Table 1. RCTs with HMA combination... | American Society of Hematology

Table 1.

RCTs with HMA combination therapy as initial therapy for MDS

Trial	Design	Disease	Investigational agent	Study arms	Patients	Primary endpoint	Anticipated study completion date
STIMULUS MDS1	Phase 2 double-blind	Intermediate- to very high-risk MDS	MBG 433: anti-TIM-3 antibody ICPI	MBG 453 + HMA	60	CR and PFS	14-Aug-23
STIMULUS MDS1	Phase 2 double-blind	Intermediate- to very high-risk MDS	MBG 433: anti-TIM-3 antibody ICPI	PBO + HMA	60	CR and PFS	14-Aug-23
PANTHER	Phase 3 open label	Intermediate- to very high-risk MDS	Pevonedistat: selective NEDD8 inhibitor induces DNA repair	Pevonedistat + aza	227	EFS	31-Mar-23
PANTHER	Phase 3 open label	Intermediate- to very high-risk MDS	Pevonedistat: selective NEDD8 inhibitor induces DNA repair	Aza	227	EFS	31-Mar-23
A18-15331	Phase 3 open label	Intermediate- to very high-risk MDS	APR-246: stabilizes mutated p53	APR-246 + aza	77	CR	1-Nov-20
A18-15331	Phase 3 open label	Intermediate- to very high-risk MDS	APR-246: stabilizes mutated p53	Aza	77	CR	1-Nov-20
HOVON 156 AML	Phase 3 open label	AML + MDS-EB2 FLT-3+	Mido and Gilt: FLT-3 inhibitor	IC + midostaurin	384	EFS	1-May-23
HOVON 156 AML	Phase 3 open label	AML + MDS-EB2 FLT-3+	Mido and Gilt: FLT-3 inhibitor	IC + gilteritinib	384	EFS	1-May-23
VERONA	Phase 3 double-blind	Intermediate- to very high-risk MDS	Venetoclax: BCL-2 inhibitor	Venetoclax + aza	250	CR and OS	26-Jan-25
VERONA	Phase 3 double-blind	Intermediate- to very high-risk MDS	Venetoclax: BCL-2 inhibitor	PBO + aza	250	CR and OS	26-Jan-25
ENHANCE	Phase 3 double-blind	Intermediate- to very high-risk MDS	Magrolimab: anti-CD47 antibody; ICPI	Magrolimab + aza	90	CR	1-Jul-25
ENHANCE	Phase 3 double-blind	Intermediate- to very high-risk MDS	Magrolimab: anti-CD47 antibody; ICPI	PBO + aza	90	CR	1-Jul-25
	Multiphase trial 1-3	MDS, AML, CMML, MDS-MPN	ASTX030: ced/aza deaminase inhibitor	Sequencing trial subcutaneous aza vs ced/aza	245	PK	1-Apr-23
LEAP	Phase 2/3 open label	MDS-EB2, AML	Midostaurin: FLT3 inhibitor nivolumab: ICPI	Midostaurin + aza	16,70	OS	1-Aug-23
				Nivolumab + aza
				Dec + LDAC
				Aza
HO-155	Phase 2 open label	MDS-EB, AML	Mido: FLT3 inhibitor	Midostaurin + dec	70	CR	1-Mar-26
HO-155	Phase 2 open label	MDS-EB, AML	Mido: FLT3 inhibitor	Dec	70	CR	1-Mar-26
Cusatuzumab combination	Phase 2 open label	Intermediate- to very high-risk MDS, CMML	Cusatuzumab: anti-CD 70 antibody	Cusatuzumab + aza	75	ORR	18-Jul-22
Cusatuzumab combination	Phase 2 open label	Intermediate- to very high-risk MDS, CMML	Cusatuzumab: anti-CD 70 antibody	Aza	75	ORR	18-Jul-22
	Phase 3	Intermediate- to very high-risk MDS	Rigosertib: Ras kinase inhibitor	Rigosertib + aza	Unknown	ORR	Unknown
	Phase 3	Intermediate- to very high-risk MDS	Rigosertib: Ras kinase inhibitor	Aza	Unknown	ORR	Unknown

