A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Condition: Purpura, Thrombocytopenic, Idiopathic
MeSH terms: Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
Intervention: Eltrombopag Tablets; Eltrombopag PfOS
Intervention description: Eltrombopag tablets will be white, round film coated tablets containing eltrombopag olamine equivalent to 12.5 mg, 25 mg, 50 mg and 75 mg of eltrombopag. The 12.5 mg tablet will be smaller than the 25 mg, 50 mg and 75 mg tablets. Subjects will receive maximum dose of 75 mg once daily (QD).; Eltrombopag PfOS is a reddish-brown to yellow powder contained inside an elongated sachet. Each sachet will contain eltrombopag olamine equivalent to 20 mg of eltrombopag per gram of powder. Subjects will receive maximum dose of 75 mg once daily (QD)
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model description: AEs were graded using the common toxicity criteria for adverse events (CTCAE) Version 4.03. If a patient reported more than one AE with the same preferred term (PT), the AE with the greatest severity was presented. If a patient reported more than one AE within the same primary system organ class (SOC), the patient was counted only once with the greatest severity at the SOC level, where applicable. An AE with missing CTCAE grade was included in the 'All grades' column of the summary tables.
In AE summaries, the primary SOC was presented alphabetically and the PTs were sorted within the primary SOC in descending frequency.
Masking: None (Open Label)
Enrollment: 9.0
Sex: All
Minimum age: 1 Year
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Chronic immune thrombocytopenic purpura, ITP, eltrombopag olamine, thrombopoietin receptor agonist, pediatrics

Summary:

      This was an open-label Phase III extension study to evaluate the long-term safety of
      eltrombopag in pediatric patients with chronic ITP who previously participated in study
      TRA115450. This study allowed dosing of eltrombopag at an individualized dose for each
      subject based upon platelet count. The starting dose was based on the subject's dose at the
      end of the TRA115450 study. The maximum dose was 75 mg daily.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Written informed consent must be obtained from the subject's guardian and accompanying
             informed assent from the subject (for children over 6 years old).

          -  Subjects must be between 1 year and <18 years of age at Day 1.

          -  Subjects must have enrolled in TRA115450/PETIT2 study.

          -  Subjects must have completed Part 1 and Part 2 of TRA115450/PETIT2 study.

          -  Female subjects of child-bearing potential (after menarche) must have a negative
             pregnancy test within 24 hours of first dose of study treatment; agree and be able to
             provide a blood or urine specimen for pregnancy testing during the study; agree to use
             effective contraception during the study and for 28 days following the last dose of
             study treatment, and not be lactating.

          -  Male subjects with a female partner of childbearing potential must agree to use
             effective contraception from 2 weeks prior to administration of the first dose of
             study treatment until 3 months after the last dose of study treatment.

        Exclusion Criteria:

          -  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).

          -  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.