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    Summary
    EudraCT Number:2021-003160-27
    Sponsor's Protocol Code Number:Sobi.PEGCET-101
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-04-05
    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 number2021-003160-27
    A.3Full title of the trial
    A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Cold Agglutinin Disease (CAD)
    Estudio multicéntrico de fase III, aleatorizado, doble ciego y controlado con un placebo, para evaluar la eficacia y la seguridad del pegcetacoplán en pacientes con enfermedad por crioaglutininas
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    “A Randomized, Double-blind, Placebo-controlled Study to Evaluate Pegcetacoplan in Patients with Cold Agglutinin Disease (CAD)”
    Estudio aleatorizado, doble ciego y controlado con un placebo, para evaluar el pegcetacoplán en pacientes con enfermedad por crioaglutininas
    A.4.1Sponsor's protocol code numberSobi.PEGCET-101
    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 SponsorSwedish Orphan Biovitrum AB
    B.1.3.4CountrySweden
    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 supportSwedish Orphan Biovitrum AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSwedish Orphan Biovitrum AB
    B.5.2Functional name of contact pointAnke Arnold
    B.5.3 Address:
    B.5.3.1Street AddressTomtebodavägen 23A
    B.5.3.2Town/ citySolna
    B.5.3.3Post codeSE-112-76
    B.5.3.4CountrySweden
    B.5.4Telephone number0041793629799
    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 Yes
    D.2.1.1.1Trade name APL-2
    D.2.1.1.2Name of the Marketing Authorisation holderEU/3/19/2201
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2201
    D.3 Description of the IMP
    D.3.1Product namePEGCETACOPLAN (APL‑2)
    D.3.2Product code 2019171-69-6
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.4EV Substance CodeSUB195466
    D.3.10 Strength
    D.3.10.1Concentration unit IU/mg international unit(s)/milligram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number90 to 1440
    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 placeboSolution for injection/infusion
    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
    Patients with Cold Agglutinin Disease (CAD)
    Pacientes con enfermedad por crioaglutininas
    E.1.1.1Medical condition in easily understood language
    Patients with Cold Agglutinin Disease (CAD)
    Pacientes con enfermedad por crioaglutininas
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate the efficacy of twice-weekly subcutaneous (s.c.) 1080-mg infusions of pegcetacoplan compared with that of placebo in patients with CAD
    Demostrar la eficacia del pegcetacoplán, administrado en dos infusiones subcutáneas semanales de 1080 mg, en comparación con un placebo, en pacientes con enfermedad por crioaglutininas
    E.2.2Secondary objectives of the trial
    • To demonstrate the effect of pegcetacoplan on the number of packed red blood cell (PRBC) transfusions in patients with CAD.
    • To demonstrate the effect of pegcetacoplan on health-related quality of life in patients with CAD.
    • To assess the effect of pegcetacoplan on clinical laboratory markers of hemolysis and transfusion dependence in patients with CAD.
    • To determine the durability of response in patients with CAD receiving pegcetacoplan.
    • To assess tolerability, safety and immunogenicity of pegcetacoplan in patients with CAD.
    • To describe long-term effect of pegcetacoplan in patients with CAD.
    •Demostrar el efecto del pegcetacoplán sobre la cantidad de transfusiones de concentrados de hematíes en pacientes con enfermedad por crioaglutininas.
    •Demostrar el efecto del pegcetacoplán sobre la calidad de vida relacionada con la salud en pacientes con enfermedad por crioaglutininas.
    •Evaluar el efecto del pegcetacoplán sobre los marcadores analíticos de hemólisis y la dependencia transfusional en pacientes con enfermedad por crioaglutininas.
    •Determinar la duración de la respuesta de los pacientes con enfermedad por crioaglutininas tratados con pegcetacoplán.
    •Evaluar la tolerabilidad, la seguridad y la inmunogenia del pegcetacoplán en pacientes con enfermedad por crioaglutininas.
    •Describir los efectos del tratamiento prolongado con pegcetacoplán en los pacientes con enfermedad por crioaglutininas.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age18 years or older.
    2. Diagnosis of primary CAD on the basis of the presence of all the following criteria:
    a Signs of hemolysis with abnormal values by at least 2 of the following hemolytic markers:
    i Reduced haptoglobin level (< LLN).
    ii Elevated LDH level (> ULN).
    iii Elevated indirect bilirubin level (> ULN; > 3 x ULN for patients with Gilbert-Meulengracht Syndrome).
    iv Increased ARC (above the ULN).
    b Monospecific direct antiglobulin test strongly positive for C3d.
    c Cold agglutinin titer ≥ 64 at 4 °C.
    3. Hb level ≤ 9 g/dL.
    4. Documented results from bone marrow biopsy within 1 year of screening with lymphoproliferative infiltration ≤ 20 %. Patients who have not received a bone marrow biopsy within 1 year of their screening visit or those patients for whom bone marrow biopsy reports are incomplete or unavailable will be required to receive a bone marrow biopsy to determine eligibility.
    5. Body weight ≤ 100 kg.
    6. Either have vaccination against Streptococcus pneumoniae, Neisseria meningitidis (Types A, C, W, Y, and B), and Haemophilus influenzae (Type B) within 2 years prior to screening or agree to receive vaccination during screening as follows:
    First dose of vaccine against N. meningitidis Types A, C, W, and Y at least 2 weeks prior to start of study drug with second dose 2 months late (Study Day 57), and then boosters every 5 years.
    First dose of the vaccine against N. meningitidis Type B at least 2 weeks prior to start of study drug with a second dose after at least 1 month (Study Day 29). First booster dose 1 year later, and then additional booster doses every 2 to 3 years.
    S. pneumoniae: pneumococcal conjugate vaccine 23 (PCV13) and/or pneumococcal polysaccharide vaccine 23 (PPSV23) as per Advisory Committee on Immunization Practices (ACIP) guidelines for adults or children with immunocompromising conditions.
    H. influenzae Type B: 1 dose at least 2 weeks prior to start of study drug.
    Vaccination is mandatory, unless documented evidence exists that patients are non responders to vaccination. Patients who were not previously vaccinated should not receive multiple vaccines on the same day.
    7. Women of childbearing potential (WOCBP), defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last IMP dose.
    Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
    8.Men must agree to the following for the duration of the study and 8 weeks after their last IMP dose:
    a Avoid fathering a child.
    b Use protocol-defined methods of contraception.
    c Refrain from donating sperm.
    9. Willing and able to give written informed consent.
    1.Edad ≥18 años.
    2.Diagnóstico de enfermedad por crioaglutininas primaria, determinada por la presencia de todos los factores siguientes:
    a)Signos de hemólisis, con valores alterados en al menos dos de los marcadores siguientes:
    i.reducción del nivel de haptoglobina (por debajo del LIN);
    ii.elevación del nivel de LDH (por encima del LSN);
    iii.elevación del nivel de bilirrubina indirecta (por encima del LSN; >3 × LSN si el paciente tiene síndrome de Gilbert Meulengracht);
    iv.elevación de la cifra absoluta de reticulocitos (por encima del LSN).
    b)Prueba directa monoespecífica de antiglobulinas fuertemente positiva para C3d.
    c)Título de crioaglutininas ≥64 a 4 °C.
    3.Nivel de hemoglobina ≤9 g/dl.
    4.Biopsia de médula ósea en el año anterior a la selección, con resultados documentados que indiquen una infiltración linfoproliferativa ≤20%. Será preciso realizar una biopsia de médula ósea, a los efectos de este criterio de inclusión, si no se ha realizado una en el año anterior a la selección o si los resultados son incompletos o no están disponibles.
    5.Peso corporal ≤100 kg.
