Table 4. TCR T-cell therapy for clinical... | American Society of Hematology

Table 4.

TCR T-cell therapy for clinical trials for AML

Trial reference	Phase/status	Product details				Disease	Disease type	Patients treated	Response	Persistence	Significant adverse events
UMIN000011519¹⁴⁷ PMID: 28860210	Phase 1 Completed	Retroviral vector with siRNA knock down of endogenous TCR, WT1 peptide vaccine	Auto	WT1	A∗24:02	Refractory AML, MDS	Active	8 (2 AML)	2/8 showed decreased blast counts in the BM (predicted leukemia regression). 2 patients with AML did not respond.	Yes, at 5 mo after treatment (4/5 patients survived for ≥12 mo).	No serious adverse events reported.
NCT01621724 EudraCT-2006-004950-25	Phase 1/2 Completed	Retroviral vector, IL-2 standard conditioning	Auto	WT1	A∗02:01	AML, CML	Active	7	4 patients showed disease responsiveness. No response in 3 patients.	Yes, for 4/7 patients at 1 y after treatment.	Febrile neutropenia (1). Anemia (1).
NCT01640301¹⁴⁸ PMID: 31235963	Phase 1/2 Terminated	EBV-specific CD8⁺ T cells, Additional IL-2 injection	Allo	WT1	A∗02:01	AML (recurrent/secondary) having undergone allo-HSCT (with no evidence of disease)	In remission	12	Maintenance of remission at median of 44 mo for 12 patients.	Yes, for 4/12 patients at 1 y after treatment.	CRS grade 3 (2). Neutropenia (2). Thrombocytopenia (2). Lymphopenia (12). Anemia (7).
NCT02550535¹¹⁵ EudraCT-2014-003111-10	Phase 1/2 Completed	Retroviral vector, Additional IL-2 injection	Auto	WT1	A∗02:01	AML, MDS	In remission	10 (AML)	Median survival of 12 mo in 6 patients with AML.	Yes, for 7/10 patients over 12 mo.	CRS (1).
NCT05066165	Phase 1/2 Terminated	CRISPR/Cas9	Auto	WT1	A∗02:01	AML	Active	2	Disease progression in both patients.	Not reported.	Febrile neutropenia (1).
NCT02770820	Phase 1/2 Terminated	EBV-specific CD8⁺ TCM/TN T cells, Additional IL-n2 injection	Auto	WT1	A∗02:01	High-risk non-M3 AML (with prior consolidation chemotherapy)	In remission	7	Not reported.	Not reported.	No serious adverse events reported.
NCT03503968¹⁴⁹ EudraCT-2017-000440-18	Phase 1/2 Terminated	-	Auto	PRAME	A∗02:01	AML, MDS, MM	Active	9	No disease progression for 1 patient. Remission at 4 wk followed by progression at 3 mo for 1 patient. Disease progression in remaining patients (7/9).	Yes, for 6/8 patients at 4 wk.	CRS grades 1-2 (2). Other SAE (not specified) (5).
NCT02743611	Phase 2/2 Terminated	Includes safety switch activated with rimiducid	Auto	PRAME	A∗02:01	Relapsed AML, MDS, uveal melanoma	Active	4	Not reported.	Not reported.	Neutropenic fever, tachypnea, CRS, pseudomonas bacteremia infection, neurotoxicity, orthostatic hypotension (1).
NCT03326921¹⁵⁰ PMID: 38683966	Phase 1 Suspended	CD8⁺ CD4⁺ TM T cells	Allo	HA-1 HA-1 (H) genotype (RS_1801284: A/G, A/A)	A∗02:01	Pediatric and adult leukemias after allo-HCT	Active/in remission	9	Reduction of marrow blasts lasting >30 days (2/9). Sustained remission (2/9). Disease progression in remaining patients (5/9).	Yes, for 8/9 patients beyond 1 y after treatment.	Neutropenia (5). Fever (3). Infection (2). Infusion reaction (1).
EudraCT-2010-024625-20¹⁵¹ PMID: 32973756	Phase 1 Terminated	EBV- or CMV-specific T cells retroviral vector	Allo	HA-1	A∗02:01	High-risk leukemia, after allo-HCT	Active (1)/in remission (4)	5	Maintenance of relapse-free survival at follow-up in 2 patients. Disease progression in 3 patients.	Yes, for 2 patients up to 21 wk.	Neutropenia. Thrombocytopenia (1).
NCT05473910¹⁵²^,¹⁵³	Phase 1 Recruiting	-	Allo	HA-1 or HA-2	A∗02:01	AML, MDS, ALL undergoing haplo-identical allo-HCT	In remission	7	Maintenance of remission at a median follow-up of 162 d in all patients (at time of reporting).	Yes, ongoing persistence at longest follow-up of 203 d.	GVHD (4).
NCT06424340	Phase I/II Recruiting	-	Auto	dNPM1	A∗02:01	AML (relapsed or refractory)	Active	-	Study ongoing	Study ongoing	Study ongoing

Trial reference	Phase/status	Product details				Disease	Disease type	Patients treated	Response	Persistence	Significant adverse events
UMIN000011519¹⁴⁷ PMID: 28860210	Phase 1 Completed	Retroviral vector with siRNA knock down of endogenous TCR, WT1 peptide vaccine	Auto	WT1	A∗24:02	Refractory AML, MDS	Active	8 (2 AML)	2/8 showed decreased blast counts in the BM (predicted leukemia regression). 2 patients with AML did not respond.	Yes, at 5 mo after treatment (4/5 patients survived for ≥12 mo).	No serious adverse events reported.
NCT01621724 EudraCT-2006-004950-25	Phase 1/2 Completed	Retroviral vector, IL-2 standard conditioning	Auto	WT1	A∗02:01	AML, CML	Active	7	4 patients showed disease responsiveness. No response in 3 patients.	Yes, for 4/7 patients at 1 y after treatment.	Febrile neutropenia (1). Anemia (1).
NCT01640301¹⁴⁸ PMID: 31235963	Phase 1/2 Terminated	EBV-specific CD8⁺ T cells, Additional IL-2 injection	Allo	WT1	A∗02:01	AML (recurrent/secondary) having undergone allo-HSCT (with no evidence of disease)	In remission	12	Maintenance of remission at median of 44 mo for 12 patients.	Yes, for 4/12 patients at 1 y after treatment.	CRS grade 3 (2). Neutropenia (2). Thrombocytopenia (2). Lymphopenia (12). Anemia (7).
NCT02550535¹¹⁵ EudraCT-2014-003111-10	Phase 1/2 Completed	Retroviral vector, Additional IL-2 injection	Auto	WT1	A∗02:01	AML, MDS	In remission	10 (AML)	Median survival of 12 mo in 6 patients with AML.	Yes, for 7/10 patients over 12 mo.	CRS (1).
NCT05066165	Phase 1/2 Terminated	CRISPR/Cas9	Auto	WT1	A∗02:01	AML	Active	2	Disease progression in both patients.	Not reported.	Febrile neutropenia (1).
NCT02770820	Phase 1/2 Terminated	EBV-specific CD8⁺ TCM/TN T cells, Additional IL-n2 injection	Auto	WT1	A∗02:01	High-risk non-M3 AML (with prior consolidation chemotherapy)	In remission	7	Not reported.	Not reported.	No serious adverse events reported.
NCT03503968¹⁴⁹ EudraCT-2017-000440-18	Phase 1/2 Terminated	-	Auto	PRAME	A∗02:01	AML, MDS, MM	Active	9	No disease progression for 1 patient. Remission at 4 wk followed by progression at 3 mo for 1 patient. Disease progression in remaining patients (7/9).	Yes, for 6/8 patients at 4 wk.	CRS grades 1-2 (2). Other SAE (not specified) (5).
NCT02743611	Phase 2/2 Terminated	Includes safety switch activated with rimiducid	Auto	PRAME	A∗02:01	Relapsed AML, MDS, uveal melanoma	Active	4	Not reported.	Not reported.	Neutropenic fever, tachypnea, CRS, pseudomonas bacteremia infection, neurotoxicity, orthostatic hypotension (1).
NCT03326921¹⁵⁰ PMID: 38683966	Phase 1 Suspended	CD8⁺ CD4⁺ TM T cells	Allo	HA-1 HA-1 (H) genotype (RS_1801284: A/G, A/A)	A∗02:01	Pediatric and adult leukemias after allo-HCT	Active/in remission	9	Reduction of marrow blasts lasting >30 days (2/9). Sustained remission (2/9). Disease progression in remaining patients (5/9).	Yes, for 8/9 patients beyond 1 y after treatment.	Neutropenia (5). Fever (3). Infection (2). Infusion reaction (1).
EudraCT-2010-024625-20¹⁵¹ PMID: 32973756	Phase 1 Terminated	EBV- or CMV-specific T cells retroviral vector	Allo	HA-1	A∗02:01	High-risk leukemia, after allo-HCT	Active (1)/in remission (4)	5	Maintenance of relapse-free survival at follow-up in 2 patients. Disease progression in 3 patients.	Yes, for 2 patients up to 21 wk.	Neutropenia. Thrombocytopenia (1).
NCT05473910¹⁵²^,¹⁵³	Phase 1 Recruiting	-	Allo	HA-1 or HA-2	A∗02:01	AML, MDS, ALL undergoing haplo-identical allo-HCT	In remission	7	Maintenance of remission at a median follow-up of 162 d in all patients (at time of reporting).	Yes, ongoing persistence at longest follow-up of 203 d.	GVHD (4).
NCT06424340	Phase I/II Recruiting	-	Auto	dNPM1	A∗02:01	AML (relapsed or refractory)	Active	-	Study ongoing	Study ongoing	Study ongoing

BM, bone marrow, CRS, cytokine release syndrome; MDS, myelodysplastic syndrome; MM, myelodysplastic syndrome; SAE, severe adverse event; siRNA, small interfering RNA; T_CM, central memory T cell; T_M, memory T cell; T_N, naive T cell.

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