. 2021 Nov 5;13(21):5555. doi: 10.3390/cancers13215555

Table 2.

List of antisense oligonucleotides in clinical trials.

No	Drug Name	Target	Condition	Clinical Trial Number	Development Stage	Chemistry	Ref.
1	Apatorsen (OGX-427)	Hsp27	Prostate, ovarian, NSCLC, breast or bladder cancer	NCT00487786	Phase I	2′-MOE PS	[99]
			Prostate cancer	NCT01120470	Phase II		[104]
			Prostate cancer	NCT01681433	Phase II		[154]
			Urologic neoplasms	NCT01454089	Phase II		[100]
			Urothelial carcinoma	NCT01780545	Phase II		[101]
			Non-squamous NSCLC	NCT01829113	Phase II		[105]
			Advanced squamous cell lung cancers	NCT02423590			[103]
			Pancreatic cancer	NCT01844817	Phase II		[102]
2	AZD4785	KRAS	NSCLC/advanced solid tumors	NCT03101839	Phase I	2′-4′ cEt	[107]
3	AZD5312 (ARRx)	Androgen receptor	Androgen receptor-dependent advanced solid tumors	NCT02144051	Phase I	PS cEt	[108]
3	AZD5312 (ARRx)	Androgen receptor	Prostate cancer/solid tumors	NCT03300505	Phase Ib/II (recruiting)	PS cEt	[109]
4	AZD9150	STAT3	DLBCL/NHL	NCT01563302	Phase I/II	cEt gapmer	[110]
			DLBCL/NHL	NCT03527147	Phase I		[155]
			HCC and metastasis	NCT01839604	Phase I		[156]
			Advanced solid malignancies	NCT03394144	Phase I		[157]
			DLBCL	NCT02549651	Phase Ib		[112]
			Advanced solid tumors	NCT03421353	Phase I		Ongoing
			Muscle-invasive bladder cancer	NCT02546661	Phase I		Ongoing
			Metastatic NSCLC	NCT03819465	Phase I		Ongoing
			NSCLC	NCT03334617	Phase II		Ongoing
			Advanced solid tumors and metastatic squamous cell carcinoma of the head and neck	NCT02499328	Phase II		Ongoing
			Advanced pancreatic, NSCLC, and mismatch repair-deficient CRC	NCT02983578	Phase II		Ongoing
5	BP1001	Grb2	Recurrent adult AML/ALL Myelodysplastic syndrome Ph1-positive CML	NCT01159028	Phase I	DNAbilize^® technology	[113]
			Solid tumors	NCT04196257	Phase I		Ongoing
			AML	NCT02781883	Phase II		Ongoing
6	c-myb AS ODN	c-myb	Hematologic malignancies	NCT00780052	Phase I	PS	[158]
6	c-myb AS ODN	c-myb	Leukemia	NCT00002592	Phase II	PS	[116]
7	EZN-2968	HIF-1	Carcinoma/lymphoma	NCT00466583	Phase I	LNA	[118]
			Liver metastases/neoplasms	NCT01120288	Phase I		[119]
			HCC	NCT02564614	Phase I		[120]
8	G3139 (Oblimersen)	Bcl-2	Waldenström Macroglobulinemia	NCT00062244	Phase 1 Phase 2	PS	[159]
			Merkel cell carcinoma	NCT00079131	Phase 2		[160]
			Solid tumors	NCT00003103	Phase 1 Phase 2		[121]
				NCT00054548	Phase 1		[161]
				NCT00543231	Phase 1
				NCT00636545	Phase 1		[161]
			Relapsed or refractory solid tumors	NCT00039481	Phase 1		[162]
			Leukemia (AML or ALL)	NCT00004862	Phase 1		[122]
			AML	NCT00039117	Phase 1		[163]
				NCT00017589	Phase 2		[125]
				NCT00085124	Phase 3		[131]
			CRC	NCT00004870	Phase 1 Phase 2		[124]
			CRC	NCT00055822	Phase 1 Phase 2		[164]
			Prostate cancer	NCT00085228	Phase 2		[165]
