Last updated: 11/03/2018 17:27:15
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 GSK Clinical Study Register is no longer maintained for this study.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.

A Three-part Study of Eltrombopag in Thrombocytopenic Subjects with Myelodysplastic Syndromes or Acute Myeloid Leukemia (Part 1: open-label, Part 2: randomized, double-blind, Part 3: extension)ASPIRE

GSK study ID
114968
Clinicaltrials.gov ID
NCT01440374
EudraCT ID
2011-000114-19
EU CT Number
Not applicable
Trial status
No longer a GSK study
No longer a GSK study
Overview
Eligibility
Locations
Study documents
Results summary
Plain language summaries
Additional information

Trial overview

Official title: A Three-part Study of Eltrombopag in Thrombocytopenic Subjects with Myelodysplastic Syndromes or Acute Myeloid Leukemia (Part 1: open-label, Part 2: randomized, double-blind, Part 3: extension)
Trial description: This is a worldwide, three-part (Part 1: open-label, Part 2: randomized, double-blind,
Part 3: extension), multi-center study to evaluate the effect of eltrombopag in subjects
with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have
thrombocytopenia due to bone marrow insufficiency from their underlying disease or
prior chemotherapy. This objective will be assessed by a composite primary endpoint
that consists of the following: the proportion of ≥Grade 3 hemorrhagic adverse events, or
platelet counts <10 Gi/L, or platelet transfusions.
Patients with MDS or AML and Grade 4 thrombocytopenia due to bone marrow
insufficiency from their underlying disease or prior chemotherapy will be enrolled in the
study. No low or intermediate-1 risk MDS subjects will be enrolled in the study.
Subjects must have had at least one of the following during the 4 weeks prior to
enrolment: platelet count <10 Gi/L, platelet transfusion, or symptomatic hemorrhagic
event. Supportive standard of care (SOC), including hydroxyurea, will be allowed as
indicated by local practice throughout the study.
The study will have 3 sequential parts. Subjects who are enrolled in Part 1
(open-label) cannot be enrolled in Part 2 of the study (randomized, double-blind);
however, subjects who complete the treatment period for Part 1 or Part 2 (8 and 12
weeks, respectively) will continue in Part 3 (extension) if the investigator determines
that the subject is receiving clinical benefit on treatment.
Primary purpose:
Not applicable
Trial design:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation:
Randomized
Primary outcomes:

Percentage of participants with clinically relevant thrombocytopenic events (CRTE) from Week 5 up to Week 12 during Part 2

Timeframe: From Week 5 up to Week 12 during Part 2

Number of participants with platelet response up to Week 8 during Part 1

Timeframe: From Baseline up to Week 8 during Part 1

Secondary outcomes:
Not applicable
Interventions:
  • Drug: eltrombopag
  • Drug: placebo
    • Enrollment:
    162
    Primary completion date:
    2015-17-03
    Observational study model:
    Not applicable
    Time perspective:
    Not applicable
    Clinical publications:
    Not applicable
    Medical condition
    Thrombocytopaenia
    Product
    eltrombopag
    Collaborators
    Not applicable
    Study date(s)
    September 2011 to December 2015
    Type
    Interventional
    Phase
    1/2

