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    Summary
    EudraCT Number:2020-005328-13
    Sponsor's Protocol Code Number:ALXN1210-TMA-315
    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:2021-06-08
    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 number2020-005328-13
    A.3Full title of the trial
    A Phase 3, Randomized, Double-blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants Who Have Thrombotic Microangiopathy Associated with a Trigger
    Studio multicentrico di fase 3, randomizzato, in doppio cieco, controllato con placebo, per valutare l’efficacia e la sicurezza di ravulizumab in partecipanti adulti affetti da microangiopatia trombotica associata a un fattore scatenante
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate efficacy and safety of Ravulizumab in adult patients with thrombotc microangiopathy
    Studio per la valutazione dell'efficacia e sicurezza di Ravulizumab in pazienti adulti affetti da microangiopatia trombotica
    A.3.2Name or abbreviated title of the trial where available
    Phase 3 Study of Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger
    Studio di fase 3 di Ravulizumab in microangiopatia trombotica associata a trigger
    A.4.1Sponsor's protocol code numberALXN1210-TMA-315
    A.5.4Other Identifiers
    Name:INDNumber:128367
    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 number0000000
    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 nameravulizumab
    D.3.2Product code [ALXN1210]
    D.3.4Pharmaceutical form Concentrate 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.3.8INN - Proposed INNRavulizumab
    D.3.9.1CAS number 1803171-55-2
    D.3.9.2Current sponsor codeALXN1210
    D.3.9.4EV Substance CodeSUB192773
    D.3.10 Strength
    D.3.10.1Concentration unit 1X 100 milligrams/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Trombotic Microangiopathy associated with a trigger
    Microangiopatia Trombotica associata ad un fattore scatenante
    E.1.1.1Medical condition in easily understood language
    TMA with a trigger
    TMA con fattore scatenante
    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 PT
    E.1.2Classification code 10043645
    E.1.2Term Thrombotic microangiopathy
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10062198
    E.1.2Term Microangiopathy
    E.1.2System Organ Class 10047065 - Vascular disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of ravulizumab versus placebo
    Valutare l’efficacia di ravulizumab versus placebo
    E.2.2Secondary objectives of the trial
    To assess Safety and tolerability of ravulizumab and additional efficacy measures
    To assess Safety and tolerability of ravulizumab and additional efficacy measures
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.18 years of age or older
    2.Body weight = 30 kilograms
    3.Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab
    4.Diagnosis of TMA (platelet count, LDH, and acute kidney injury) associated with a trigger such as autoimmune, solid organ transplant or drug induced
    5.Vaccinated against meningococcal infection (N meningitidis), within 3 years prior to, or at the time of, randomization. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics for at least 2 weeks after the vaccination. If participant cannot receive the meningococcal vaccine, then participant must be willing to receive antibiotic prophylaxis coverage against N meningitidis during the entire Treatment Period and for 8 months following the final dose of study drug. Additional vaccination (Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae) may be considered based on individual patient condition
    1. Il soggetto deve avere un’età =18 anni al momento della firma del consenso informato.
    2. Peso corporeo =30 kg allo screening.
    3. I partecipanti di sesso femminile in età fertile e i partecipanti di sesso maschile devono seguire le linee guida sulla contraccezione specificate nel protocollo, iniziando dallo screening e continuando fino ad almento 8 mesi dopo l'ultima dose di ravulizumab
    4. La diagnosi di MAT entro =14 giorni prima dello screening è associata ad almeno 1 dei seguenti fattori scatenanti:
    a. malattia autoimmune (nefrite lupica, MAT associata a sclerosi sistemica [SSc-MAT]);
    b. infezione;
    c. trapianto di organo solido (rene, pancreas, fegato, cuore, intestino tenue);
    d. farmaci;
    e. ipertensione maligna.
    6. Vaccinazione contro l’infezione da meningococco (N. meningitidis) nei 3 anni precedenti o al momento della randomizzazione. I partecipanti che iniziano il trattamento con il farmaco dello studio meno di 2 settimane dopo aver ricevuto un vaccino anti-meningococco devono ricevere un’appropriata profilassi antibiotica per almeno 2 settimane dopo la vaccinazione. Se il partecipante non può ricevere il vaccino anti-meningococco, deve essere disposto a ricevere una copertura profilattica antibiotica contro N. meningitidis durante l’intero periodo di trattamento e per 8 mesi dopo la dose finale del farmaco in studio.
    7. I partecipanti con MAT associata a nefrite lupica, sclerosi sistemica o trapianto di organo solido devono essere vaccinati contro Haemophilus influenzae di tipo b (Hib) e Streptococcus pneumoniae prima della randomizzazione, secondo le attuali linee guida nazionali/locali sulla vaccinazione.
