Efficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults With Chronic Immune Thrombocytopenia (Amendment 02)
To demonstrate that the efficacy of avatrombopag (in addition to standard of care) is superior to placebo (in addition to standard of care) for the treatment of adult participants with chronic immune thrombocytopenia (idiopathic thrombocytopenic purpura) (ITP) as measured by cumulative number of weeks of platelet response over 6 months of once daily treatment in adults participants who received at least 1 prior ITP therapy.
To evaluate the safety and tolerability of long-term therapy with avatrombopag in participants with chronic ITP (cITP).
Drug: Standard of care
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
|Official Title:||A Phase 3, Multicenter, 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 Chronic Immune Thrombocytopenia (Idiopathic Thrombocytopenia Purpura)|
- Cumulative Number Of Weeks Of Platelet Response Over 6 Months of Treatment [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Proportion of Participants with a Platelet Response At Day 8 [ Time Frame: 8 days ] [ Designated as safety issue: No ]Platelet response rate at Day 8 defined by the proportion of participants with a platelet response >= 50 X 10^9/L (liter) at Day 8. Subjects with missing platelet counts at Day 8 or use of a rescue therapy before or on Day 8 will be considered platelet nonresponders.
|Study Start Date:||February 2012|
|Study Completion Date:||March 2014|
|Primary Completion Date:||November 2013 (Final data collection date for primary outcome measure)|
Active Comparator: Avatrombopag (Core Study)
Avatrombopag was administered orally as 5 mg, 10 mg, 20 mg, 30 mg, or 40 mg in a flexible dose design for 26 weeks. Participants received blinded therapy at a starting dose of 20 mg avatrombopag, one daily and they were allowed to have their dose titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on their response to study drug.
Other Names:Drug: Standard of care
Placebo Comparator: Placebo (Core Study)
Placebo was administered as 5 mg, 10 mg, 20 mg, 30 mg or 40 mg in a flexible dose design for 26 weeks. Placebo was administered orally at a starting dose of 20 mg, once daily. Afterwards the dose could be titrated up (maximum dose of 40 mg avatrombopag) or down (minimum dose of 5 mg avatrombopag) depending on the participant's response to the study drug; placebo titration was used to maintain the blind.
|Drug: Placebo Drug: Standard of care|
Experimental: Avatrombopag (Open-Label Extension)
Participants who met all eligibility criteria requirements of extension phase and who discontinued the core study because of lack of treatment effect continued into the extension phase. Avatrombopag was administered to participants who entered extension phase, with a starting dose of 20 mg avatrombopag, once daily for 76 weeks and underwent dose titration.
Other Names:Drug: Standard of care
Please refer to this study by its ClinicalTrials.gov identifier: NCT01438840
|Adelaide, Australia, ����|
|Bedford Park, Australia|
|Antwerpen, Belgium, ����|
|Plovdiv, Bulgaria, ����|
|Sofia, Bulgaria, ����|
|Brno, Czech Republic, ����|
|Hradec Kralove, Czech Republic|
|Praha, Czech Republic|
|Praha, Czech Republic, ����|
|Rotterdam, Netherlands, ����|
|Christchurch, New Zealand|
|Palmerston North, New Zealand, ����|
|Chorzow, Poland, ����|
|Krakow, Poland, ����|
|Wroclaw, Poland, ����|
|Singapore, Singapore, ����|
|Johannesburg, South Africa, ����|
|Johannesburg, South Africa|
|Dnipropetrovsk, Ukraine, ����|
|Ivano-Frankivsk, Ukraine, ����|
|Lviv, Ukraine, ����|
|Study Director:||Joe McIntosh||Eisai Inc.|