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A Three-part Study of Eltrombopag in Thrombocytopenic Subjects With Myelodysplastic Syndromes or Acute Myeloid Leukemia

NCT01440374 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 162

Last updated 2017-06-05

Study results available
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Summary

This was 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 was 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 were enrolled in the study. No low or intermediate-1 risk MDS subjects were 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, was allowed as indicated by local practice throughout the study. The study had 3 sequential parts. Subjects who were enrolled in Part 1 (open-label) cannot be enrolled in Part 2 of the study (randomized, double-blind); however, subjects who completed the treatment period for Part 1 or Part 2 (8 and 12 weeks, respectively) continued in Part 3 (extension) if the investigator determined that the subject was receiving clinical benefit on treatment.

Conditions

  • Thrombocytopaenia

Interventions

DRUG

eltrombopag

100 mg daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg (150 mg for subjects of East Asian heritage)

DRUG

placebo

100 mg matching placebo daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg matching placebo (150 mg for subjects of East Asian heritage)

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
RANDOMIZED
Purpose
SUPPORTIVE_CARE
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
110 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-09-30
Primary Completion
2015-03-31
Completion
2015-12-31

Countries

  • United States
  • Argentina
  • Belgium
  • Brazil
  • Canada
  • Czechia
  • Germany
  • Greece
  • Hong Kong
  • Hungary
  • Ireland
  • Israel
  • Italy
  • Mexico
  • Netherlands
  • Poland
  • Puerto Rico
  • Russia
  • South Korea
  • Spain
  • Taiwan
  • Thailand

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01440374 on ClinicalTrials.gov