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)
    Estudio para evaluar la eficacia y seguridad de mitapivat en sujetos con Alfa- o Beta-talasemia no dependiente de transfusiones (ENERGIZE)
    A.4.1Sponsor's protocol code numberAG348-C-017
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04770753
    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 SponsorAgios 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 supportAgios Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAgios Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointScientific Communications
    B.5.3 Address:
    B.5.3.1Street Address88 Sidney Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02139-4169
    B.5.3.4CountryUnited States
    B.5.6E-mailmedinfo@agios.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 nameMitapivat, Mitapivat sulfate
    D.3.2Product code AG-348
    D.3.4Pharmaceutical form 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 INNMitapivat
    D.3.9.1CAS number 2151847-10-6
    D.3.9.2Current sponsor codeAG-348 sulfate hydrate
    D.3.9.3Other descriptive nameAGI-1480, AGX-0841, AG-348, AG-348 hemisulfate
    D.3.9.4EV Substance CodeSUB181978
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboTablet
    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
    Non–Transfusion-Dependent Alpha- or Beta-Thalassemia
    α- o β-talasemia no dependiente de transfusiones
    E.1.1.1Medical condition in easily understood language
    Thalassemia-genetic blood disorder where not enough hemoglobin is produced.2Types-α &β
    Non-transfusion-dependent thalassemias-Patients who do not require lifelong regular transfusions
    Talasemia-Trastorno genético sanguíneo donde no se produce suficiente hemoglobina.2Tipos-α&β no dependientes de transfusión-Pacientes que no requieren transfusiones regulares de por vida
    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 LLT
    E.1.2Classification code 10074356
    E.1.2Term Non-transfusion dependent thalassemia
    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 compare the effect of mitapivat versus placebo on anemia in subjects with α- or
    β- non–transfusion-dependent thalassemia (NTDT)
    Comparar el efecto de mitapivat frente al placebo sobre la anemia en sujetos con α- o β-talasemia no dependiente de transfusiones (TNDT)
    E.2.2Secondary objectives of the trial
    Key Secondary Objectives
    • To compare the effect of mitapivat versus placebo on fatigue
    • To compare the effect of mitapivat versus placebo on additional measures of anemia

    Secondary Objectives
    • To evaluate the effect of mitapivat versus placebo on anemia and markers of hemolysis and erythropoiesis
    • To evaluate the effect of mitapivat versus placebo on additional measures of fatigue
    • To evaluate the effect of mitapivat versus placebo on physical activity
    • To evaluate the effect of mitapivat versus placebo on iron metabolism
    • To evaluate the safety of mitapivat
    • To evaluate the pharmacokinetic and pharmacodynamic effects of mitapivat
    Objetivos secundarios clave:

    - Comparar el efecto de mitapivat frente al placebo sobre la fatiga.

    - Comparar el efecto de mitapivat frente al placebo en mediciones adicionales de anemia.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects are eligible to be included in the study only if all the following criteria apply:
    1. ≥18 years of age at the time of providing informed consent.
    2. Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene
    mutations, HbE/β-thalassemia, or α-thalassemia/HbH disease) based on Hb electrophoresis, Hb high-performance liquid chromatography, and/or DNA analysis from the subject’s medical record. If this information is not available from the subject’s
    medical record, the test(s) can be performed by a local laboratory during the Screening
    Period. If a local laboratory is unable to perform the test(s), results from the comprehensive α- and β-globin genotyping performed by the study central laboratory can be used.
    3. Hb concentration ≤10.0 g/dL, based on an average of at least 2 Hb concentration
    measurements (separated by ≥7 days) collected during the Screening Period.
    4. Non–transfusion dependent, defined as ≤5 RBC units during the 24-week period before randomization, and no RBC transfusions ≤8 weeks before providing informed consent or during the Screening Period.
    5. If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before
    randomization.
    6. Women of childbearing potential (WOCBP) and men with partners who are WOCBP
    must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug for women and 90 days after the last dose of study drug for men. The second form of contraception can be an acceptable barrier method.
