Primary Immune Thrombocytopenia
Conditions
Keywords
Drug Therapy
Brief summary
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.
Detailed description
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.
Interventions
DRUGPlacebo
TAK-079 placebo-matching SC injection.
DRUGTAK-079
TAK-079 SC injection.
Sponsors
Takeda
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Diagnosed with ITP that has persisted for ≥3 months, diagnosed in accordance to The American Society of Hematology 2011 Evidence-based Practice Guideline for Immune Thrombocytopenia or the International Consensus Report on The Investigation and Management of Primary Immune Thrombocytopenia as locally applicable. 2. Has a mean platelet count of \<30,000/μL (and individually ≤35,000/μL) on at least 2 measurements at least 1 week apart during screening. 3. Diagnosis of ITP supported by a prior response to an ITP therapy (other than a thrombopoietin receptor agonists \[TPO-RA\]) that achieved a platelet count of ≥50,000/μL. 4. If receiving standard background treatment for ITP, treatment should be stable in dose and frequency for at least 4 weeks before dosing. 1. Permitted standard background treatments may include: 1 oral corticosteroid; ±1 immunosuppressant from the following list: azathioprine, danazol, dapsone, cyclosporine, mycophenolate mofetil, mycophenolate sodium; ±1 TPO-RA (romiplostim, eltrombopag, avatrombopag); ±fostamatinib. Corticosteroids, including dexamethasone, must be given as oral, daily or every-other-day therapy as opposed to pulse therapy. 2. The dose of any permitted standard background therapy must be expected to remain stable through the study, unless dose reduction is required because of toxicities.
Exclusion criteria
1. Use of anticoagulants or any drug with antiplatelet effect (such as aspirin) within 3 weeks before screening. 2. Has a history of any thrombotic or embolic event within 12 months before screening. 3. Has a history of splenectomy within 3 months before screening. 4. Use of intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin or anti-D immunoglobulin treatment within 4 weeks of screening, or an expectation that any therapy besides the participant's standard background therapies may be used for treatment of thrombocytopenia (e.g., a rescue therapy) between screening and dosing. 5. Diagnosed with chronic obstructive pulmonary disease (COPD) or asthma, and a prebronchodilatory forced expiratory volume in 1 second (FEV1) \<50% of predicted normal. 6. Use of rituximab or any monoclonal antibody (mAb) for immunomodulation within 4 months before first dosing. Note: Participants with prior exposure to rituximab must have cluster of differentiation (CD) 19 counts within the normal range at screening. 7. Use of immunosuppressants (such as cyclophosphamide, vincristine) other than permitted oral immunosuppressants within 6 months before first dosing. 8. Has been diagnosed with myelodysplastic syndrome. 9. Has received a live vaccine within 4 weeks before screening or has any live vaccine planned during the study. 10 Has had an opportunistic infection ≤12 weeks before initial study dosing or is currently undergoing treatment for a chronic opportunistic infection, such as tuberculosis (TB), pneumocystis pneumonia, cytomegalovirus, herpes simplex virus, herpes zoster, or atypical mycobacteria.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Up to Week 32 in each Period of the study | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Platelet Response at Weeks 16 and 32 | 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. |
| Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | 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. |
| Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | 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. |
| Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | 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. |
Countries
Bulgaria, China, Croatia, Germany, Greece, Italy, Japan, Slovenia, Spain, Ukraine, United States
Participant flow
Recruitment details
Participants took part in the study at 24 investigative sites globally from 09 November 2020 to 29 April 2024.
Pre-assignment details
Participants who had persistent/chronic primary immune thrombocytopenia (ITP) were randomized to receive either mezagitamab (TAK-079) or matching placebo in Part A or Part B of this study.
