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    EudraCT Number:2018-003775-35
    Sponsor's Protocol Code Number:SHP655-201
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-04-30
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2018-003775-35
    A.3Full title of the trial
    A Phase 2, multicenter, randomized, placebo-controlled, double-blind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, randomized, placebo-controlled, double-blind study of
    rADAMTS-13 (SHP655) in the treatment of patients with aTTP
    A.4.1Sponsor's protocol code numberSHP655-201
    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 SponsorBaxalta Innovation GmbH
    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.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 pointArchana Savla
    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+16175888208
    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/3/08/588
    D.3 Description of the IMP
    D.3.1Product nameRecombinant A Disintegrin And Metalloproteinase with Thrombospondin Type-1 Motifs 13 (rADAMTS13)
    D.3.2Product code SHP 655 (BAX 930)
    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 codeSHP655
    D.3.9.3Other descriptive nameBAX930 - RECOMBINANT HUMAN ADAMTS13
    D.3.9.4EV Substance CodeSUB183727
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNapadamtase alfa
    D.3.9.2Current sponsor codeSHP655
    D.3.9.3Other descriptive nameBAX930 - RECOMBINANT HUMAN ADAMTS13
    D.3.9.4EV Substance CodeSUB183727
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1500
    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 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 infusion
    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 (aTTP)
    E.1.1.1Medical condition in easily understood language
    acquired blood disorder
    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
    1. Assess the PK of ADAMTS-13 in aTTP subjects treated for an acute episode by daily plasma exchange (PEX), immunosuppressant therapy, with or without SHP655 supplementation
    2. Study the PK/PD relationship between ADAMTS-13 activity levels and pathophysiological biomarkers, as well as clinical efficacy parameters
    E.2.2Secondary objectives of the trial
    PK/PD Objectives
    1. Evaluate changes in levels of ADAMTS-13 binding and inhibitory autoantibodies from baseline in response to daily PEX, with or without SHP655 supplementation, during the acute episode and up to 30 days after the resolution of the acute aTTP episode
    2. Evaluate ADAMTS-13 activity levels in subjects up to 30 days after acute aTTP episode remission
    3. Specify dose(s) of SHP655 needed to achieve and maintain adequate plasma levels of rADAMTS-13

    Safety/Efficacy Objectives
    1. Assess the safety and immunogenicity of two regimens of SHP655 supplementation administered during an acute TTP episode in subjects undergoing PEX treatment and immunosuppressant therapy

    2. Evaluate the occurrences of aTTP related complications, aTTP relapses, exacerbations and end-organ function improvement

