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    Summary
    EudraCT Number:2010-019375-30
    Sponsor's Protocol Code Number:ALX-0681-2.1/10
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-07-27
    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 ConcernedAustria - BASG
    A.2EudraCT number2010-019375-30
    A.3Full title of the trial
    A Phase II single blind, randomized, placebo controlled trial to study the efficacy and safety of anti-von Willebrand factor Nanobody administered as adjunctive treatment to patients with acquired thrombotic thrombocytopenic purpura.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase II single blind, randomized, placebo controlled trial to study the efficacy and safety of anti-von Willebrand factor Nanobody administered as adjunctive treatment to patients with acquired thrombotic thrombocytopenic purpura.
    A.4.1Sponsor's protocol code numberALX-0681-2.1/10
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01151423
    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
    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 organisationAblynx
    B.5.2Functional name of contact pointJosefin-Beate Holz
    B.5.3 Address:
    B.5.3.1Street AddressTechnologiepark 21
    B.5.3.2Town/ cityZwijnaarde
    B.5.3.3Post code9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number329262 0000
    B.5.5Fax number329262 0002
    B.5.6E-mailjosi.holz@ablynx.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 nameAnti-von Willebrand Factor Nanobody, Caplacizumab
    D.3.2Product code ALX-0081
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 915810- 67-2
    D.3.9.2Current sponsor codeALX-0081 Nanobody
    D.3.9.3Other descriptive nameAnti-von Willebrand Factor Nanobody
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5.0
    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 injection
    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
    Acquired thrombotic thrombocytopenic purpura (TTP)
    E.1.1.1Medical condition in easily understood language
    Blood disease where one of the factors involved in blood clotting does not work properly, resulting in inappropriate clumping of platelets, leading to organ damage, platelet depletion, and bleeding.
    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 14.1
    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
    Reduction of time-to-response, defined by the achievement of laboratory blood marker response, confirmed at 48 hours after the initial reporting of this response
    E.2.2Secondary objectives of the trial
    •Improvement in number of subjects responding to therapy
    •Reduction PE procedure-related items
    •Reduction time to resolution or improvement of signs and symptoms
    typical of TTP, incl. blood markers
    •Reduction of number of exacerbations (defined as recurrent
    thrombocytopenia following a response and requiring a re-initiation of
    daily PE treatment after ≥ 1 day but ≤ 30 days after last daily PE) and relapses (defined as de novo event of TTP occurring later than 30 days after last daily PE)
    •Improvement of cognitive level at steady state post acute phase (adults only)
    •Improvement of clinical symptoms and organ function
    •Reduction in mortality within PE treatment period and within subsequent study drug treatment period (incl. tapering)
    •Reduction of concomitant treatment-related complications
    •Safety and immunogenicity evaluation of adjunctive treatment with ALX-0081
    •Determination of pharmacokinetic and pharmacodynamic characteristics of ALX 0081 in patients with acquired TTP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. 18 years of age or older (adults) or aged 12 to < 18 years (adolescents)
    2. Male or female willing to accept an acceptable contraceptive regimen
    3. Patients with clinical diagnosis of TTP
    4. Necessitating PE
    5. Subject accessible to follow-up
    6. Obtained, signed and dated informed consent and assent (if applicable, for adolescents)
    E.4Principal exclusion criteria
    1. Platelet count greater or equal to 100,000/µL
    2. Severe active infection indicated by sepsis (requirement for pressors with or without positive blood cultures)
    3. Clinical evidence of enteric infection with E. coli 0157 or related organism
    4. Anti-phospholipid syndrome
    5. Diagnosis of disseminated intravascular coagulation (DIC)
    6. Pregnancy or breast-feeding
    7. Haematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy
    8. Known congenital TTP
    9. Active bleeding or high risk of bleeding
    10. Uncontrolled arterial hypertension
    11. Known chronic treatment with anticoagulant treatment that can not be stopped safely, including but not limited to:
    •vitamin K antagonists
    •heparin or low molecular weight heparin (LMWH)
