Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   39572   clinical trials with a EudraCT protocol, of which   6487   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2017-001773-17
    Sponsor's Protocol Code Number:FGCL-4592-082
    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:2017-08-08
    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 number2017-001773-17
    A.3Full title of the trial
    A Phase 3 Randomized Double-Blind Placebo-Controlled Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Patients with Lower Risk Myelodysplastic Syndrome (MDS) with Low Red Blood Cell (RBC) Transfusion Burden (LTB)
    Estudio en fase III, aleatorizado, doble ciego y controlado con placebo para investigar la eficacia y seguridad de roxadustat (FG-4592) en el tratamiento de la anemia en pacientes con riesgo inferior de síndrome mielodisplásico (SMD) y carga baja de transfusión (LTB) de eritrocitos.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and efficacy study of roxadustat to treat anemia in patients with lower risk Myelodysplastic Syndrome (MDS), who require 1 to 4 packs of Red Blood Cells through transfusion every 8-weeks.
    Estudio de seguridad y eficacia de roxadustat en el tratamiento de pacientes con riesgo inferior de síndrome mielodisplásico que requieren de 1 a 4 bolsas de eritrocitos en un periodo de 8 semanas.
    A.4.1Sponsor's protocol code numberFGCL-4592-082
    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 SponsorFibroGen, 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 supportFibroGen, Inc.
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportAstraZeneca
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFibroGen, Inc.
    B.5.2Functional name of contact pointClinical Development
    B.5.3 Address:
    B.5.3.1Street Address409 Illinois Street
    B.5.3.2Town/ citySan Francisco
    B.5.3.3Post codeCA 94158
    B.5.3.4CountryUnited States
    B.5.4Telephone number14159781200
    B.5.6E-mail082MDSstudy@Fibrogen.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 nameRoxadustat
    D.3.2Product code FG-4592 - 20 mg
    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 INNRoxadustat
    D.3.9.1CAS number 808118-40-3
    D.3.9.2Current sponsor codeFG-4592/ASP1517/AZD9941
    D.3.9.3Other descriptive nameRoxadustat
    D.3.9.4EV Substance CodeSUB169531
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRoxadustat
    D.3.2Product code FG-4592 - 50 mg
    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 INNRoxadustat
    D.3.9.1CAS number 808118-40-3
    D.3.9.2Current sponsor codeFG-4592/ASP1517/AZD9941
    D.3.9.3Other descriptive nameROXADUSTAT
    D.3.9.4EV Substance CodeSUB169531
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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: 3
    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 nameRoxadustat
    D.3.2Product code FG-4592 - 100 mg
    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 INNRoxadustat
    D.3.9.1CAS number 808118-40-3
    D.3.9.2Current sponsor codeFG-4592/ASP1517/AZD9941
    D.3.9.3Other descriptive nameROXADUSTAT
    D.3.9.4EV Substance CodeSUB169531
    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.IMP: 4
    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 nameRoxadustat
    D.3.2Product code FG-4592 - 150 mg
    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 INNRoxadustat
    D.3.9.1CAS number 808118-40-3
    D.3.9.2Current sponsor codeFG-4592/ASP1517/AZD9941
    D.3.9.3Other descriptive nameROXADUSTAT
    D.3.9.4EV Substance CodeSUB169531
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 3
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 4
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    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
    Anemia due to Myelodysplastic Syndrome (MDS) in International Prognostic Scoring System – Revised Very Low, Low, or Intermediate Risk with <5% Blasts, and has low red blood cell transfusion burden (requires 1 to 4 packed red blood cell units per 8-week period)
    Anemia debido al síndrome mielodisplásico (SMD) según el sistema internacional de puntuación del pronóstico revisado (IPSS-R), <5 % de mieloblastos (riesgo inferior de SMD) y carga baja de transfusión de eritrocitos (necesidad de 1 a 4 bolsas de eritrocitos [pRBC] en un periodo de 8 semanas).
    E.1.1.1Medical condition in easily understood language
    Anemia in patients with myelodysplastic syndrome (MDS)
    Anemia en pacientes con síndrome mielodisplásico (SMD)
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of roxadustat in the treatment of anemia in patients with lower risk MDS who have a low burden of RBC transfusion.
