Table 3 Partial FDA-approved drugs and clinical trials for the treatment of nonneoplastic diseases, including NDDs and metabolic diseases

From: p53: from understanding its structure to advances in therapeutic targeting

Regimen	Identifier	Phase	Disease	N	Status	Study period	URL
Targeting the missense mutation of p53
APR-246	NCT00900614	I	Multiple cancers	36	Completed	2009/05–2010/10	https://clinicaltrials.gov/search?term=NCT00900614
APR-246	NCT02999893	I/II	Oesophageal carcinoma	5	Terminated	2017/4/11–2021/02/07	https://clinicaltrials.gov/search?term=NCT02999893
APR-246 plus aza	NCT03745716	III	MDS	154	Completed	2019/01/11–2022/01/14	https://clinicaltrials.gov/search?term=NCT03745716
	NCT03931291	II	AML or MDS	33	Completed	2019/09/16–2022/01/14	https://clinicaltrials.gov/search?term=NCT03931291
	NCT03072043	I/II	Multiple cancers	55	Completed	2017/05/18–2021/12/08	https://clinicaltrials.gov/search?term=NCT03072043
	NCT03588078	I/II	Multiple cancers	53	Unknown	2018/09/15–2021/05/15	https://clinicaltrials.gov/search?term=NCT03588078
APR-246 plus pembrolizumab	NCT04383938	I/II	Advanced solid tumor	37	Completed	2020/05/07–2025/05/13	https://clinicaltrials.gov/search?term=NCT04383938
APR-246 plus aza and venetoclax	NCT04214860	I	Myeloid malignancy	51	Completed	2019/12/13–2022/01/14	https://clinicaltrials.gov/search?term=NCT04214860
APR-246 plus PLD	NCT03268382	II	Ovarian cancer	36	Completed	2017/07/31–2019/07/10	https://clinicaltrials.gov/search?term=NCT03268382
APR-246 plus carboplatin/PLD	NCT02098343	I/II	Ovarian cancer	247	Completed	2014/3–2019/04	https://clinicaltrials.gov/search?term=NCT02098343
APR-246 plus dabrafenib	NCT03391050	I/II	Melanoma	3	Terminated	2018/01/18–2018/08/08	https://clinicaltrials.gov/search?term=NCT03391050
ATO	NCT04695223	II	Multiple cancers	30	Unknown	2021/01/01–2021/10/31	https://clinicaltrials.gov/search?term=NCT04695223
ATO	NCT04869475	II	Refractory solid tumors	20	Unknown	2021/04/20–2024/04/30	https://clinicaltrials.gov/search?term=NCT04869475
ATO plus decitabine	NCT03855371	I	MDS, AML	5	Unknown	2018/01/10–2024/07	https://clinicaltrials.gov/search?term=NCT03855371
MDM2 inhibitors
RG7112	NCT01164033	I	Neoplasms	76	Completed	2010/08–2013/05	https://clinicaltrials.gov/search?term=NCT01164033
	NCT00623870	I	Hematologic neoplasms	116	Completed	2008/05–2012/11	https://clinicaltrials.gov/search?term=NCT00623870
	NCT01677780	I	Leukemia	11	Completed	2012/11/28–2017/06/26	https://clinicaltrials.gov/search?term=NCT01677780
RG7112 plus doxorubicin	NCT01605526	I	Sarcoma	23	Completed	2012/05–2013/06	https://clinicaltrials.gov/search?term=NCT01605526
RG7112 plus ara-C	NCT01635296	I	Leukemia	43	Completed	2012/07–2013/10	https://clinicaltrials.gov/search?term=NCT01635296
RG7388	NCT03287245	I	PV	27	Terminated	2018/02/21–2020/03/03	https://clinicaltrials.gov/search?term=NCT03287245
	NCT03362723	I	Solid tumors	48	Completed	2017/11/27–2019/06/11	https://clinicaltrials.gov/search?term=NCT03362723
	NCT02828930	I	Solid tumors	8	Completed	2016/09/08–2017/06/19	https://clinicaltrials.gov/search?term=NCT02828930
RG7388 plus ara-C	NCT02545283	III	AML	447	Terminated	2015/12/30–2020/04/24	https://clinicaltrials.gov/search?term=NCT02545283