    •non-acetyl salicylic acid non-steroidal anti-inflammatory molecules
    12. Severe or life threatening clinical condition other than TTP that would impair participation in the trial
    13. Subjects with malignancies resulting in a life expectation of less than 3 months
    14. Subjects with known or suspected bone marrow carcinosis
    15. Subjects who cannot comply with study protocol requirements and procedures.
    16. Known hypersensitivity to the active substance or to excipients of the study drug
    17. Severe liver impairment, corresponding to grade 3 toxicity defined by the CTCAE scale. For the key liver parameters, this is defined as follows:
    •bilirubin > 3 x ULN (need to differentiate isolated increase in indirect bilirubin due to haemolysis, this is not an exclusion parameter but disease related)
    •alanine aminotransferase/aspartate aminotransferase (ALT/AST) > 5 x ULN
    •alkaline phosphatase (AP) > 5 x ULN
    •gamma glutamyl transpeptidase (GGT) > 5 x ULN
    18. Severe chronic renal impairment, as defined by GFR < 30 mL/min
    E.5 End points
    E.5.1Primary end point(s)
    Time-to-response, based on the following criteria:
    - Recovery of platelets ≥ 150,000/µL
    - This response must be confirmed at 48 hours after the initial reporting of platelet recovery equal to or above 150,000/µL by a de novo measure of platelets ≥ 150,000/µL and lactate dehydrogenase (LDH) ≤ 2 X upper limit of normal (ULN)
    E.5.1.1Timepoint(s) of evaluation of this end point
    - initial timepoint: time when platelets have recovered to ≥ 150,000/µL
    - this is to be confirmed at 48 hrs after initial timepoint
    E.5.2Secondary end point(s)
    • Number of subjects with complete remission (defined as confirmed platelet response and absence of exacerbation)
    • Number of (subjects with) exacerbations of TTP (defined as recurrent thrombocytopenia following a confirmed platelet response and requiring a re-initiation of daily PE treatment after ≥ 1 day but ≤ 30 days after the last daily PE) and time to first exacerbation of TTP
    • Number of subjects relapsing of TTP (defined as de novo event of TTP
    that occurs later than 30 days after the last daily PE)
    • Number of daily PE sessions, number of plasma units administered and
    number of days of daily PE
    • Resolution of non-focal neurological symptoms as defined by neurocognitive function at complete remission, measured by a neurocognitive test battery (adults only)
    • Resolution or improvement (improvement of ≥ 1 grade in the Common Terminology Criteria for Adverse Events (CTCAE) v4.0 scale) of TTPrelated signs and symptoms as captured on physical examination and as adverse events, at complete remission and at end of the study drug treatment period (including tapering)(by number of unique subjects and by total number of adverse events (AEs)) Total mortality within the PE treatment period and within the subsequent study drug treatment period (including tapering)
    • Incidence of PE treatment-related AEs, such as, but not restricted to: haemorrhage from catheter insertion, sepsis, catheter thrombosis, pneumothorax, fluid overload, hypoxia, hypotension, anaphylactoid reactions and transfusion related acute lung injury (TRALI)
    • Incidence and severity of ALX-0081 treatment-emergent AEs and relationship to study drug
    • Development of anti-drug antibodies (ADA) ≤ 30 days post-last study drug treatment
    • PK and PD profile
    E.5.2.1Timepoint(s) of evaluation of this end point
    All secondary endpoints achieved within 30 day period after end of study
    drug treatment
    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 Yes
    E.8.1.4Double blind No
    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 EEA30
    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
    Canada
    Israel
    Russian Federation
    Switzerland
    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
    Study period ends when the last patient completes the 12-month follow-up visit (12 months after last administration of study drug).
    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 years3
    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 Yes
    F.1.1Number of subjects for this age range: 5
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 105
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    See Protocol Section 11.3
    In the clinical setting of this trial investigating the acute phase of TTP, a more severe intensity of physical and/or mental incapacity is expected as these are part of the symptoms of acute TTP.
    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 70
    F.4.2.2In the whole clinical trial 115
    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)
    Treatment after the end of the trial will be the normal treatment of the condition.
    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 Decision2010-08-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-12-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-03-14
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