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    Summary
    EudraCT Number:2019-004055-37
    Sponsor's Protocol Code Number:ALXN1830-WAI-201
    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:2020-05-10
    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 number2019-004055-37
    A.3Full title of the trial
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Study of ALXN1830 in Patients with Warm Autoimmune Hemolytic Anemia
    Estudio en fase II, aleatorizado, doble ciego, controlado con placebo, de ALXN1830 en pacientes anemia hemolítica autoinmunitaria por anticuerpos calientes
    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-201
    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 number3493272 31 27
    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.3.2Product code ALXN1830
    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 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 number50
    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 placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    WAIHA
    AHAIC
    E.1.1.1Medical condition in easily understood language
    WAIHA
    AHAIC)
    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 adult subjects with WAIHA
    Determinar la eficacia de ALXN1830 en comparación con el placebo en sujetos adultos con AHAIC
    E.2.2Secondary objectives of the trial
    - Assess the effect of ALXN1830 on the need for transfusions compared to placebo
    - Assess the effect of ALXN1830 on quality of life compared to placebo
    - Assess the effect on ALXN1830 on anemia compared to placebo
    - Assess the effect of ALXN1830 on hemolysis compared to placebo
    - Assess the effect of ALXN1830 on corticosteroid usage compared to placebo
    - Assess the safety and tolerability of ALXN1830 compared to placebo
    - Assess the effect of ALXN1830 on serum total IgG levels compared to placebo
    - Evaluar el efecto de ALXN1830 sobre la necesidad de transfusiones en comparación con el placebo
    - Evaluar el efecto de ALXN1830 sobre la calidad de vida en comparación con el placebo
    - Evaluar el efecto de ALXN1830 sobre la anemia en comparación con el placebo
    - Evaluar el efecto de ALXN1830 sobre la hemólisis en comparación con el placebo
    - Evaluar el efecto de ALXN1830 sobre el uso de corticoesteroides en comparación con el placebo
    - Evaluar la seguridad y tolerabilidad de ALXN1830 en comparación con el placebo
    - Evaluar el efecto de ALXN1830 sobre los niveles de IgG total en suero en comparación
    con el placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject must be at least 18 years of age at the time of signing the informed consent
    2. Primary or secondary WAIHA, diagnosed 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
    6. Positive direct antiglobulin test (Coombs) (IgG positive who are positive or negative for the presence of complement C3 [C3]) at Screening
    7. Evidence of active hemolysis including any one of the below:
    a. LDH > upper limit of normal (ULN) or
    b. Haptoglobin < lower limit of normal (LLN) or
    c. Indirect bilirubin > ULN
    8. Total IgG > 500 mg/dL at Screening
    1. El sujeto debe tener al menos 18 años de edad en el momento de la firma del consentimiento informado
    2. AHAIC primaria o secundaria, diagnosticada al menos 6 semanas antes de la selección
    3. Haber fracasado o no haber tolerado al menos una pauta de tratamiento anterior para la AHAIC, por ejemplo, corticoesteroides, rituximab, azatioprina, ciclofosfamida, ciclosporina, micofenolato de mofetilo, danazol o vincristina
    4. Hemoglobina <10 g/dl y ≥6 g/dl
    6. Resultado positivo en la prueba de antiglobulina directa (prueba de Coombs) (IgG positivas con o sin presencia del complemento C3 [C3]) en la selección
    7. Indicios de hemólisis activa, incluidos cualquiera de los siguientes:
    a. LDH > límite superior de la normalidad (LSN) o
    b. Haptoglobina < límite inferior de la normalidad (LIN) o
    c. Bilirrubina indirecta > LSN
    8. IgG total >500 mg/dl en la selección
    E.4Principal exclusion criteria
    1. Packed RBC or whole blood transfusions in the 2 weeks prior to Screening
    2. History of cold antibody 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. History of leukemia, Hodgkin, or non-Hodgkin lymphoma. Exception: low-grade chronic lymphocytic leukemia (CLL) with no enlargement of lymph nodes, spleen, or liver, and normal platelet count as per central lab, not requiring treatment for CLL, no more than 3 years since the diagnosis of CLL and after discussion with the Sponsor’s Medical Monitor.
    6. 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 subject had not been disease-free for at least 5 years
    7. Active infection or history of recurrent systemic infections in the 2 years prior to Screening
    9. Systemic lupus erythematosus (SLE) or other autoimmune disease that is not stable or not well controlled on current therapy
    1. Transfusiones de concentrado de eritrocitos o sangre completa en las 2 semanas anteriores a la selección
    2. Antecedentes de AHAI por anticuerpos fríos, síndrome de aglutininas frías, AHAI de tipo mixto o hemoglobinuria paroxística por frío
    3. Antecedentes de anemia hemolítica autoinmunitaria inducida por fármacos o relacionada con infección
    4. Deficiencia de hierro, ácido fólico o vitamina B12
    5. Antecedentes de leucemia o de linfoma de Hodgkin o no Hodgkin. Excepción: leucemia linfocítica crónica (LLC) de bajo grado sin engrosamiento de los ganglios linfáticos, el bazo o el hígado, y con un recuento de plaquetas normal según el laboratorio central; no requiere tratamiento para la LLC; no han transcurrido más de 3 años desde el diagnóstico de LLC; y después de consultarlo con el supervisor médico del promotor.
