Table 1.

Studies investigating Tpo receptor agonists in bone marrow failure syndromes

Study	Patient population	Trial identification*	Start date
Upfront studies
Phase 2, single-arm, multicenter, open label study to investigate the efficacy and safety of eltrombopag and CsA as first-line therapy (SOAR). Sponsor Novartis (conducted worldwide)	Severe and very severe AA	NCT02998645	05/11/2017
	≥6 y
Phase 1/2, single-arm, open label, nonrandomized, prospective study of eltrombopag + horse ATG + CsA. Sponsor NHLBI. Conducted at the NIH	Severe and very severe AA	NCT01623167	06/12/2012
	≥2 y
Phase 2, multicenter, randomized study of eltrombopag combined with horse ATG + CsA vs horse ATG and CsA as first-line treatment in pediatric SAA. Sponsor Federal Research Institute of Pediatric Hematology, Oncology and Immunology. Conducted at the Russian Federation	Severe and very severe AA	NCT03413306	12/20/2016
	2-18 y of age
Randomized clinical trial of the use of eltrombopag in children with idiopathic aplastic anemia. Sponsor Assiut University. Conducted in Egypt	Moderate and severe AA	NCT03243656	12/2017
	1-18 y of age
Phase 2, eltrombopag; nonrandomized, single arm, open label, dose escalation to maximum 300 mg/d. Sponsor University of Utah and Novartis as collaborators. Conducted in Utah	Moderate, severe, and very severe AA	NCT01703169	11/2012
	≥18 y
Phase 2, eltrombopag; nonrandomized, single arm, open label. Sponsor NHLBI. Conducted at the NIH	Moderate AA and unilineage bone marrow failure syndromes	NCT01328587	04/01/2011
	≥18 y
Phase 3, horse ATG + CsA + eltrombopag; prospective, randomized, open label (RACE). Sponsor EBMT. Novartis and Pfizer as collaborators. Conducted in Europe	Severe and very severe AA	NCT02099747	03/2015
	≥15 y
Phase 2, eltrombopag + rabbit ATG + CsA; nonrandomized, single arm, open label. Sponsor Novartis. Conducted in Japan	Moderate, severe, and very severe AA	NCT02404025	03/2015
	≥18 to ≤75 y
Efficacy and safety of eltrombopag in combination with CsA in moderate aplastic anemia: prospective, randomized, multicenter study. Sponsor University of Ulm. Conducted in Germany	Moderate AA	NCT02773225	01/27/2015
	≥18 y
Second-line studies
Phase 2, eltrombopag; nonrandomized, single arm, open label. Sponsor NHLBI. Conducted at the NIH	AA refractory to standard IST	NCT00922883	05/29/2009
	≥12 y
Phase 2, eltrombopag; nonrandomized, single arm, open label. Extended fixed dosing (150 mg) in SAA. Sponsor NHLBI. Conducted at the NIH	Severe and very severe AA	NCT01891994	06/28/2013
	≥2 to ≤100 y
Phase 2, eltrombopag; nonrandomized, open label. Sponsor Novartis. Conducted in Japan	Refractory, moderate, or severe AA	NCT02148133	06/23/2014
	≥18 to <80 y
Phase 2, open label, multicenter, intrapatient dose escalation study of eltrombopag + IST. Sponsor Novartis. Conducted in the United States, Hong Kong, the Russian Federation, and the United Kingdom	Treatment-naïve, relapsed, or refractory SAA	NCT03025698	09/30/2017
	1-18 y of age
Phase 2, romiplostim; nonrandomized, single arm, open label. Sponsor Kyowa Hakko Kirin Co., Ltd. Conducted in the Republic of Korea and Japan	Refractory AA to at least on course of IST	NCT02773290	05/2016
	≥20 y
Phase 2, randomized, open label, parallel, comparative, dose-finding study to evaluate the efficacy and safety of romiplostim in SAA. Sponsor Kyowa Hakko Kirin Co., Ltd. Conducted in the Republic of Korea	Refractory SAA	NCT02094417	04/14/2014
	≥19 y

NHLBI, National Heart, Lung and Blood Institute.

^*Trial identification, sponsor, collaborator(s), and sites were accessed at www.clinicaltrials.gov on September 1, 2018.