Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
Thrombotic Thrombocytopenic Purpura (TTP)
About this trial
This is an expanded access trial for Thrombotic Thrombocytopenic Purpura (TTP) focused on measuring Drug Therapy
Eligibility Criteria
Inclusion Criteria: Participant or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (signed by legal representative if participants is <18 years of age). Participant has a documented diagnosis of severe congenital ADAMTS13 deficiency, defined by: Confirmed molecular genetic testing, or ADAMTS13 activity and a negative test for ADAMTS13 antibodies. Participant is currently on a prophylactic regimen or has a documented history of at least 1 TTP event. Standard of Care (SoC) is considered inadequate, or the participant is intolerant to SoC*. SoC are plasma-based therapies, e.g., fresh frozen plasma or S/D treated plasma Exclusion Criteria: Participant is currently eligible to enter into an interventional clinical trial for cTTP. There is a suitable alternative commercially available treatment for the treatment of cTTP. Participant has been diagnosed with any other thrombotic microangiopathies (TMAs) like disorder (microangiopathic hemolytic anemia), including acquired TTP. Participant has a known hypersensitivity/allergies/intolerance or contraindications to TAK-755 or its excipients, such as hamster proteins. Participant has a medical history or presence of a functional ADAMTS13 inhibitor. Participant has a medical history of genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including Participants who are human immunodeficiency virus (HIV)-positive with an absolute cluster of differentiation 4 (CD4) count <200 per millimeter cube (<200/mm^3) or who are receiving chronic immunosuppressive drugs