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    Summary
    EudraCT Number:2019-001882-34
    Sponsor's Protocol Code Number:C-935788-058
    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:2019-12-02
    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 number2019-001882-34
    A.3Full title of the trial
    A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
    Estudio de extensión abierto de fase III del fostamatinib disódico en el tratamiento de la anemia hemolítica autoimmune por anticuerpos calientes
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a Phase 3 multi-center, open-label extension study to evaluate the long-term safety and efficacy of fostamatinib (R788) in subjects with wAIHA who have completed 24 weeks of participation in study C-935788-057.
    Estudio de extensión abierto y multicéntrico de fase III para evaluar la eficacia y seguridad a largo plazo del fostamatinib(R788) en pacientes con anemia hemolítica autoimmune por anticuerpos calientes que han completado 24 semanas de su participación en el estudio C-935788-057.
    A.4.1Sponsor's protocol code numberC-935788-058
    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 SponsorRigel 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 supportRigel Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharm-Olam International (Spain) S.L.U
    B.5.2Functional name of contact pointRegulatory Department
    B.5.3 Address:
    B.5.3.1Street AddressC/Antracita, 7 Planta 1 A Nave 6
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28045
    B.5.3.4CountrySpain
    B.5.4Telephone number+34 91-145 91 10
    B.5.5Fax number+34 91-434-27 73
    B.5.6E-mailregulatory.spain@pharm-olam.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 nameFostamatinib Disodium
    D.3.2Product code R935788
    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 INNFostamatinib
    D.3.9.1CAS number 914295-16-2
    D.3.9.2Current sponsor codeFostamatinib Disodium R935788
    D.3.9.3Other descriptive nameR788, fostamatinib disodium hexahydrate (in USPI/FPI)
    D.3.9.4EV Substance CodeSUB32625
    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: 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 nameFostamatinib Disodium
    D.3.2Product code R935788
    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 INNFostamatinib
    D.3.9.1CAS number 914295-16-2
    D.3.9.2Current sponsor codeFostamatinib Disodium R935788
    D.3.9.3Other descriptive nameR788, fostamatinib disodium hexahydrate (in USPI/FPI)
    D.3.9.4EV Substance CodeSUB32625
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Warm antibody autoimmune hemolytic anemia (wAIHA)
    Anemia hemolítica autoinmune por anticuerpos calientes (AHAIc)
    E.1.1.1Medical condition in easily understood language
    Autoimmune hemolytic anemia (AIHA) is an acquired disorder manifested by autoantibody- mediated red blood cell (RBC) destruction.
    La anemia hemolítica autoinmune (AHAI) es un trastorno adquirido que se manifiesta por la destrucción de los glóbulos rojos mediada por autoanticuerpos.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003825
    E.1.2Term Autoimmune hemolytic anemia
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is:
    • To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA)
    El objetivo principal del presente estudio es:
    • Evaluar la seguridad a largo plazo del fostamatinib en pacientes con anemia hemolítica autoinmune (AHAI) por anticuerpos calientes
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are:
    • To estimate the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA)
    • To estimate the durability of response in subjects receiving fostamatinib for wAIHA
    • To assess steroid use in subjects with warm antibody autoimmune hemolytic anemia (wAIHA) treated with fostamatinib.
    Los objetivos secundarios del presente estudio son:
    • Estimar la eficacia del fostamatinib en pacientes con AHAI por anticuerpos calientes
    • Estimar la duración de la respuesta en pacientes que reciben fostamatinib para tratar la AHAI por anticuerpos calientes
    • Evaluar el uso de corticoesteroides en pacientes con AHAI por anticuerpos calientes tratados con fostamatinib.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject must be willing and able to give written informed consent by signing an IRB approved Informed Consent Form prior to undergoing any study-specific procedures.
    2. Subject must have completed all 24 weeks of participation in the study C-935788-057.
    3. Female subjects must be either post-menopausal for at least 1 year or surgically sterile; or, if of childbearing potential, must not be pregnant or lactating and must agree to use a highly effective method of birth control throughout the duration of the trial and for 14 days following the last dose. Acceptable methods of birth control are defined as: hormonal contraception (pill, injection or implant) used consistently for at least 30 days prior to baseline an intrauterine device (IUD), or intrauterine hormone-releasing system (IUS), or true abstinence (i.e. abstinence is in line with the preferred and usual lifestyle of the subject).
    4. In the investigator’s opinion, the subject has the ability to understand the nature of the study and any hazards of participation and to communicate satisfactorily with the investigator.
    1. Los pacientes deberán estar dispuestos y en la capacidad de otorgar su consentimiento informado por escrito, mediante la firma de un Documento de consentimiento informado aprobado por un comité de ética, antes de la realización de cualquier procedimiento propio del estudio.
