Table 2.

Clinical trials undergoing for avatrombopag (E5501, AKR 501, YM477) for the treatment of various thrombocytopenia (www.clinicaltrials.gov).

Sr. No	Trial identifier (number)	Phase of the study	Trial design	Intervention/treatment	Primary endpoints expected outcome	Status
1	NCT03326843	Phase III	Open-label study to evaluate the efficacy and safety of avatrombopag for the treatment of subjects with thrombocytopenia scheduled for a surgical procedure	Avatrombopag 60 mg	Proportion of subjects who achieve a platelet count >100 × 109/L on procedure day	Terminated
2	NCT03471078	Phase III	Randomized, double-blind, placebo-controlled study with an open-label extension to evaluate the efficacy and safety of avatrombopag for the treatment of chemotherapy-induced thrombocytopenia in subjects with active nonhematological cancers	Avatrombopag Placebo oral tablet	Proportion of subjects who do not require platelet transfusion, dose reduction in chemotherapy by 15%, or chemotherapy delay by ⩾4 days	Recruiting
3	NCT03554759	Phase IV	An observation, cohort study of the use of avatrombopag in patients with thrombocytopenia associated with CLD undergoing a procedure	Avatrombopag	Change in platelet count up to 8 days after the last dose of avatrombopag	Terminated
4	NCT02227693	Phase II	Randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy, safety, and pharmacokinetics of once-daily oral avatrombopag in Japanese subjects with CLDs and thrombocytopenia	Avatrombopag Placebo	Proportion of subjects who had platelet count ⩾50 × 109/L and at least 20 × 109/L increase from baseline at visit 4	Completed
5	NCT01976104 and NCT01972529	Phase III	Randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of once-daily oral avatrombopag for the treatment of adults with thrombocytopenia associated with liver disease prior to an elective procedure	Avatrombopag Placebo	Percentage of participants who did not require a platelet transfusion up to 7 days following a scheduled procedure	Completed
6	NCT01438840	Phase III	Randomized, double-blind, placebo-controlled, parallel-group trial with an open-label extension phase to evaluate the efficacy and safety of oral E5501 plus standard of care for the treatment of thrombocytopenia in adults with CITP/idiopathic thrombocytopenic purpura	Avatrombopag Placebo Standard of care	Number of weeks with platelet count greater ⩾50 × 109/L during 6-month treatment period	Completed
7	NCT00914927	Phase II	Randomized, placebo-controlled, double-blind, parallel-group study to evaluate the efficacy, safety, and population PKs of once-daily oral E5501 tablets used up to 7 days in subjects with CLD and thrombocytopenia prior to elective surgical or diagnostic procedures	Avatrombopag Placebo	Percentage of participants experiencing response, ie, a participant having an increase of at least 20 000/mm3 platelet count from baseline and a platelet count >50 000/mm3 at least once during day 4 through day 8	Completed
8	NCT02039076	Phase I	Randomized, open-label, 5-treatment-period study to evaluate the PK and PD of avatrombopag following a single-dose administration of avatrombopag in the fed and fasted condition to healthy Japanese and white subjects	Avatrombopag	Primary outcome: PK profiles of avatrombopag—Cmax, AUC, and t1/2	Completed
9	NCT01355289	Phase II	Randomized, placebo-controlled, double-blind, parallel-group study, with an open-label extension, to evaluate the efficacy, safety, and pharmacokinetics of E5501 in subjects with chronic hepatitis C virus–related thrombocytopenia who are potential candidates for antiviral treatment	Avatrombopag Placebo PEG-IFN Telaprevir Ribavirin	Number of participants who achieved platelet response ⩾100 × 109/L) by day 21 of treatment period	Completed
10	NCT01433978	Phase III	Randomized, double-blind, active-controlled, parallel-group trial with an open-label extension phase to evaluate the efficacy and safety of oral E5501 vs eltrombopag, in adults with CITP	Eltrombopag Avatrombopag Standard of care	Change from baseline in local platelet count for the 6-month treatment period (day 5, day 8, weeks 2, 3, 4, 6, 8, 10, 12, 14, 16, 18, 19, 20, 22, 23, 24, 25, 26)	Terminated
11	NCT00441090	Phase II	Randomized, double-blind, placebo-controlled, dose-ranging, parallel-group study of AKR-501 tablets taken orally once daily for 28 days in patients with ITP	Placebo Avatrombopag tablets	Response rate to avatrombopag on day 28	Completed
12	NCT00625443	Phase II	This is a nonrandomized study used to determine the safety and efficacy of AKR-501 administered in participants with chronic ITP who were enrolled into and completed 28 days of study treatment in Protocol 501-CL-003 (NCT00441090)	Placebo Avatrombopag tablets	Number of participants with treatment-emergent adverse events—from day 1 to 6 months while receiving the treatment and 7 months after discontinuation of treatment Incidence of severe (Grade 3 or 4) treatment-emergent adverse events—from day 1 to 6 months while receiving the treatment and 7 months after discontinuation of treatment Incidence of drug-related treatment-emergent adverse events—from day 1 to 6 months while receiving the treatment and 7 months after discontinuation of treatment	Completed

Abbreviations: CITP indicates chronic immune thrombocytopenia; CLD, chronic liver disease; ITP, immune thrombocytopenia; PD, pharmacodynamics; PEG-IFN, pegylated interferon; PK, pharmacokinetics.