A phase 3, randomized, controlled study of prophylactic and on-demand
- Conditions
- severe congenital thrombotic thrombocytopenicpurpura (cTTP, Upshaw-Schulman Syndrome [USS],hereditary thrombotic thrombocytopenic purpura [hTTP]
- Registration Number
- JPRN-jRCT2080224906
- Lead Sponsor
- Baxalta now part of Shire(ICCC: IQVIA Services Japan K.K.)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- recruiting
- Sex
- All
- Target Recruitment
- 4
1) Participant or legally authorized representative has provided signed informed consent greater than or equal to (>=)18 years of age) and/or assent form (signed by legal representative if participants is less than (<)18 years of age).
2)Participant is 0 to 70 years of age, inclusive, at the time of screening. (Participants <18 years of age will be enrolled only after at least 5 adults (>=18 years of age) each have at least 10 exposures with BAX 930 and reviewed by the DMC. In France, no participants younger than 18 years of age will be enrolled into the study before the first adult participant has been treated with BAX 930 for a minimum of 6 months.
3)Participant has a documented diagnosis of severe hereditary ADAMTS13 deficiency, defined as:Confirmed by molecular genetic testing, documented in participant history or at screening, and
A disintegrin and metalloprotease with a thrombospondin type 1 motif, member 13 (ADAMTS13) activity <10% as measured by the fluorescent resonance energy transfer- von Willebrand factor73 (FRETS-VWF73) assay, documented in participant history or at screening (participants currently receiving SoC prophylactic therapy may exceed 10% ADAMTS13 activity at screening).
Note: Participants currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion
4)Participant does not display any severe thrombotic thrombocytopenic purpura (TTP) signs (platelet count <100,000/ microliter (uL) and elevation of lactate dehydrogenase (LDH) greater than (>2)x upper limit of normal (ULN)) at screening. (Prophylactic cohort only).
5)Participant is currently on a prophylactic dosing regimen or has a documented history of at least 1 TTP event and an ability to tolerate SoC prophylactic dosing (prophylactic cohort only).
1) Participant has been diagnosed with any other TTP-like disorder (microangiopathic hemolytic anemia), including acquired TTP.
2) Participant has known hypersensitivity to hamster proteins.
3)Participant has experienced an acute TTP episode less than 30 days prior to screening (prophylactic cohort only).
4) Participant has a medical history or presence of a functional ADAMTS13 inhibitor at screening.
5) 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/ Cubic Millimeter (mm^3) or who are receiving chronic immunosuppressive drugs.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method