The primary objective of this study was to assess the efficacy of 3 dose levels of lusutrombopag (0.5 mg, 0.75 mg, and 1.0 mg) and placebo on platelet count.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
20
Tablet
Tablet
Investigator
Anaheim, California, United States
Investigator
Los Angeles, California, United States
Percentage of Participants With a Response
Responders were participants with one of the following: 1. achieved a platelet count of ≥ 50,000 cells/µL after 6 weeks of dosing; or 2. prematurely withdrawn due to a platelet count \> 400,000 cells/µL prior to Day 42. Participants were counted as non-responders if any of the following conditions held: * The above conditions were not satisfied; * They received rescue medications; * They satisfied the above conditions after receiving restricted medications during the treatment period; * They had achieved a platelet count of ≥ 50,000 cells/µL before Week 6 but not after Week 6; or * They withdrew for any reason other than a platelet count \> 400,000 cells/µL.
Time frame: Week 6
Change From Baseline in Platelet Count at Week 6
Time frame: Baseline and Week 6
Duration of Response
Duration of response was defined as the percentage of the cumulative time a platelet count was ≥ 50,000 cells/µL during the treatment period.
Time frame: 6 weeks
Percentage of Participants Who Achieved a Platelet Count of ≥ 30,000 Cells/µL and Doubled the Baseline Platelet Count After 6 Weeks of Dosing
Time frame: Week 6
Percentage of Participants Who Achieved a Platelet Count of ≥ 50,000 Cells/µL and Doubled the Baseline Platelet Count After 6 Weeks of Dosing
Time frame: Week 6
Number of Participants With Worst Severity of Bleeding Associated With ITP During the Treatment Period,
Bleeding assessments were performed by the Investigator according to the World Health Organization (WHO) criteria bleeding scale: Grade 0: no bleeding; Grade 1: petechial bleeding; Grade 2: mild blood loss (clinically significant); Grade 3: gross blood loss, requires transfusion (severe); Grade 4: debilitating blood loss, retinal or cerebral associated with fatality. For each participant, the most severe WHO bleeding grade observed during the 6-week treatment period is reported.
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Investigator
Washington D.C., District of Columbia, United States
Investigator
Boynton Beach, Florida, United States
Investigator
Jacksonville, Florida, United States
Investigator
Atlanta, Georgia, United States
Investigator
Riverdale, Georgia, United States
Investigator
Metairie, Louisiana, United States
Investigator
Bethesda, Maryland, United States
Investigator
Boston, Massachusetts, United States
...and 9 more locations
Time frame: 6 weeks
Number of Participants Who Received Rescue Medication During the Treatment Period
Time frame: 6 weeks
Number of Participants With Adverse Events (AEs)
An AE is defined as any untoward medical occurrence in a subject administered a pharmaceutical product during the course of a clinical investigation, including any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product (IP), whether or not thought to be related to the IP. AEs reported after initial study drug administration were considered treatment-emergent. A serious adverse event is defined as any AE that resulted in death, was life-threatening, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect or an important medical event that, based upon medical judgment, may jeopardize the participant or require medical or surgical intervention to prevent one of the outcomes listed above. A treatment-related AE is any AE determined by the investigator to be possibly related, probably related, or definitely related to study drug.
Time frame: 6 weeks
Lusutrombopag Plasma Concentration
Plasma concentrations of lusutrombopag were determined using a validated liquid chromatography mass spectrometry method. The lower limit of quantification (LOQ) for the plasma assay for lusutrombopag was 0.1 ng/mL.
Time frame: Days 8, 22, and 36, after dosing
Plasma Concentration of Metabolite S-888711 Deshexyl
Plasma concentrations of the major metabolite S-888711 deshexyl were determined using a validated liquid chromatography mass spectrometry method. The lower limit of quantification (LOQ) for the plasma assay for S-888711 deshexyl was 0.1 ng/mL.
Time frame: Days 8, 22, and 36, after dosing