    2. Los pacientes deberán haber concluido la totalidad de las 24 semanas de participación en el estudio C-935788-057.
    3. Las pacientes deberán encontrarse en período postmenopáusico por lo menos durante un año o haberse sometido a esterilización quirúrgica. En el caso de las pacientes con capacidad de quedar embarazadas, no deberán estar embarazadas o en período de lactancia y deberán aceptar utilizar un método anticonceptivo muy eficaz en el transcurso del ensayo y durante los 14 días siguientes a la administración de la última dosis. Los métodos anticonceptivos aceptables son: métodos anticonceptivos hormonales (píldora, inyección o implante) utilizados de forma continua durante un mínimo de 30 días antes de la visita basal, dispositivo intrauterino (DIU) o sistema intrauterino (SIU) liberador de un tratamiento anticonceptivo hormonal, o abstinencia verdadera (es decir, la abstinencia está en concordancia con la forma de vida preferida y habitual de la paciente).
    4. A juicio del investigador, el paciente es capaz de entender la naturaleza del estudio y los riesgos de la participación, así como de comunicarse de forma satisfactoria con el investigador.
    E.4Principal exclusion criteria
    1. Any subject who discontinued participation in Study C-935788-057 prior to Week 24.
    1. Pacientes que hayan dejado de participar en el estudio C-935788-057 antes de la semana 24.
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints:
    • Adverse events
    • Changes from baseline in selected laboratory values per CTCAE criteria V 5.0
    • Changes from baseline in blood pressure over time

    Efficacy Endpoints:
    • Durable hemoglobin response (Yes/No) within 24 weeks of treatment
    A hemoglobin response is defined as a hemoglobin level of >10 g/dL and ≥2 g/dL higher than the baseline (i.e, hemoglobin value prior to fostamatinib exposure), during the previous 4 weeks, the steroid dose is not higher than the baseline level, and rescue medication is not administered. A durable response is defined as a hemoglobin response on at least 3 scheduled visits during the first 24 weeks of this study.
    • Hemoglobin response at 48 weeks (Yes/No) defined as subjects who achieve a durable response, as above, and have a hemoglobin response at 48 weeks
    • Hemoglobin response by week 24
    • Partial hemoglobin response, defined as a Hgb change from baseline of ≥2 g/dL
    • Subjects who achieve steroid dose < 10 mg/day (prednisone equivalent)
    • Change from baseline in hemoglobin values over time
    • Change from baseline in steroid dose
    • Subjects requiring any rescue during this study
    • Overall duration of response and duration of first response
    • Subjects who decrease any concomitant wAIHA therapy
    Criterios de valoración de la seguridad:
    • Acontecimientos adversos
    • Cambios con respecto a los valores basales en las pruebas analíticas seleccionadas, según los criterios terminológicos comunes para acontecimientos adversos (CTCAE) V 5.0
    • Cambios con respecto al valor basal en la presión arterial en el transcurso del tiempo

    Criterios de valoración de la eficacia:
    • Respuesta hemoglobínica duradera (sí/no) en las 24 semanas de tratamiento
    Una respuesta hemoglobínica se define como un valor de hemoglobina >10 g/dl y a su vez superior en ≥2 g/dl al valor basal (es decir, el valor de hemoglobina antes de la exposición al fostamatinib) si, durante las 4 semanas anteriores, la dosis de corticoesteroides no se incrementó respecto al nivel basal y no se administró tratamiento de rescate). Una respuesta duradera se define como una respuesta hemoglobínica presente en por lo menos tres visitas programadas en el transcurso de las 24 semanas de duración de este estudio.
    • Respuesta hemoglobínica a las 48 semanas (sí/no), definida como pacientes con una respuesta duradera, como se definió antes, y una respuesta hemoglobínica a las 48 semanas.
    • Respuesta hemoglobínica en la semana 24
    • Respuesta hemoglobínica parcial, definida como un cambio en el valor de la hemoglobina ≥2 g/dl con respecto al valor basal
    • Pacientes que alcanzan una dosis de corticoesteroides <10 mg/día (prednisona o equivalente)
    • Cambio en el transcurso del tiempo en los valores de hemoglobina con respecto al valor basal
    • Cambio en la dosis de corticoesteroides con respecto a la dosis basal
    • Pacientes que necesiten algún tratamiento de rescate a lo largo de este estudio
    • Duración global de la respuesta y duración de la primera respuesta
    • Pacientes a quienes se les haya disminuido algún medicamento concurrente para la AHAI por anticuerpos calientes
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 week evaluation
    Semana 24 de evaluación
    E.5.2Secondary end point(s)
    None
    Ninguno
    E.5.2.1Timepoint(s) of evaluation of this end point
    None
    Ninguno
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    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
    Austria
    Belarus
    Belgium
    Bulgaria
    Canada
    Czech Republic
    Denmark
    France
    Georgia
    Germany
    Hungary
    Italy
    Netherlands
    Norway
    Romania
    Russian Federation
    Serbia
    Spain
    Ukraine
    United Kingdom
    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
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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: 81
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 81
    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
    Ninguno
    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 Decision2020-01-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-12-17
    P. End of Trial
    P.End of Trial StatusOngoing
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