Study of a new medication for childhood chronic immune thrombocytopenic purpura, ITP, a blood disorder of low platelet counts that can lead to bruising easily, bleeding gums, and/or bleeding inside the body.

Phase 1

• Conditions: 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: 14.1Level: PTClassification code 10021245Term: Idiopathic thrombocytopenic purpuraSystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2011-002184-17-CZ
• Lead Sponsor: Glaxosmithkline Research and Development Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-12-08
• Last Update Posted: 14 November 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 75

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 <18 years of age at Day 1.
3. Subjects will have a confirmed diagnosis of chronic ITP for at least 1 year, at screening, according to the guidelines published in the International Working Group Report [Rodeghiero, 2009].
4. A peripheral blood smear or bone marrow examination will support the diagnosis of ITP with no evidence of other causes of thrombocytopenia.
5. Subjects must be refractory or have relapsed after at least one prior ITP therapy, or subjects must be unable, for a medical reason, to continue other ITP treatments.
6. Subjects must have a Day 1 (or within 48 hours prior) platelet count <30 Gi/L.
7. Previous therapy for ITP with immunoglobulins (IVIg and anti-D) must have been completed at least 2 weeks prior to Day 1, or these therapies must have been completed at least 1 week prior to Day 1 and have been clearly ineffective.
8. Previous treatment for ITP with splenectomy, rituximab and cyclophosphamide must have been completed at least 4 weeks prior to Day 1.
9. Subjects treated with concomitant ITP medication (e.g. corticosteroids or azathioprine) must be receiving a dose that has been stable for at least 4 weeks prior to Day 1.
10. Subjects must have a complete blood count (CBC) not suggestive of another hematological disorder.
11. Subjects must have the following laboratory results:
• prothrombin time international normalized ratio (INR) and activated partial thromboplastin time (aPTT) within 80 to 120% of the normal range.
• clinical chemistries that do NOT exceed the upper limit of normal (ULN) reference range by more than 20% for the following: creatinine, ALT, AST, total bilirubin, and alkaline phosphatase.
• total albumin that is not below the lower limit of normal (LLN) by more than 10%.
12. 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, as defined in Section 7.3.3, during the study and for 28 days following the last dose of study treatment, and
• not be lactating.
13. Male subjects with a female partner of childbearing potential must agree to use effective contraception as described in Section 7.3.3 from 2 weeks prior to administration of the first dose of study treatment until 3 months after the last dose of study treatment.
14. In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.

Are the trial subjects under 18? yes
Number of subjects for this age range: 75
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified