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    Summary
    EudraCT Number:2022-001177-31
    Sponsor's Protocol Code Number:EFC16521
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-06-29
    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 ConcernedGermany - PEI
    A.2EudraCT number2022-001177-31
    A.3Full title of the trial
    An open-label, single-arm, multicenter study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy without first line therapeutic plasma exchange in adults with immune-mediated thrombotic thrombocytopenic purpura
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Caplacizumab and immunosuppressive therapy without first line therapeutic plasma exchange in adults with immune-mediated thrombotic thrombocytopenic purpura
    A.4.1Sponsor's protocol code numberEFC16521
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1244-0426
    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 SponsorSanofi-Aventis Recherche & Développement
    B.1.3.4CountryFrance
    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 supportSanofi-Aventis Recherche & Développement
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi-Aventis Deutschland GmbH
    B.5.2Functional name of contact point
    B.5.3.4CountryGermany
    B.5.6E-mailmedinfo.de@sanofi.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 Yes
    D.2.1.1.1Trade name Cablivi®
    D.2.1.1.2Name of the Marketing Authorisation holderAblynx NV
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameCablivi®
    D.3.2Product code ALX0081
    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 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 codeALX0081
    D.3.9.3Other descriptive nameNanobody directed towards the human A1 domain of von Willebrand factor
    D.3.9.4EV Substance CodeSUB181150
    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.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 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.IMP: 2
    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 Yes
    D.2.1.1.1Trade name Cablivi®
    D.2.1.1.2Name of the Marketing Authorisation holderAblynx NV
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameCablivi®
    D.3.2Product code ALX0081
    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 IMPSubcutaneous 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 codeALX0081
    D.3.9.3Other descriptive nameNanobody directed towards the human A1 domain of von Willebrand factor
    D.3.9.4EV Substance CodeSUB181150
    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.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 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
    Thrombotic Thrombocytopenic Purpura
    E.1.1.1Medical condition in easily understood language
    Thrombotic Thrombocytopenic Purpura
    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 the efficacy of caplacizumab in combination with immunosuppressive therapy (IST) without therapeutic plasma exchange (TPE) in adults with immune mediated thrombotic thrombocytopenic purpura (iTTP)
    E.2.2Secondary objectives of the trial
    To evaluate:
    - the need for therapeutic plasma exchange in adult participants with an episode of iTTP treated with caplacizumab and IST
    - the safety of caplacizumab in combination with IST without 1st line TPE in adults with iTTP
    - the effect of treatment with caplacizumab and IST without 1st line TPE on clinical response
    - the effect of treatment with caplacizumab and IST without 1st line TPE on restoring platelet counts
    - the effect of treatment with caplacizumab and IST without 1st line TPE on refractory disease
    - the effect of treatment with caplacizumab and IST without 1st line TPE on clinically relevant iTTP-related events consisting of iTTP-related mortality
    - the effect of treatment with caplacizumab and IST without 1st line TPE on clinically relevant iTTP-related events consisting of exacerbation of iTTP
    - the effect of treatment with caplacizumab and IST without 1st-line TPE on clinically relevant iTTP-related events consisting of relapse of iTTP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants with a clinical diagnosis of iTTP (initial or recurrent), which includes thrombocytopenia, microangiopathic hemolytic anemia (eg, presence of schistocytes in peripheral blood smear) and relatively preserved renal function. The iTTP diagnosis should be confirmed by ADAMTS13 testing within 48 hours (2 days).
    Participants with a clinical diagnosis of iTTP and a French TMA score of 1 or 2.

    A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:
    Is a woman of nonchildbearing potential (WONCBP),
    OR
    Is a woman of childbearing potential (WOCBP) and agrees to use an acceptable contraceptive method during the overall treatment period and for at least 2 months after the last study drug administration.

    Male participants with female partners of childbearing potential must agree to follow the contraceptive guidance as per protocol during the overall treatment period and for at least 2 months after last study drug administration.
    E.4Principal exclusion criteria
    Platelet count ≥100 × 10^9/L.

    Serum creatinine level >2.26 mg/dL (200 ╬╝mol/L) in case platelet count is >30 × 10^9/L. (to exclude possible cases of atypical HUS).

    Known other causes of thrombocytopenia including but not limited to:
    • Clinical evidence of enteric infection with E. coli 0157 or related organism.
    • Atypical HUS.
    • Hematopoietic stem cell, bone marrow or solid organ transplantation-associated thrombotic microangiopathy.
    • Known or suspected sepsis.
    • Diagnosis of disseminated intravascular coagulation.

    Congenital TTP (known at the time of study entry).

    Clinically significant active bleeding or known co-morbidities associated with high risk of bleeding (excluding thrombocytopenia).

    Inherited or acquired coagulation disorders.

    Malignant arterial hypertension.

    Participants requiring or expected to require invasive procedures immediately (eg, stroke requiring thrombolytic therapy, those who need mechanical ventilation, etc.).

    Those presenting with severe neurological or cardiac disease.

    Clinical condition other than that associated with TTP, with life expectancy <6 months, such as end-stage malignancy.

    Known chronic treatment with anticoagulants and anti-platelet drugs that cannot be stopped (interrupted) safely, including but not limited to:
    • vitamin K antagonists.
    • direct-acting oral anticoagulants.
    • heparin or low molecular weight heparin (LMWH).
    • non-steroidal anti-inflammatory molecules other than acetyl salicylic acid.

    Participants who were previously enrolled in this clinical study (study EFC16521).

    Participants who received an investigational drug, or device, other than caplacizumab, within 30 days of anticipated IMP administration or 5 half-lives of the previous investigational drug, whichever is longer.

    Positive result on COVID test.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants achieving Remission without requiring therapeutic plasma exchange (TPE); Remission is defined as sustained Clinical Response (sustained platelet count ≥150 × 10^9/L and lactate dehydrogenase [LDH] <1.5 × upper limit of normal [ULN] and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits) with either (a) no TPE and no anti- von Willebrand factor (anti-vWF) therapy for ≥30 days (Clinical Remission), or (b) with attainment of a disintegrin and metalloproteinase with a thrombospondin type 1 motif13 (ADAMTS13) ≥50% (Complete ADAMTS13 remission), whichever occurs first.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Overall study period from day 1 to day 168 (treatment period + 12 weeks of follow-up)
    E.5.2Secondary end point(s)
    1. Proportion of participants achieving Remission
    2. Proportion of participants who require TPE
    3. The occurrence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs)
    4. Proportion of participants achieving Clinical Response; Clinical Response is defined as sustained platelet count ≥150 × 10^9/L and LDH <1.5 × ULN and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits.
    5. Proportion of participants achieving Clinical Response
    6. Time to platelet count response; Platelet count response defined as time from start of treatment to initial platelet count ≥150 × 10^9/L that is sustained for ≥2 days
    7. Proportion of participants refractory to therapy; Refractory to therapy defined as lack of sustained platelet count increment (over 2 consecutive days) or platelet counts <50 × 10^9/L and persistently elevated LDH (>1.5 × ULN) despite 5 days of treatment
    8. Proportion of participants with TTP-related death
    9. Proportion of participants with TTP-related death
    10. Proportion of participants with a clinical exacerbation of iTTP; Clinical Exacerbation is defined as after a Clinical Response and before a Clinical Remission, platelet count decreases to <150 × 10^9/L (with other causes of thrombocytopenia excluded), with or without clinical evidence of new or progressive ischemic organ injury, within 30 days of stopping TPE or anti vWF therapy.
    11. Proportion of participants with a clinical exacerbation of iTTP
    12. Proportion of participants with a clinical relapse of iTTP; Clinical Relapse is defined as after a Clinical Remission, platelet count decreases to <150 × 10^9/L (with other causes of thrombocytopenia ruled out), with or without clinical evidence of new ischemic organ injury. A Clinical Relapse must be confirmed by documentation of severe ADAMTS13 deficiency (<10%).
    13. Proportion of participants with a clinical relapse of iTTP
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Overall study period from day 1 to day 168
    2. On-treatment period from day 1 to day 84
    3. Treatment-emergent (TE) period from day 1 to day 112
    4. On-treatment period from day 1 to day 84
    5. Overall study period from day 1 to day 168
    6. From day 1 to day 168
    7. On-treatment period from day 1 to day 84
    8. On-treatment period from day 1 to day 84
    9. Overall study period from day 1 to day 168
    10. On-treatment period from day 1 to day 84
    11. Overall study period from day 1 to day 168
    12. On-treatment period from day 1 to day 84
    13. Overall study period from day 1 to day 168
    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 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 No
    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.8.3 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA32
    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
    Switzerland
    Canada
    Japan
    United Kingdom
    United States
    Austria
    Belgium
    Czechia
    France
    Germany
    Greece
    Italy
    Netherlands
    Spain
    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
    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 months8
    E.8.9.1In the Member State concerned days20
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days23
    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: 58
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 39
    F.4.2.2In the whole clinical trial 64
    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)
    None
    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 Decision2022-08-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-15
    P. End of Trial
    P.End of Trial StatusOngoing
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