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    Summary
    EudraCT Number:2020-003675-18
    Sponsor's Protocol Code Number:ANX005-wAIHA-02
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-08-02
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-003675-18
    A.3Full title of the trial
    A Phase 2, Open-Label, Repeat Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Proof-of-Concept of Intravenous ANX005 in Subjects with Warm Autoimmune Hemolytic Anemia
    Studio in aperto, di fase 2, a dosi ripetute per valutare la sicurezza, la tollerabilità, la farmacocinetica, la farmacodinamica e la prova di fattibilità di ANX005 mediante iniezione endovenosa in soggetti con anemia emolitica autoimmune calda
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Assess the Safety, Tolerability, Efficacy, PK, PD and POC of Intravenous ANX005 in Patients With wAIHA
    Studio per valutare la sicurezza, la tollerabilità, l'efficacia, PK, PD e POC di ANX005 per via endovenosa in pazienti con wAIHA
    A.3.2Name or abbreviated title of the trial where available
    Study with intravenous ANX005 in patients with wAIHA
    Studio con ANX005 per via endovenosa in pazienti con wAIHA
    A.4.1Sponsor's protocol code numberANX005-wAIHA-02
    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 SponsorAnnexon, 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 supportAnnexon, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAnnexon, Inc.
    B.5.2Functional name of contact pointClinical Trials Informations
    B.5.3 Address:
    B.5.3.1Street Address180 Kimbali Way
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16508225500
    B.5.5Fax number+16506369773
    B.5.6E-mailinfo@annexonbio.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 nameANX005
    D.3.2Product code [ANX005]
    D.3.4Pharmaceutical form Powder for solution for injection/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.3.9.2Current sponsor codeANX005
    D.3.9.3Other descriptive nameRecombinant humanized monoclonal antibody: IgG4 (heavy chain); kappa (light chain)
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    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
    anemia emolitica autoimmune calda
    E.1.1.1Medical condition in easily understood language
    warm autoimmune hemolytic anemia
    anemia emolitica autoimmune calda
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    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 No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this repeat dose Phase 2 study is as follows:
    • To evaluate the safety, tolerability, and proof of concept of two once-weekly intravenous infusions of 100 mg/kg ANX005 in subjects with wAIHA.
    L'obiettivo principale di questo studio di Fase 2 è di valutare la sicurezza, tollerabilità e prova di fattibilità di due infusioni endovenose una volta alla settimana di 100 mg/kg di ANX005 in soggetti con anemia emolitica autoimmune calda (wAIHA)
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are as follows:
    • To evaluate the pharmacokinetic (PK) profile of ANX005 in subjects with wAIHA
    • To evaluate the effect of ANX005 on classical complement pathway inhibition as measured by complement system related biomarkers in subjects with wAIHA
    • To assess the effect of 2 once-weekly doses of ANX005 on the following disease activity markers in subjects with wAIHA:
    Hemoglobin,
    Lactate dehydrogenase (LDH),
    Total and Indirect bilirubin,
    Haptoglobin,
    Reticulocyte count, Platelet count,
    CRP, Direct Antibody Test (DAT).
    • Valutare il profilo farmacocinetico (PK) di ANX005 in soggetti con wAIHA;
    • Valutare l'effetto di ANX005 sull'inibizione della via classica del complemento come misurato dai biomarker relativi al sistema del complemento in soggetti con wAIHA
    • Valutare l'effetto di 2 dosi una volta a settimana di ANX005 sui seguenti marker di attività della malattia in soggetti con wAIHA:
    o Emoglobina
    o Lattato deidrogenasi (LDH)
    o Bilirubina indiretta e totale
    o Aptoglobina
    o Conta dei reticolociti
    o Conta delle piastrine
    o CRP
    o Test anticorpi diretti (DAT)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or non-pregnant, non-lactating female = 18 years of age on the day of signing informed consent
    2. Diagnosis of wAIHA at least 3 months prior to screening
    3. Hgb level > 10.0 g/dL (pre-transfusion) with no alternative explanation for anemia apart from wAIHA at screening
    4. Positive direct antiglobulin test (DAT) = 1 + for C3d and IgG
    5. Evidence of classical complement pathway activation by one of the
    below:
    a. Serum complement component 4 (C4) below the lower limit of normal
    b. CH50 below the lower limit of normal
    6. Evidence of active hemolysis based on at least one of the following:
    a. LDH above the ULN
    b. Indirect bilirubin above the ULN
    c. Haptoglobin below the LLN
    7. Vaccinations against encapsulated bacterial organisms (N. meningitidis, H. influenzae and S. pneumoniae) within 5 years prior to screening or subject must be willing
    to complete vaccinations at least 2 weeks prior to dosing with ANX005.
    Documentation of vaccinations must be received at the study site in advance of the scheduled screening visit
    1. Maschio o femmina non gravida, non in allattamento di età = 18 anni il giorno della firma del consenso informato
    2. Diagnosi di wAIHA almeno 3 mesi prima dello screening
    3. Livello di Hgb> 10,0 g / dL (pre-trasfusione) senza alternative spiegazioni per l'anemia a parte wAIHA allo screening
    4. Test dell'antiglobulina diretta positivo (DAT) = 1 + per C3d e IgG
    5. Prova dell'attivazione della via classica del complemento da parte di uno dei sotto:
    a. Componente del complemento sierico 4 (C4) al di sotto del limite inferiore della norma
    b. CH50 al di sotto del limite inferiore del normale
    6. Evidenza di emolisi attiva basata su almeno uno dei seguenti:
    a. LDH sopra l'ULN
    b. Bilirubina indiretta al di sopra dell'ULN
    c. Aptoglobina al di sotto del LLN
    7. Vaccinazioni contro organismi batterici incapsulati (N. meningitidis, H. influenzae e S.pneumoniae) entro 5 anni prima dello screening o il soggetto deve essere disponibile
    completare le vaccinazioni almeno 2 settimane prima della somministrazione di ANX005.