    3. Evaluate the time to aTTP related complications, aTTP relapses, exacerbations and end-organ function improvement

    4. Evaluate the occurrence of procedure related adverse events
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject or legally authorized representative voluntarily signs informed consent. For subjects unable to provide consent, a fully recognized medical proxy may be used according to local laws.
    2. Subject is 18 to 75 years old at the time of screening.
    3. Subject has been diagnosed with primary or secondary autoimmune aTTP based on the following criteria:
    a. Thrombocytopenia [drop in platelet count ≥50% or platelet count < 100,000/μL];
     No more than 3 subjects per arm may be enrolled with a screening platelet count ≥50,000/ μL.
    b. Microangiopathic hemolytic anemia [elevation of lactate dehydrogenase (LDH) >2-fold or by presence or increase of schistocytes in peripheral blood smear].
    4. Willingness to fully comply with study procedures and requirements, and intention to initiate plasma exchange (PEX). Subjects may be provisionally entered into the trial and undergo randomization pending the results of the ADAMTS-13 activity, anti-ADAMTS-13 antibody, and genetic testing for cTTP.
    5. If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study. 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.
    E.4Principal exclusion criteria
    1. Subject has been diagnosed with congenital TTP.
    2. Subject has plasma ADAMTS-13 activity> 10% of normal at the central lab; if screening samples are not taken until after the first PEX, ADAMTS-13 activity from the local lab is permitted to determine
    3. Subject has been diagnosed with another cause of thrombotic microangiopathy (TMA) including: DIC, disseminated malignancy, malignant hypertension, hematopoietic stem cell transplantation, shiga toxin-related
    and atypical HUS, drug toxicity (e.g. gemcitabine, mitomycin C, clopidogrel) and pregnancy-related thrombocytopenia syndromes (e.g. HELLP, eclampsia).
    4. Subject has been exposed to another IP within 30 days prior to enrollment or is scheduled to participate in another clinical study involving IP or investigational device during the course of the study.
    5. Subject has received caplacizumab within 1 months of study enrollment.
    6. Subject is human immunodeficiency virus positive (HIV+) with unstable disease or CD4+ count ≤200 cells/mm3 within 3 months screening.
    7. Subjects with conditions of severe immunodeficiency.
    8. Subject has had a previous aTTP event in the past 30 days.
    9. Subject has another underlying progressive fatal disease and/or life expectancy of less than 3 months.
    10. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
    11. 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. However, a fully recognized medical proxy will be permitted to provide consent.
    12. If female, subject is pregnant or lactating.
    13. Subject is a family member or employee of the Sponsor or investigator.
    14. Any contraindication to PEX, methylprednisolone and/or rituximab as per prescribing information.
    15. Known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent
    molecule ADAMTS-13, hamster protein, or other constituents of SHP655.
    E.5 End points
    E.5.1Primary end point(s)
    1. ADAMTS-13 activity levels
    2. Platelet count and LDH levels
    E.5.1.1Timepoint(s) of evaluation of this end point
    Various time points throughout the study
    E.5.2Secondary end point(s)
    PK/PD Endpoints
    1. The PK/PD temporal relationship of efficacy parameters (e.g., platelet count, LDH levels), as a function of ADAMTS-13 activity
    2. The ADAMTS-13 binding and inhibitory autoantibody levels in response to daily PEX, with or without SHP655 supplementation, during the acute TTP episode and up to 30 days after resolution
    3. ADAMTS-13 activity levels in subjects receiving additional SHP655 for up to 30 days after the resolution of the TTP episode
    4. The relationship between ADAMTS-13 activity and end-organ disease status (e.g., renal, neurologic, and cardiac)
    5. PK parameters such as incremental recovery, area under the curve, systemic and antibody induced clearance, maximum ADAMTS-13 activity between PEX or SHP 655 infusions, and trough levels prior PEX
    6. Occurrence of ADAMTS-13 activity trough levels >10%

    Safety/Efficacy endpoints
    1. Time to normalization of platelet count, defined as platelet count ≥ 150,000/μL, which must be confirmed by a second normal platelet count ≥ 150,000/μL and LDH <2 ULN 48 hours following initial normalization
    2. Occurrence of remission, defined as a normal platelet count and LDH <2 ULN for at least 48 hours following initial normalization of platelet count (acute episode period)
    3. Time to first exacerbation (aTTP episode ≤30 days following remission)
    4. Time to relapse (aTTP episode >30 days following remission)
    5. Occurrence of exacerbation
    6. Occurrence of relapse
    7. Occurrence of major clinical events related to TTP including: a. Death, b. Stroke, c. MI
    d. Organ dysfunction not normalized within the 90-day observation period; i. Chronic renal insufficiency, ii. Neurologic impairment, iii. Neurocognitive deficits.
    8. Incidence of major clinical events related to PEX, including clinically relevant bleeding (modified ITP score) or thrombosis at the site of line insertion, adverse reactions to plasma, including citrate reactions, allergic reactions, and TRALI
    9. Changes in the titer of binding and inhibitory antibodies to ADAMTS-13
    10. Occurrence of antibodies to SHP655
    11. Incidence of AEs and SAEs, and specifically product-related AEs and SAEs
    12. Clinically relevant changes in vital signs, clinical chemistry, and hematology
    E.5.2.1Timepoint(s) of evaluation of this end point
    Various time points throughout the study
    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 No
    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. 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 No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    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 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: 30
    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 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 33
    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
    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 Decision2019-10-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-08-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-08-05
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