Trial	Design	Disease	Investigational agent	Study arms	Patients	Primary endpoint	Anticipated study completion date
STIMULUS MDS1	Phase 2 double-blind	Intermediate- to very high-risk MDS	MBG 433: anti-TIM-3 antibody ICPI	MBG 453 + HMA	60	CR and PFS	14-Aug-23
STIMULUS MDS1	Phase 2 double-blind	Intermediate- to very high-risk MDS	MBG 433: anti-TIM-3 antibody ICPI	PBO + HMA	60	CR and PFS	14-Aug-23
PANTHER	Phase 3 open label	Intermediate- to very high-risk MDS	Pevonedistat: selective NEDD8 inhibitor induces DNA repair	Pevonedistat + aza	227	EFS	31-Mar-23
PANTHER	Phase 3 open label	Intermediate- to very high-risk MDS	Pevonedistat: selective NEDD8 inhibitor induces DNA repair	Aza	227	EFS	31-Mar-23
A18-15331	Phase 3 open label	Intermediate- to very high-risk MDS	APR-246: stabilizes mutated p53	APR-246 + aza	77	CR	1-Nov-20
A18-15331	Phase 3 open label	Intermediate- to very high-risk MDS	APR-246: stabilizes mutated p53	Aza	77	CR	1-Nov-20
HOVON 156 AML	Phase 3 open label	AML + MDS-EB2 FLT-3+	Mido and Gilt: FLT-3 inhibitor	IC + midostaurin	384	EFS	1-May-23
HOVON 156 AML	Phase 3 open label	AML + MDS-EB2 FLT-3+	Mido and Gilt: FLT-3 inhibitor	IC + gilteritinib	384	EFS	1-May-23
VERONA	Phase 3 double-blind	Intermediate- to very high-risk MDS	Venetoclax: BCL-2 inhibitor	Venetoclax + aza	250	CR and OS	26-Jan-25
VERONA	Phase 3 double-blind	Intermediate- to very high-risk MDS	Venetoclax: BCL-2 inhibitor	PBO + aza	250	CR and OS	26-Jan-25
ENHANCE	Phase 3 double-blind	Intermediate- to very high-risk MDS	Magrolimab: anti-CD47 antibody; ICPI	Magrolimab + aza	90	CR	1-Jul-25
ENHANCE	Phase 3 double-blind	Intermediate- to very high-risk MDS	Magrolimab: anti-CD47 antibody; ICPI	PBO + aza	90	CR	1-Jul-25
	Multiphase trial 1-3	MDS, AML, CMML, MDS-MPN	ASTX030: ced/aza deaminase inhibitor	Sequencing trial subcutaneous aza vs ced/aza	245	PK	1-Apr-23
LEAP	Phase 2/3 open label	MDS-EB2, AML	Midostaurin: FLT3 inhibitor nivolumab: ICPI	Midostaurin + aza	16,70	OS	1-Aug-23
				Nivolumab + aza
				Dec + LDAC
				Aza
HO-155	Phase 2 open label	MDS-EB, AML	Mido: FLT3 inhibitor	Midostaurin + dec	70	CR	1-Mar-26
HO-155	Phase 2 open label	MDS-EB, AML	Mido: FLT3 inhibitor	Dec	70	CR	1-Mar-26
Cusatuzumab combination	Phase 2 open label	Intermediate- to very high-risk MDS, CMML	Cusatuzumab: anti-CD 70 antibody	Cusatuzumab + aza	75	ORR	18-Jul-22
Cusatuzumab combination	Phase 2 open label	Intermediate- to very high-risk MDS, CMML	Cusatuzumab: anti-CD 70 antibody	Aza	75	ORR	18-Jul-22
	Phase 3	Intermediate- to very high-risk MDS	Rigosertib: Ras kinase inhibitor	Rigosertib + aza	Unknown	ORR	Unknown
	Phase 3	Intermediate- to very high-risk MDS	Rigosertib: Ras kinase inhibitor	Aza	Unknown	ORR	Unknown

CR, complete remission; EFS, event-free survival; Gilt, gilteritinib; IC, induction chemotherapy; ICPI, immune checkpoint inhibitor; LDAC, low-dose ara-c; Mido, midostaurin; MPN, myeloproliferative neoplasm; ORR, overall response rate; PBO, placebo; PFS, progression-free survival; PK, pharmacokinetics.

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