To assess the efficacy of eltrombopag, relative to placebo, in achieving platelet counts of = 50 Gi/L, when administered to pediatric subjects with previously treated chronic ITP during the first 12 weeks of Part 1, the randomized treatment period. MedDRA version: 14.1 Level: PT Classification code 10021245 Term: Idiopathic thrombocytopenic purpura System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Product Name: ELTROMBOPAG
Product Code: SB-497115
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: ELTROMBOPAG
CAS Number: 496775-62-3
Current Sponsor code: SB-497115
Other descriptive nam
Sponsors
Glaxosmithkline Research and Development Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written informed consent must be obtained from the subject’s guardian and accompanying informed assent from the subject (for children over 6 years old). 2. Subjects must be between 1 year and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subjects with any clinically relevant abnormality, other than ITP, identified on the screening examination or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another primary diagnosis (e.g. Thrombocytopenia is secondary to another disease). 2. Subjects with concurrent or past malignant disease, including myeloproliferative disorder. 3. Subjects expected not to be suitable for continuation of their current therapy for at least 13 additional weeks. 4. Subjects with a history of platelet agglutination abnormality that prevents reliable measurement of platelet counts. 5. Subjects with a diagnosis of secondary immune thrombocytopenia, including those with laboratory or clinical evidence of HIV infection, anti-phospholipid antibody syndrome, chronic hepatitis B infection, hepatitis c virus infection, or any evidence of active hepatitis at the time of subject screening. 6. Subjects with Evans syndrome (autoimmune thrombocytopenia and autoimmune hemolysis). 7. Subjects with known inherited thrombocytopenia (e.g. MYH9 disorders). 8. Subjects treated with any medication that affects platelet function (including but not limited to aspirin, clopidogrel and/or NSAIDS) or anti-coagulants for >3 consecutive days within 2 weeks of Day 1, or subjects treated with any prohibited medication as described in Section 6.2. 9. Subjects who have received treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding Day 1. 10. Subjects who have previously received eltrombopag or any other thrombopoietin receptor agonist. 11. Any subject considered to be a child in care, defined as one who has been placed under the control or protection of an agency, organization, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation. This can include a child cared for by foster parents or living in a care home or institution, provided that the arrangement falls within the definition above. The definition of a child in care does not include a child who is adopted or who has an appointed legal guardian. 12. Subjects who have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to eltrombopag or excipients that contraindicates their participation. 13. Any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions that could interfere with the subject’s safety or compliance to the study procedures.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): The odds of achieving platelet counts = 50 Gi/L during the first 12 weeks of Part 1, the randomized treatment period, for subjects receiving eltrombopag relative to placebo.;Timepoint(s) of evaluation of this end point: Primary endpoint will be based on platelet counts obtained through the first 12 weeks of Part 1.;Main Objective: To assess the efficacy of eltrombopag, relative to placebo, in achieving platelet counts of = 50 Gi/L, when administered to pediatric subjects with previously treated chronic ITP during the first 12 weeks of Part 1, the randomized treatment period.;Secondary Objective: • To describe the efficacy of eltrombopag in achieving platelet counts = 50 Gi/L when administered to pediatric subjects with previously treated chronic ITP. • To assess the efficacy of eltrombopag in achieving sustained platelet counts = 50 Gi/L when administered to pediatric subjects with previously treated chronic ITP. • To describe the effect of eltrombopag on reduction and/or interruption of concomitant ITP therapies, when administered for 24 weeks to pediatric subjects with previously treated chronic ITP. • To describe the effect of eltrombopag on the need for rescue ITP medication when administered to pediatric subjects with previously treated chronic ITP. • To assess the efficacy of eltrombopag in decreasing the incidence and severity of bleeding symptoms when administered in pediatric subjects with previously treated chronic. Please refer to the protocol for The remaining objectives P12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The proportion of subjects receiving eltrombopag, compared to placebo, who achieve platelet counts = 50 Gi/L for at least 6 out of 8 weeks, between weeks 5-12 of Part 1. Weighted mean platelet change (area under the platelet-time curve divided by duration), for subjects receiving eltrombopag relative to placebo, from baseline to Week 12 of Part 1. The proportion of subjects receiving eltrombopag, compared to placebo, who achieve platelet counts = 50 Gi/L at any time during the first 6 weeks of Part 1. The proportion of subjects receiving eltrombopag, compared to placebo, who achieve platelet counts = 50 Gi/L at any time during the first 12 weeks of Part 1. The proportion of subjects achieving platelet counts = 50 Gi/L at any time during Part 2. Maximum period of time with platelet counts continuously = 50 Gi/L for subjects receiving eltrombopag relative to placebo during the first 12 weeks of Part 1. The proportion of weeks in which subjects achieve platelet counts = 50 Gi/L, between weeks 4-24 of Part 2. Maximum period of time with platelet counts continuously = 50 Gi/L during Part 2. The proportion of subjects who reduced or discontinued baseline concomitant ITP medications during Part 2. The proportion of subjects receiving eltrombopag, relative to placebo, who required protocol-defined rescue treatment during Part 1. The proportion of subjects who required protocol-defined rescue treatment during Part 2. Incidence and severity of symptoms associated with ITP, including bleeding, bruising and petechiae, measured using the World Health Organization (WHO) Bleeding Scale for subjects receiving eltrombopag relative to placebo, during Part 1. Incidence and severity of symptoms associated with ITP, including bleeding, bruising and petechiae, measured using the WHO Bleeding Scale during Part 2. Safety and tolerability parameters including blood pressure and heart rate, ophthalmic examinations, clinical laboratory assessments and | — |
Countries
Argentina, Chile, Czech Republic, Germany, Hong Kong, Israel, Italy, Poland, Russian Federation, Spain, Taiwan, Thailand, United Kingdom, United States
Contacts
Public ContactClinical Trial Helpdesk
GlaxoSmithKline Research & Development
Outcome results
None listed