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    Summary
    EudraCT Number:2020-003348-10
    Sponsor's Protocol Code Number:TAK-755-3002
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-09-14
    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 ConcernedUK - MHRA
    A.2EudraCT number2020-003348-10
    A.3Full title of the trial
    A Phase 3b, prospective, open-label, multicenter, single treatment arm, continuation study of the safety and efficacy of TAK-755 (rADAMTS-13, also known as BAX 930/SHP655) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP; Upshaw Schulman Syndrome, or hereditary thrombotic thrombocytopenic purpura)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A continuation study to evaluate the prophylactic and on demand treatment of congenital Thrombotic Thrombocytopenic Purpura (cTTP) with the drug TAK-755 (rADAMTS-13, also known as BAX 930/SHP655)
    A.3.2Name or abbreviated title of the trial where available
    Baxalta TAK-755-3002 Continuation study to 281102
    A.4.1Sponsor's protocol code numberTAK-755-3002
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/324/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxalta Innovations GmbH
    B.1.3.4CountryAustria
    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 supportBaxalta Innovations GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxalta US Inc.
    B.5.2Functional name of contact pointEmily Skelton
    B.5.3 Address:
    B.5.3.1Street Address650 East Kenadall Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02191
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 617 588-8426
    B.5.6E-mailemily.skelton@takeda.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 nameRecombinant A Disintegrin and Metalloproteinase Thrombospondin Type-1 Motifs 13
    D.3.2Product code TAK-755 orBAX930 or SHP655
    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 INNApadamtase alfa
    D.3.9.2Current sponsor codeTAK-755 (SHP655 or BAX930)
    D.3.9.3Other descriptive nameRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNApadamtase alfa
    D.3.9.2Current sponsor codeTAK-755 (BAX930 or SHP655)
    D.3.9.3Other descriptive nameRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    congenital Thrombotic thrombocytopenic purpura (TTP)
    E.1.1.1Medical condition in easily understood language
    congenital Thrombotic thrombocytopenic purpura
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    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 long-term safety and tolerability of TAK-755 (rADAMTS-13) in terms of related adverse events (AEs) and serious adverse events (SAEs) in both the prophylactic and the on-demand cohorts.
    E.2.2Secondary objectives of the trial
    1. To evaluate the efficacy of TAK-755 in the treatment of acute thrombotic thrombocytopenic purpura (TTP) episodes as measured by the
    a) number of acute TTP events responding to treatment
    and
    b) time to resolution of the acute TTP in either the prophylactic or the on-demand cohorts.
    2. To evaluate the incidence of isolated TTP manifestations including thrombocytopenia, microangiopathic hemolytic anemia, renal dysfunction, neurologic signs and symptoms, and abdominal pain in the prophylactic cohort.
    3. To evaluate the proportion of subjects that require dose modification and supplemental dose in the prophylactic cohort.
    4. To evaluate the incidence of subacute manifestations in subjects receiving prophylactic treatment.
    For more secondery objectives please refer to Protocol pages 10,11.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who have completed TAK-755 Phase 3 pivotal study (281102) and who meet ALL of the following criteria are eligible for this study:
    1. Subject or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (<18 years of age).
    2. Subject is 0 to 70 years of age at the time of screening of the 281102 study.
    3. Subject has been diagnosed with severe congenital ADAMTS-13 deficiency.
    4. Subject does not display any severe TTP signs (platelet count <100,000/µL and elevation of LDH >2 × upper limit of normal [ULN]) at screening (prophylactic cohort only).
    5. Subjects ≥16 years of age must have a Karnofsky score ≥70% and subjects <16 years of age must have a Lansky score ≥80%.
    6. If female of childbearing potential, subject presents with a negative serum or urine pregnancy test confirmed not more than 7 days before the first IP administration and agrees to employ highly effective birth control measures for the duration of the study and to undergo quarterly pregnancy testing.
    7. Sexually active males must use an accepted and effective method of contraception during the treatment and until a minimum of 16 days after the last dose administered.
    8. Subject is willing and able to comply with the requirements of the protocol.

    TAK-755 naïve subjects can only be enrolled in this continuation after enrollment of the adult subjects in the prophylactic arm of the TAK-755 Phase 3 pivotal study (281102) has been completed. TAK-755 naïve pediatric subjects can be enrolled after enrollment of the respective age cohort into the pivotal Phase 3 study (281102) has been completed. Subjects in the UK who are naïve to TAK-755 may not be enrolled in this study.
    TAK-755 naïve subjects and subjects who were enrolled into the on-demand cohort of the TAK-755 Phase 3 pivotal study (281102) who meet ALL of the following criteria are eligible for this study:
    1. Subject is naïve to TAK-755 or was enrolled into the on-demand cohort of the TAK-755 Phase 3 pivotal study (281102) for treatment of an acute event but did not receive prophylactic treatment.
