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    Summary
    EudraCT Number:2018-003534-32
    Sponsor's Protocol Code Number:MOM-M254-001
    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:2019-04-12
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2018-003534-32
    A.3Full title of the trial
    A 4-part Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of M254 in healthy volunteers and in patients with immune thrombocytopenic purpura
    Estudio en fase I/II de 4 partes para evaluar la seguridad, tolerabilidad, farmacocinética y farmacodinámica de M254 en voluntarios sanos y en pacientes con púrpura trombocitopénica inmunitaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenic purpura (ITP) patients
    Seguridad y tolerabilidad de M254 en voluntarios sanos y pacientes con púrpura trombocitopénica inmunitaria (PTI)
    A.4.1Sponsor's protocol code numberMOM-M254-001
    A.5.4Other Identifiers
    Name:CRO codeNumber:MMT102EC-170091
    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 SponsorMomenta 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 supportMomenta Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMomenta Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address301 Binney Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrialinfo@momentapharma.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 nameN/A
    D.3.2Product code M254
    D.3.4Pharmaceutical form Solution for infusion
    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 INNN/A
    D.3.9.1CAS number 2214289-68-4
    D.3.9.2Current sponsor codeM254
    D.3.9.3Other descriptive namehypersialylated immunoglobulin
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number3 to 1000
    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) Yes
    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 Yes
    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 RoleComparator
    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 Yes
    D.2.1.1.1Trade name Privigen®
    D.2.1.1.2Name of the Marketing Authorisation holderCSL Behring
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 namePrivigen
    D.3.4Pharmaceutical form Infusion
    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 INNn.a.
    D.3.9.1CAS number 9007-83-4
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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) Yes
    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 Yes
    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 placeboInfusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Immune thrombocytopenic purpura
    púrpura trombocitopénica inmunitaria
    E.1.1.1Medical condition in easily understood language
    Immune thrombocytopenic purpura
    púrpura trombocitopénica inmunitaria
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10074667
    E.1.2Term Immune thrombocytopenic purpura
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A:
    To assess the safety and tolerability of a single ascending dose of intravenous administration of M254 in healthy volunteers.

    Part B:
    To assess the safety and tolerability of a single intravenous administration of M254 in idiopathic. thrombocytopenic purpura (ITP) patients compared to 1000 mg/kg intravenous immunoglobulin (IVIg).

    Part C:
    To assess the safety of a single intravenous administration of M254 compared to 1000 mg/kg of IVIg.
    To characterize the PD of single intravenous administration of M254 compared to 1000 mg/kg IVIg.

    Part D:
    To assess the safety and tolerability of repeated intravenous administration of M254 in ITP patients.
    Parte A:
    Evaluar la seguridad y la tolerabilidad de dosis ascendentes únicas de administración intravenosa de M254 en voluntarios sanos

    Parte B:
    Evaluar la seguridad y la tolerabilidad de una administración intravenosa única de M254 en pacientes con púrpura trombocitopénica idiopática (PTI) en comparación con 1000 mg/kg de inmunoglobulina intravenosa (IgIV)

    Parte C:
    Evaluar la seguridad de una administración intravenosa única de M254 en comparación con 1000 mg/kg de IgIV
    Caracterizar la FD de una administración intravenosa única de M254 en comparación con 1000 mg/kg de IgIV

    Parte D:
    Evaluar la seguridad y la tolerabilidad de la administración intravenosa repetida de M254 en pacientes con TPI
    E.2.2Secondary objectives of the trial
    Part A:
    To characterize the pharmacokinetics (PK) of a single intravenous administration of M254 at different doses in healthy volunteers.

    Part B:
    To characterize the PK of a single intravenous administration of M254 at different doses in ITP patients.

    Part C:
    To characterize the PK of a single intravenous administration of M254 at different doses.

    Part D:
    To characterize the PK of repeated intravenous doses of M254 in ITP patients.
    To assess PD of repeated intravenous doses of M254 in ITP patients.
    Parte A:
    Caracterizar la farmacocinética (FC) de una administración intravenosa única de M254 a diferentes dosis en voluntarios sanos

    Parte B:
    Caracterizar la FC de una administración intravenosa única de M254 a diferentes dosis en pacientes con PTI

    Parte C:
    Caracterizar la FC de una administración intravenosa única de M254 a diferentes dosis

    Parte D:
    Caracterizar la FC de dosis intravenosas repetidas de M254 en pacientes con TPI
    Evaluar la FD de dosis intravenosas repetidas de M254 en pacientes con TPI
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participant must be ≥ 18 and ≤ 55 years of age inclusive, at the time of signing the informed consent form (ICF).
    2. Good health as indicated by medical history (without hemolysis or thrombosis that may impact current study), physical examination, vital signs (with systolic blood pressure below 140 mmHg), clinical laboratory tests, and 12-lead electrocardiogram, and all abnormal findings are assessed as not clinically significant by the Investigator.
    3. Body weight must be between 50 and 110 kg, inclusive, and body mass index (BMI) between 18.5 and 30 kg/m2, inclusive, at screening.
    4. Healthy male and females are eligible.
    a.Male participants:
    • If male, surgically or biologically sterile. If not sterile, agreement to use an acceptable form of birth control with sexual partner or abstain from sexual relations for 100 days following the last treatment.
    b. Female participants:
    • A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies:
    ◦ Not a woman of childbearing potential (WOCBP) (Surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or of non-childbearing potential (i.e., postmenopausal for at least 1 year).
    OR
    ◦ A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 90 days after the last dose of study intervention.
    • Of child-bearing potential, with a fertile male sexual partner, willing to use 2 adequate methods of contraception from 30 days prior to dosing until 90 days after the last dose of study treatment. Adequate contraception is defined as using hormonal contraceptives or an intrauterine device combined with at least 1 of the following forms of contraception: a diaphragm or cervical cap, or a condom. Also, total abstinence, in accordance with the lifestyle of the subject, is acceptable.
    5. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
    6. Ability to speak, read, and understand primary medical care language(s) at the site.
    7. If female, a negative serum pregnancy test at screening and a negative urine or serum pregnancy test at check-in to the clinic for the baseline visit, and not nursing or planning a pregnancy through 90 days following the last dose of study medication.
    Patients:
    1. Participant must be 18 to 64 years of age, inclusive, at the time of signing the ICF.
    2. Diagnosed with ITP (with or without splenectomy) according to the Hematology Guidelines for at least 3 months prior to screening.
    3. Platelet count ≥ 15 × 10^9/L and < 50 × 10^9/L within 96 hours prior to start of study drug infusion on Day 1
    4. Body weight must be < 110 kg
    5. Maintenance immunosuppressive therapy, steroid therapy, cyclosporine A, mycophenolate mofetil, azathioprine, thrombopoietin receptor agonists, or danazol are allowed, but the dosages of all these medications must be stable for at least 4 weeks prior to Visit 1 (Day 1).
    6. No history of clotting disorder other than ITP including no myocardial infarction within the last 6 months. No arrhythmia known to increase the risk of thrombotic events (eg, atrial fibrillation).
    For inclusion criteria patients nr. 07 - 10 please see protocol; no more possibility here.
    1. El participante debe tener entre 18 y 55 años de edad, ambos inclusive, en el momento de firmar el formulario de consentimiento informado (FCI).
    2. Buena salud según lo indicado en el historial médico (sin hemólisis o trombosis que puedan afectar el estudio actual), la exploración física, las constantes vitales (con presión arterial sistólica por debajo de 140 mmHg), las pruebas analíticas clínicas y el electrocardiograma de 12 derivaciones, y todos los hallazgos anómalos se evalúan como no clínicamente significativos por el investigador.
