The purpose of this study is to determine the efficacy, safety and tolerability, of AKR-501 (avatrombopag) tablets, as compared to placebo, in the treatment of participants with chronic Idiopathic Thrombocytopenic Purpura (ITP).
This is a Phase 2, multi-center, double-blind, randomized, placebo-controlled, dose-ranging, parallel-group study. The pharmacokinetic (PK) and pharmacokinetic/pharmacodynamic (PK/PD) relationship of avatrombopag will also be studied. Approximately 65 eligible participants will be randomized in a 3:3:3:3:1 ratio in a double-blinded fashion into one of five parallel treatment groups to receive daily doses of either avatrombopag 2.5, 5, 10 or 20 mg or placebo for 28 days, respectively. Each avatrombopag dosing group will consist of 15 participants while the placebo group will consist of 5 participants. All study participants will be evaluated weekly (Days 3, 5, 7, 14, 21 and 28) for safety, efficacy, and (Days 7, 14, 21, and 28) avatrombopag PK while receiving study treatment with a final assessment for safety and effectiveness to be done 2 weeks after the last study dose (Day 42). At the completion of Visit Day 28±1, participants who complete 28±1 days of study dosing will be assessed for eligibility to enroll into the rollover Study 501-CL-004 (NCT00625443) based on this visit.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
64
Placebo tablets 2.5, 5, 10 and 20 mg taken orally once daily for 28 days.
Avatrombopag tablets 2.5, 5, 10 and 20 mg taken orally once daily for 28 days.
Responder Rate (RR) to Avatrombopag on Day 28
Platelet counts were measured from the participant's blood, which was drawn at their Screening Visit B (Day -4 to Day 1, Baseline), and on study drug treatment Day 28. The RR was defined as the percentage of participants with a Day 1 platelet count of less than 30,000/milliliter (mL) who reached a platelet count of greater than or equal to 50,000/mL on Day 28 of study medication, together with the percentage of participants using steroids who had a Day 1 platelet count greater than or equal to 30,000/mL but less than 50,000/mL who reached a platelet count of greater than or equal to 20,000/mL higher than their Day 1 platelet count on Day 28 of study medication. The RR was summarized by treatment group using the method of last observation carried forward (LOCF).
Time frame: Day-4 to Day 1, Baseline, Day 28
Change in Platelet Count From Baseline
Platelet counts were measured from the participant's, blood which was drawn at their Screening Visit B (Day -4 to Day 1, Baseline), and on study drug treatment Days 7, 14, 21, and 28. The unit of measure was "K/mm\^3", where "K = platelets x 1000 = platelets x 10\^3" and "mm\^3 = cubic milliliter= microliter".
Time frame: Day -4 to Day 1, Baseline, Day 7, Day 14, Day 21, Day 28
Responder Rate to Avatrombopag by Visit
Platelet counts were measured from the participant's blood, which was drawn at their Screening Visit B (Day -4 to Day 1, Baseline), and on study drug treatment Days 7, 14, and 21. The RR was summarized by treatment group using the method of LOCF. Day 28 was not included with this data because it was reported as a primary outcome measure.
Time frame: Day -4 to Day 1, Baseline, Day 7, Day 14, and Day 21
Percentage of Participants With Platelet Counts Greater Than or Equal to 50,000/mL by Visit
Platelet counts were measured from the participant's blood, which was drawn at their Screening Visit B (Day -4 to Day 1, Baseline), and on study drug treatment Days 7, 14, 21, and 28.
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Time frame: Day -4 to Day 1, Baseline, Day 7, Day 14, Day 21, and Day 28
Percentage of Participants With Platelet Counts Greater Than or Equal to 100,000/mL by Visit
Platelet counts were measured from the participant's, blood which was drawn at their Screening Visit B (Day -4 to Day 1, Baseline), and on study drug treatment Days 7, 14, 21, and 28.
Time frame: Day -4 to Day 1, Baseline, Day 7, Day 14, Day 21, and Day 28
Percentage of Participants Whose Platelet Counts Doubled From Baseline by Visit
Platelet counts were measured from the participant's, blood which was drawn at their Screening Visit B (Day -4 to Day 1, Baseline), and on study drug treatment Days 7, 14, 21, and 28.
Time frame: Day -4 to Day 1, Baseline, Day 7, Day 14, Day 21, and Day 28