    La documentazione delle vaccinazioni deve essere ricevuta presso il sito dello studio in anticipo dalla visita di screening programmata
    E.4Principal exclusion criteria
    1. History of or current lymphoma or lymphoproliferative disorder requiring therapy
    2. Elevated aspartate aminotransferase or alanine aminotransferase levels > 2.5 times the upper limit of normal at screening
    3. Platelet count < 30 x 109/L
    4. History of cold agglutinin disease or IgM cold agglutinin titer > 1:64
    5. History of solid organ, bone marrow, or stem cell transplantation
    6. History of splenectomy within the 3 months prior to screening
    7. Other wAIHA treatments:
    - Received rituximab or other anti-CD20 monoclonal antibody < 3 months prior to screening
    - Receiving steroids > 1 mg/kg of prednisone or equivalent daily at screening
    8. Signs and symptoms of, or a diagnosis consistent with, a chronic autoimmune disorder not resulting from primary wAIHA and/or an ANA titer of = 1:160
    9. Known genetic deficiencies of the complement cascade system
    1. Anamnesi o attuale linfoma o disturbo linfoproliferativo che richiede terapia
    2. Aspartato aminotransferasi o alanina aminotransferasi elevati livelli> 2,5 volte superiore il limite di normalità allo screening
    3. Conta piastrinica <30 x 109 / L
    4. Anamnesi di malattia da agglutinine fredde o titolo di agglutinine fredde IgM> 1:64
    5. Storia di trapianto di organi solidi, midollo osseo o cellule staminali
    6. Storia di splenectomia nei 3 mesi precedenti lo screening
    7. Altri trattamenti wAIHA:
    - Ricevuto rituximab o altro anticorpo monoclonale anti-CD20 <3 mesi prima dello screening
    - Ricezione dose giornaliera di steroidi> 1 mg / kg di prednisone o equivalente allo screening
    8. Segni e sintomi di, o una diagnosi coerente con, un cronico disturbo autoimmune non derivante da wAIHA primario e / o un titolo ANA di = 1: 160
    9. Carenze genetiche note del sistema a cascata del complemento
    E.5 End points
    E.5.1Primary end point(s)
    Adverse events, infusion related reactions, safety laboratory assessments (serum chemistry, hematology, urine analysis), and vital signs.
    Eventi avversi, reazioni correlate all'infusione, laboratorio di sicurezza valutazioni (chimica del siero, ematologia, analisi delle urine) e segni vitali.
    E.5.1.1Timepoint(s) of evaluation of this end point
    10 weeks
    10 settimane
    E.5.2Secondary end point(s)
    Pharmacokinetic (PK) endpoints are:
    -Maximum observed serum concentration (Cmax)
    -Observed time to Cmax (Tmax)
    -Area under the ANX005 serum concentration-time curve to the last
    sample (AUC0-t) and
    extrapolated through infinity
    -Accumulation ratio
    -Terminal half-life (t1/2)
    -Terminal elimination rate constant
    -Clearance (CL)
    -Volume of distribution (Vss)
    Pharmacodynamics (PD) Endpoints:
    -Hemoglobin
    -Lactate dehydrogenase (LDH)
    -Total and Indirect bilirubin
    -Haptoglobin
    -Reticulocyte count
    -Platelet count
    -Direct Antibody Test (DAT)
    Gli endpoint farmacocinetici (PK) sono:
    -Massima concentrazione sierica osservata (Cmax)
    -Tempo osservato a Cmax (Tmax)
    -Area sotto la curva concentrazione sierica-tempo fino all'ultimo ANX005
    campione (AUC0-t) e
    estrapolato all'infinito
    -Rapporto di accumulo
    -Emivita terminale (t1 / 2)
    -Velocità di eliminazione del terminale costante
    -Cancellazione (CL)
    -Volume di distribuzione (Vss)
    Endpoint farmacodinamici (PD):
    -Emoglobina
    -Lattato deidrogenasi (LDH)
    -Bilirubina totale e indiretta
    -Haptoglobin
    -Conteggio dei reticolociti
    -Conteggio piastrine
    -Test diretto degli anticorpi (DAT)
    E.5.2.1Timepoint(s) of evaluation of this end point
    10 weeks
    10 settimane
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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, Proof-of-Concept of Intravenous ANX005 in Subjects with Warm Autoimmune Hemolytic Anemia
    Tollerabilità, prova di fattibilità di ANX005 endovena in soggetti con anemia emolitica autoimmune calda
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Studio a dosi ripetute in aperto
    Open repeat dose study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Austria
    France
    Germany
    Hungary
    Italy
    Netherlands
    Norway
    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
    LVLS
    LVLS
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 9
    F.4.2.2In the whole clinical trial 12
    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)
    Once study participation is completed, the subject can discuss with their personal physician about treatment options.
    Una volta completata la partecipazione allo studio, il soggetto potrà discutere con il proprio medico personale sulle opzioni di trattamento.
    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 Decision2020-12-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-06
    P. End of Trial
    P.End of Trial StatusOngoing
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