    E.4Principal exclusion criteria
    1.Any known gene mutation that causes aHUS
    2.Postpartum aHUS
    3.Known CKD with eGFR = 45 mL/min/1.73 m2 by CKD-EPI equation (Levey, 2009) due to any cause
    4.TMA due to hematopoietic stem cell transplantation = 12 months of Screening
    5.Primary and secondary glomerular diseases other than lupus
    6.Diagnosis of primary antiphospholipid antibody syndrome
    7.Shiga toxin-producing Escherichia coli infections including but not limited to Shiga toxin-related hemolytic uremic syndrome
    8.Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13' (ADAMTS13) deficiency (activity < 5%)
    9.Positive direct Coombs test
    10.Diagnosis of disseminated intravascular coagulation (DIC)
    11.Presence of sepsis according within 7 days prior to or during Screening
    12.Presence of monoclonal gammopathy including but not limited to multiple myeloma
    13.Known bone marrow insufficiency or failure evidenced by cytopenias
    14.Unresolved N meningitidis infection
    15.History of malignancy within 5 years of Screening with the exception of nonmelanoma skin cancer or carcinoma in situ of the cervix that has been treated with no evidence of recurrence
    16.Use of any complement inhibitors within the past 3 years
    1. Qualsiasi mutazione genica nota che causi SEUa (Sezione 10.10)
    2. SEUa post-parto
    3. Malattia renale cronica (MRC) nota, con eGFR =45 ml/min/1,73 m2 in base all’equazione CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) (Levey, 2009) dovuta a qualsiasi causa
    4. MAT dovuta a trapianto di cellule staminali ematopoietiche =12 mesi dallo screening
    5. Malattie glomerulari primarie e secondarie diverse dal lupus
    6. Diagnosi di sindrome da anticorpi antifosfolipidi primaria
    7. Infezioni da ceppi di Escherichia coli produttori della tossina di Shiga, tra cui, a titolo esemplificativo ma non esaustivo, sindrome emolitico-uremica correlata alla tossina di Shiga
    8. Deficit noto, familiare o acquisito, di “una disintegrina e metalloproteinasi con motivo trombospondina di tipo 1, membro 13” (ADAMTS13) (attività <5%)
    9. Test di Coombs diretto positivo
    10. Diagnosi di coagulazione intravascolare disseminata (CID) secondo i criteri di punteggio della Società internazionale sulla trombosi e l’emostasi (International Society on Thrombosis and Haemostasis, [ISTH]) (Sezione 10.10)
    11. Presenza di sepsi secondo la definizione del Terzo Consenso Internazionale (Singer, 2016) nei 7 giorni precedenti o durante lo screening
    12. Presenza di gammopatia monoclonale, compreso, senza limitazione, il mieloma multiplo
    13. Insufficienza o compromissione nota del midollo osseo evidenziata da citopenie
    14. Infezione da N. meningitidis non risolta
    15. Anamnesi di tumore maligno nei 5 anni precedenti lo screening, ad eccezione del carcinoma cutaneo non-melanoma o del carcinoma in situ del collo dell’utero che è stato trattato senza evidenza di recidiva
    16. Uso di qualsiasi inibitore del complemento negli ultimi 3 anni
    E.5 End points
    E.5.1Primary end point(s)
    Complete TMA response
    Complete TMA response
    E.5.1.1Timepoint(s) of evaluation of this end point
    throughout week 26
    throughout week 26
    E.5.2Secondary end point(s)
    1. Time to complete TMA repsonse
    2. Hematologic response
    3. Renal response
    4. TMA response duration and TMA relapse
    5. Change in kidney function
    1. Time to complete TMA repsonse
    2. Hematologic response
    3. Renal response
    4. TMA response duration and TMA relapse
    5. Change in kidney function
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 26 and Week 52
    Settimana 26 e 52
    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 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
    Immunogenicity
    Immunogenicity
    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 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 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 concerned2
    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 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
    Canada
    Japan
    Korea, Republic of
    Taiwan
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    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
    Last Visit Last Subject
    Ultima visita ultimo paziente
    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 days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 79
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 21
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 100
    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)
    Ravulizumab will not be administered to participants after completion of 26 week treatment period. Upon completion of the last study visit, participants will return to the care of their treating physician.
    Ravulizumab will not be administered to participants after completion of 26 week treatment period. Upon completion of the last study visit, participants will return to the care of their treating physician.
    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 Decision2021-04-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-16
    P. End of Trial
    P.End of Trial StatusOngoing
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