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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43800   clinical trials with a EudraCT protocol, of which   7272   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2015-001098-42
    Sponsor's Protocol Code Number:ALX0681-C301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-07-31
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2015-001098-42
    A.3Full title of the trial
    A Phase III double-blind, randomized, parallel group, multicenter placebo-controlled trial to study the efficacy and safety of caplacizumab in patients with acquired thrombotic thrombocytopenic purpura.
    Ensayo clínico de fase III, doble ciego, aleatorizado de grupos paralelos, multicéntrico, controlado con placebo, para estudiar la eficacia y la seguridad de caplacizumab en pacientes con púrpura trombocitopénica trombótica adquirida
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase III trial with caplacizumab in patients with acquired thrombotic thrombocytopenic purpura.
    Ensayo Clínico de fase III con caplacizumab en pacientes con púrpura trombocitopénica trombótica adquirida
    A.4.1Sponsor's protocol code numberALX0681-C301
    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 SponsorAblynx NV
    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.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
    B.5.3 Address:
    B.5.3.1Street AddressC/Antracita, 7 Planta 1 A Nave 6
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28045
    B.5.4Telephone number34914342777
    B.5.5Fax number34914342773
    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)
    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.3.9.3Other descriptive nameALX-0081
    D.3.9.4EV Substance CodeSUB91244
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number12.5
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder and solvent for solution for injection
    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
    Acquired Thrombotic thrombocytopenic purpura
    Púrpura trombocitopénica trombótica adquirida
    E.1.1.1Medical condition in easily understood language
    TTP is a rare condition in which the blood becomes ?sticky? and forms clots within the blood vessels in different parts of the body.
    La PTT es una enfermedad rara que se caracteriza por la viscosidad excesiva de la sangre y por la formación de coágulos en los vasos sanguíneos de distintas partes del cuerpo.
    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 18.0
    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 efficacy of caplacizumab in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis.
    Evaluar la eficacia de caplacizumab en la restauración más rápida del número normal de plaquetas como medida de la prevención de trombosis microvascular adicional
    E.2.2Secondary objectives of the trial
    -To evaluate prevention of recurrence of presenting TTP episode after cessation of daily PE, including prolongation of caplacizumab treatment beyond 30 days post-daily PE treatment based on clinical assessment of underlying disease and ADAMTS13 activity
    -To evaluate mortality rate
    -To evaluate the effect of caplacizumab on biomarkers for organ damage: LDH, cTnI, and serum creatinine.
    -Evaluar la prevención de recidiva del episodio de PTT de presentación tras el cese del intercambio de plasma (IP) diario, incluyendo la prolongación del tratamiento con caplacizumab después del periodo de 30 días posterior al tratamiento de IP diario, basándose en la evaluación clínica de la enfermedad subyacente y en la actividad de ADAMTS13
    -evaluar la tasa de mortalidad
    -evaluar el efecto de caplacizumab sobre los biomarcadores de daño orgánico: lactato deshidrogenasa (LDH), troponina I cardiaca (TnIc) y creatinina sérica.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Adult male or female ? 18 years of age at the time of signing the informed consent form (ICF)
    2.Clinical diagnosis of acquired TTP (initial or recurrent), which includes thrombocytopenia and microscopic evidence of red blood cell fragmentation (e.g. schistocytes)
    3.Requires initiation of daily PE treatment and has received PE treatment prior to randomization .
    1-Adulto varón o mujer ? 18 años de edad en el momento de la firma del formulario de consentimiento informado (FCI)
    2-Diagnóstico clínico de PTT adquirida (inicial o recurrente), que incluye trombocitopenia y evidencia microscópica de fragmentación eritrocitaria (p. ej., esquistocitos)
    3-Requiere instauración del tratamiento de IP diario y ha recibido tratamiento de IP antes de la aleatorización.
    E.4Principal exclusion criteria
    1.Platelet count ?100×109/L
    2.Serum creatinine level >200 µmol/L in case platelet count is > 30×109/L
    3.Known other causes of thrombocytopenia
    4.Congenital TTP (known at the time of study entry)
    5.Pregnancy or breast-feeding.
    1.Número de plaquetas ?100×109/l
    2.Nivel de creatinina sérica >200 µmol/l en caso de que el número de plaquetas sea > 30×109/l
    3.Otras causas conocidas de trombocitopenia
    4.PTT congénita (conocida en el momento de la entrada en el estudio)
    5.Embarazo o lactancia.
    E.5 End points
    E.5.1Primary end point(s)
    Time to platelet count response defined as initial platelet count ? 150×109/L with subsequent stop of daily PE within 5 days.
    Tiempo hasta la respuesta en el número de plaquetas definida como un número inicial de plaquetas ? 150×109/l con la consiguiente interrupción del IP diario en los siguientes 5 días.
    E.5.1.1Timepoint(s) of evaluation of this end point
    daily PE period
    Periodo de IP diario
    E.5.2Secondary end point(s)
    1-Proportion of subjects with an exacerbation and/or relapse of TTP as well as the number of such events
    2-Proportion of subjects with treatment-emergent clinically significant TTP-related events as well as the number of such events.
    3-Area under the curve (AUC) of platelet count
    4-Time to lactate dehydrogenase (LDH) ? 2 x upper limit of normal (ULN)
    5-Time to cardiac Troponin I (cTnI) ? 1 x ULN
    6-Time to serum creatinine ? 1 x ULN
    7-Mortality Rate
    8-(Serious)adverse events
    1.Proporción de pacientes con exacerbación y/o recidiva de la PTT así como el número de dichos acontecimientos.
    2.Proporción de pacientes con acontecimientos relacionados con la PTT clínicamente significativos así como el número de dichos acontecimientos.
    3.Área bajo la curva (AUC) del número de plaquetas.
    4.Tiempo hasta una lactato deshidrogenasa (LDH) ? 2 x límite superior normal (LSN)
    5.Tiempo hasta una troponina I cardiaca (TnIc) ? 1 x ULN.
    6.Tiempo hasta una creatinina sérica ? 1 x ULN.
    7.Tasa de mortalidad
    8. Acontecimientos Adversos (Graves)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-During study drug treatment and during follow-up (FU)
    2, 4, 5 &6- During the overall study period.
    3- until Day 5
    7-Initial daily PE period, full study drug treatment period, FU period and overall study period
    8-During the overall study period
    1.Durante el tratamiento con el fármaco del estudio y durante el seguimiento.
    2., 4, 5 y 6. Durante el periodo global del ensayo.
    3. hasta el día 5
    7. Periodo de IP diario inicial, periodo completo de tratamiento con el fármaco del estudio, periodo de seguimiento y periodo global del estudio.
    8. Durante el periodo global del ensayo.
    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. 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA47
    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
    Czech Republic
    New Zealand
    United Kingdom
    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
    Última visita del último paciente.
    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 months0
    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 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: 89
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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. of subjects incapable of giving consent
    Adults with a clinical diagnosis of acquired TTP who require initiation of daily PE treatment.It is possible that patients were brought to the hospital unconscious and therefore unable to sign or give their consent.
    Adultos con un diagnóstico clínico de PTT adquirida que requieren instauración del tratamiento de IP diario.Es posible que los pacientes fueran llevados al hospital inconscientes y por lo tanto no puedan firmar ni dar su consentimiento.
    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)
    Standar of care
    Práctica Clínica Habitual
    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 Decision2015-10-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-10-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-08-16
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