    6.Vacunación contra Streptococcus pneumoniae, Neisseria meningitidis (tipos A, C, W, Y y B) y Haemophilus influenzae (tipo B) en los dos años anteriores, o administración de las siguientes vacunas en la fase de selección:
    oprimera dosis de la vacuna contra N. meningitidis de los tipos A, C, W e Y al menos dos semanas antes de iniciar el tratamiento en estudio, y una segunda dosis al cabo de dos meses (día 57 del ensayo), y a continuación refuerzos cada cinco años;
    oprimera dosis de la vacuna contra N. meningitidis de tipo B al menos dos semanas antes de iniciar el tratamiento en estudio, y una segunda dosis al cabo de al menos un mes (día 29 del ensayo); primera dosis de refuerzo al cabo de un año y a continuación refuerzos adicionales cada dos o tres años;
    oS. pneumoniae: vacuna antineumocócica conjugada tridecavalente (PCV 13) o icositrivalente (PPSV 23), según lo estipulado en las directrices del Comité Asesor sobre Prácticas de Inmunización de los CDC estadounidenses (ACIP) para los adultos y menores con trastornos asociados a inmunodeficiencias;
    oH. influenzae de tipo B: una dosis al menos dos semanas antes de comenzar a recibir el fármaco en estudio.
    La vacunación es obligatoria, salvo que se haya documentado que el paciente no responde a la vacunación. Los pacientes que no estén vacunados no deben recibir más de una vacuna el mismo día.
    7.En el caso de las mujeres en edad fértil (definidas como las que hayan pasado la menarquia y NO presenten esterilidad irreversible ni sean posmenopáusicas), negativo en la prueba del embarazo en el momento de la selección y voluntad de utilizar los métodos anticonceptivos definidos en el protocolo durante toda su participación en el ensayo y durante las ocho semanas siguientes a la última dosis del medicamento en investigación.
    Nota: Se entenderá que es posmenopáusica la mujer que lleve 12 meses consecutivos sin menstruar, siempre que no concurra otra causa médica.
    8.En el caso de los varones, voluntad de respetar los compromisos siguientes durante toda su participación en el ensayo y durante las ocho semanas siguientes a la última dosis del medicamento en investigación:
    a)no engendrar descendencia;
    b)utilizar los anticonceptivos definidos en el protocolo;
    c)abstenerse de donar semen.
    9.Voluntad y capacidad de otorgar el consentimiento informado por escrito.
    E.4Principal exclusion criteria
    1. Have received other anticomplement therapies (approved or investigational) within 5 half-lives of the agent prior to randomization (e.g., eculizumab within 10 weeks, ravulizumab within 36 weeks or sutimlimab within 4 weeks) and are not able or willing to refrain from using them during the study.
    2. Treatment with rituximab monotherapy within 12 weeks prior to randomization, or rituximab combination therapies (e.g., with bendamustine, fludarabine, other cytotoxic drugs or ibrutinib) within 16 weeks prior to randomization.
    3. Use of prohibited medications as described in the protocol. The list of acceptable medications and required stable regimen periods are outlined in the study protocol.
    4. Diagnosis of systemic lupus erythematosus or other autoimmune diseases with antinuclear antibodies.
    5. History of an aggressive lymphoma or presence of a lymphoma requiring therapy.
    6. Have received an organ transplant.
    7. Cold agglutinin syndrome secondary to Mycoplasma pneumoniae, Epstein-Barr virus or other specific causative infection.
    8. HIV or hepatitis C virus detectable by polymerase chain reaction at screening or documented in the patient’s medical record.
    9. Chronic inactive hepatitis B virus with viral loads > 1000 IU/mL (> 5000 copies/mL) at screening or documented in the patient’s medical record. Eligible patients who are chronic active carriers (≤ 1000 IU/mL) must receive prophylactic antiviral treatment (e.g., entecavir, tenofovir, lamivudine) according to local country guidelines.
