Table 1.

Summary of the main clinical interventions and results.

	Clinical development	Main results
Small molecule TGF-β inhibitors
Galunisertib (LY2157299)	PhII (NCT01373164): galunisertib + gemcitabine vs. gemcitabine in PAC	Trend to OS benefit
	PhII (NCT01246986): monotherapy in HCC	OS benefit in patients with >20% decrease in TGF-β1, AFP, and CDH1 levels from baseline
	PhII (NCT01246986): combination with sorafenib in HCC	Ongoing
	PhIb (NCT02734160): combination with durvalumab in PAC	Ongoing
	PhIb (NCT02423343): combination with nivolumab in glioblastoma, NSCLC and HCC	Ongoing
Monoclonal antibodies against TGF-β
Fresolimumab (GC1008)	PhIb (NCT00356460): monotherapy in melanoma and RCC	Preliminary evidence of antitumor activity
	PhII (NCT01401062): combination with radiotherapy in mBC	Ongoing
	PhI/II (NCT02581787): combination with radiotherapy in early stage NSCLC	Ongoing
Vaccinations strategies
Lucanix (Belagenpumatucel-L)	PhIII (NCT00676507): monotherapy as maintenance therapy in NSCLC	OS benefit in patients with prior radiotherapy and/or randomized within 12 weeks of chemotherapy completion
Vigil (Gemogenovatucel-T)	PhII/III (NCT02346747): monotherapy as maintenance in high-risk ovarian cancer	Preliminary evidence of improved relapse-free survival
	PhIIb (NCT02511132): monotherapy vs. gemcitabine + docetaxel in Ewing's sarcoma	Ongoing
	PhIII (NCT02639234): combination with nivolumab in NSCLC after platinum-based therapy	Ongoing
Antisense oligonucleotides
Trabedersen	PhIIb (NCT00761280): monotherapy vs. SOC in refractory GBM or anaplastic astrocytoma	Non significative OS benefit
	PhI/II (NCT00844064): monotherapy in melanoma	Preliminary evidence of improved overall survival

GBM: glioblastoma multiforme; HCC: hepatocellular carcinoma; mBC: metastatic breast cancer; NSCLC: non-small cell lung cancer; PAC: pancreatic adenocarcinoma; PhII: phase II trial; RCC: renal cell carcinoma.