    2. Subject or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (<18 years of age).
    3. Subject is 0 to 70 years of age at the time of screening.
    4. Subject has been diagnosed with severe congenital ADAMTS-13 deficiency defined as:
    a. Confirmed by molecular genetic testing, documented in subject history or at screening, and
    b. ADAMTS-13 activity <10% as measured by the fluorescence resonance energy transfer (FRETS)-Von Willebrand factor (VWF)73 assay, documented in subject history or at screening. Subjects currently receiving standard of care prophylactic therapy may exceed 10% ADAMTS 13 activity at screening.
    5. Subjects currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion.
    6. Subject does not display any severe TTP signs (platelet count <100,000/µL and elevation of LDH >2 × ULN) at screening (prophylactic cohort only).
    7. Subjects ≥16 years of age must have a Karnofsky score ≥70% and subjects <16 years of age must have a Lansky score ≥80%.
    8. Subject is hepatitis C virus negative (HCV­) as confirmed by antibody or polymerase chain reaction testing OR HCV positive (HCV+) if their disease is chronic but stable.
    9. If female of childbearing potential, subject presents with a negative serum or urine pregnancy test confirmed not more than 7 days before the first IP administration and agrees to employ highly effective birth control measures for the duration of the study and to undergo quarterly pregnancy testing.
    10. Sexually active males must use an accepted and effective method of contraception during treatment and until a minimum of 16 days after the last dose administered.
    11. Subject is willing and able to comply with the requirements of the protocol.
    E.4Principal exclusion criteria
    The subject will be excluded from the study if any of the following exclusion criteria are met.
    For both subjects who have completed TAK-755 Phase 3 pivotal study (281102) and TAK 755 naïve subjects:
    1. Subject has been diagnosed with any other TTP-like disorder (microangiopathic hemolytic anemia), including immune-mediated TTP.
    2. Known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS-13, hamster protein, or other constituents of TAK-755.
    3. Subject has a medical history or presence of a functional ADAMTS-13 inhibitor at screening.
    4. Subject has a medical history of a genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including subjects who are human immunodeficiency virus positive with an absolute cluster of differentiation 4 (CD4) count <200/mm3 or who are receiving chronic immunosuppressive drugs.
    5. Subject has a history of significant neurological events, such as major stroke, indicating that a relapse might have severe consequences, as judged by the investigator.
    6. Subject has been diagnosed with severe cardiovascular disease (New York Heart Association classes 3 to 4).
    7. Subject with end stage renal disease requiring chronic dialysis.
    8. Subject has been diagnosed with hepatic dysfunction, as evidenced by, but not limited to, any of the following:
    a. Serum alanine aminotransferase ≥2 × ULN
    b. Severe hypoalbuminemia <24 g/L
    c. Portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices).
    9. In the opinion of the investigator, the subject has another clinically significant concomitant disease that may pose additional risks for the subject.
    10. Subject has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to enrollment. Use of corticosteroids in conjunction with administration of fresh frozen plasma to prevent allergic manifestations is permitted.
    11. Subject has an acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, bronchial asthma) at the time of screening (prophylactic cohort only).
    12. Subject is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
    13. Subject has a history of drug and/or alcohol abuse within the last 2 years.
    14. Subject has a progressive fatal disease and/or life expectancy of ≤3 months.
    15. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
    16. Subject suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
    17. Subject is a family member or employee of the sponsor or investigator.
    18. If female, subject is pregnant or lactating at the time of enrollment.
    19. In the UK only: Subjects who are naive to TAK-755.
    E.5 End points
    E.5.1Primary end point(s)
    There is no primary efficacy endpoint for this study, as the primary objective of the study is long-term safety.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Various timepoints throughout the study
    E.5.2Secondary end point(s)
    1. Prophylactic cohort:
    Efficacy analysis for the prophylactic cohort will provide the number and incidence rate of acute TTP events in total and by enrollment status (“Naïve” or having completed the Phase 3 pivotal study [281102]), with the corresponding 95%, 2-sided confidence intervals (CIs).
    2. Prophylactic and on-demand cohorts:
    For acute TTP events, the number and proportion of acute events responding to treatment will be summarized, along with the 2-sided 95% CIs for the pooled prophylactic and on-demand cohort data as well as by cohort.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Various timepoints throughout the study
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA26
    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
    Australia
    Austria
    Belgium
    Brazil
    Canada
    China
    Colombia
    Czechia
    Denmark
    France
    Germany
    Hungary
    India
    Israel
    Italy
    Japan
    Korea, Republic of
    Netherlands
    Norway
    Poland
    Portugal
    Russian Federation
    Serbia
    Spain
    Sweden
    Switzerland
    Taiwan
    Thailand
    Turkey
    Ukraine
    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
    last subject last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days10
    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: 18
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 3
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 4
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 46
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 88
    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 treatment or use of study drug once it is commercially available.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-11-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-17
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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