    10. Presence of an active malignant disease within the last 12 months other than skin basal cell carcinoma or in situ carcinoma of the cervix. A low-grade lymphoproliferative bone marrow disorder not requiring therapy by itself is not defined as a malignant disease in this context.
    11. A monospecific direct antiglobulin test result of IgG > 1+.
    12. Presence or suspicion of liver dysfunction as indicated by elevated alanine aminotransferase (ALT) > 2.5 x ULN, or direct bilirubin levels > 2 x ULN .
    13. Presence or suspicion of severe recurrent or chronic infections that, in the opinion of the investigator, increase the patient’s risk by participating in the study.
    14. Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days prior to screening period.
    15. If breastfeeding, is unwilling to discontinue for the duration of study and for at least 8 weeks after the final IMP dose.
    16. Inability to cooperate with study procedures.
    17. Any disease(s), psychiatric condition, metabolic dysfunction, or findings from a physical examination or clinical laboratory test result that would cause reasonable suspicion of a disease or condition that may jeopardize the patient’s wellbeing, that may increase the risk associated with study participation, that may affect the interpretation of the results, or that would make the patient unsuitable for this study.
    1.Haber recibido otros tratamientos anti complemento (autorizados o en fase de investigación) antes de la aleatorización, en un período equivalente a cinco semividas del producto (p. ej., eculizumab, 10 semanas; ravulizumab, 36 semanas; y sutimlimab, 4 semanas) y falta de voluntad o incapacidad para no seguir estos tratamientos durante el ensayo.
    2.Tratamiento con rituximab en monoterapia en las 12 semanas antes de la aleatorización o politerapias con rituximab (p. ej., con bendamustina, fludarabina, otros citotóxicos o ibrutinib) en las 16 semanas antes de la aleatorización.
    3.Administración de medicamentos prohibidos por el protocolo. En el protocolo se enumeran los medicamentos aceptables y el correspondiente período durante el cual debe haberse mantenido estable la posología.
    4.Diagnóstico de lupus eritematoso sistémico u otras enfermedades autoinmunitarias con anticuerpos antinucleares.
    5.Antecedentes de linfoma agresivo o presencia de un linfoma que requiera tratamiento.
    6.Haber recibido un trasplante de órgano.
    7.Síndrome de crioaglutininas secundario a una infección por Mycoplasma pneumoniae, virus de Epstein Barr u otros agentes causales.
    8.VIH o virus de la hepatitis C detectable mediante reacción en cadena de la polimerasa en la selección o documentado en la historia clínica.
    9.Virus de la hepatitis B crónico inactivo, con carga vírica >1000 UI/ml (>5000 copias por mililitro) en la selección o documentado en la historia clínica. Los pacientes que sean portadores crónicos activos (≤1000 UI/ml) deben seguir profilaxis antiviral (p. ej., entecavir, tenofovir o lamivudina) según los protocolos nacionales.
    10.Enfermedad maligna activa en los últimos 12 meses, a excepción del carcinoma basocelular de la piel y el carcinoma in situ de cuello uterino. A los efectos de este criterio de exclusión, no se entenderá como enfermedad maligna un trastorno linfoproliferativo de la médula ósea, de bajo grado, que no requiera tratamiento.
    11.Prueba directa monoespecífica de antiglobulinas con resultado de IgG >1+.
    12.Presencia o sospecha de disfunción hepática por la elevación de la alanina aminotransferasa (ALT) >2,5 × LSN o por niveles de bilirrubina directa >2 × LSN.
    13.Presencia o sospecha de infecciones severas, recurrentes o crónicas que, en opinión del investigador, acrecienten el riesgo del paciente en caso de participar en el ensayo.
    14.Participación en otros ensayos experimentales con medicamentos o exposición a otros fármacos, productos sanitarios o procedimientos en investigación en los 30 días antes de la fase de selección.
    15.En caso de lactancia materna, falta de voluntad para interrumpir la lactancia durante la participación y durante al menos ocho semanas después de la última dosis del medicamento en investigación.
    16.Incapacidad de colaborar con las actividades del protocolo.
    17.Cualquier afección, enfermedad psiquiátrica, disfunción metabólica o alteración detectada en la exploración física o en los análisis clínicos que permita sospechar cabalmente de la presencia de una dolencia o trastorno que suponga un riesgo para el bienestar del paciente, que incremente el riesgo asociado a la participación en el ensayo, que pueda afectar a la interpretación de los resultados o que contraindique la participación del paciente en el ensayo.
    E.5 End points
    E.5.1Primary end point(s)
    Response to treatment at Week 24.
    Response is defined as:
    • An increase in hemoglobin (Hb) of ≥ 1.5 g/dL from Baseline or Hb
    normalization at Week 16; AND
    • Maintenance of this effect from Week 16 to Week 24; AND
    • The absence of PRBC transfusions (between Week 5 and Week 24).
    Note: Hb normalization is defined as within normal range (between the defined
    upper and lower limits of normal [ULN and LLN]), as set by the testing
    laboratory.
    Respuesta al tratamiento a la semana 24.
    Se define respuesta como:
    •un incremento de la hemoglobina ≥1,5 g/dl respecto del valor inicial
    o la normalización a la semana 16; Y ADEMÁS
    •el mantenimiento de este efecto entre la semana 16 y la semana 24; Y ADEMÁS
    •la ausencia de transfusiones de concentrados de hematíes (desde la semana 5 hasta la semana 24).
    Nota: Por normalización de la hemoglobina se entiende un valor situado dentro del intervalo normal (entre los límites superior e inferior de la normalidad, LSN y LIN), según los intervalos del laboratorio que realice los análisis.
    E.5.1.1Timepoint(s) of evaluation of this end point
    • Transfusion avoidance (Yes/No) from Week 5 to Week 24.
    • Change from Baseline to Week 24 in Hb level.
    • Change from Baseline to Week 24 in the Functional Assessment of Cancer Therapy–Anemia/Fatigue (FACT-An) score.
    •Ausencia de transfusiones (sí/no) entre la semana 5 y la semana 24.
    •Variación del nivel de hemoglobina a la semana 24.
    •Variación de la puntuación FACT An (Functional Assessment of Cancer Therapy Anemia/Fatigue) a la semana 24.
    E.5.2Secondary end point(s)
    Part A:
    • Number of PRBC transfusions from Week 5 to Week 24.
    • Change from Baseline to Week 24 in the following:
    • Lactate dehydrogenase (LDH) level.
    • Haptoglobin level.
    • Indirect bilirubin level.
    • Absolute reticulocyte counts (ARC).
    • D-dimer level.
    • Normalization of markers of hemolysis at Week 24, specifically:
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Time to normalization from Baseline to Week 24 for the following:
    • Hb level.
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Number of PRBC units transfused from Week 5 to Week 24.
    • Change from Baseline to Week 24 in the following:
    • 12-Item Short Form Survey (SF-12) score.
    • 5-Level EuroQol 5-Dimension (EQ-5D-5L) score.
    Part B:
    • Change from Baseline to Week 48 in the following:
    • Hb level.
    • LDH level.
    • Haptoglobin level.
    • Indirect bilirubin level.
    • ARC.
    • D-dimer level.
    • Normalization of markers of hemolysis at Week 48, specifically:
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Time to normalization from Baseline to Week 48 for the following:
    • Hb level.
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Durability of response for patients randomized to pegcetacoplan who achieve the primary endpoint at Week 24 (patients are considered to
    maintain response until they receive a blood transfusion or they experience Hb decrease by at least 1.5 g/dL from the highest level achieved or to below 10.0 g/dL).
    • Change from Baseline to Week 48 in the following:
    • FACT-An score.
    • SF-12 score.
    • EQ-5D-5L score.
    Parte A:
    •Número de transfusiones de concentrados de hematíes entre la semana 5 y la semana 24.
    •Variación a la semana 24 de los parámetros siguientes:
    • nivel de lactato deshidrogenasa (LDH);
    • nivel de haptoglobina;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos;
    • nivel de dímero D.
    •Normalización de los marcadores de hemólisis a la semana 24, concretamente:
    • nivel de LDH;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    •Tiempo hasta la normalización, entre el inicio y la semana 24, de los parámetros siguientes:
    • nivel de hemoglobina;
    • nivel de LDH;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    •Unidades de concentrado de eritrocitos transfundidas entre la semana 5 y la semana 24.
    •Variación a la semana 24 de los parámetros siguientes:
    • puntuación del cuestionario de salud abreviado de 12 ítems (SF 12);
    • puntuación del cuestionario EuroQoL de 5 niveles y 5 dimensiones (EQ 5D 5L).
    Parte B:
    •Variación a la semana 48 de los parámetros siguientes:
    • nivel de hemoglobina;
    • nivel de LDH;
    • nivel de haptoglobina;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    • nivel de dímero D.
    •Normalización de los marcadores de hemólisis a la semana 48, concretamente:
    • nivel de LDH;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    •Tiempo hasta la normalización, entre el inicio y la semana 48, de los parámetros siguientes:
    • nivel de hemoglobina;
    • nivel de LDH;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    •Duración de la respuesta de los pacientes asignados al pegcetacoplán que consiguen el criterio principal a la semana 24 (se considerará que la respuesta se mantiene hasta que el paciente reciba una transfusión de sangre o hasta que se reduzca el nivel de hemoglobina ≥1,5 g/dl respecto al nivel más alto alcanzado o por debajo de 10,0 g/dl).
    •Variación a la semana 48 de los parámetros siguientes:
    • puntuación del cuestionario FACT An;
    • puntuación del cuestionario SF 12;
    • puntuación del cuestionario EQ 5D 5L;
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part C:
    • Change from Baseline to Week 96 in the following:
    • Hb level.
    • LDH level.
    • Haptoglobin level.
    • Indirect bilirubin level.
    • ARC.
    • D-dimer level.
    • Normalization of markers of hemolysis at Week 96, specifically:
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Time to normalization from Baseline to Week 96 for the following:
    • Hb level.
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Change from Baseline to Week 96 in the following:
    • FACT-An score.
    • SF-12 score.
    • EQ-5D-5L score
    Parte C:
    •Variación a la semana 96 de los parámetros siguientes:
    • nivel de hemoglobina;
    • nivel de LDH;
    • nivel de haptoglobina;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    • nivel de dímero D.
    •Normalización de los marcadores de hemólisis a la semana 96, concretamente:
    • nivel de LDH;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    •Tiempo hasta la normalización, entre el inicio y la semana 96, de los parámetros siguientes:
    • nivel de hemoglobina;
    • nivel de LDH;
    • nivel de bilirrubina indirecta;
    • cifra absoluta de reticulocitos.
    •Variación a la semana 96 de los parámetros siguientes:
    • puntuación del cuestionario FACT An;
    • puntuación del cuestionario SF 12;
    • puntuación del cuestionario EQ 5D 5L;
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) 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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    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
    Canada
    Georgia
    Japan
    Russian Federation
    Ukraine
    United States
    Austria
    France
    Germany
    Italy
    Norway
    United Kingdom
    Bulgaria
    Spain
    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
    The end of study is when all randomized patients have terminated the 6week safety follow-up period and performed the EOS visit or the Early termination visit in case of discontinuation for any reason from the study before completion.
    El final del estudio se producirá cuando todos los pacientes aleatorizados terminen el período de seguimiento de seguridad de 6 semanas y hayan realizado la visita de final de estudio (EoS) o la visita de terminación anticipada en caso de interrupción del estudio por cualquier motivo antes de la finalización.
    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 months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months11
    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) 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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 60
    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 Decision2022-04-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-31
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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