RG7388 plus selinexor	NCT05952687	I	Multiple cancers	52	Withdrawn	2024/03–2025/09	https://clinicaltrials.gov/search?term=NCT05952687
RG7388 plus dxm and ixazomib citrate	NCT02633059	I/II	MM	33	Completed	2015/12/30–2022/11/01	https://clinicaltrials.gov/search?term=NCT02633059
RG7388 plus ara-C plus daunorubicin	NCT03850535	I/II	AML	24	Terminated	2019/03/25–2020/09/10	https://clinicaltrials.gov/search?term=NCT03850535
RG7388 plus pegasys	NCT02407080	I	PV, Thrombocythemia	13	Completed	2015/04–2018/12/19	https://clinicaltrials.gov/search?term=NCT02407080
RG7388 plus immunochemotherapy	NCT03135262	I/II	Lymphoma	29	Terminated	2017/06/15–2020/04/30	https://clinicaltrials.gov/search?term=NCT03135262
Regimen	Identifier	Phase	Disease	N	Status	Study period	URL
RG7388 plus R and obinutuzumab	NCT02624986	I/II	NHL	25	Terminated	2015/12/23–2019/05/20	https://clinicaltrials.gov/search?term=NCT02624986
RG7388 plus venetoclax and cobimetinib	NCT02670044	I	AML	88	Completed	2016/03/09–2020/12/10	https://clinicaltrials.gov/search?term=NCT02670044
RG7388 plus cobimetinib and atezolizumab	NCT03566485	I/II	Breast cancer	12	Terminated	2018/07/10–2020/12/10	https://clinicaltrials.gov/search?term=NCT03566485
RG7388 plus chemotherapy	NCT04589845	II	Solid tumors	920	Recruiting	2021/01/18–2032/09/25	https://clinicaltrials.gov/search?term=NCT04589845
RG7388 plus chemotherapy	NCT03555149	I/II	Colorectal cancer	96	Terminated	2018/09/27–2022/09/26	https://clinicaltrials.gov/search?term=NCT03555149
RG7388 plus targeted compounds	NCT03158389	I/II	Glioblastoma	228	Completed	2018/05/07–2023/02/22	https://clinicaltrials.gov/search?term=NCT03158389
AMG-232	NCT01723020	I	Advanced malignancy	107	Completed	2012/12/27–2017/09/25	https://clinicaltrials.gov/search?term=NCT01723020
	NCT03107780	I	Glioblastoma	86	Active, not recruiting	2018/07/09–2026/02/07	https://clinicaltrials.gov/search?term=NCT03107780
	NCT03217266	I	Soft tissue sarcoma	46	Active, not recruiting	2018/06/20–2026/02/28	https://clinicaltrials.gov/search?term=NCT03217266
AMG-232 plus trametinib	NCT02016729	I	Advanced malignancy	36	Completed	2014/04/01–2017/07/31	https://clinicaltrials.gov/search?term=NCT02016729
AMG-232 plus trametinib and dabrafenib	NCT02110355	I	Advanced malignancy	31	Completed	2014/12/19–2018/12/27	https://clinicaltrials.gov/search?term=NCT02110355
AMG-232 plus venetoclax and decitabine	NCT03041688	I	AML	58	Active, not recruiting	2018/02/08–2026/06/30	https://clinicaltrials.gov/search?term=NCT03041688
AMG-232 plus immunotherapy	NCT03031730	I	MM	40	Terminated	2018/06/14–2024/07/24	https://clinicaltrials.gov/search?term=NCT03031730
AMG-232 plus ara-C and idarubicin	NCT04190550	I	AML	24	Active, not recruiting	2021/02/04–2026/06/30	https://clinicaltrials.gov/search?term=NCT04190550
APG-115	NCT02935907	I	Multiple cancers	50	Completed	2016/10–2019/06/28	https://clinicaltrials.gov/search?term=NCT02935907
APG-115 plus APG-2575	NCT05701306	I	Multiple cancers	100	Recruiting	2023/02/28–2027/12/31	https://clinicaltrials.gov/search?term=NCT05701306
APG-115 plus APG-2575	NCT04496349	II	T-Prolymphocytic Leukemia	36	Recruiting	2021/07/12–2027/05/31	https://clinicaltrials.gov/search?term=NCT04496349
