Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body.

Last updated: February 19, 2015
Sponsor: GlaxoSmithKline
Overall Status: Completed

Phase

3

Condition

Lupus

Thrombocytopenic

Immune Thrombocytopenia (Itp)

Treatment

N/A

Clinical Study ID

NCT01520909
115450
2011-002184-17
  • Ages 1-17
  • All Genders

Study Summary

The purpose of this study is to investigate the efficacy, safety and tolerability of eltrombopag in children with previously treated chronic immune thrombocytopenia who are between 1 and 17 years of age. This is a 2 part study. In part 1, patients will be randomized to receive either eltrombopag or placebo for 13 weeks. All patients who complete part 1 will enter part 2. In part 2, all patients will receive 24 weeks of eltrombopag.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Written informed consent must be obtained from the patient's guardian and accompanyinginformed assent from the patient (for children over 6 years old)

  • Patients must be between 1 year and <18 years of age at Day 1

  • Patients will have a confirmed diagnosis of chronic ITP for at least 1 year, atscreening, according to the guidelines published in the International Working GroupReport

  • A peripheral blood smear or bone marrow examination will support the diagnosis of ITPwith no evidence of other causes of thrombocytopenia.

  • Patients must be refractory or have relapsed after at least one prior ITP therapy, orpatients must be unable, for a medical reason, to continue other ITP treatments.

  • Patients must have a Day 1 (or within 48 hours prior) platelet count <30 Gi/L.

  • Previous therapy for ITP with immunoglobulins (IVIg and anti-D) must have beencompleted at least 2 weeks prior to Day 1, or these therapies must have been completedat least 1 week prior to Day 1 and have been clearly ineffective.

  • Previous treatment for ITP with splenectomy, rituximab and cyclophosphamide must havebeen completed at least 4 weeks prior to Day 1.

  • Patients treated with concomitant ITP medication (e.g. corticosteroids orazathioprine) must be receiving a dose that has been stable for at least 4 weeks priorto Day 1.

  • Patients must have a complete blood count (CBC) not suggestive of anotherhematological disorder.

  • Patients must have the following laboratory results:

  • prothrombin time international normalized ratio (INR) and activated partialthromboplastin time (aPTT) within 80 to 120% of the normal range.

  • clinical chemistries that do NOT exceed the upper limit of normal reference range bymore than 20% for the following: creatinine, ALT, AST, total bilirubin, and alkalinephosphatase.

  • total albumin that is not below the lower limit of normal by more than 10%.

  • Female patients 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 duringthe study,

  • agree to use effective contraception during the study and for 28 days following thelast dose of study treatment, and not be lactating.

  • Male patients with a female partner of childbearing potential must agree to useeffective contraception from 2 weeks prior to administration of the first dose ofstudy treatment until 3 months after the last dose of study treatment.

  • In France, a patient will be eligible for inclusion in this study only if eitheraffiliated to or a beneficiary of a social security category.

Exclusion

Exclusion Criteria:

  • Patients with any clinically relevant abnormality, other than ITP, identified on thescreening examination or any other medical condition or circumstance, which in theopinion of the investigator makes the patient unsuitable for participation in thestudy or suggests another primary diagnosis (e.g. Thrombocytopenia is secondary toanother disease).

  • Patients with concurrent or past malignant disease, including myeloproliferativedisorder.

  • Patients expected not to be suitable for continuation of their current therapy for atleast 13 additional weeks.

  • Patients with a history of platelet agglutination abnormality that prevents reliablemeasurement of platelet counts.

  • Patients with a diagnosis of secondary immune thrombocytopenia, including those withlaboratory or clinical evidence of HIV infection, anti-phospholipid antibody syndrome,chronic hepatitis B infection, hepatitis c virus infection, or any evidence of activehepatitis at the time of subject screening.

  • Patients with Evans syndrome (autoimmune thrombocytopenia and autoimmune hemolysis).

  • Patients with known inherited thrombocytopenia (e.g. MYH9 disorders).

  • Patients treated with any medication that affects platelet function (including but notlimited to aspirin, clopidogrel and/or NSAIDS) or anti-coagulants for >3 consecutivedays within 2 weeks of Day 1.

  • Patients who have received treatment with an investigational drug within 30 days or 5half-lives (whichever is longer) preceding Day 1.

  • Patients who have previously received eltrombopag or any other thrombopoietin receptoragonist.

  • Any patient considered to be a child in care, defined as one who has been placed underthe control or protection of an agency, organization, institution or entity by thecourts, the government or a government body, acting in accordance with powersconferred on them by law or regulation. This can include a child cared for by fosterparents or living in a care home or institution, provided that the arrangement fallswithin the definition above. The definition of a child in care does not include achild who is adopted or who has an appointed legal guardian.

  • Patients who have a known immediate or delayed hypersensitivity reaction oridiosyncrasy to drugs chemically related to eltrombopag or excipients thatcontraindicates their participation.

  • Any serious and/or unstable pre-existing medical, psychiatric disorder, or otherconditions that could interfere with the patient's safety or compliance to the studyprocedures.

Study Design

Total Participants: 92
Study Start date:
March 01, 2012
Estimated Completion Date:
January 31, 2014

Study Description

This is a two part, double-blind, randomized, placebo-controlled and open-label Phase III study to investigate the efficacy, safety and tolerability of eltrombopag in pediatric patients with previously treated chronic ITP. In Part 1, patients will be randomized to receive eltrombopag or placebo in a 13-week double-blind, placebo-controlled treatment period. After completing Part 1, patients will begin Part 2, in which they will receive eltrombopag in an open-label manner during a 24-week treatment period.

