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    Summary
    EudraCT Number:2016-001503-23
    Sponsor's Protocol Code Number:ALX0681-C302
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-01-18
    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 number2016-001503-23
    A.3Full title of the trial
    Prospective Follow-up Study for Patients who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)
    Studio di Follow-up Prospettico per i Pazienti che hanno Completato lo Studio ALX0681-C301 (HERCULES), per Valutare la Sicurezza e l'Efficacia di Caplacizumab a Lungo Termine (Post-HERCULES)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Follow-up Study for Patients who Completed Study ALX0681-C301 (Post-HERCULES)
    Studio di Follow-up per i Pazienti che hanno completato lo studio ALX0681-C301 (Post-HERCULES)
    A.3.2Name or abbreviated title of the trial where available
    Follow-up Study for Patients who Completed Study ALX0681-C301 (Post-HERCULES)
    Studio di Follow-up per i Pazienti che hanno completato lo studio ALX0681-C301 (Post-HERCULES)
    A.4.1Sponsor's protocol code numberALX0681-C302
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/060/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorABLYNX NV
    B.1.3.4CountryBelgium
    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 supportAblynx NV
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharm-Olam International (Spain) S.L.U.
    B.5.2Functional name of contact pointRegulatory Department
    B.5.3 Address:
    B.5.3.1Street AddressC/Antracita, 7 Planta 1-Nave 6
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28045
    B.5.3.4CountrySpain
    B.5.4Telephone number0034914342777
    B.5.5Fax number0034914342773
    B.5.6E-mailregulatory.spain@pharm-olam.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/09/629
    D.3 Description of the IMP
    D.3.1Product nameCaplacizumab (an anti-von Willebrand Factor Nanobody ALX-0081)
    D.3.2Product code ALX-0081
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous bolus use (Noncurrent)
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCAPLACIZUMAB
    D.3.9.1CAS number 915810-67-2
    D.3.9.2Current sponsor codeALX-0081
    D.3.9.4EV Substance CodeSUB91244
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number11
    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 Yes
    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
    Acquired thrombotic thrombocytopenic purpura (TTP)
    Porpora Trombotica Trombocitopenica acquisita (PTT)
    E.1.1.1Medical condition in easily understood language
    TPP is a rare condition in which the blood becoms "sticky" and forms clots within the blood vessels in different parts of the body.
    la PTT ¿ una condizione rara in cui il sangue diviene 'appiccicoso' e forma coaguli all'interno dei vasi sanguigni in vari distretti del corpo
    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 10043648
    E.1.2Term Thrombotic thrombocytopenic purpura
    E.1.2System Organ Class 10005329 - Blood and lymphatic system 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 evaluate long-term safety and efficacy of caplacizumab
    ¿To evaluate safety and efficacy of repeated use of caplacizumab
    ¿To characterize long term impact of TTP
    ¿ Valutare la sicurezza e l¿efficacia a lungo termine di caplacizumab
    ¿ Valutare la sicurezza e l¿efficacia dell¿uso ripetuto di caplacizumab
    ¿ Caratterizzare l¿impatto a lungo termine della TTP
    E.2.2Secondary objectives of the trial
    Not applicable
    non applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each potential subject must satisfy all of the following criteria to be enrolled in the study:
    1.Completed the Final (28 day) FU visit in Study ALX0681-C301.
    2.Is =18 years of age at the time of signing the informed consent form (ICF).
    3.Provided informed consent prior to initiation of any study specific activity/procedure.
    Per essere arruolato nello studio, ogni potenziale soggetto deve soddisfare tutti i seguenti criteri:
    1. Aver completato la Visita finale di FU (28 giorni) nello Studio ALX0681-C301.
    2. Avere = 18 anni al momento della sottoscrizione del modulo di consenso informato (ICF).
    3. Aver fornito il consenso informato prima di iniziare qualunque attività/procedura specifica dello studio.
    E.4Principal exclusion criteria
    The criteria for exclusion are the following:
    1.Not being able/willing to comply with the study protocol procedures.
    2.Currently enrolled in a clinical study with another investigational drug or device.
    I criteri di esclusione sono i seguenti:
    1. Incapacità/mancata disponibilità ad aderire al protocollo dello studio.
    2. Arruolamento in corso in uno studio clinico con un altro farmaco o dispositivo sperimentale.
    E.5 End points
    E.5.1Primary end point(s)

    • Proporzione di soggetti con gli eventi correlati a PTT , numero di eventi correlati con la TTP e tempo all'evento
    • Tasso di mortalità durante lo studio
    • Proporzione dei soggetti con recidiva della patologia, numero di recidive e tempo alla recidiva
    • Proporzione di soggetti che hanno riportato eventi tromboembolici gravi , numero di eventi e tempo all'evento
    • Funzioni cognitive
    • Qualità della vita
    • Immunogenicità del trattamento (ripetuto) con caplacizumab
    • Proportion of subjects with TTP-related events , number of TTP-related events and time to event
    • Mortality rate during the study
    • Proportion of subjects with recurrence of disease, number of recurrences, and time to recurrence
    • Proportion of subjects with reported major thromboembolic events ,number of events, and time to event
    • Cognitive function
    • Quality of life
    • Immunogenicity of (repeated) treatment with caplacizumab
    E.5.1.1Timepoint(s) of evaluation of this end point
    from screening until last follow-up visit
    dallo screening fino all'ultima visita di follow-up
    E.5.2Secondary end point(s)
    Not applicable
    Non applicabile
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    Non applicabile
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 prospettico di follow-up per i pazienti che hanno completato lo studio ALX0681-C301
    Prospective follow-up for subject who completed study ALX0681-C301
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Israel
    Turkey
    United States
    Austria
    Belgium
    Czechia
    France
    Hungary
    Italy
    Spain
    Switzerland
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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: 95
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 9
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 92
    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)
    Standard of care
    standard of care
    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 Decision2016-09-06
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
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