APG-115 plus toripalimab	NCT04785196	I/II	Advanced solid tumor	95	Recruiting	2021/05/26–2027/01	https://clinicaltrials.gov/search?term=NCT04785196
APG-115 plus 5-aza	NCT04358393	I/II	AML, MDS, CMML	69	Recruiting	2020/12/04–2025/12/30	https://clinicaltrials.gov/search?term=NCT04358393
APG-115 plus pembrolizumab	NCT03611868	I/II	Advanced solid tumors	224	Recruiting	2018/08/29–2025/03/30	https://clinicaltrials.gov/search?term=NCT03611868
APG-115 plus carboplatin	NCT03781986	I/II	Gland cancer	34	Active, not recruiting	2019/10/28–2025/12	https://clinicaltrials.gov/search?term=NCT03781986
APG-115 plus ara-C and aza	NCT04275518	I	AML, MDS	102	Recruiting	2020/07/06–2025/12/31	https://clinicaltrials.gov/search?term=NCT04275518
Dual MDM2 and MDM4 inhibitors
ALRN-6924	NCT02264613	I/II	Multiple cancers	149	Completed	2014/10–2020/04	https://clinicaltrials.gov/search?term=NCT02264613
ALRN-6924 plus ara-C	NCT02909972	I	AML, MDS	55	Completed	2016/09–2019/08	https://clinicaltrials.gov/search?term=NCT02909972
ALRN-6924 plus ara-C	NCT03654716	I	Pediatric cancer	21	Completed	2018/11/01–2023/07/17	https://clinicaltrials.gov/search?term=NCT03654716
Cancer Immunotherapy targeting p53
p53-SLP	NCT01639885	I/II	Recurrent ovarian cancer	15	Completed	2011/08–2014/01	https://clinicaltrials.gov/search?term=NCT01639885
p53-SLP plus cyclophosphamide	NCT00844506	II	Ovarian cancer	19	Completed	2008/10–2009/07	https://clinicaltrials.gov/search?term=NCT00844506
MVAp53	NCT02432963	I	Multiple cancers	11	Active, not recruiting	2016/06/14–2025/10/31	https://clinicaltrials.gov/search?term=NCT02432963
MVAp53	NCT01191684	I	Multiple cancers	12	Completed	2011/10–2013/08	https://clinicaltrials.gov/search?term=NCT01191684
MVAp53 plus gemcitabine	NCT02275039	I	Multiple cancers	12	Completed	2015/01–2018/04/23	https://clinicaltrials.gov/search?term=NCT02275039
MVAp53 plus pembrolizumab	NCT03113487	II	Multiple cancers	29	Active, not recruiting	2018/2/1–2025/06/03	https://clinicaltrials.gov/search?term=NCT03113487
DC–p53 plus ipilimumab or nivolumab	NCT03406715	II	SCLC	14	Terminated	2018/3/15–2022/05/16	https://clinicaltrials.gov/search?term=NCT03406715
Tedopi plus nivolumab and docetaxel	NCT04884282	II	NSCLC	105	Recruiting	2021/10/12–2025/05/17	https://clinicaltrials.gov/search?term=NCT04884282
Gene therapy targeting p53
SGT-53	NCT00470613	I	Neoplasm	25	Completed	2008/02–2016/12	https://clinicaltrials.gov/search?term=NCT00470613
SGT-53 plus paclitaxel and gemcitabine	NCT02340117	II	Pancreatic cancer	28	Active, not recruiting	2015/01–2026/12	https://clinicaltrials.gov/search?term=NCT02340117

Aza azacitidine, MDS myelodysplastic syndromes, AML acute myeloid leukemia, PLD pegylated liposomal doxorubicin hydrochloride, Ara-C cytarabine, PV polycythemia vera, CNS central nervous system, Dxm dexamethasone, MM multiple myeloma, R rituximab, NHL non-Hodgkin’s lymphoma, CMML chronic myelomonocytic leukemia, SCLC small-cell lung cancer, NSCLC non-small-cell lung cancer

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Table 3 Partial FDA-approved drugs and clinical trials for the treatment of nonneoplastic diseases, including NDDs and metabolic diseases

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