Idiopathic Thrombocytopenic Purpura
Conditions
Keywords
Idiopathic Thrombocytopenic Purpura, ITP, Chronic Idiopathic Thrombocytopenic Purpura
Brief summary
The purpose of this study is to determine the safety and efficacy of AKR-501 (avatrombopag) administered in participants with chronic Idiopathic Thrombocytopenic Purpura (ITP) who were enrolled into and completed 28 days of study treatment in Protocol 501-CL-003 (NCT00441090).
Detailed description
Participants eligible to enroll into this rollover protocol will begin study treatment within 2-5 days of their Day 28 study termination visit in Protocol 501-CL-003 (NCT00441090). Participants who met the primary efficacy response criterion in Protocol 501-CL-003 will continue receiving the same study treatment to which they were assigned in the previous protocol in a double-blinded manner, these being one of the following 5 treatments: * avatrombopag 2.5 mg daily * avatrombopag 5 mg daily * avatrombopag 10 mg daily * avatrombopag 20 mg daily * placebo Participants who did not meet the primary efficacy response criterion in Protocol 501-CL-003 who otherwise meet the eligibility criteria for this rollover protocol will be offered open label avatrombopag 10 mg daily. This is a parallel group, rollover study.
Interventions
DRUGBlinded (placebo)
Placebo Orally, once daily administered under fasting conditions (at least 1 hr prior to or at least 2 hours after a meal or snack) Duration - 6 months
DRUGOpen Label (Avatrombopag tablets)
Dose 10 mg Orally, once daily administered under fasting conditions (at least 1 hr prior to or at least 2 hours after a meal or snack) Duration - 6 months
DRUGBlinded (Avatrombopoag tablets)
Dose: 2.5, 5, 10, or 20 mg Orally, once daily administered under fasting conditions (at least 1 hr prior to or at least 2 hours after a meal or snack) Duration - 6 months
Sponsors
Eisai Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patients who completed 28 days of study treatment in Protocol 501-CL-003. 2. No significant safety or tolerability concerns from the patient's participation of Protocol 501-CL-003 as determined by the Investigator. 3. Received medical monitor approval for enrollment into this study. 4. Patients receiving maintenance corticosteroids may be enrolled, as long as the corticosteroids have been administered at a stable dose and the Investigator does not foresee the need to change the steroid dose during study participation. Patients should remain on this stable corticosteroid dose during study participation. 5. Women of child-bearing potential must have a negative serum pregnancy test at the Day 28 assessment in Protocol 501-CL-003. (Childbearing potential is defined as any woman who has not been surgically sterilized and is pre-menopausal or peri-menopausal i.e., any menstrual flow within 12 months of Screening Visit A for Protocol 501-CL-003). 6. Women of child-bearing potential must agree to practice a medically approved form of contraception (one of the following must be used: condoms (male or female) with a spermicidal agent, diaphragm or cervical cap with a spermicidal agent, IUD,hormonal contraception, abstinence). 7. Willing and able to provide written informed consent.