    7. Written informed consent before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study.
    1. Edad ≥18 años en el momento de otorgar el consentimiento informado.
    2. Diagnóstico documentado de talasemia (β-talasemia con o sin mutaciones en el gen de α-globina, enfermedad de la HbE/β-talasemia o α-talasemia/HbH) basado en una prueba de electroforesis de Hb, cromatografía líquida de alta resolución de Hb y/o el análisis de ADN disponible en la historia clínica del sujeto. Si esta información no se encuentra disponible en la historia clínica del sujeto, la(s) prueba(s) se puede(n) realizar en un laboratorio local durante el periodo de selección. En el caso de que la(s) prueba(s) no se pueda(n) realizar en un laboratorio local, se pueden utilizar los resultados del genotipado completo de α- y β-globina realizado por el laboratorio central del estudio.
    3. Concentración de Hb ≤10,0 g/dl, basada en la media de al menos 2 mediciones de la concentración de Hb (con un intervalo de ≥7 días) obtenidas durante el periodo de selección.
    4. Sujetos no dependientes de transfusiones, lo que se define como ≤5 unidades de eritrocitos durante el periodo de 24 semanas antes de la aleatorización y no haber recibido ninguna transfusión de eritrocitos ≤8 semanas antes de otorgar el consentimiento informado ni durante el periodo de selección.
    5. En el caso de tratamiento con hidroxiurea, la dosis de hidroxiurea debe haber permanecido estable durante ≥16 semanas antes de la aleatorización.
    6. Las mujeres con capacidad de concebir (MCC), así como los hombres con parejas que son MCC, no deben mantener relaciones sexuales que puedan provocar el embarazo como parte de su estilo de vida habitual o deben aceptar el uso de 2 métodos anticonceptivos, 1 de los cuales debe considerarse altamente eficaz, desde el momento de otorgar el consentimiento informado, durante el estudio y durante 28 días tras la última dosis del fármaco del estudio en las mujeres y 90 días tras la última dosis del fármaco del estudio en los hombres. El segundo método anticonceptivo puede ser un método de barrera aceptable.
    7. Haber otorgado el consentimiento informado por escrito antes de realizar cualquier procedimiento relacionado con el estudio y estar dispuesto a cumplir todos los procedimientos del estudio durante este.
    E.4Principal exclusion criteria
    Subjects are excluded from the study if any of the following criteria apply:
    1. Pregnant or breastfeeding.
    2. Documented history of homozygous or heterozygous HbS or HbC.
    3. Prior exposure to gene therapy or prior bone marrow or stem cell transplantation.
    4. Currently receiving treatment with luspatercept; the last dose must have been
    administered ≥12 weeks before screening.
    5. Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥12 weeks before screening.
    6. History of any malignancy, except for nonmelanomatous skin cancer in situ, cervical
    carcinoma in situ, or breast carcinoma in situ. Subjects must not have active disease or
    received anticancer treatment ≤5 years before providing informed consent.
    7. History of active and/or uncontrolled cardiac or pulmonary disease ≤6 months before providing informed consent, including but not limited to:
    a. New York Heart Association Class III or IV heart failure or clinically significant
    dysrhythmia
    b. Myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or
    thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism
    c. Heart rate–corrected QT interval using Fridericia’s method ≥450 milliseconds, except
    for right or left bundle branch block
    d. Severe pulmonary fibrosis as defined by severe hypoxia, evidence of right-sided heart failure, and radiographic pulmonary fibrosis >50%
    e. Severe pulmonary hypertension as defined by severe symptoms associated with
    hypoxia, right-sided heart failure, and oxygen indicated
    8. Hepatobiliary disorders, including but not limited to:
    a. Liver disease with histopathological evidence of cirrhosis or severe fibrosis
    b. Clinically symptomatic cholelithiasis or cholecystitis (prior cholecystectomy is not
    exclusionary)
    c. History of drug-induced cholestatic hepatitis
    d. Aspartate aminotransferase (AST) >2.5 × upper limit of normal (ULN); unless due to
    hemolysis and hepatic iron deposition) and alanine aminotransferase (ALT) >2.5 ×
    ULN (unless due to hepatic iron deposition)
    9. Estimated glomerular filtration rate <45 mL/min/1.73 m2 by Chronic Kidney Disease
    Epidemiology Collaboration creatinine equation.
    10. Nonfasting triglycerides >440 mg/dL (5 mmol/L).
    11. Active infection requiring systemic antimicrobial therapy at the time of providing
    informed consent. If antimicrobial therapy is required during the Screening Period,
    screening procedures should not be performed while antimicrobial therapy is being
    administered, and the last dose of antimicrobial therapy must be administered ≥7 days
    before randomization.
    12. Positive test for hepatitis C virus (HCV) antibody (Ab) with evidence of active HCV
    infection, or positive test for hepatitis B surface antigen (HBsAg).
    13. Positive test for HIV-1 Ab or HIV-2 Ab.
    14. History of major surgery (including splenectomy) ≤16 weeks before providing informed consent and/or a major surgical procedure planned during the study.
    15. Current enrollment or past participation (≤12 weeks before administration of the first dose of study drug or a time frame equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational
    treatment or device.
    16. Receiving strong CYP3A4/5 inhibitors that have not been stopped for ≥5 days or a time frame equivalent to 5 half-lives (whichever is longer), or strong CYP3A4 inducers that have not been stopped for ≥4 weeks or a time frame equivalent to 5 half-lives (whichever is longer), before administration of the first dose of study drug.
    17. Receiving anabolic steroids, including testosterone preparations, that have not been stopped for ≤4 weeks before administration of the first dose of study drug.
    18. Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate).
    19. Any medical, hematological, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data.
    1. Estar embarazada o en periodo de lactancia
    2. Antecedentes documentados de HbS o HbC homocigota o heterocigota
    3. Exposición previa a una terapia génica o trasplante anterior de médula ósea o células madre
    4. Sujetos que actualmente están recibiendo tratamiento con luspatercept; la última dosis se debe haber administrado ≥12 semanas antes de la selección
    5. Sujetos que actualmente están recibiendo tratamiento con factores estimulantes hematopoyéticos; la última dosis se debe haber administrado ≥12 semanas antes de la selección
    6. Antecedentes de neoplasia maligna, excepto cáncer de piel no melanocítico in situ, carcinoma in situ de cuello uterino o carcinoma de mama in situ. Los sujetos no deben tener enfermedad activa ni haber recibido tratamiento antineoplásico ≤5 años antes de otorgar el consentimiento informado.
    7. Antecedentes de enfermedad cardíaca o pulmonar activa y/o no controlada ≤6 meses antes de otorgar el consentimiento informado, incluidos, entre otros, los siguientes: a. Insuficiencia cardíaca clase III o IV según la Asociación del Corazón de Nueva York (New York Heart Association) o arritmia clínicamente significativa.