    Evaluar la eficacia de roxadustat para el tratamiento de la anemia en pacientes con riesgo inferior de SMD y carga baja de transfusiones de eritrocitos.
    E.2.2Secondary objectives of the trial
    - To evaluate the safety of roxadustat
    - To evaluate the impact of roxadustat on RBC transfusion requirements
    - To evaluate pharmacokinetics (PK) and pharmacodynamics (PD) of roxadustat in MDS patients
    - To evaluate effect of roxadustat on quality of life parameters
    -Evaluar la seguridad de roxadustat
    -Evaluar la influencia de roxadustat en la necesidad de transfusiones de eritrocitos
    -Evaluar la farmacocinética (FC) y farmacodinámica (FD) de roxadustat en los pacientes con SMD
    -Evaluar el efecto de roxadustat en los parámetros de la calidad de vida
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of primary MDS of >/=16 weeks duration (confirmed by bone marrow aspirate/biopsy within 16 weeks prior to treatment Day 1), classified by the IPSS-R as very low, low, or intermediate risk with <5% bone marrow blasts (documented within 16 weeks prior to randomization).
    2. RBC transfusion requirement of either
    a. 2 to 4 pRBC over the 8-weeks prior to randomization, or
    b. 1 pRBC during the 8-weeks prior to randomization. Patients with 1 pRBC must have a documented history of requiring 1pRBC/8-weeks in 2 consecutive periods of 8 weeks in the 16 weeks preceding registration/randomization
    3. No prior use of recombinant erythropoietins or analogues (erythropoiesis-stimulating agents, ESAs), or received a life-time ESA exposure of less than 28 days, AND received no ESA within the 12 weeks prior to day 1 randomization. ESAs include but are not limited to any recombinant human erythropoietin and other drugs listed in Appendix I of the Protocol.
    4. Pre-transfusion Hb =/<10.0 g/dL during Screening
    5. Age >/=18 years
    6. Body weight >/=45 kg
    7. ECOG performance status of 0 or 1 at last screen visit
    8. Must be capable of giving written informed consent
    1. Diagnóstico de SMD primaria de >/=16 semanas de duración (confirmado a partir de aspirado/biopsia medular en el plazo de las 16 semanas anteriores al día 1 de tratamiento), riesgo muy bajo, bajo o medio según el IPSS-R y <5 % de mieloblastos (documentado en el plazo de las 16 semanas anteriores a la aleatorización).
    2. Necesidad de transfusión de eritrocitos, ya sea
    a. 2 a 4 pRBC en el periodo de las 8 semanas anteriores a la aleatorización, o
    b. 1 pRBC durante las 8 semanas anteriores a la aleatorización. Los pacientes con 1 pRBC deben presentar antecedentes documentados de necesitar 1 pRBC/8 semanas en 2 periodos consecutivos de 8 semanas en las 16 semanas anteriores al registro/aleatorización
    3. Sin uso anterior de eritropoyetinas recombinantes o análogos (factores estimulantes de la eritropoyesis [FEE]) o haber estado expuesto a FEE menos de 28 días durante toda la vida Y no haber recibido FEE en el plazo de las 12 semanas anteriores al día 1 de aleatorización. Entre los FEE se incluyen, entre otros, todas las eritropoyetinas humanas recombinantes y demás fármacos indicados en el anexo I.