Connect with a study center

  • GSK Investigational Site

    Capital Federal, Buenos Aires 1425
    Argentina

    Site Not Available

  • GSK Investigational Site

    Santiago, 8380418
    Chile

    Site Not Available

  • GSK Investigational Site

    Brno, 613 00
    Czech Republic

    Site Not Available

  • GSK Investigational Site

    Olomouc, 775 20
    Czech Republic

    Site Not Available

  • GSK Investigational Site

    Ostrava, 708 52
    Czech Republic

    Site Not Available

  • GSK Investigational Site

    Praha 5, 154 00
    Czech Republic

    Site Not Available

  • GSK Investigational Site

    Freiburg, Baden-Wuerttemberg 79106
    Germany

    Site Not Available

  • GSK Investigational Site

    Mainz, Rheinland-Pfalz 55131
    Germany

    Site Not Available

  • GSK Investigational Site

    Berlin, 13353
    Germany

    Site Not Available

  • GSK Investigational Site

    Pokfulam,
    Hong Kong

    Site Not Available

  • GSK Investigational Site

    Shatin,
    Hong Kong

    Site Not Available

  • GSK Investigational Site

    Beer-Sheva, 84101
    Israel

    Site Not Available

  • GSK Investigational Site

    Haifa, 31048
    Israel

    Site Not Available

  • GSK Investigational Site

    Petah-Tikva, 49202
    Israel

    Site Not Available

  • GSK Investigational Site

    Ramat Gan, 52621
    Israel

    Site Not Available

  • GSK Investigational Site

    Rehovot, 76100
    Israel

    Site Not Available

  • GSK Investigational Site

    Tel-Aviv, 64239
    Israel

    Site Not Available

  • GSK Investigational Site

    Bologna, Emilia-Romagna 40138
    Italy

    Site Not Available

  • GSK Investigational Site

    Roma, Lazio 00165
    Italy

    Site Not Available

  • GSK Investigational Site

    Monza, Lombardia 20900
    Italy

    Site Not Available

  • GSK Investigational Site

    Pavia, Lombardia 27100
    Italy

    Site Not Available

  • GSK Investigational Site

    Torino, Piemonte 10126
    Italy

    Site Not Available

  • GSK Investigational Site

    Bari, Puglia 70124
    Italy

    Site Not Available

  • GSK Investigational Site

    Padova, Veneto 35128
    Italy

    Site Not Available

  • GSK Investigational Site

    Bydgoszcz,
    Poland

    Site Not Available

  • GSK Investigational Site

    Gdansk, 80-952
    Poland

    Site Not Available

  • GSK Investigational Site

    Lodz, 91-738
    Poland

    Site Not Available

  • GSK Investigational Site

    Lublin, 20-093
    Poland

    Site Not Available

  • GSK Investigational Site

    Zabrze, 41-800
    Poland

    Site Not Available

  • GSK Investigational Site

    Krasnodar, 350007
    Russian Federation

    Site Not Available

  • GSK Investigational Site

    Moscow, 117198
    Russian Federation

    Site Not Available

  • GSK Investigational Site

    Saint Petersburg, 198205
    Russian Federation

    Site Not Available

  • GSK Investigational Site

    Barcelona, 08035
    Spain

    Site Not Available

  • GSK Investigational Site

    Madrid, 28007
    Spain

    Site Not Available

  • GSK Investigational Site

    Murcia (El Palmar), 30120
    Spain

    Site Not Available

  • GSK Investigational Site

    Málaga, 29011
    Spain

    Site Not Available

  • GSK Investigational Site

    Málaga, 29011
    Spain

    Site Not Available

  • GSK Investigational Site

    Pamplona, 31008
    Spain

    Site Not Available

  • GSK Investigational Site

    Valencia, 46026
    Spain

    Site Not Available

  • GSK Investigational Site

    Tainan, 704
    Taiwan

    Site Not Available

  • GSK Investigational Site

    Taoyuan, 333
    Taiwan

    Site Not Available

  • GSK Investigational Site

    Bangkok,
    Thailand

    Site Not Available

  • GSK Investigational Site

    Khon Kaen, 40002
    Thailand

    Site Not Available

  • GSK Investigational Site

    Songkla, 90110
    Thailand

    Site Not Available

  • GSK Investigational Site

    Birmingham, B4 6NH
    United Kingdom

    Site Not Available

  • GSK Investigational Site

    Cardiff, CF14 4XW
    United Kingdom

    Site Not Available

  • GSK Investigational Site

    London, SW17 0QT
    United Kingdom

    Site Not Available

  • GSK Investigational Site

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • GSK Investigational Site

    Romford, RM7 0AG
    United Kingdom

    Site Not Available

  • GSK Investigational Site

    Sheffield, S10 2TH
    United Kingdom

    Site Not Available

  • GSK Investigational Site

    Los Angeles, California 90027
    United States

    Site Not Available

  • GSK Investigational Site

    St. Petersburg, Florida 33701
    United States

    Site Not Available

  • GSK Investigational Site

    Brooklyn, New York 11219
    United States

    Site Not Available

  • GSK Investigational Site

    Salt Lake City, Utah 84113
    United States

    Site Not Available

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