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 TAK-755-3002 Continuation study to 281102

Baxalta Innovations GmbH0 sites88 target enrollmentSeptember 14, 2020

Conditionscongenital Thrombotic thrombocytopenic purpura (TTP)Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: congenital Thrombotic thrombocytopenic purpura (TTP)
Sponsor: Baxalta Innovations GmbH
Enrollment: 88
Status: Active, not recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

September 14, 2020

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Baxalta Innovations GmbH

Eligibility Criteria

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.

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: