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: A Study of EltromboPag In Myelodysplastic SyndRomes and AcutE Myeloid Leukemia

Phase 2

Completed

• Conditions: thrombocytopenia; decreased number of thrombocytes; 10018849

• Registration Number: NL-OMON39136
• Lead Sponsor: GlaxoSmithKline BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 6 May 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 2

Inclusion Criteria

* Age 18 years and above.
* 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.
* Grade 4 thrombocytopenia (platelet counts <25 Gi/L) due to bone marrow insufficiency (or platelet count *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 are not eligible.
* Prior systemic treatment for malignancy, with the exception of hydroxyurea, must have been discontinued 4-8 weeks prior to entry into the study (see protocol for details).
* Subjects with a prior stem cell transplant (SCT) must have relapsed after the SCT.
* Female participants and female partners of male participants of child-bearing potential (after menarche): subject must not be sexually active or is practicing an acceptable method of contraception.

Exclusion Criteria

* MDS and an IPSS of low or intermediate-1 risk.
* Acute promyelocytic or megakaryocytic leukemia or AML secondary to a myeloproliferative neoplasm.
* History of treatment with romiplostim or other TPO-R agonists.
* Leukocytosis *25,000/uL.
* Pregnancy and breast feeding. Inadequate contraception, if relevant.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The composite primary endpoint of the randomized part of the study consists of<br /><br>the following: the proportion of * Grade 3 hemorrhagic events, or platelet<br /><br>counts <10 Gi/L, or platelet transfusions.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Number of platelet transfusions, effects on hematologic improvement, platelet<br /><br>counts, bleeding symptoms, disease progression, overall survival. Safety and<br /><br>tolerability. Medical resource utilization. quality of life. Population PK.</p><br>