    4. Hb antes de la transfusión =/<10,0 g/dl durante la selección
    5. Edad >/=18 años
    6. Peso corporal >/=45 kg
    7. Estado funcional según el Grupo Oncológico Cooperativo de la Costa Este de los EE. UU. (ECOG) de 0 o 1 en la última visita de selección (anexo F)
    8. Debe ser capaz de otorgar el consentimiento informado por escrito
    E.4Principal exclusion criteria
    1. Diagnosis of secondary MDS associated with prior chemotherapy, extensive radiation therapy (>25% of bone marrow reserve), and or/other significant chemical or radiation exposure
    2. Previous diagnosis of IPSS-R high risk or very high risk MDS
    3. Planned myeloablative or craniospinal radiation during the study
    4. Prior bone marrow or stem cell transplantation (SCT)
    5. Significant myelofibrosis (>2+ fibrosis)
    6. MDS associated with 5q(del) cytogenetic abnormality
    7. Screen erythropoietin level >400 mIU/mL
    8. Alanine aminotransferase (ALT) AND aspartate aminotransferase (AST) >3 × upper limit of normal (ULN), and total bilirubin (Tbili) > 1.5 × ULN
    9. Azacitidine, decitabine, thalidomide, lenalidomide, granulocyte colony-stimulating factor (G-CSF), or luspatercept, or any investigational drugs within 8-weeks prior to Day 1 Treatment or plans to use any of these medications during the course of clinical trial participation
    10. Anticipated use of dapsone at any dose amount or chronic use of acetaminophen or paracetamol > 2.0 g/day during the study
    11. Clinically significant anemia due to non-MDS etiologies such as iron deficiency, vitamin B12 or folate deficiency, autoimmune or hereditary hemolysis or anemia or hemorrhage or hereditary anemia such as sickle cell anemia or thalassemia
    12. Active infection(s) requiring antibiotic therapy
    13. Cockroft-Gault calculated creatinine clearance <30 mL/min
    14. Thromboembolic event (deep vein thrombosis (DVT)), pulmonary embolism, myocardial infarction, stroke, transient ischemic attack (TIA) within previous 6 months
    15. Current condition requiring anticoagulants
    16. Significant heart disease, including New York Heart Association (NYHA) Class III or IV congestive heart failure, uncontrolled hypertension or hypotension, or significant valvular or endocardial disease that would put the patient at risk for thromboembolism
    17. Clinically significant or uncontrolled ongoing inflammatory/autoimmune disease (e.g., rheumatoid arthritis, Crohn’s disease, celiac disease, etc.)
    18. History of significant liver disease or active liver disease
    19. Major surgery planned during the treatment period
    20. Known, active or chronic gastrointestinal bleeding
    21. Known human immunodeficiency virus (HIV), hepatitis B or hepatitis C infection
    22. Clinically significant or uncontrolled medical condition that would affect the patient's ability to participate in the study or confound the study's efficacy or safety results
    23. History of leukemia or other active malignancy except localized and non-metastatic squamous or basal cell carcinoma of the skin, or cervical intraepithelial neoplasm; patients with a history of cured malignancy with no evidence of malignancy for at least 5 years) are eligible
    24. Previous recipients of roxadustat or another hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI)
    1. Diagnóstico de SMD secundario asociado a quimioterapia anterior, radioterapia considerable (>25 % de la reserva medular) u otro tipo de exposición significativa a químicos o a radiación
    2. Diagnóstico anterior de SMD de riesgo alto o muy alto según el IPSS-R (apéndice G)
    3. Previsión de radiación mielosupresora o craneoespinal durante el estudio
    4. Trasplante anterior de médula ósea o de células madre (TCM)
    5. Mielofibrosis significativa (fibrosis >2+)
    6. SMD asociado a la anomalía citogenética 5q(del)
    7. Nivel de eritropoyetina en la selección >400 mUI/ml
    8. Alanina aminotransferasa (ALT) Y aspartato aminotransferasa (AST) >3 × límite superior de la normalidad (LSN) y bilirrubina total (BT) >1,5 × LSN (anexo H)
    9. Tratamiento con azacitidina, decitabina, talidomida, lenalidomida, factor estimulante de las colonias de granulocitos (G-CSF), luspatercept u otros fármacos en investigación en el plazo de las 8 semanas anteriores al día 1 de tratamiento o previsión de uso de estos medicamentos durante el transcurso de la participación en el ensayo clínico
    10. Uso previsto de dapsona a cualquier dosis o uso crónico de paracetamol >2,0 g/día durante el estudio
    11. Anemia de importancia clínica de etiología distinta al SMD, como la deficiencia de hierro, la deficiencia de vitamina B12 o deficiencia de folato, hemólisis autoinmunitaria o hereditaria, anemia, hemorragias o anemias hereditarias como la anemia drepanocítica o la talasemia
    12. Infección o infecciones activas que requieran tratamiento con antibióticos
    13. Aclaramiento de creatinina según la fórmula Cockroft-Gault <30 ml/min
    14. Acontecimiento tromboembólico (trombosis venosa profunda [TVP]), embolia pulmonar, infarto de miocardio, accidente cerebrovascular, accidente isquémico transitorio (AIT) en el plazo de los 6 meses anteriores
    15. Enfermedad actual que requiera tratamiento con anticoagulantes
    16. Cardiopatía significativa, incluyendo la insuficiencia cardíaca congestiva de clase III o IV según la Asociación de Cardiología de Nueva York (NYHA), hipertensión o hipotensión no controlada, o valvulopatía o enfermedad endocárdica que pudiera poner en riesgo de tromboembolia al paciente
    17. Enfermedad autoinmunitaria/inflamatoria en curso de importancia clínica o no controlada (p. ej., artritis reumatoide, enfermedad de Crohn, celiaquía, etc.)