Exclusion criteria
1. Women who are pregnant and/or lactating. 2. Use of the following drugs or treatments: * Rituximab * Azathioprine, Cyclosporine A, or other immunosuppressant therapy * Aspirin, Aspirin-containing compounds, Salicylates,Anticoagulants, Non-steroidal anti-inflammatory drugs(NSAIDs)(including Cyclooxygenase-2 \[COX-2\] specific NSAIDs), clopidogrel; ticlopidine; and any drugs that affect platelet function. * Danazol * Rh0(D) immune globulin (WinRho®) or intravenous immunoglobulin (IVIG). 3. Inability to comply with protocol requirements or give informed consent, as determined by the Investigator. For more information regarding inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment. | A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of study drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. Related AEs included those whose relationship was categorized as possible or probably by the investigator. Dose interruption includes dose decreased, dose previously stopped, permanently stopped, or temporarily stopped. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag). |
| Incidence of Severe (Grade 3 or 4) TEAEs | Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment. | A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag). |
| Incidence of Drug-Related TEAEs | Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment. | Drug-related TEAEs included those whose relationship was categorized as possible or probably by the investigator. A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4 | Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly. |
| Percentage of Participants Who Maintained Response-Level Platelet Count | Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4 | Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly. |
| Median Platelet Counts at Selected Analysis Timepoints | Day 1, Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4 | Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly. |
| Number of Participants With Changes in Concomitant Steroid Use | Day 1 through last 8 weeks of the Treatment Period | A participant who used steroids at study entry was considered to have permanently discontinued steroid use if they had no steroid use during the last 8 weeks of the study Treatment Period. A participant who used steroids at study entry was considered to have decreased concomitant steroid medication by at least 50% if they permanently discontinued steroids, or in no dose of steroid was higher than 50% of their baseline steroid dose during the last 8 weeks of the study Treatment Period. |
| Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response | Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4 | Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly. |
| Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4 | Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly. |
| Percentage of Participants Who Maintained a Platelet Count of 100,000/mm^3 or Higher by Response Status | Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4 | Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly. |
Countries
United States
Participant flow
Pre-assignment details
After Protocol Amendment 4, this study implemented a flexible dose regimen and was considered to have an open-label, uncontrolled, single-arm design. Due to these limitations in study design, a single grouping method with 3 subgroups was implemented for reporting participant disposition.
Participants by arm
| Arm | Count |
|---|---|
| Lower 1/3 Avatrombopag Dose Group Participants in this group took less than 8.85 mg avatrombopag orally once daily under fasting conditions (at least 1 hour before and 2 hours after a meal or snack). Grouping of avatrombopag dose into lower 1/3, middle 1/3, and upper 1/3 was based on the average daily dose received during the combined active treatment periods of studies 501-CL-003 and 501-CL-004. | 12 |
| Middle 1/3 Avatrombopag Dose Group Participants in this group took greater than or equal to 8.85 mg avatrombopag to less than 13.5 mg avatrombopag orally once daily under fasting conditions (at least 1 hour before and 2 hours after a meal or snack). Grouping of avatrombopag dose into lower 1/3, middle 1/3, and upper 1/3 was based on the average daily dose received during the combined active treatment periods of studies 501-CL-003 and 501-CL-004. | 26 |
| Upper 1/3 Avatrombopag Dose Group Participants in this group took greater than or equal to 13.5 mg avatrombopag orally once daily under fasting conditions (at least 1 hour before and 2 hours after a meal or snack). Grouping of avatrombopag dose into lower 1/3, middle 1/3, and upper 1/3 was based on the average daily dose received during the combined active treatment periods of studies 501-CL-003 and 501-CL-004. | 15 |
| Total | 53 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 1 | 4 | 0 |
| Overall Study | Non-compliance with protocol | 0 | 1 | 0 |
| Overall Study | Other | 0 | 1 | 1 |
| Overall Study | Physician Decision | 0 | 4 | 2 |
| Overall Study | Platelet count increase >=500,000/mm3 | 1 | 0 | 0 |
| Overall Study | Withdrawal by Subject | 0 | 1 | 2 |
Baseline characteristics
| Characteristic | Lower 1/3 Avatrombopag Dose Group | Middle 1/3 Avatrombopag Dose Group | Upper 1/3 Avatrombopag Dose Group | Total |
|---|---|---|---|---|
| Age, Continuous | 44.7 Years STANDARD_DEVIATION 21.09 | 52.5 Years STANDARD_DEVIATION 18.44 | 50.4 Years STANDARD_DEVIATION 15.65 | 50.1 Years STANDARD_DEVIATION 18.25 |
| Sex: Female, Male Female | 7 Participants | 20 Participants | 11 Participants | 38 Participants |
| Sex: Female, Male Male | 5 Participants | 6 Participants | 4 Participants | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 12 | 0 / 26 | 0 / 15 |
| other Total, other adverse events | 12 / 12 | 24 / 26 | 14 / 15 |
| serious Total, serious adverse events | 2 / 12 | 4 / 26 | 3 / 15 |
Outcome results
Primary
Incidence of Drug-Related TEAEs
Drug-related TEAEs included those whose relationship was categorized as possible or probably by the investigator. A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).
