Table 2.

Some examples of clinical trials of ITZ on different cancers

Cancer	Patients No.	Study type	Therapeutic regimen of ITZ	Reported pathway	Treatment effects	Adverse effects	References
Basal cell carcinoma	29 (19 treated, 10 controls)	Phase 2	200 mg twice daily for 1 month, or 100 mg twice daily for average 2.3 months	Hh pathway inhibition	Cell proliferation inhibited by 45%, and tumor area reduced by 24%	Fatigue (grade 2), and heart failure (grade 4)	6
Metastatic basal cell carcinoma	5	Phase 2	400 mg daily on days 6–18, with arsenic trioxide on days 1–5	Hh pathway inhibition	GLI1 mRNA reduced by 75%	Fatigue (grade 2), and heart failure (grade 4)	107
Advanced basal cell carcinoma	1	Case report	200 mg daily for 2 weeks with 200 mg daily of sonidegib after failure with vismodegib	Hh pathway inhibition	Tumor regressed	—	7
Stage III unresectable pancreatic adenocarcinoma	1	Case report	200 mg daily for 9 months	Hh pathway inhibition	Tumor size reduced and resected	—	3
Metastatic pancreatic cancer	38	Retrospective analysis	400 mg daily on days −2 to +2 in combination with other chemotherapy (docetaxel, gemcitabine and carboplatin) every 2 weeks for 3–11 cycles	Hh pathway inhibition, angiogenesis and P-gp inhibition	The mean overall survival was 11.4 months	—	103
Biochemically recurrent prostate cancer	1	Case report	600 mg daily for 5 months	—	PSA fell by >50%	Hypoaldosteronism, hyperbilirubinaemia	98
Metastatic castration-resistant prostate cancer	46 (17 in low dose group, 29 in high dose group)	Phase 2	Low dose: 200 mg daily; High dose: 600 mg daily until disease progression or toxicity	Hh pathway inhibition	Disease progression in 15 (low dose) and 22 (high dose)	Fatigue, anorexia, rash, hypertension, and hypokalaemia (all in Grade 3)	11
Progressive nonsquamous non-small -cell lung cancer	23 (15 treated with ITZ, 8 controls)	Phase 2	pemetrexed 500 mg/m2 on day 1, with or without ITZ 200 mg daily, on a 21-day cycle, for 3 months	—	Overall survival longer	—	104
Recurrent clear cell ovarian carcinoma	9	Retrospective analysis	400 mg daily on days −2 to +2 with chemotherapy (docetaxel and carboplatin) on day 1, repeated every 2 weeks	—	The overall survival was 1047 days, and progression free survival was 544 days	Deranged liver function (Grade 1), and anorexia (Grade 2)	95
Recurrent triple-negative breast cancer	13	Retrospective analysis	400 mg daily on days −2 to +2 with chemotherapy (docetaxel and carboplatin) on day 1, repeated every 2 weeks	—	The overall survival was longer for 20.4 months, and progression free survival was 10.8 months	Fatigue, insomnia, nausea, and vomiting	102

Abbreviations: ITZ, itraconazole; Hh pathway, hedgehog pathway; GLI, glioma-associated oncogene homolog.