    18. Antecedentes de hepatopatía significativa o hepatopatía activa
    19. Previsión de cirugía mayor durante el periodo de tratamiento
    20. Hemorragia gastrointestinal conocida, activa o crónica
    21. Infección conocida por el virus de la inmunodeficiencia humana (VIH), hepatitis B o hepatitis C
    22. Enfermedad de importancia clínica o no controlada que pudiera afectar a la capacidad del paciente para participar en el estudio o confundir los resultados de eficacia o seguridad del estudio
    23. Antecedentes de leucemia u otra neoplasia maligna activa, excepto carcinoma epidermoide o basocelular localizado y no metastásico, o neoplasia intraepitelial cervical; son aptos los pacientes con antecedentes de neoplasia maligna curada sin indicios de neoplasia maligna durante un mínimo de 5 años
    24. Pacientes que hayan tomado anteriormente roxadustat u otro inhibidor de la prolil hidroxilasa del factor inducible por hipoxia (HIF-PHI)
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the proportion of patients who achieved transfusion-independence (TI) >/=56 consecutive days in the first 28 weeks of treatment
    El criterio principal de valoración de la eficacia es la proporción de pacientes que han logrado la independencia de la transfusión (TI) >/=56 días consecutivos en las primeras 28 semanas de tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    The first 28 weeks of treatment
    A las 28 semanas de tratamiento.
    E.5.2Secondary end point(s)
    - Proportion of patients who achieved >/=50% reduction in number of RBC transfusion over any 8 weeks compared to their baseline
    - Cumulative number of patient-exposure-week of TI
    - Cumulative number of pRBC packs transfused
    - Proportion of patients who achieved TI for >/=20 Weeks (120 Days)
    - Mean change from baseline in Physical Function as measured by Patient Reported Outcomes Measurement Information System (PROMIS)
    - Mean change from baseline in PROMIS Fatigue score.
    - Mean change from baseline in Euroqol Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) assessment
    -Proporción de pacientes que han logrado una reducción >/=50 % en el número de transfusiones de eritrocitos a lo largo de cualquier periodo de 8 semanas en comparación con sus valores iniciales
    -Número acumulado de semanas de exposición del paciente (patient-exposure-week) de TI
    -Número de acumulado de bolsas de pRBC utilizadas
    -Proporción de pacientes que han logrado la IP durante >/=20 semanas (120 días)
    -Media de la variación con respecto al inicio en la función física según lo medido por el sistema de medición de información de resultados notificados por el paciente (PROMIS)
    -Media de la variación con respecto al inicio en la puntuación de fatiga del PROMIS
    -Media de la variación con respecto al inicio según el cuestionario sobre salud y calidad de vida EuroQoL de cinco dimensiones y cinco niveles (EQ-5D-5L)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary Efficacy Endpoints (all secondary and exploratory endpoints will be analyzed at both for the first 28 weeks of treatment and for the end of the 52-week study treatment period)
    Criterios secundarios de valoración de la eficacia (todos los criterios de valoración secundarios y exploratorios se analizarán tanto en las primeras 28 semanas de tratamiento como al final del periodo de tratamiento del estudio de 52 semanas):
    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 Yes
    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) 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 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 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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA46
    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
    France
    Germany
    Israel
    Italy
    Korea, Republic of
    Netherlands
    Poland
    Spain
    Taiwan
    United Kingdom
    United States
    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 visit of last subject
    Última visita de último paciente
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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 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: 60
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 243
    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 state48
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 126
    F.4.2.2In the whole clinical trial 303
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-09-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-29
    P. End of Trial
    P.End of Trial StatusOngoing
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA