No longer a GSK study

Study Description:

This product has been transferred to Novartis. GSK Clinical Study Register is no longer maintained for this study. The most up to date information is available on clinicaltrials.gov.

This is an open-label, non-randomized, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/ microliter who are refractory to anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST), who have relapsed after ATG-based IST, or who are ineligible for ATG-based IST. Eltrombopag is expected to improve trilineage blood cells and decrease transfusion frequency based on the result from the previous study in patients with severe AA. This study will use the hematologic response rate, defined as the proportion of subjects showing improvement in at least one of the three blood cell lineages or a decrease in blood transfusion volume, as the primary endpoint. Twenty patients will be enrolled in the study. Treatment with eltrombopag will be started at 25 milligram (mg)/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 100 mg/day. Response assessment will be performed at 3 months after starting the study treatment (Week 13). Subjects in whom the treatment is assessed as effective will be continued with the study treatment. Subjects in whom the treatment is assessed as effective (when meeting any of the response criteria) at 6 months after starting the study treatment (Week 26) may enter the extension phase and continue the treatment with eltrombopag. The primary endpoint is the hematologic response rate at Week26.

GSK Study ID:

200926


ClinicalTrials.gov Identifier:

NCT02148133


EudraCT Number:

Not Available


Study Overview

Medical Conditions

Heart, Blood & Circulatory - Other

Product

eltrombopag

Collaborators

N/A


Date

July 2014 to December 2016

Type

Interventional

Phase

2/3


Gender

Both

Age

18 Year - 79 Year

Accepts Healthy Volunteers

none


Study Documents

No documents available
This study has Protocol summary on ClinicalTrial.gov. Click here to learn more.

Locations

  • Central Contact :

    Not available

  • Central Contact Phone:

    877-379-3718

  • Central Contact Email:

    [email protected]


Study Design

  • Primary Purpose: Treatment
  • Allocation: N\A
  • Study Design: Single Group Assignment
  • Study Classification: Safety/Efficacy Study
  • Masking: Not Available
  • Masked Subject: No
  • Masked Caregiver: No
  • Masked Investigator: No
  • Masked Assessor: No
Primary Outcomes:

  • Neutrophil count
    Timeframe: Baseline to Week 26

  • Hemoglobin
    Timeframe: Baseline to Week 26

  • Platelet count
    Timeframe: Baseline to Week 26

Secondary Outcomes:

  • Proportion of subjects with reduced volume of transfusion (platelet and RBC)
    Timeframe: Up to 2.5 years

  • Pharmacokinetics (PK) of eltrombopag
    Timeframe: Up to Week 26

  • Change in neutrophil count in the absence of G-CSF
    Timeframe: Up to 2.5 years

  • Frequency and volume of transfusion (platelet and RBC)
    Timeframe: Up to 2.5 years

  • Proportion of subjects who become transfusion independent
    Timeframe: Up to 2.5 years

  • Time to hematologic response and duration (any response according to the response criteria for the primary endpoint)
    Timeframe: Up to 2.5 years

  • Number and proportion of subjects with adverse events (AE)
    Timeframe: Up to 2.5 years

  • Change in hemoglobin in the absence of RBC transfusion
    Timeframe: Up to 2.5 years

  • Number and proportion of subjects with bleeding and severity of bleeding
    Timeframe: Up to 2.5 years

  • Change in platelet count in the absence of platelet transfusion
    Timeframe: Up to 2.5 years

  • Hematologic response in terms of the platelet count, hemoglobin level, and neutrophil count (any response according to the response criteria for the primary endpoint)
    Timeframe: Week13

Interventions:

  • drug: Eltrombopag 12.5 mg
  • drug: Eltrombopag 25 mg

Keyword:

  • Thrombocytopenia, Eltrombopag, Aplastic Anemia (AA), Thrombopoietin
Inclusion / Exclusion Criteria: Click to view inclusion/exclusion criteria:
Enrollment: 21