Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
NCT ID: NCT03922308
Last Updated: 2022-12-01
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
28 participants
INTERVENTIONAL
2019-10-09
2021-08-05
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Standard of Care (SoC) + Placebo
Participants received SoC daily PEX followed by placebo immediately and 12 +/- 1 hours after completion of PEX until remission was achieved (up to approximately 6 months).
Placebo
Participants will receive injection of placebo matched to SHP655.
Standard of Care
Participants will receive PEX as Standard of Care (SOC).
SoC + SHP655 + Placebo
Participants received SoC daily PEX and SHP655 40 +/- 4 international units per kilogram (IU/kg), IV injection, QD, immediately after PEX and placebo 12 +/- 1 hours after completion of PEX until remission was achieved (up to approximately 6 months).
Placebo
Participants will receive injection of placebo matched to SHP655.
SHP655
Participants will receive injection of SHP655.
Standard of Care
Participants will receive PEX as Standard of Care (SOC).
SoC + SHP655
Participants received SoC daily PEX and SHP655 40 +/- 4 IU/kg, IV injection, BID, immediately after PEX and 12 +/- 1 hours after completion of PEX until remission was achieved (up to approximately 6 months).
SHP655
Participants will receive injection of SHP655.
Standard of Care
Participants will receive PEX as Standard of Care (SOC).
Interventions
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Placebo
Participants will receive injection of placebo matched to SHP655.
SHP655
Participants will receive injection of SHP655.
Standard of Care
Participants will receive PEX as Standard of Care (SOC).
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participant is 18 to 75 years old at the time of screening.
* Participant has been diagnosed with primary or secondary autoimmune acquired thrombotic thrombocytopenic purpura (aTTP) based on the following criteria:
a) Thrombocytopenia \[drop in platelet count \>=50% or platelet count \<100,000/microlitre (μL)\] i) No more than 3 participants per arm may be enrolled with a screening platelet count \>= 50,000/μL.
b) Microangiopathic hemolytic anemia \[elevation of lactate dehydrogenase (LDH) \>2-fold or by presence or increase of schistocytes in peripheral blood smear\].
* Willingness to fully comply with study procedures and requirements, and intention to initiate plasma exchange (PEX). Participants 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 congenital thrombotic thrombocytopenic purpura (cTTP).
* If female of childbearing potential, participant 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.
Exclusion Criteria
* Participant 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 eligibility.
* Participant 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).
* Participant 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.
* Participant has received caplacizumab within 1 month prior to study enrollment.
* Participant is human immunodeficiency virus positive (HIV+) with unstable disease or CD4+ count \<=200 cells/mm\^3 within 3 months screening.
* Participants with conditions of severe immunodeficiency.
* Participant has had a previous aTTP event in the past 30 days.
* Participant has another underlying progressive fatal disease and/or life expectancy of less than 3 months.
* Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures
* Participant 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.
* If female, participant is pregnant or lactating.
* Participant is a family member or employee of the Sponsor or investigator.
* Any contraindication to PEX, methylprednisolone and/or rituximab as per prescribing information.
* Known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS-13, hamster protein, or other constituents of SHP655.
18 Years
75 Years
ALL
No
Sponsors
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Takeda Development Center Americas, Inc.
INDUSTRY
Shire
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Shire
Locations
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The University of Alabama at Birmingham
Birmingham, Alabama, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States
Stephenson Cancer Center
Oklahoma City, Oklahoma, United States
Medical University of South Carolina (MUSC)
Charleston, South Carolina, United States
London Health Sciences Centre (LHSC) - University Hospital
London, Ontario, Canada
St. Michael's Hospital
Toronto, Ontario, Canada
CHU de Reims - Hôpital Maison Blanche
Reims, Marne, France
Hopital Conception
Marseille, , France
Chu Saint-Antoine
Paris, , France
CHU de Rouen
Seine Maritime, , France
Hamatologie, Onkologie, Hämostaseologie
Frankfurt, , Germany
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, , Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, , Italy
Hospital General Universitario de Alicante
Alicante, , Spain
Hospital General Universitario Gregorio Maranon
Madrid, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Hospital Dr. Peset
Valencia, , Spain
Hospital Universitari i Politècnic la Fe
Valencia, , Spain
University Hospital of Wales
Cardiff, West Glamorgan, United Kingdom
Royal Liverpool University Hospital
Liverpool, , United Kingdom
1st Floor, UCLH-Haematology
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol: Original
Document Type: Study Protocol: Protocol Amendment 1
Document Type: Study Protocol: Protocol Amendment 2
Document Type: Study Protocol: Protocol Amendment 3
Document Type: Study Protocol: Protocol Amendment 4
Document Type: Statistical Analysis Plan
Related Links
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To obtain more information on the study, click here/on this link
Other Identifiers
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2018-003775-35
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
SHP655-201
Identifier Type: -
Identifier Source: org_study_id
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