Table 2.

Pharmacological treatments for β-thalassemia

Trial identifier	Phase/status	Enrolled patients	Drug product	Route of administration	Target	Sponsor/center
NCT01571635 [49]	Phase 2 Active, not recruiting	16 TDT 30 NTDT	Sotatercept (ACE-011)	Subcutaneous	Ineffective erythropoiesis	Celgene Multicenter international sites
NCT01749540 [51]	Phase 2 Completed	31 TDT 33 NTDT	Luspatercept (ACE-536)	Subcutaneous	Ineffective erythropoiesis	Acceleron Pharma Multicenter international sites
NCT02604433 [52]	Phase 3 Active, not recruiting	336 TDT	Luspatercept (ACE-536)	Subcutaneous	Ineffective erythropoiesis	Celgene and Acceleron Pharma Multicenter international sites
NCT03342404	Phase 2 Active, not recruiting	145 NTDT	Luspatercept (ACE-536)	Subcutaneous	Ineffective erythropoiesis	Celgene and Acceleron Pharma Multicenter international sites
NCT02049450 [56]	Phase 2 Completed	30 TDT	Ruxolitinib	Oral	Ineffective erythropoiesis through inhibition of JAK2	Novartis Pharmaceuticals Multicenter international sites
NCT03381833	Phase 2 Recruitingb,c	100a TDT	LJPC-401	Subcutaneous	Iron metabolism through synthetic human hepcidin	La Jolla Pharmaceutical Company Multicenter international sites
NCT03802201	Phase 2 Recruitingb,c	192a TDT and NTDT	PTG-300	Subcutaneous	Iron metabolism through synthetic human hepcidin	Protagonist Therapeutics
NCT04364269	Phase 2 Not yet recruiting	36a NTDT	VIT-2763	Oral	Iron metabolism through ferroportin inhibition	Vifor Multicenter international sites
NCT04059406	Phase 2a Not yet recruiting	36a NTDT	IONIS TMPRSS6-LRx	Subcutaneous	Iron metabolism through TMPRSS6 inhibition	Ionis Pharmaceuticals
NCT04176653	Phase 1 Withdrawn due to COVID-19b	TDT and low-risk MDS	SLN124	Subcutaneous	Iron metabolism through TMPRSS6 inhibition	Silence Therapeutics

TDT transfusion-dependent thalassemia, NTDT non transfusion-dependent thalassemia, MDS myelodysplastic syndrome, COVID-19 Coronavirus disease 19

^aEstimated enrollment

^bAs of 31 May 2020, from ClinicalTrials.gov

^cThis study was recently stopped and closed