NCT00903422

Eltrombopag Treatment of Thrombocytopenia in Subjects With Advanced Myelodysplastic Syndrome (MDS) or Secondary Acute Myeloid Leukemia After MDS (sAML/MDS)

Completed Phase 1 Last updated 13 November 2017

What this trial tests

Phase 1 trial testing eltrombopag olamine in Myelodysplastic Syndrome in 98 participants. Completed in 5 December 2013.

Timeline

14 May 2009

Primary endpoint
26 June 2012

5 December 2013

Quick facts

Lead sponsor	GlaxoSmithKline
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	treatment
Enrollment	98
Start date	14 May 2009
Primary completion	26 June 2012
Estimated completion	5 December 2013
Sites	65 locations across Denmark, France, Hong Kong, Italy, Taiwan, United Kingdom, Germany, South Korea

Drugs / interventions tested

• eltrombopag olamine — full drug profile →
• Placebo

Conditions studied

• Myelodysplastic Syndrome — all drugs for Myelodysplastic Syndrome →

Sponsor

GlaxoSmithKline — full company profile →

Who can join

18 and older, any sex, with Myelodysplastic Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study will evaluate the safety and tolerability of eltrombopag in the treatment of low platelet counts in adult subjects with advanced myelodysplastic syndrome (MDS), secondary acute myeloid leukemia after MDS (sAML/MDS), or de novo AML that are relapsed, refractory or ineligible to receive azacitidine, decitabine, intensive chemotherapy or autologous/allogeneic stem cell transplantation. This is a placebo-controlled study in which patients will receive study medication daily for 6 months, during which time the dose of study medication may be adjusted based upon individual platelet counts and bone marrow blast counts. All subjects will receive best standard of care (platelet transfusions, mild chemotherapy, cytokines, valproic acid, all-trans retinoic acid, ESAs or G-CSF) in addition to study medication. Subjects taking placebo may be allowed to crossover to eltrombopag treatment if a clinically and statistically significant improvement in bone marrow blast counts is seen in subjects treated with eltrombopag.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Iron and leukemia: new insights for future treatments.
   Wang F, Lv H, Zhao B, Zhou L, et al · · 2019 · cited 104× · PMID 31519186 · DOI 10.1186/s13046-019-1397-3
2. Safety and tolerability of eltrombopag versus placebo for treatment of thrombocytopenia in patients with advanced myelodysplastic syndromes or acute myeloid leukaemia: a multicentre, randomised, placebo-controlled, double-blind, phase 1/2 trial.
   Platzbecker U, Wong RS, Verma A, Abboud C, et al · · 2015 · cited 55× · PMID 26686043 · DOI 10.1016/s2352-3026(15)00149-0
3. Retinoid receptor signaling and autophagy in acute promyelocytic leukemia.
   Orfali N, McKenna SL, Cahill MR, Gudas LJ, et al · · 2014 · cited 36× · PMID 24694321 · DOI 10.1016/j.yexcr.2014.03.018
4. Safety and Efficacy of Eltrombopag and Romiplostim in Myelodysplastic Syndromes: A Systematic Review and Meta-Analysis.
   Meng F, Chen X, Yu S, Ren X, et al · · 2020 · cited 14× · PMID 33324559 · DOI 10.3389/fonc.2020.582686
5. Thrombopoietin mimetics for patients with myelodysplastic syndromes.
   Dodillet H, Kreuzer KA, Monsef I, Skoetz N. · · 2017 · cited 13× · PMID 28962071 · DOI 10.1002/14651858.cd009883.pub2
6. Alternatives, and adjuncts, to prophylactic platelet transfusion for people with haematological malignancies undergoing intensive chemotherapy or stem cell transplantation.
   Desborough M, Estcourt LJ, Doree C, Trivella M, et al · · 2016 · cited 10× · PMID 27548292 · DOI 10.1002/14651858.cd010982.pub2
7. The synergistic antileukemic effects of eltrombopag and decitabine in myeloid leukemia cells.
   Shi M, Xu F, Yang X, Bai Y, et al · · 2019 · cited 9× · PMID 31564981 · DOI 10.2147/cmar.s213931
8. Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature.
   Capecchi M, Serpenti F, Giannotta J, Pettine L, et al · · 2021 · cited 6× · PMID 34650908 · DOI 10.3389/fonc.2021.680411