Table 1.

Current targeted therapies and immunotherapies targets under evaluation in preclinical and/or clinical development in North America and Europe for rhabdomyosarcoma.

Molecular target	Drug	Phase	Clinicaltrialsregister.eu identifier (European)	Clinicaltrials.gov identifier (USA)
BRD4	JQ1, OTX015	Preclinical		NA
CHD4	ED2-AD101 (SMARCA5/CHD4 dual inhibitor)	Preclinical		NA
HDAC	Entinostat, Vorinostat	Clinical (I/II)	2008-008513-19; 2018-000127-14	NCT02780804 (Entinostat
ALK	Critotinib	Clinical (II)	2011-001988-52
PLK1	Volasertib	Preclinicial		NA
GSK3β	Tideglusib, LY2090314, 9-ING-41	Preclinical		NA
PI3K/mTOR	Omipalisib, Temsirolimus	Clinical (I/II)	2007-000371-42	NCT00106353; NCT01222715
MEK1	Cobimetinib	Clinical (I/II)	2014-004685-25
FGFR	Erdafitinib	Clinical (II)		NCT03210714; NCT03155620
IGF-1R	R1507 (mAb)	Clinical (II)	2007-003940-30	NCT00642941
VEGF	Bevacizumab (mAb)	Clinical (II)	2013-003595-12	NCT01222715
Multi-RTKs	Regorafenib	Clinical (II)	2013-003579-36	NCT01900743
SMO	LDE225, Erismodegib, Vismodegib, Sonidegib	Clinical (II)	2010-019348-37	NCT01125800
NOTCH	RO4929097, MK0572, brontictuzumab (mAb), tarextumab (mAb)	Preclinical		NA
CDK4/6	Palbociclib, Ribociclib, Abemaciclib	Preclinical		NA
Wee1	AZD1775	Clinical (I/II)		NCT02095132
PARP	Olaparib, Iniparib, Veliparib	Clinical (II)		NCT03155620; NCT03233204
Bcl-2	Venetoclax, ABT-737	Preclinical		NA
XIAP	Smac mimetics (LCL161)	Preclinical		NA
Immunotherapy
B7-H3	Enoblituzumab (mAb)	Clinical (I)		NCT02982941
HER2	Autologous HER2-specific CAR T cells	Clinical (I)		NCT00902044
EGFR	Autologogous EGFR-specific CAR T cells	Clinical (I)		NCT03618381
CTLA-4	Ipilimumab	Clinical (I/II)		NCT02304458; NCT01445379
PD-1/PD-L1	Nivolumab, Pembrolizumab, Atezolizumab	Clinical (I/II)	2014-004697-41; 2018-000127-14	NCT02304458