Primary immune thrombocytopenia (ITP) is a rare disease that results in low levels of platelets - the cells that help blood clot. The main aim of the study is to check for side effects from taking TAK-079 at three different dose levels. Another aim is to learn if TAK-079 can increase the platelet count in people with ITP. In addition to receiving stable background therapy for ITP, participants will receive an injection of either TAK-079 or a placebo once a week for 2 months. A placebo looks like TAK-079 but will not have any medicine in it. After treatment, all participants will be followed-up for another 2 months. Then, participants who received TAK-079 will continue to be followed-up for an extra 4 months. Participants who received the placebo and would like to receive TAK-079 may be able to do this in an extension period in the study.
The drug being tested in this study is called TAK-079. TAK-079 is being tested to treat people who have primary immune thrombocytopenia (ITP). This study will evaluate the safety and biologic activity of TAK-079 or matching placebo in combination with stable ITP background therapy. The study will enroll approximately 36 to 54 participants. In Part A of the study, participants will be randomly assigned (by chance, like flipping a coin) to one of the three treatment groups. Those who received placebo in this period will have the choice to receive TAK-079 after a safety follow-up period and will be randomized to one of the two open-label TAK-079 treatment arms. An unblinded safety review will take place once a minimum of 24 evaluable participants are available for analysis in Part A to decide whether to open enrollment into Part B. In Part B participants will be randomly assigned to one of two treatment groups. Those who received placebo in this period will have the choice to receive study drug after a safety follow-up period in a single open-label TAK-079 treatment arm. This multi-center trial will be conducted worldwide. All participants will be followed for at least 8 weeks in a Safety Follow-up Period, and a 16-week Long-term Follow-up Period after the 8 weeks of treatment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
41
Arizona Clinical Research Center - Hunt - PPDS
Tucson, Arizona, United States
University of Florida
Gainesville, Florida, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Medical Center
Boston, Massachusetts, United States
Percentage of Participants With at Least One Grade 3 or Higher Treatment Emergent Adverse Event (TEAE), Treatment Emergent Serious Adverse Event (SAE), and TEAEs Leading to TAK-079 Discontinuation
An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with the treatment. SAE means any untoward medical occurrence that at any dose: a) results in death; b) is life-threatening; c) requires inpatient hospitalization or prolongation of an existing hospitalization; d) results in persistent or significant disability or incapacity; e) is a congenital anomaly/birth defect; f) is a medically important event. TEAEs were defined as an AE having a start date and time equal to or later than the start date and time of the first dose of investigational medicinal product (IMP). Percentages were rounded off to the nearest single decimal place.
Time frame: Up to Week 32 in each Period of the study
Percentage of Participants With Platelet Response at Weeks 16 and 32
Platelet response is defined as a platelet count ≥50,000/microliter (μL) and ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
Time frame: At Weeks 16 and 32
Percentage of Participants With Complete Platelet Response at Weeks 16 and 32
Complete platelet response is defined as a platelet count ≥100,000/μL on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
Time frame: At Weeks 16 and 32
Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32
A clinically meaningful platelet response is defined as a platelet count ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
Time frame: At Weeks 16 and 32
Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32
A hemostatic platelet response is defined for participants with a baseline platelet count of \<15,000/μL who achieved a platelet count of ≥30,000/μL and ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.
Time frame: At Weeks 16 and 32
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