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)

Baxalta Innovations GmbH0 sites77 target enrollmentSeptember 30, 2021

Overview

No summary available.

Registry

who.int

Start Date

September 30, 2021

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•Applies to Subjects who have completed the TAK\-755 Phase 3 pivotal study (Study 281102\) in the prophylactic cohort
•Subjects who have completed TAK\-755 Study 281102 (in the prophylactic cohort 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.

•The subject will be excluded from the study if any of the following exclusion criteria are met.
•Applies to subjects who have completed TAK\-755 Phase 3 pivotal study (Study 281102\):
•1\. Known life\-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS\-13, hamster protein, or other constituents of TAK\-755\.
•2\. Subject has presence of a functional ADAMTS\-13 inhibitor at screening.
•3\. In the opinion of the investigator, the subject has another clinically significant concomitant disease that may pose additional risks for the subject.
•4\. Subject is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
•5\. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
•6\. 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.
•7\. Subject is a family member or employee of the sponsor or investigator
•Applies to naïve subjects and non\-naïve on\-demand cohort subjects: