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    Summary
    EudraCT Number:2021-001211-90
    Sponsor's Protocol Code Number:ALXN1830-WAI-202
    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:2021-07-02
    Trial results View 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 number2021-001211-90
    A.3Full title of the trial
    A Phase 2, Multiple Ascending Dose, Randomized, Double-Blind, Placebo-Controlled Study of ALXN1830 Administered Subcutaneously in Patients with Warm Autoimmune Hemolytic Anemia (WAIHA)
    Estudio en fase II, de dosis múltiples ascendentes, aleatorizado, doble ciego y controlado con placebo de ALXN1830 administrado por vía subcutánea en pacientes con anemia hemolítica autoinmunitaria por anticuerpos calientes (WAIHA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    ALXN1830 in Patients with Warm Autoimmune Hemolytic Anemia
    ALXN1830 en pacientes con anemia hemolítica autoinmunitaria por anticuerpos calientes
    A.4.1Sponsor's protocol code numberALXN1830-WAI-202
    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 SponsorAlexion 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 supportAlexion Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address103-105 Rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    B.5.3.4CountryFrance
    B.5.4Telephone number3493 2723019
    B.5.5Fax number33747100611
    B.5.6E-mailclinicaltrials.eu@alexion.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 nameALXN1830
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNORILANOLIMAB
    D.3.9.1CAS number 2066544-85-0
    D.3.9.2Current sponsor codeALXN1830
    D.3.9.3Other descriptive nameSYNT001
    D.3.9.4EV Substance CodeSUB197997
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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 infusion
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Warm Autoimmune Hemolytic Anemia (WAIHA)
    Anemia hemolítica autoinmunitaria por anticuerpos calientes
    E.1.1.1Medical condition in easily understood language
    Autoimmune disorder characterized by the premature destruction of healthy red blood cells
    Trastorno autoinmunitario caracterizado por la destrucción prematura de eritrocitos sanos
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Determine the efficacy of ALXN1830 compared to placebo in the treatment of WAIHA
    Determinar la eficacia de ALXN1830 en comparación con placebo en el tratamiento de la WAIHA.
    E.2.2Secondary objectives of the trial
    1. Assess the effect of ALXN1830 on the need for transfusions compared to placebo
    2. Assess the effect on ALXN1830 on anemia compared to placebo
    3. Evaluate the efficacy of ALXN1830 after 4 weeks compared to placebo
    4. Assess the effect on ALXN1830 on hemolysis compared to placebo
    5. Assess the effect of ALXN1830 on corticosteroid usage compared to placebo
    6. Assess the safety and tolerability of ALXN1830 compared to placebo
    7. Assess the immunogenicity of ALXN1830 compared to placebo
    8. Assess the pharmacokinetics (PK) of ALXN1830
    9. Assess the effect of ALXN1830 on serum total IgG levels compared to placebo
    10. Assess the effect of ALXN1830 on other PD biomarkers compared to placebo
    1.Evaluar el efecto de ALXN1830 en comparación con placebo sobre la necesidad de transfusiones de sangre.
    2. Evaluar el efecto de ALXN1830 en comparación con placebo sobre la anemia.
    3. Evaluar la eficacia de ALXN1830 en comparación con placebo después de 4 semanas.
    4. Evaluar el efecto de ALXN1830 en comparación con placebo sobre la hemolisis.
    5. Evaluar el efecto de ALXN1830 en comparación con placebo sobre el uso de corticosteroides.
    6. Evaluar la seguridad y la tolerabilidad de ALXN1830 en comparación con el placebo.
    7. Evaluar la inmunogenicidad de ALXN1830 en comparación con el placebo.
    8. Evaluar la farmacocinética (FC) de ALXN1830.
    9. Evaluar el efecto de ALXN1830 sobre las concentraciones séricas de IgG total en comparación con placebo.
    10. Evaluar el efecto de ALXN1830 sobre otros biomarcadores FD en comparación con placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participant must be at least 18 years of age inclusive, at the time of signing the informed consent
    2. Diagnosed with primary or secondary WAIHA at least 6 weeks prior to Screening
    3. Failed or have not tolerated at least one prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine
    4. Hemoglobin < 10 g/dL and ≥ 6 g/dL at Screening
    5. Positive direct antiglobulin test (Coombs) (IgG positive who are positive or negative for the presence of complement C3 [C3]) at Screening
    6. Evidence of active hemolysis
    1. El participante debe tener 18 años o más en el momento de firmar el consentimiento informado.
    2. Diagnóstico de WAIHA primaria o secundaria al menos 6 semanas antes de la selección
    3. Fracaso o intolerancia al menos a una pauta de tratamiento previa de la WAIHA; por ejemplo, corticosteroides, rituximab, azatioprina, ciclofosfamida, ciclosporina, micofenolato mofetilo, danazol o vincristina
    4. Hemoglobina <10 g/dl y ≥6 g/dl en la selección
    5. Prueba de antiglobulina directa (Coombs) positiva (IgG positiva que sea positiva o negativa para la presencia del complemento C3 [C3]) en la selección
    6. Signos de hemólisis activa
    E.4Principal exclusion criteria
    1. Packed RBC or whole blood transfusions in the 2 weeks prior to Screening
    2. History of cold antibody autoimmune hemolytic anemia (AIHA), cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria
    3. History of drug-induced or infection-related immune hemolytic anemia
    4. Iron, folic acid, or vitamin B12 deficiency
    5. Participants with Evan’s syndrome
    6. History of leukemia, Hodgkin, or non Hodgkin lymphoma
    7. History of solid malignancy other than basal cell or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, or other malignancy for which the participant had not been disease free for at least 5 years
    8. Active infection or history of recurrent systemic infections in the 2 years prior to Screening
    10. Systemic lupus erythematosus (SLE) or other autoimmune disease that is not stable or not well controlled on current therapy
    1. Transfusiones de concentrados de eritrocitos o de sangre completa en las 2 semanas previas a la selección
    2. Antecedentes de anemia hemolítica autoinmunitaria (AIHA) por anticuerpos por frío, síndrome de aglutinina por frío, AIHA de tipo mixto o hemoglobinuria paroxística por frío
    3. Antecedentes de anemia hemolítica inmunitaria inducida por fármacos o relacionada con infecciones
    4. Deficiencia de hierro, ácido fólico o vitamina B12
    5. Participantes con síndrome de Evan
    6. Antecedentes de leucemia, linfoma de Hodgkin o linfoma no hodgkiniano.
    7. Antecedentes de neoplasia maligna sólida distinta del carcinoma basocelular o espinocelular de la piel, carcinoma in situ de cuello uterino u otra neoplasia maligna por cuya causa el participante no haya estado libre de enfermedad durante al menos 5 años
    8. Infección activa o antecedentes de infecciones sistémicas recurrentes en los 2 años previos a la selección
    10. Lupus eritematoso sistémico (LES) u otra enfermedad autoinmunitaria que no se mantenga estable o no esté bien controlada con el tratamiento actual
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants achieving a ≥ 2 g/dL increase in Hgb from Baseline (Day 1) to the end of Primary Treatment, without requiring any increase in the dose of an existing WAIHA medication after Day 1 and without pRBC transfusions after Day 14
    Proporción de participantes que experimenten un aumento ≥2 g/dl de la Hb entre el momento basal (día 1) y el final del tratamiento principal, sin necesidad de aumentar la dosis de una medicación existente para la WAIHA después del día 1 y sin transfusiones de concentrado de eritrocitos después del día 14.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of Primary Treatment (Day 57 or Day 85)
    Final del tratamiento principal (día 57 o día 85)
    E.5.2Secondary end point(s)
    1. Total number of units of pRBCs transfused:
    2. Durability: number of Hgb measurements ≥ 2 g/dL
    4. Proportion of participants who require new WAIHA rescue medication or any increase in the dose of an existing WAIHA medication or pRBC transfusions for the treatment of anemia
    5. Proportion of participants achieving a ≥ 2 g/dL increase in Hgb, without requiring any increase in the dose of an existing WAIHA medication
    6. Markers of hemolysis: absolute and percentage change in serum lactate dehydrogenase (LDH) levels, absolute reticulocyte count, serum indirect bilirubin, and serum haptoglobin
    7. Total corticosteroid usage from Baseline (Day 1) to the end of Primary Treatment
    13. Incidence of AEs, SAEs and AESIs over time
    15. Serum concentrations of ALXN1830 over time
    16. Serum total IgG levels
    1. Número total de unidades de concentrado de eritrocitos transfundidas.
    2. Durabilidad: número de mediciones de Hb ≥2 g/dl.
    4. Proporción de participantes que necesitan nueva medicación de rescate para la WAIHA o cualquier aumento de la dosis de una medicación existente para la WAIHA o transfusiones de concentrado de eritrocitos para el tratamiento de la anemia.
    5. Proporción de participantes que experimenten un aumento ≥2 g/dl de la Hb sin necesidad de aumentar la dosis de una medicación existente para la WAIHA.
    6. Marcadores de hemolisis: cambio absoluto y porcentual de la concentración sérica de lactato deshidrogenasa (LDH), el recuento absoluto de reticulocitos, la bilirrubina indirecta sérica y la haptoglobina sérica.
    7. Uso total de corticosteroides desde el momento basal (día 1) hasta el final del tratamiento principal.
    13. Incidencia de AA, AAG y AAIE a lo largo del tiempo.
    15. Concentraciones séricas de ALXN1830 a lo largo del tiempo.
    16. Concentraciones séricas de IgG total.
    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 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 Yes
    E.6.10Pharmacogenetic Yes
    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) 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 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 trial3
    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 EEA10
    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
    France
    Germany
    Italy
    Korea, Republic of
    Spain
    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
    Date when the last participant completes the last visit (including follow-up)
    Fecha en la que el último participante complete la última vista (incluido el seguimiento)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months7
    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: 21
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 15
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 36
    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)
    Upon completion of the last study visit, participants will return to the care of their treating physician
    Una vez finalizada la última visita del estudio, los participantes volverán a la asistencia de su médico
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-08-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-04
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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