    b. Infarto de miocardio o angina de pecho inestable; accidente cerebrovascular hemorrágico, embólico o trombótico; trombosis venosa profunda; o embolia pulmonar o arterial
    c. Intervalo QT corregido para la frecuencia card mediante el método de Fridericia ≥450 milisegundos, excepto en el caso de bloq. de la rama derecha o izquierda
    d. Fibrosis pulmonar grave, definida por hipoxia grave, indicios de insuficiencia cardíaca derecha y fibrosis pulmonar radiográfica >50 %
    e. Hipertensión pulmonar grave, definida por la presencia de síntomas graves con hipoxia, insuficiencia cardíaca derecha e indicación de oxígeno
    8. Trastornos hepatobiliares, incluidos, entre otros, los siguientes:
    a. Enfermedad hepática con indicios histopatológicos de cirrosis o fibrosis grave
    b. Colelitiasis o colecistitis clínicamente sintomática (la colecistectomía previa no es motivo de exclusión)
    c. Antecedentes de hepatitis colestásica inducida por fármacos
    d. Aspartato aminotransferasa >2,5 × límite superior de la normalidad (LSN) (a menos que el aumento se deba a hemólisis y depósitos hepáticos de hierro) y alanina aminotransferasa >2,5 × LSN (a menos que el aumento se deba a depósitos hepáticos de hierro)
    9. Tasa de filtración glomerular estimada <45 ml/min/1,73 m2 calculada mediante la ecuación de la creatinina desarrollada por el grupo de colaboración de epidemiología de insuficiencia renal crónica (Chronic Kidney Disease Epidemiology Collaboration)
    10. Triglicéridos sin ayuno >440 mg/dl (5 mmol/l)
    11. Infección activa que requiere tratamiento antimicrobiano sistémico en el momento de otorgar el consentimiento informado. Si el sujeto requiere tratamiento antimicrobiano durante el periodo de selección, no se deben realizar los procedimientos de selección mientras se esté administrando el tratamiento antimicrobiano, y la última dosis del tratamiento antimicrobiano se debe administrar ≥7 días antes de la aleatorización
    12. Resultado positivo en la prueba de anticuerpos (Ac) contra el virus de la hepatitis C (VHC) con indicios de infección activa por VHC, o resultado positivo en la prueba del antígeno de superficie de la hepatitis B
    13. Resultado positivo en la prueba de Ac contra el VIH-1 o Ac contra el VIH-2
    14. Antecedentes de cirugía mayor (incluida la esplenectomía) ≤16 semanas antes de otorgar el consentimiento informado y/o tener programado someterse a una intervención quirúrgica mayor
    15. Sujetos incluidos en la actualidad o que anteriormente han participado (≤12 semanas antes de la administración de la primera dosis del fármaco del estudio o durante un periodo de tiempo equivalente a 5 semividas del fármaco del estudio en fase de investigación, el período que sea más largo) en cualquier otro estudio clínico con un tratamiento o dispositivo en investigación
    16. Sujetos que reciben inhibidores potentes del P450 (CYP)3A4/5 cuyo tratamiento no se haya interrumpido ≥5 días o durante un periodo de t. equivalente a 5 semividas (el período que sea más largo) o inductores potentes del CYP3A4 cuyo tratamiento no se haya interrumpido ≥4 semanas o durante un periodo de t. equivalente a 5 semividas (el período que sea más largo) antes de la administración de la primera dosis del fármaco del estudio
    17. Sujetos que reciben esteroides anabólicos, incluidas preparaciones de testosterona, cuya administración no se haya interrumpido ≤4 semanas antes de la administración de la primera dosis del fármaco del estudio