Participants by arm
| Arm | Count |
|---|---|
| Part A & B: Double Blind Period: Placebo Participants received TAK-079 placebo-matching injection SC, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Placebo-assigned participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32. | 13 |
| Part A: Double Blind Period: TAK-079 100 mg Participants received TAK-079 100 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32. | 9 |
| Part A: Double Blind Period: TAK-079 300 mg Participants received TAK-079 300 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32. | 8 |
| Part B: Double Blind Period: TAK-079 600 mg Participants received TAK-079 600 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants were then followed up for another 16 weeks in an unblinded LFP up to Week 32. | 11 |
| Total | 41 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 | FG005 | FG006 |
|---|---|---|---|---|---|---|---|---|
| Double Blind Period (Main Study) | Reason Not Specified | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
| Double Blind Period (Main Study) | Withdrawal by Subject | 1 | 1 | 0 | 2 | 0 | 0 | 0 |
Baseline characteristics
| Characteristic | Part A & B: Double Blind Period: Placebo | Part A: Double Blind Period: TAK-079 100 mg | Part A: Double Blind Period: TAK-079 300 mg | Part B: Double Blind Period: TAK-079 600 mg | Total |
|---|---|---|---|---|---|
| Age, Continuous | 38.8 years STANDARD_DEVIATION 15.86 | 49.0 years STANDARD_DEVIATION 14.45 | 52.3 years STANDARD_DEVIATION 16.59 | 48.4 years STANDARD_DEVIATION 19.68 | 46.2 years STANDARD_DEVIATION 17.03 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 12 Participants | 9 Participants | 8 Participants | 9 Participants | 38 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 0 Participants | 0 Participants | 2 Participants | 3 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) White | 11 Participants | 9 Participants | 8 Participants | 9 Participants | 37 Participants |
| Sex: Female, Male Female | 9 Participants | 5 Participants | 5 Participants | 9 Participants | 28 Participants |
| Sex: Female, Male Male | 4 Participants | 4 Participants | 3 Participants | 2 Participants | 13 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk |
|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 13 | 0 / 9 | 0 / 8 | 0 / 11 | 0 / 4 | 0 / 4 | 0 / 4 |
| other Total, other adverse events | 9 / 13 | 7 / 9 | 5 / 8 | 6 / 11 | 2 / 4 | 4 / 4 | 2 / 4 |
| serious Total, serious adverse events | 1 / 13 | 2 / 9 | 0 / 8 | 2 / 11 | 1 / 4 | 0 / 4 | 0 / 4 |
Outcome results
Primary
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
Population: The Safety Analysis Set included all participants who received at least 1 dose of study drug.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Part A & B: Double Blind Period: Placebo | 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 | TEAEs Leading to TAK-079 Discontinuation | 0 percentage of participants |
| Part A & B: Double Blind Period: Placebo | 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 | Treatment Emergent SAE | 7.7 percentage of participants |
| Part A & B: Double Blind Period: Placebo | 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 | Grade 3 or Higher TEAE | 23.1 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | 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 | Treatment Emergent SAE | 22.2 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | 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 | Grade 3 or Higher TEAE | 22.2 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | 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 | TEAEs Leading to TAK-079 Discontinuation | 22.2 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | 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 | TEAEs Leading to TAK-079 Discontinuation | 0 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | 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 | Grade 3 or Higher TEAE | 0 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | 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 | Treatment Emergent SAE | 0 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | 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 | Treatment Emergent SAE | 18.2 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | 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 | Grade 3 or Higher TEAE | 27.3 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | 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 | TEAEs Leading to TAK-079 Discontinuation | 18.2 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | 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 | Treatment Emergent SAE | 25.0 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | 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 | Grade 3 or Higher TEAE | 0 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | 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 | TEAEs Leading to TAK-079 Discontinuation | 25.0 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | 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 | Grade 3 or Higher TEAE | 0 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | 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 | TEAEs Leading to TAK-079 Discontinuation | 0 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | 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 | Treatment Emergent SAE | 0 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | 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 | TEAEs Leading to TAK-079 Discontinuation | 0 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | 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 | Treatment Emergent SAE | 0 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | 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 | Grade 3 or Higher TEAE | 25.0 percentage of participants |
Secondary
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
