Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Completed · Phase 2 · Has a placebo group

Conditions studied: Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

In brief

The purpose of this study is to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment of acquired thrombotic thrombocytopenic purpura (aTTP) participants.

Key facts

Study ID

NCT03922308

Run by

Shire

People needed

28

Starts

2019-10-09

Expected to finish

2021-08-05

Last updated by the study team

2022-12-01

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participant or legally authorized representative voluntarily signs informed consent. For participants unable to provide consent, a fully recognized medical proxy may be used according to local laws.
• 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.

You may not qualify if…

• Participant has been diagnosed with congenital TTP.
• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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