Table 1.

Clinical trials of AZNs in both hematologic malignancies and solid tumors.

Study	Identifier	Study Population	Intervention(s)	Primary Outcome Measure(s)	Primary Outcome Measure Results
Jabbour et al., 2017 [74]	NCT1720225	IPSS low- or intermediate 1-risk MDS or CMML	Decitabine 20 mg/m2 IV 1 h daily for three days OR azacitidine 75 mg/m2 IV 1 h daily or subcutaneously for 3 days (repeated every 4 weeks)	Overall response rate (ORR)	Decitabine ORR: 70% Azacitidine ORR: 49% (p = 0.03)
Sallman et al., 2021 [75]	NCT03745716	IPSS low- or intermediate 1-risk MDS, at least one TP53 mutation and isolated deletion of 5q, failure of prior treatment with at least 4 full cycles of lenalidomide	Dose escalation of APR-246 via IV starting at 50 mg/kg lean body weight; azacitidine administered subcutaneously or via IV at 75 mg/m2	Complete response (CR) rate	CR for MDS patients:50% CR for AML patients:36%
Kuang et al., 2022 [76]	NCT02260440	Histologically confirmed metastatic colorectal cancer previously treated with currently approved standard therapies	Pembrolizumab 200 mg every 21 days; azacitidine 100 mg subcutaneous injection on days 1–5 every 21 days	Overall response rate (ORR)	ORR: 3%
Oza et al., 2020 [77]	NCT01696032	Platinum-resistant histologically or cytologically confirmed recurrent ovarian cancer; high-grade serous or grade 2–3 endometroid/mixed cell/clear cell epithelial ovarian cancer; primary peritoneal carcinoma; fallopian tube cancer	Guadecitabine 30 mg/m2 subcutaneously once-daily on days 1–5 and carboplatin IV AUC 4 on Day 8 OR treatment of choice	Progression free survival (PFS)	Median PFS: 16.3 vs. 9.1 weeks, p = 0.076-month PFS rate: 37% vs. 11%, p = 0.003
Tawbi et al., 2013 [78]	NCT00715793	Non-resectable stage IIIB/C or stage IV metastatic melanoma; either no prior therapy or have progressed despite prior therapies	Decitabine 0.075 or 0.15 mg/kg IV daily for 5 days/week for 2 weeks; TMZ orally 75 mg/m2 for weeks 2–5 of a 6-week cycle	Overall response rate (ORR)	ORR: 18%
Olivieri et al., 2011 [79]	NCT00661726	Beta-thalassemia as confirmed by DNA testing; transfusion independent for at least 120 days; red blood cell folate levels above lower limit of normal	Decitabine 0.2 mg/kg subcutaneously twice a week for 12 weeks	Number of patients with increase in Hb at least 1.5 g/dL and change in total hemoglobin from baseline to peak	# of patients: 2/5 Change in Hb: 7.88 +/−0.88 g/dL (baseline) to 9.04 +/−0.77 g/dL (peak)
Tang et al., 2021 [80]	NCT02487563	Platelet count ≤ 30 × 109/L persistently at day 60 post-HSCT or later; neutrophil and hemoglobin were well recovered; full donor chimerism was achieved; no response to conventional treatments for a duration of at least 4 weeks;	Low-dose decitabine (15 mg/m2 daily IV for 3 consecutive days [days 1–3]) plus recombinant human thrombopoietin (300 U/kg daily); decitabine alone; or conventional treatment	Response rate of platelet recovery	Combination: 66.7% Decitabine alone: 73.3% Conventional: 19.4% (p < 0.001)