Time frame: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
Population: Safety population included all participants who received at least one dose of study drug and had at least one post-treatment safety assessment. The term post-treatment refers to any assessment timepoint after the participant received at least one dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Fatigue | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Headache | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Diarrhoea | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Vomiting | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Abdominal pain upper | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Hypoaesthesia | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Vision blurred | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Mood swings | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Haematuria | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Menorrhagia | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Dyspnoea | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Leukocytosis | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Anaemia | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Thrombocytopenia | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Splenomegaly | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Visual disturbance | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Arthropathy | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Epistaxis | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Pain in extremity | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Oedema peripheral | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Nausea | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Platelet count increased | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Rash | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Back pain | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Muscular weakness | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Decreased appetite | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Alanine aminotransferase increased | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Hyperlipidaemia | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Dizziness | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Platelet count decreased | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Arthralgia | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Nasal congestion | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Platelet count decreased | 2 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Headache | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Thrombocytopenia | 2 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Splenomegaly | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Vision blurred | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Nausea | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Diarrhoea | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Vomiting | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Fatigue | 4 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Oedema peripheral | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Platelet count increased | 2 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Decreased appetite | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Arthralgia | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Arthropathy | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Muscular weakness | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Anaemia | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Leukocytosis | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Visual disturbance | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Abdominal pain upper | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Alanine aminotransferase increased | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Hyperlipidaemia | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Back pain | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Pain in extremity | 2 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Mood swings | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Dizziness | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Hypoaesthesia | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Haematuria | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Menorrhagia | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Dyspnoea | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Epistaxis | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Nasal congestion | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Rash | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Alanine aminotransferase increased | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Hyperlipidaemia | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Rash | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Platelet count decreased | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Arthralgia | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Platelet count increased | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Menorrhagia | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Fatigue | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Nasal congestion | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Pain in extremity | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Vomiting | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Visual disturbance | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Diarrhoea | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Headache | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Nausea | 2 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Thrombocytopenia | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Mood swings | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Splenomegaly | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Anaemia | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Dyspnoea | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Dizziness | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Leukocytosis | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Hypoaesthesia | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Epistaxis | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Muscular weakness | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Vision blurred | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Abdominal pain upper | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Arthropathy | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Back pain | 2 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Oedema peripheral | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Decreased appetite | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Drug-Related TEAEs | Haematuria | 0 Participants |
Primary
Incidence of Severe (Grade 3 or 4) TEAEs
A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).
Time frame: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
Population: Safety population included all participants who received at least one dose of study drug and had at least one post-treatment safety assessment. The term post-treatment refers to any assessment timepoint after the participant received at least one dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Thrombocytopenia | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Leukocytosis | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Fatigue | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Chest pain | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Platelet count increased | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Alanine aminotransferase increased | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Back pain | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Epistaxis | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Splenomegaly | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Haemorrhagic diathesis | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Idiopathic thrombocytopenic purpura | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Diarrhoea | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Mitral valve incompetence | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Vomiting | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Oedema peripheral | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Asthenia | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Pyrexia | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Platelet count decreased | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Aspartate aminotransferase increased | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hyperuricaemia | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Dizziness | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hypoaesthesia | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Cerebrovascular accident | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Haemorrhage intercranial | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hypotension | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Pelvic venous thrombosis | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Haemorrhagic diathesis | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Platelet count increased | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Pelvic venous thrombosis | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Thrombocytopenia | 2 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Splenomegaly | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hypoaesthesia | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Platelet count decreased | 2 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hypotension | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Fatigue | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Haemorrhage intercranial | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Oedema peripheral | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Dizziness | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Idiopathic thrombocytopenic purpura | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Asthenia | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Leukocytosis | 1 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Mitral valve incompetence | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Diarrhoea | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Vomiting | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Chest pain | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Epistaxis | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Pyrexia | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Alanine aminotransferase increased | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Aspartate aminotransferase increased | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hyperuricaemia | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Back pain | 0 Participants |
| Middle 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Cerebrovascular accident | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Chest pain | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hyperuricaemia | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Epistaxis | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Platelet count decreased | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Pelvic venous thrombosis | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Dizziness | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Pyrexia | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Thrombocytopenia | 4 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Platelet count increased | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Cerebrovascular accident | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Splenomegaly | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Mitral valve incompetence | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Haemorrhage intercranial | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Haemorrhagic diathesis | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Alanine aminotransferase increased | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Idiopathic thrombocytopenic purpura | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Leukocytosis | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hypotension | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Aspartate aminotransferase increased | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Diarrhoea | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Vomiting | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Back pain | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Fatigue | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Hypoaesthesia | 1 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Asthenia | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Incidence of Severe (Grade 3 or 4) TEAEs | Oedema peripheral | 0 Participants |
Primary
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of study drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. Related AEs included those whose relationship was categorized as possible or probably by the investigator. Dose interruption includes dose decreased, dose previously stopped, permanently stopped, or temporarily stopped. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).
Time frame: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
Population: Safety population included all participants who received at least one dose of study drug and had at least one post-treatment safety assessment. The term post-treatment refers to any assessment timepoint after the participant received at least one dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Treatment-related TEAEs | 5 Participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Severe (Grade 3-4) TEAEs | 4 Participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Dose interruption due to TEAE | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Serious TEAEs | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Withdrawal of study drug due to TEAE | 2 Participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Deaths | 0 Participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Serious treatment-related TEAEs | 1 Participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | TEAEs | 12 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Dose interruption due to TEAE | 3 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Withdrawal of study drug due to TEAE | 4 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | TEAEs | 24 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Severe (Grade 3-4) TEAEs | 7 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Treatment-related TEAEs | 15 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Serious TEAEs | 4 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Serious treatment-related TEAEs | 2 Participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Deaths | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Serious TEAEs | 3 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Deaths | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Withdrawal of study drug due to TEAE | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Serious treatment-related TEAEs | 0 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | TEAEs | 14 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Treatment-related TEAEs | 7 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Severe (Grade 3-4) TEAEs | 6 Participants |
| Upper 1/3 Avatrombopag Dose Group | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Dose interruption due to TEAE | 2 Participants |
Secondary
Median Platelet Counts at Selected Analysis Timepoints
Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly.
Time frame: Day 1, Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
Population: Full analysis set (FAS) included all participants who completed 501-CL-003 and received study drug in the current study, except baseline values and ITP-directed medications taken during the 2-week period before the first dose of avatrombopag in the current study. Data was summarized using the observed case (OC) method.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 2 | 126.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 18 | 119.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 10 | 85.5 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 20 | 92.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 6 | 104.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 22 | 109.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 12 | 88.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 24 | 154.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 4 | 113.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 1 | 59.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 14 | 84.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 2 | 25.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 8 | 137.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 3 | 27.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 16 | 143.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 4 | 35.0 Platelets x 1000/mm^3 |
| Lower 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Day 1 | 55.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 4 | 28.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Day 1 | 23.5 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 2 | 54.5 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 4 | 38.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 6 | 47.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 8 | 45.5 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 10 | 72.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 12 | 72.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 14 | 77.5 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 16 | 46.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 18 | 78.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 20 | 68.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 22 | 78.5 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Week 24 | 66.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 1 | 40.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 2 | 22.0 Platelets x 1000/mm^3 |
| Middle 1/3 Avatrombopag Dose Group | Median Platelet Counts at Selected Analysis Timepoints | Follow-up Week 3 | 36.0 Platelets x 1000/mm^3 |
Secondary
Number of Participants With Changes in Concomitant Steroid Use
A participant who used steroids at study entry was considered to have permanently discontinued steroid use if they had no steroid use during the last 8 weeks of the study Treatment Period. A participant who used steroids at study entry was considered to have decreased concomitant steroid medication by at least 50% if they permanently discontinued steroids, or in no dose of steroid was higher than 50% of their baseline steroid dose during the last 8 weeks of the study Treatment Period.
Time frame: Day 1 through last 8 weeks of the Treatment Period
Population: FAS. Data was summarized using the OC method. Only those participants who had data available at both Baseline and post-Baseline were analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Changes in Concomitant Steroid Use | Permanently Discontinued | 4 Number of participants |
| Lower 1/3 Avatrombopag Dose Group | Number of Participants With Changes in Concomitant Steroid Use | Decreased by greater than or equal to 50% | 7 Number of participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Changes in Concomitant Steroid Use | Permanently Discontinued | 4 Number of participants |
| Middle 1/3 Avatrombopag Dose Group | Number of Participants With Changes in Concomitant Steroid Use | Decreased by greater than or equal to 50% | 6 Number of participants |
Secondary
Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response
Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly.
Time frame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
Population: FAS. Data was summarized using the OC method.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response | Durable Platelet Response | 72.0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response | Transient Platelet Response | 16.0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response | Overall Response | 88.0 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response | Durable Platelet Response | 35.7 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response | Transient Platelet Response | 28.6 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response | Overall Response | 64.3 Percentage of participants |
Secondary
Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit
Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly.
Time frame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
Population: FAS. Data was summarized using the OC method.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 4 | 58.3 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 8 | 52.4 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 12 | 42.9 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 16 | 63.6 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 20 | 40.0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 24 | 71.4 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 1 | 18.2 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 2 | 0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 3 | 25.0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 4 | 23.1 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 2 | 59.1 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 2 | 25.0 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 1 | 27.3 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 8 | 15.4 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 4 | 13.3 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 12 | 35.0 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 2 | 33.3 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 16 | 17.6 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 4 | 18.5 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 20 | 25.0 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Follow up Week 3 | 11.1 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit | Week 24 | 35.3 Percentage of participants |
Secondary
Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit
Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly.
Time frame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
Population: FAS. Data was summarized using the OC method.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 12 | 85.7 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 24 | 81.0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 8 | 90.5 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 1 | 54.5 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 16 | 81.8 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 2 | 10.0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 4 | 79.2 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 3 | 41.7 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 20 | 90.0 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 4 | 38.5 Percentage of participants |
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 2 | 86.4 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 4 | 33.3 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 2 | 53.6 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 4 | 44.4 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 8 | 46.2 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 12 | 65.0 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 16 | 47.1 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 20 | 66.7 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Week 24 | 70.6 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 1 | 45.5 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 2 | 33.3 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit | Follow up Week 3 | 44.4 Percentage of participants |
Secondary
Percentage of Participants Who Maintained a Platelet Count of 100,000/mm^3 or Higher by Response Status
Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly.
Time frame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
Population: FAS. Data was summarized using the OC method.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Maintained a Platelet Count of 100,000/mm^3 or Higher by Response Status | 32.0 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Maintained a Platelet Count of 100,000/mm^3 or Higher by Response Status | 0 Percentage of participants |
Secondary
Percentage of Participants Who Maintained Response-Level Platelet Count
Platelet count (PC) was measured from blood draws. Based on avatrombopag response in study 501-CL-003, participants were divided into two subgroups; 1) Responders, participants who met the primary efficacy response; pretreatment PC was less than 30,000/mm\^3 and increased to greater than or equal to 50,000/mm\^3 after treatment with avatrombopag, and for participants on steroids, pretreatment PC was greater than or equal to 30,000/mm\^3 but less than 50,000/mm\^3 and increased to a PC greater than or equal to 20,000/mm\^3 above their pretreatment values and 2) Nonresponders, participants who did not meet the primary efficacy response and those who were in the placebo arm. Open-label dose escalation of avatrombopag, as well as reductions in ITP-directed concomitant therapy (e.g. steroids) was allowed for all participants. During the periods of avatrombopag dose escalation and/or ITP-directed concomitant medication reduction, platelet sampling was done weekly.
Time frame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
Population: FAS. Data was summarized using the OC method.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Lower 1/3 Avatrombopag Dose Group | Percentage of Participants Who Maintained Response-Level Platelet Count | 56.0 Percentage of participants |
| Middle 1/3 Avatrombopag Dose Group | Percentage of Participants Who Maintained Response-Level Platelet Count | 0 Percentage of participants |