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    Summary
    EudraCT Number:2021-001211-90
    Sponsor's Protocol Code Number:ALXN1830-WAI-202
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-10-22
    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 ConcernedItaly - Italian Medicines Agency
    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)
    Studio di fase 2, a dose multipla crescente, randomizzato, in doppio cieco, controllato con placebo su ALXN1830 somministrato per via sottocutanea in pazienti affetti da anemia emolitica autoimmune calda (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 in pazienti con anemia emolitica autoimmune calda
    A.3.2Name or abbreviated title of the trial where available
    N/A
    N/A
    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 INCORPORATED
    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 number0033787148158
    B.5.5Fax number0033747100611
    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.3.2Product code [N/A]
    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.3.9.4EV 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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 placeboConcentrate for solution for injection/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 emolitica autoimmune calda (WAIHA)
    E.1.1.1Medical condition in easily understood language
    Autoimmune disorder characterized by the premature destruction of healthy red blood cells
    Disturbo autoimmune caratterizzato dalla distruzione prematura dei globuli rossi sani
    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
    Determinare l'efficacia di ALXN1830 rispetto al placebo nel trattamento di 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. Valutare l'effetto di ALXN1830 sulla necessità di trasfusioni rispetto al placebo
    2. Valutare l'effetto di ALXN1830 sull'anemia rispetto al placebo
    3. Valutare l'efficacia di ALXN1830 dopo 4 settimane rispetto al placebo
    4. Valutare l'effetto di ALXN1830 sull'emolisi rispetto al placebo
    5. Valutare l'effetto di ALXN1830 sull'uso di corticosteroidi rispetto al placebo
    6. Valutare la sicurezza e la tollerabilità di ALXN1830 rispetto al placebo
    7. Valutare l'immunogenicità di ALXN1830 rispetto al placebo
    8. Valutare la farmacocinetica (PK) di ALXN1830
    9. Valutare l'effetto di ALXN1830 sui livelli sierici di IgG totali rispetto al placebo
    10. Valutare l'effetto di ALXN1830 su altri biomarcatori PD rispetto al 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. Il partecipante deve avere almeno 18 anni compresi, al momento della firma del consenso informato
    2. Diagnosticato con WAIHA primario o secondario almeno 6 settimane prima dello screening
    3. Ha fallito o non ha tollerato almeno un precedente regime di trattamento WAIHA, ad esempio corticosteroidi, rituximab, azatioprina, ciclofosfamide, ciclosporina, micofenolato mofetile, danazolo o vincristina
    4. Emoglobina < 10 g/dL e = 6 g/dL allo Screening
    5. Positivo al test dell'antiglobulina diretto (Coombs) (IgG positivi che sono positivi o negativi per la presenza del complemento C3 [C3]) allo Screening
    6. Evidenza di emolisi attiva
    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. Trasfusioni di globuli rossi o sangue intero nelle 2 settimane precedenti lo screening
    2. Storia di anemia emolitica autoimmune da anticorpi freddi (AIHA), sindrome da agglutinine fredde, AIHA di tipo misto o emoglobinuria parossistica fredda
    3. Storia di anemia emolitica immunitaria indotta da farmaci o correlata all'infezione
    4. Carenza di ferro, acido folico o vitamina B12
    5. Partecipanti con la sindrome di Evan
    6. Storia di leucemia, linfoma di Hodgkin o non Hodgkin
    7. Storia di tumore solido diverso da carcinoma basocellulare o a cellule squamose della pelle, carcinoma in situ della cervice o altri tumori per i quali il partecipante non era libero da malattia da almeno 5 anni
    8. Infezione attiva o storia di infezioni sistemiche ricorrenti nei 2 anni precedenti lo Screening
    10. Lupus eritematoso sistemico (LES) o altra malattia autoimmune che non è stabile o non ben controllata con la terapia attuale
    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
    Percentuale di partecipanti che hanno ottenuto un aumento di = 2 g/dL di Hgb dal basale (giorno 1) alla fine del trattamento primario, senza richiedere alcun aumento della dose di un farmaco WAIHA esistente dopo il giorno 1 e senza trasfusioni di globuli rossi dopo il giorno 14
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of Primary Treatment (Day 57 or Day 85)
    Fine del trattamento primario (giorno 57 o giorno 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. Numero totale di unità di PRBC trasfuse:
    2. Durabilità: numero di misurazioni di Hgb = 2 g/dL
    4. Percentuale di partecipanti che richiedono nuovi farmaci di salvataggio WAIHA o qualsiasi aumento della dose di un farmaco WAIHA esistente o pRBC
    trasfusioni per il trattamento dell'anemia
    5. Percentuale di partecipanti che ottengono un aumento di Hgb = 2 g/dL, senza richiedere alcun aumento della dose di un farmaco WAIHA esistente
    6. Marcatori di emolisi: variazione assoluta e percentuale dei livelli sierici di lattato deidrogenasi (LDH), conta assoluta dei reticolociti, siero
    bilirubina indiretta e aptoglobina sierica
    7. Utilizzo totale di corticosteroidi dal basale (giorno 1) alla fine del trattamento primario
    13. Incidenza di AE, SAE e AESI nel tempo
    15. Concentrazioni sieriche di ALXN1830 nel tempo
    16. Livelli sierici totali di IgG
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study
    Durante lo studio
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Korea, Republic of
    United States
    France
    Germany
    Italy
    Spain
    United Kingdom
    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)
    Data in cui l'ultimo partecipante completa l'ultima visita (incluso il follow-up)
    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 state3
    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
    Al termine dell'ultima visita di studio, i partecipanti torneranno alle cure del proprio medico curante
    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 Decision2021-08-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-20
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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