    3. El peso corporal debe estar entre 50 y 110 kg, inclusive, y el índice de masa corporal (IMC) entre 18,5 y 30 kg/m2, inclusive, en el momento de la selección.
    4. Son aptos los hombres y las mujeres sanos.
    a. Participantes del sexo masculino:
    • Si es hombre, quirúrgica o biológicamente estéril. Si no es estéril, acuerdo para usar una forma aceptable de regulación de la natalidad con la pareja sexual o abstenerse de tener relaciones sexuales durante 100 días después del último tratamiento.
    b. Participantes del sexo femenino:
    • Una participante del sexo femenino es apta para participar si no está embarazada, no está en periodo de lactancia y se cumple, al menos, una de las siguientes condiciones:
    ◦ No es una mujer con capacidad de concebir (esterilizada quirúrgicamente mediante histerectomía, ooforectomía bilateral o ligadura de trompas bilateral) o sin capacidad de concebir (es decir, postmenopáusica durante al menos 1 año).
    O
    ◦ Una mujer con capacidad de concebir que esté de acuerdo en seguir la guía anticonceptiva durante el período de tratamiento y, al menos, los 90 días después de la última dosis de intervención del estudio.
    • Con capacidad de concebir, con una pareja sexual masculina fértil, dispuesta a usar 2 métodos anticonceptivos adecuados desde 30 días antes de la dosis hasta 90 días después de la última dosis del tratamiento del estudio. La anticoncepción adecuada se define como el uso de anticonceptivos hormonales o de un dispositivo intrauterino combinado con, al menos, una de las siguientes formas de anticoncepción: un diafragma o capuchón cervical, o un condón. Además, se acepta la abstinencia total, de acuerdo con el estilo de vida del sujeto.
    5. Capaz de dar un consentimiento informado firmado que incluya el cumplimiento de los requisitos y las restricciones enumerados en el FCI y en este protocolo.
    6. Capacidad para hablar, leer y entender el idioma o los idiomas de atención médica primaria en el centro.
    7. Si es mujer, una prueba de embarazo en suero negativa en el momento de la selección y una prueba de embarazo en orina o suero negativa en la entrada en la clínica para la visita inicial, y no estar amamantando o planificando un embarazo hasta 90 días después de la última dosis de la medicación del estudio.
    Pacientes:
    1. El participante debe tener entre 18 y 64 años de edad, ambos inclusive, en el momento de firmar el FCI.
    2. Diagnóstico de PTI (con o sin esplenectomía), de acuerdo con las Guías de Hematología, durante al menos 3 meses antes de la selección.
    3. Recuento plaquetario ≥15 × 10^9/l y <50 × 10^9/l en las 96 horas previas al inicio de la infusión del fármaco del estudio el Día 1
    4. El peso corporal debe ser <110 kg
    5. La terapia inmunosupresora de mantenimiento, la terapia con esteroides, la ciclosporina A, el micofenolato mofetil, la azatioprina, los agonistas de los receptores de trombopoyetina o el danazol están permitidos, pero las dosis de todos estos medicamentos deben ser estables durante al menos 4 semanas antes de la Visita 1 (Día 1).
    6. Ningún antecedente de trastorno de coagulación aparte de la PTI, incluyendo ningún infarto de miocardio en los últimos 6 meses. No se conoce la presencia de arritmias que aumenten el riesgo de episodios trombóticos (p. ej., fibrilación auricular).
    Para los criterios de inclusión, los pacientes con n.° 07 - 10 ver el protocolo; aquí no hay más posibilidades.
    E.4Principal exclusion criteria
    1. Previously received M254.
    2. History of any drug allergy, hypersensitivity, or intolerance to any drug product that in the opinion of the Investigator would place the subject at particular risk and compromise the safety of the subject in the
    study.
    3. History or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, oncologic, psychiatric disease, or any other condition, that in the opinion of the Investigator would jeopardize the safety of the subject or the validity of the study results.
    4. History of splenectomy, asthma (with the exception of childhood asthma that has resolved), chronic obstructive pulmonary disease, or recurrent or current gastrointestinal or respiratory infections.
    5. Any illness within 5 days, or clinically significant airway infections within 30 days, prior to first study drug dosing.
    6. On fluid restriction.
    7. Any prescription medication(s) within 14 days of dose administration (or 5 half-lives, whichever is longer) or any non-prescribed systemic or topical medication (including any herbal product) within 7 days prior to dose administration.
    8. Plans to participate in another clinical trial while enrolled in this study and/or received an investigational drug and/or device within 60 days prior to dose administration.
    9. Positive urine drug screen at screening.
    10. Positivity for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV) at screening.
    11. History or current diagnosis of substance dependence (except nicotine and caffeine) or alcohol abuse over the past 2 years, according to the criteria of the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision (DSM-IV-TR).
    12. Smokes or has smoked more than 5 cigarettes per day in the last 90 days and is unable to stop smoking during in-patient observation period in clinic.
    13. Unwilling to abstain from alcohol for at least 24 hours prior to dosing with study medication until the time of discharge from the study unit and at least 24 hours prior to each ambulatory visit.
    14. Donation or significant loss of whole blood (480 mL or more) within 30 days or plasma within 14 days prior to admission.
    15. Vaccination within 1 month before dosing, or plans to receive vaccination within 3 months after the last dose.
    16. Veins unsuitable for cannulation or multiple venipunctures on either arm.
    17. Patients with any prior history of arterial or venous thrombosis, AND ≥ 2 of the following risk factors: hormone replacement therapy, systemic contraception (containing estrogen), smoking, diabetes, hypercholesterolemia, medication for hypertension, cancer, hereditary thrombophilic disorders (eg, Factor V Leiden, antithrombin III deficiency, antiphospholipid syndrome, etc), hyperviscosity (cryoglobulines and chylomicronemia)
    18. Patients with selective IgA deficiency with known anti-IgA antibodies.
    Patients:
    1. Patients with any prior history of arterial or venous thrombosis, AND ≥ 2 of the following risk factors: hormone replacement therapy, systemic contraception (containing estrogen), smoking, diabetes, hypercholesterolemia, medication for hypertension, cancer, hereditary thrombophilic disorders (eg, Factor V Leiden, antithrombin III deficiency, antiphospholipid syndrome, etc), hyperviscosity (cryoglobulines and chylomicronemia)
    2. Any clinically relevant abnormality, other than ITP, which in the opinion of the Investigator makes the patient unsuitable for participation in the study.
    3. Female patients who are nursing or pregnant at screening or pre-dose on Day 1.
    4. History of alcohol/drug abuse or dependence within 12 months of the study.
    5. Rituximab within 3 months of screening
    6. Patient has consumed aspirin, aspirin-containing compounds, salicylates, anticoagulants, quinine or non-steroidal anti-inflammatories (NSAIDs) for > 3 consecutive days within 2 weeks of the study start and until the end of the study.
    7. Consumption of any herbal or dietary supplements, excluding vitamin or mineral supplements, within 1 week of the study start.
    8. If known from prior medical history, non-response to IVIg. Nonresponse to IVIg is defined as increase of platelet count did not rise above 50 × 10^9/L within 14 days of treatment.
    9. History of platelet agglutination that prevents reliable measurement of platelet counts.
    10. Any laboratory or clinical evidence for HIV infection.
    For exclusion criteria patients nr. 11 - 17 please see protocol; no more possibility here.
    1.Tratamiento previo con M254
    2.Antecedentes de alergia farmacológica, hipersensibilidad o intolerancia a cualquier fármaco que,en opinión del investigador,pondría en riesgo al paciente y comprometería su seguridad en el estudio
    3.Antecedentes o presencia de enfermedad cardiovascular, pulmonar,hepática,renal,hematológica,gastrointestinal,endocrina,inmunológica,dermatológica,neurológica,oncológica,psiquiátrica o cualquier otra afección que,en opinión del Investigador,ponga en peligro la seguridad del paciente o la validez de los resultados del estudio
    4.Antecedentes de esplenectomía, asma(con excepción del asma infantil que se ha resuelto),EPOC o infecciones gastrointestinales o respiratorias recurrentes o actuales
    5.Cualquier enfermedad en los 5 días,o infecciones de vías respiratorias clínicamente significativas en los 30 días,previos a la primera dosis del fármaco del estudio.
    6.Con restricción de líquidos
    7.Cualquier medicamento recetado en los 14 días previos a la administración de la dosis(o 5 semividas,lo que sea más largo)o cualquier medicamento tópico o sistémico sin receta (incluyendo cualquier producto a base de hierbas)en los 7 días previos a la administración de la dosis
    8.Planes para participar en otro ensayo clínico mientras esté inscrito en este estudio y/o haya recibido un fármaco y/o dispositivo en investigación en los 60 días previos a la administración de la dosis
    9.Prueba de orina positiva en el momento de la selección.
    10.Positivo para el virus de la inmunodeficiencia humana (VIH),el antígeno de superficie de la hepatitis B(HBsAg)o el virus de la hepatitis C(VHC)en el momento de la selección
    11.Antecedentes o diagnóstico actual de dependencia de sustancias(excepto nicotina y cafeína)o abuso de alcohol en los últimos 2 años,de acuerdo con los criterios del Manual de Diagnóstico y Estadística de Trastornos Mentales,4.ª Edición, Revisión de Texto (DSM-IV-TR)
    12.Fumar actualmente o haber fumado más de 5 cigarrillos al día en los últimos 90 días y no poder dejar de fumar durante el período de observación ingresado en la clínica
    13.No estar dispuesto a abstenerse de consumir alcohol durante al menos 24 horas antes de tomar la medicación del estudio y hasta el momento del alta de la unidad de estudio y al menos 24 horas antes de cada visita ambulatoria
    14.Donación o pérdida significativa de sangre completa(480 ml o más)en los 30 días o plasma en los 14 días previos al ingreso
    15.Vacunación en el mes previo a la administración de la dosis, o planes para recibir la vacuna en los 3 meses posteriores a la última dosis
    16.Venas no aptas para canulación o múltiples venopunciones en ambos brazos
    17.Pacientes con antecedentes de trombosis arterial o venosa,Y ≥2 de los siguientes factores de riesgo:tratamiento sustitutivo hormonal,anticoncepción sistémica (con estrógeno), abaquismo,diabetes,hipercolesterolemia, medicamentos para la hipertensión,cáncer,trastornos trombofílicos hereditarios(p. ej., Factor V Leiden, insuficiencia de antitrombina III, síndrome antifosfolípido, etc.), hiperviscosidad (crioglobulinas y quilomicronemia).
    18.Pacientes con insuficiencia selectiva de IgA y anticuerpos anti-IgA conocidos
    Pacientes:
    1.Pacientes con antecedentes de trombosis arterial o venosa,Y ≥2 de los siguientes factores de riesgo: tratamiento sustitutivo hormonal,anticoncepción sistémica(con estrógeno),tabaquismo,diabetes,hipercolesterolemia, medicamentos para la hipertensión,cáncer,trastornos trombofílicos hereditarios (p.ej.,Factor V Leiden, insuficiencia de antitrombina III, síndrome antifosfolípido, etc.),hiperviscosidad(crioglobulinas y quilomicronemia)
    2.Cualquier anomalía clínicamente relevante, distinta de la PTI, que, en opinión del investigador, haga que el paciente no sea apto para participar en el estudio
    3.Pacientes de sexo femenino en periodo de lactancia o embarazadas durante la selección o antes de la dosis el día 1
    4.Antecedentes de abuso o dependencia de alcohol/drogas en los 12 meses posteriores al estudio
    5.Rituximab en los 3 meses posteriores a la selección
    6.El paciente ha consumido aspirina, compuestos que contienen aspirina, salicilatos, anticoagulantes, quinina o antinflamatorios no esteroideos(AINE)durante más de 3 días consecutivos en las 2 semanas posteriores al inicio del estudio y hasta el final del mismo
    7.Consumo de cualquier complemento herbario o alimenticio, salvo complementos vitamínicos o minerales, en la semana posterior al inicio del estudio
    8.Si se conoce por el historial médico anterior, falta de respuesta a la IgIV. La falta de respuesta a la IgIV se define como un aumento del recuento plaquetario que no superó los 50 × 10^9/l en los 14 días posteriores al tratamiento.
    9.Antecedentes de aglutinación plaquetaria que impide la medición fiable de los recuentos plaquetarios.
    10.Cualquier indicio clínico o analítico de infección por VIH.
    Para los criterios de exclusión,los pacientes con n.° 11 - 17 ver el protocolo;aquí no hay más posibilidades
    E.5 End points
    E.5.1Primary end point(s)
    Part A:
    Incidence and severity of adverse events (AEs) following administration of M254 at single dose levels.
    Clinically significant changes in clinical safety labs, vital signs, and electrocardiograms (ECGs) with M254 administration.

    Part B:
    Incidence and severity of AEs following administration of M254 at single dose levels and 1000 mg/kg IVIg.
    Clinically significant changes in clinical safety labs, vital signs, and ECGs following M254 administration and 1000 mg/kg IVIg.

    Part C:
    Incidence and severity of AEs of M254 and 1000 mg/kg IVIg.
    Clinically significant changes in clinical safety labs, vital signs, and ECGs following M254 administration and 1000 mg/kg IVIg.
    Platelet response after M254 administration compared to IVIg.

    Part D:
    Incidence and severity of AEs with repeated administration of M254.
    Clinically significant changes in clinical safety labs, vital signs, and ECGs with M254 administration.
    Parte A:
    Incidencia e intensidad de acontecimientos adversos (AA) después de la administración de M254 en niveles de dosis única
    Cambios clínicamente significativos en las pruebas analíticas de seguridad clínica, las constantes vitales y los electrocardiogramas (ECG) con la administración de M254

    Parte B:
    Incidencia e intensidad de AA tras la administración de M254 en niveles de dosis única y 1000 mg/kg de IgIV
    Cambios clínicamente significativos en las pruebas analíticas de seguridad clínica, las constantes vitales y los ECG después de la administración de M254 y 1000 mg/kg de IgIV

    Parte C:
    Incidencia e intensidad de AA de M254 y 1000 mg/kg de IgIV
    Cambios clínicamente significativos en las pruebas analíticas de seguridad clínica, las constantes vitales y los ECG después de la administración de M254 y 1000 mg/kg de IgIV
    Respuesta plaquetaria después de la administración de M254 en comparación con IgIV

    Parte D:
    Incidencia e intensidad de AA con la administración repetida de M254
    Cambios clínicamente significativos en las pruebas analíticas de seguridad clínica, las constantes vitales y los ECG con la administración de M254
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study
    A lo largo del estudio
    E.5.2Secondary end point(s)
    Part A:
    Measurements of PK parameters of M254 following the administration of a single intravenous dose.

    Part B:
    Measurements of PK parameters of M254 following the administration of a single intravenous dose.

    Part C:
    Measurements of PK parameters of M254 following the administration of a single intravenous dose.

    Part D:
    Measurements of PK parameters of M254 following the administration of a repeated intravenous doses.
    Platelet response after repeated M254 administration.
    Parte A:
    Mediciones de los parámetros de FC de M254 después de la administración de una dosis intravenosa única

    Parte B:
    Mediciones de los parámetros de FC de M254 después de la administración de una dosis intravenosa única

    Parte C:
    Mediciones de los parámetros de FC de M254 después de la administración de una dosis intravenosa única

    Parte D:
    Mediciones de los parámetros de FC de M254 después de la administración de dosis intravenosas repetidas
    Respuesta plaquetaria después de la administración repetida de M254
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study
    A lo largo 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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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
    Tolerability
    Tolerabilidad
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    El estudio es doble ciego para la parte A(Solo en Holanda),pero es abierto para el resto del estudio
    Study is double blind for Part A (Netherlands only), but it is open for the remainder of the trial.
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Belgium
    France
    Hungary
    Italy
    Netherlands
    Poland
    Spain
    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
    LVLS
    Ultima visita del ultimo paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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 years2
    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: 70
    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 Yes
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 62
    F.4.2.2In the whole clinical trial 70
    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
    Ningumo
    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 Decision2019-06-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-05
    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
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