    Participation criteria

    Sex
    Female & Male
    Age
    18 - 110 Year
    Accepts healthy volunteers
    none
    • Adult subjects (18 years of age or older) with MDS or AML (bone marrow blasts ≤50%) with thrombocytopenia due to bone marrow insufficiency from the disease or prior treatment. Subjects with transient thrombocytopenia due to active treatment with disease modifying agents or chemotherapy (except for hydroxyurea) are excluded
    • Subjects must have Grade 4 thrombocytopenia (platelet counts <25 Gi/L) due to bone marrow insufficiency (or Grade 4 thrombocytopenia, but platelet count greater than or equal to 25 Gi/L due to platelet transfusion). In addition, subjects must have had at least one of the following during the 4 week screening period: platelet transfusion, or symptomatic bleeding or platelet count <10 Gi/L. Subjects whose thrombocytopenia below 10 Gi/L is due to causes other than bone marrow insufficiency (e.g., fever, infection, autoimmune disease) are not eligible.
    • Subjects with MDS and an IPSS of low or intermediate-1 risk at screening.
    • Subjects with a diagnosis of acute promyelocytic or megakaryocytic leukemia or AML secondary to a myeloproliferative neoplasm.
    Inclusion and exclusion criteria
    Inclusion criteria:
    • Adult subjects (18 years of age or older) with MDS or AML (bone marrow blasts ≤50%) with thrombocytopenia due to bone marrow insufficiency from the disease or prior treatment. Subjects with transient thrombocytopenia due to active treatment with disease modifying agents or chemotherapy (except for hydroxyurea) are excluded
    • Subjects must have Grade 4 thrombocytopenia (platelet counts <25 Gi/L) due to bone marrow insufficiency (or Grade 4 thrombocytopenia, but platelet count greater than or equal to 25 Gi/L due to platelet transfusion). In addition, subjects must have had at least one of the following during the 4 week screening period: platelet transfusion, or symptomatic bleeding or platelet count <10 Gi/L. Subjects whose thrombocytopenia below 10 Gi/L is due to causes other than bone marrow insufficiency (e.g., fever, infection, autoimmune disease) are not eligible.
    • Subjects must have platelet count, bleeding and platelet transfusion data available over a period of at least 4 weeks prior to randomization.
    • Prior systemic treatment for malignancy, with the exception of hydroxyurea, must have been discontinued prior to entry into the study: at least 4 weeks before Day 1 for the following: chemotherapy, demethylating agents (azacitidine or decitabine), lenalidomide, thalidomide, clofarabine and IL-11(oprelvekin); at least 8 weeks before Day 1 for antithymocyte/antilymphocyte globulin.
    • Subjects with a prior stem cell transplant (SCT) must have relapsed after the SCT.
    • Subjects must be stable and, in the opinion of the investigator, be expected to complete a 12 week treatment period.
    • ECOG Status 0-2.
    • Subject must be able to understand and comply with protocol requirements and instructions.
    • Subject has signed and dated an informed consent form.
    • Adequate baseline organ function defined by the criteria below: total bilirubin ≤ 1.5xULN except for Gilbert’s syndrome or cases clearly not indicative of inadequate liver function (i.e. elevation of indirect (hemolytic) bilirubin in the absence of ALT abnormality), ALT ≤ 3xULN, creatinine ≤ 2.5xULN
    • Women must be either of non-child bearing potential or women with child-bearing potential and men with reproductive potential must be willing to practice acceptable methods of birth control during the study Women of childbearing potential must have a negative serum or urine pregnancy test within 7 days prior to the first dose of study treatment.
    • 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.
    Exclusion criteria:
    • Subjects with MDS and an IPSS of low or intermediate-1 risk at screening.
    • Subjects with a diagnosis of acute promyelocytic or megakaryocytic leukemia or AML secondary to a myeloproliferative neoplasm.
    • History of treatment with romiplostim or other TPO-R agonists.
    • Subjects with a QTc >480 msec (QTc >510 msec for subjects with Bundle Branch Block).
    • Leukocytosis ≥25,000/uL on Day 1 of treatment with study medication.
    • Subjects with known thrombophilic risk factors. Exception: Subjects for whom the potential benefits of participating in the study outweigh the potential risks of thromboembolic events, as determined by the investigator.
    • Female subjects who are nursing or pregnant (positive serum or urine β-human chorionic gonadotropin [β-hCG] pregnancy test) at screening or pre-dose on Day 1.
    • Current alcohol or drug abuse.
    • Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
    • Active and uncontrolled infections (e.g. sepsis, hepatitis B, hepatitis C).
    • Subjects infected with Human Immunodeficiency Virus (HIV).
    • Subjects with liver cirrhosis (as determined by the investigator).
    • Subjects receiving or planned to receive any prohibited medication.
    • Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to eltrombopag or excipient (microcrystalline cellulose, mannitol, polyvinylpyrrolidone, sodium starch glycolate, magnesium stearate, hypromellose, titanium dioxide, polyethylene glycol 400 and polysorbate 80) that contraindicates the subjects’ participation.
    • In France, subjects who have participated in any study using an investigational drug during the previous 30 days.

    Trial location(s)

    This study does not involve prospective enrollment of participants.

    Study documents

    No study documents available.

    Results overview

    Study Results yet to be posted

    Recruitment status
    No longer a GSK study
    Actual primary completion date
    2015-17-03
    Actual study completion date
    Not applicable

    Plain language summaries

    Plain language summaries of clinical trial results for Phase 2-4 clinical trials that were initiated on or after January 2022 will be posted by GSK within one year following study completion.

    Additional information about the trial

    Additional information
    Not applicable
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