    18. Alergia conocida a mitapivat o a cualquiera de los excipientes presentes.
    19. Cualquier trastorno médico, hematológico, psicológico o de la conducta, o cualquier tratamiento anterior o actual que, en opinión del investigador, pueda constituir un riesgo inaceptable en relación con la participación en el estudio y/o confundir la interpretación de los datos del estudio
    E.5 End points
    E.5.1Primary end point(s)
    • Hemoglobin (Hb) response, defined as a ≥1.0 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with baseline
    Respuesta de la hemoglobina (Hb), definida como un aumento de ≥1,0 g/dl en la concentración media de Hb desde la semana 12 hasta la semana 24 con respecto al inicio
    E.5.1.1Timepoint(s) of evaluation of this end point
    Please refer to the schedule of assessment
    Consulte el calendario de evaluación
    E.5.2Secondary end point(s)
    Key Secondary Endpoints
    • Change from baseline in average Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) subscale score from Week 12 through Week 24
    • Change from baseline in average Hb concentration from Week 12 through Week 24

    Secondary Endpoints
    • Hb 1.5+ response, defined as a ≥1.5 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with baseline
    • Change from baseline in indirect bilirubin, lactate dehydrogenase, and haptoglobin at Week 24
    • Change from baseline in reticulocytes and erythropoietin at Week 24
    • Improvement in the Patient Global Impression of Severity (PGIS)-Fatigue by at least 1 category at Weeks 12, 16, 20, and 24 compared with baseline, or “no change” if no or mild fatigue at baseline
    • Improvement in the Patient Global Impression of Change (PGIC)-Fatigue at Weeks 12, 16, 20, and 24, or “no change” if no or mild fatigue at baseline
    • Change from baseline in the 6-minute walk test (6MWT) distance at Week 24
    • Change from baseline in markers of iron metabolism, including serum ferritin and transferrin saturation, at Week 24
    • Type, severity, and relationship of adverse events (AEs) and serious adverse events
    • Plasma or blood concentrations and pharmacokinetic parameters of mitapivat and pharmacodynamic parameters, including adenosine triphosphate (ATP) and 2,3-diphosphoglycerate (2,3-DPG)
    Objetivos secundarios clave:
    - Cambio con respecto al inicio en la puntuación media de la subescala de Evaluación Funcional para el Tratamiento de Enfermedades Crónicas: Fatiga (Functional Assessment of Chronic Illness Therapy-Fatigue, FACIT: Fatiga) desde la semana 12 hasta la semana 24.
    - Cambio con respecto al inicio en la concentración media de Hb desde la semana 12 hasta la semana 24.
    Objetivos secundarios:
    - Respuesta de 1,5+ de la Hb, lo que se define como un aumento ≥1,5 g/dl en la concentración media de Hb desde la semana 12 hasta la semana 24 con respecto al inicio.
    - Cambio con respecto al inicio en la bilirrubina indirecta, lactato deshidrogenasa y haptoglobina en la semana 24.
    - Cambio con respecto al inicio en los reticulocitos y la eritropoyetina en la semana 24.
    - Mejoría de al menos 1 categoría en la escala de Impresión global de la intensidad por el paciente (Patient Global Impression of Severity, PGIS): Fatiga en las semanas 12, 16, 20 y 24 con respecto al inicio, o “sin cambio” en caso de ausencia de fatiga o presencia de fatiga leve al inicio.
    - Mejoría en la escala de Impresión global del cambio por el paciente (Patient Global Impression of Change, PGIC): Fatiga en las semanas 12, 16, 20 y 24, o “sin cambio” en caso de ausencia de fatiga o presencia de fatiga leve al inicio.
    - Cambio con respecto al inicio en la distancia caminada en la prueba de marcha de 6 minutos (PM6M) en la semana 24.
    - Cambio con respecto al inicio en marcadores del metabolismo del hierro, incluidas la ferritina sérica y la saturación de transferrina, en la semana 24.
    - Tipo, intensidad y relación de los acontecimientos adversos (AA) y acontecimientos adversos graves.
    - Concentraciones sanguíneas o plasmáticas y parámetros farmacocinéticos de mitapivat y parámetros farmacodinámicos, incluidos trifosfato de adenosina (ATP) y 2,3-difosfoglicerato (2,3-DPG).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please refer to the schedule of assessment
    Consulte el calendario de evaluación
    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 Yes
    E.6.13.1Other scope of the trial description
    Targeted genotyping, exploratory analysis of biomarkers
    Genotipado dirigido, análisis exploratorio de biomarcadores
    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 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 No
    E.8.1.6Cross over Yes
    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 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 EEA31
    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
    Brazil
    Canada
    Lebanon
    Malaysia
    Saudi Arabia
    Taiwan
    Thailand
    Turkey
    United States
    Bulgaria
    Denmark
    Germany
    Greece
    Italy
    Netherlands
    Spain
    United Kingdom
    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 years5
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days18
    E.8.9.2In all countries concerned by the trial years7
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 167
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 52
    F.4.2.2In the whole clinical trial 171
    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)
    NA
    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 Decision2021-10-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-27
    P. End of Trial
    P.End of Trial StatusOngoing
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