Population: The Full Analysis Set consisted of all randomized participants who had received at least 1 dose of study drug. Number analyzed indicates the number of participants with data available for analyses at the specified time point. All participants in the 'Part A \& B: Double Blind Period: Placebo' arm either transitioned to OLE Period or discontinued from the study prior to Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Part A & B: Double Blind Period: Placebo | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 16 | 30.77 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 16 | 66.67 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 32 | 66.67 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 16 | 75.00 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 32 | 75.00 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 32 | 90.91 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 16 | 90.91 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 16 | 75.00 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 32 | 75.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 16 | 50.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 32 | 50.00 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 32 | 25.00 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 | Week 16 | 25.00 percentage of participants |
p-value: =0.1295% CI: [-7.22, 67.75]Barnard's test
p-value: =0.0695% CI: [-0.83, 73.77]Barnard's test
p-value: =0.00395% CI: [21.33, 82.42]Barnard's test
Secondary
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
Population: The Full Analysis Set consisted of all randomized participants who had received at least 1 dose of study drug. Number analyzed indicates the number of participants with data available for analyses at the specified time point. All participants in the 'Part A \& B: Double Blind Period: Placebo' arm either transitioned to OLE Period or discontinued from the study prior to Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Part A & B: Double Blind Period: Placebo | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 16 | 0 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 16 | 55.56 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 32 | 55.56 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 16 | 50.00 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 32 | 50.00 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 32 | 81.82 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 16 | 81.82 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 16 | 0 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 32 | 25.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 16 | 25.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 32 | 25.00 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 32 | 25.00 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 | Week 16 | 25.00 percentage of participants |
p-value: =0.00195% CI: [25.11, 81.51]Barnard's test
p-value: =0.00495% CI: [19.63, 78.95]Barnard's test
p-value: <0.00195% CI: [51.24, 94.99]Barnard's test
Secondary
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
Population: The Full Analysis Set included all randomized participants who had received at least 1 dose of study drug. Overall number of participants indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses at the specified time point. All participants in the 'Part A \& B: Double Blind Period: Placebo' arm either transitioned to OLE Period or discontinued from the study prior to Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Part A & B: Double Blind Period: Placebo | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 16 | 0 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 32 | 40.00 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 16 | 40.00 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 32 | 25.00 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 16 | 25.00 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 16 | 100.00 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 32 | 100.00 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 32 | 100.00 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 16 | 100.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 32 | 100.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 16 | 100.00 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 32 | 0 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 | Week 16 | 0 percentage of participants |
p-value: =0.18795% CI: [-16.28, 78.17]Barnard's test
p-value: =0.31595% CI: [-28.85, 71.78]Barnard's test
p-value: =0.00495% CI: [35.12, 100]Barnard's test
Secondary
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
Population: The Full Analysis Set included all randomized participants who had received at least 1 dose of study drug. Number analyzed indicates the number of participants with data available for analyses at the specified time point. All participants in the 'Part A \& B: Double Blind Period: Placebo' arm either transitioned to OLE Period or discontinued from the study prior to Week 32.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Part A & B: Double Blind Period: Placebo | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 16 | 23.08 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 16 | 66.67 percentage of participants |
| Part A: Double Blind Period: TAK-079 100 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 32 | 66.67 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 16 | 62.50 percentage of participants |
| Part A: Double Blind Period: TAK-079 300 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 32 | 62.50 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 32 | 90.91 percentage of participants |
| Part B: Double Blind Period: TAK-079 600 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 16 | 90.91 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 16 | 50.00 percentage of participants |
| Part A: Open-label Extension (OLE) Period: TAK-079 100 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 32 | 50.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 16 | 50.00 percentage of participants |
| Part A: OLE Period: TAK-079 300 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 32 | 50.00 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 32 | 25.00 percentage of participants |
| Part B: OLE Period: TAK-079 600 mg | Percentage of Participants With Platelet Response at Weeks 16 and 32 | Week 16 | 25.00 percentage of participants |
p-value: =0.05695% CI: [0.81, 73.35]Barnard's test
p-value: =0.08895% CI: [-4.24, 71.38]Barnard's test
p-value: =0.00195% CI: [29.21, 87.29]Barnard's test