    6. Antecedentes de neoplasia maligna sólida distinta al carcinoma basocelular o espinocelular de la piel, carcinoma de cuello uterino in situ u otra neoplasia maligna de la que el sujeto no haya estado libre durante al menos 5 años
    7. Infección activa o antecedentes de infecciones sistémicas recurrentes en los 2 años anteriores a la selección
    9. Lupus eritematoso sistémico (LES) u otra enfermedad autoinmunitaria que no sea estable o no esté bien controlada con el tratamiento actual
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects achieving a ≥ 2 g/dL increase in Hgb, without requiring increase in any WAIHA medication after Day 1 and without pRBC transfusions after Day 14.
    Proporción de sujetos que logran un aumento ≥2 g/dl en la Hgb, sin necesidad de aumentar ninguna medicación para la AHAIC después del día 1 y sin transfusiones de concentrado de eritrocitos (CE) después del día 14.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline (Day 1) to Day 92 (after 13 weeks of treatment)
    Desde el inicio (día 1) hasta el día 92 (después de 13 semanas de tratamiento)
    E.5.2Secondary end point(s)
    - Total number of units of pRBCs transfused from Day 15 to Day 92
    - Change in quality of life (QoL) assessed via EQ-5D-5L and Functional Assessment of Cancer Therapy Anemia (FACT-AN) from baseline to Day 92
    - Durability: number of weekly Hgb measurements ≥ 2 g/dL over baseline
    - Proportion of subjects who require new WAIHA rescue medication or increase in the dose of an existing WAIHA medication or pRBC transfusions (Day 15 to Day 92) for the treatment of anemia
    - Markers of hemolysis: absolute and percentage change from baseline to Day 92 in serum lactate dehydrogenase (LDH) levels, absolute reticulocyte count, serum indirect bilirubin, serum haptoglobin
    - Total corticosteroid use from Day 1 to Day 92
    - Safety and tolerability of ALXN1830 compared to placebo assessed by physical examinations, vital signs, ECGs, laboratory assessments, and incidence of AEs, SAEs and AESIs
    - Serum concentration of ALXN1830 over time
    - Mean and percentage decrease in serum total IgG levels by dose group (ALXN1830 vs placebo) and time point
    - Número total de unidades de CE transfundidas desde el día 15 hasta el día 92
    - Cambio en la calidad de vida (CdV) evaluada mediante el EQ-5D-5L y la evaluación funcional del tratamiento contra el cáncer-anemia (Functional Assessment of Cancer Therapy Anemia, FACT-AN) desde el inicio hasta el día 92
    - Durabilidad: número de mediciones semanales de Hgb ≥2 g/dl con respecto al inicio
    - Proporción de sujetos que necesitan una nueva medicación de rescate para la AHAIC, un aumento en la dosis de un medicamento ya existente para la AHAIC o transfusiones de CE (desde el día 15 hasta el día 92) para el tratamiento de la anemia
    - Marcadores de hemólisis: cambio absoluto y porcentual desde el inicio hasta el día 92 en los niveles séricos de lactato deshidrogenasa (LDH), recuento absoluto de reticulocitos, bilirrubina indirecta en suero, haptoglobina en suero
    - Uso total de corticoesteroides desde el día 1 hasta el día 92
    - Seguridad y tolerabilidad de ALXN1830 en comparación con el placebo evaluadas mediante exploraciones físicas, constantes vitales, ECG, pruebas analíticas e incidencia de AA, AAG y AAEI
    - Concentración sérica de ALXN1830 a lo largo del tiempo
    - Disminución media y porcentual de los niveles de IgG total en suero por grupo de dosis (ALXN1830 frente a placebo) y punto temporal
    E.5.2.1Timepoint(s) of evaluation of this end point
    The timepoint(s) is precised for each secondary end point at E.5.2
    Los puntos temporales para cada criterio de valoración secundario se detallan en E.5.2
    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 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) 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 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA38
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Canada
    France
    Germany
    Italy
    Korea, Republic of
    Netherlands
    Russian Federation
    Spain
    Taiwan
    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 years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 19
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 13
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 32
    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)
    Patients will return to the care of their treating physician at the completion of study participation.
    Los pacientes volverán a estar al cuidado de su médico responsable cuando finalice su participación en el estudio.
    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 Decision2020-05-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-05-28
    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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