			HCC	NCT00047229	Phase 2		[166]
			Recurrent SCLC	NCT00005032	Phase 1 Phase 2		[123]
			Extensive stage SCLC	NCT00017251	Phase 1		[128]
			Extensive stage SCLC	NCT00042978	Phase 2		[128]
			CML	NCT00049192	Phase 2		[167]
			NHL	NCT00086944	Phase 1 Phase 2		[168]
			Recurrent B-cell NHL	NCT00054639	Phase 2		[168]
			CLL	NCT00078234	Phase 1 Phase 2		[169]
				NCT00021749	Phase 1 Phase 2		[129]
				NCT00024440	Phase 3		[130]
			Metastatic RCC	NCT00059813	Phase 2		[170]
			Advanced esophageal, gastro-esophageal junction and gastric cancer	NCT00064259	Phase 1 Phase 2		[171]
			Melanoma	NCT00409383	Phase 1		Unknown
				NCT00542893	Phase 1		[172]
				NCT00518895	Phase 3		[173]
				NCT00016263	Phase 3		[126]
				NCT00070343	Not applicable		Ongoing
			NSCLC	NCT00030641	Phase 2 Phase 3		Ongoing
			Multiple myeloma and plasma cell neoplasm	NCT00049374	Phase 2		[174]
			Multiple myeloma and plasma cell neoplasm	NCT00017602	Phase 3		[127]
			DLBCL	NCT00070083	Phase 1
9	GTI-2040	R2 component of RNR	Breast cancer	NCT00068588	Phase 2	PS	[135]
			AML	NCT00070551	Phase 1		[175]
			AML	NCT00565058	Phase 2		[136]
			Acute leukemia, high-grade myelodysplastic syndromes, or refractory or blastic phase CML	NCT00459212	Phase 1		[176]
			RCC	NCT00056173	Phase 1 Phase 2		[134]
			NSCLC, prostate cancer, or other solid tumors	NCT00074022	Phase 1 Phase 2		[137]
			CRC or other solid tumors	NCT00084643	Phase 1		[138]
			Prostate cancer	NCT00087165	Phase 2		[139]
			Metastatic or unresectable solid tumors	NCT00078962	Phase 1		[177]
10	ISIS 3521	Pkc-Alpha	NSCLC/ melanoma (skin)	NCT00003989	Phase II	PS
			NSCLC	NCT00017407	Phase III
				NCT00042679	Phase II		[178]
				NCT00034268	Phase III		[142]
11	ISIS 5132	C-raf	Ovarian cancer	NCT00003892	Phase II	PS	[144]
12	ISIS 3521 + ISIS 5132	Pkc-Alpha C-raf	Breast cancer	NCT00003236	Phase II		[179]
13	LErafAON	Raf-1	Advanced solid tumors	NCT00024661	Phase I	Liposome encapsulated PS	[146]
				NCT00100672	Phase I		[180]
				NCT00024648	Phase I		[147]
14	OGX-011	Clusterin	Cancer	NCT01497470	Phase I	2′-MOE PS	[181]
			NSCLC	NCT00138658	Phase I/II		[182]
			NSCLC	NCT01630733	Phase III		Ongoing
			Prostate cancer	NCT00054106	Phase I		[148]
				NCT00258388	Phase II		[151]
				NCT00138918	Phase II		[181]
				NCT00327340	Phase II		[152,183]
				NCT01578655	Phase III		[153]
				NCT01188187	Phase III		[184,185]
			Breast cancer	NCT00258375	Phase II		[150]
			Solid tumors	NCT00471432	Phase I		[149]

Hepatocellular carcinoma—HCC; small cell lung cancer—SCLC; non-small cell lung cancer—NSCLC; diffuse large B cell lymphoma—DLBCL; non-Hodgkin’s lymphoma—NHL; colorectal cancer—CRC; acute myeloid leukemia—AML; acute lymphoblastic leukemia—ALL; chronic myelogenous leukemia—CML; chronic lymphocytic leukemia—CLL; renal cell carcinoma—RCC.