Table 2.

Tisagenlecleucel: past, current, and projected trials (pivotal trials leading to approval are shown in Table 1).

Primary disease	Primary study population	Description	Publication	Main sponsor	Clinical trial identifier	Alias	Design	Location	Phase	Actual or estimated enrollment	Status
B-ALL
R/R B-ALL	Adult	Randomized comparison blinatumomab and inotuzumab		Novartis		OBERON	Multicenter	Multinational	III	220	NYR
B-ALL	Pediatric	tisagenlecleu cel if MRD+ following first line treatment		Novartis		CASSI OPEIA	Single center	UPENN/CHOP	II	140	R
R/R B-ALL	Pediatric	Optimization study of tocilizumab for CRS		UPENN			Single center	UPENN/CHOP	Pilot	80	ANR
R/R B-ALL	Pediatric	Expanded access/compassionate use		Novartis			Multicenter	Multinational	II
R/R B-ALL & DLBCL	Adult & pediatric	tisagenlecleu cel-axicabtagene ciloleucel head-to-head in DLBCL		MCC			Single center	MCC	II	120	R
R/R B-ALL & DLBCL	Pediatric	CTL119		UPENN			Single center	ACC/UPENN
B-ALL & DLBCL	Adult & pediatric	managed access program		Novartis			Multicenter	United States	NA	NA	NA
R/R B-ALL	Adult	Allogeneic CART19		UPENN			Single center	ACC/UPENN	I	2	Completed
R/R B-ALL	Adult			UPENN			Single center	ACC/UPENN	II	30	Completed
R/R B-ALL	Pediatric	CTL119 for very high risk subsets of pediatric B-ALL		UPENN			Single center	UPENN/CHOP	II	85	R
R/R B-ALL	Adult	CART22 alone or in combination with CTL119		UPENN			Single center	UPENN	I	18	R
Lymphoma
R/R NHL	Pediatric			Novartis		BIANCA	Multicenter	United States & Spain	II	35	R
R/R NHL	Adult			Novartis		BELINDA	NA	NA	III	318	NYR
R/R DLBCL	Adult	tisagenlecleu cel-ibrutinib combination therapy		Novartis			Single center	UPENN	Ib	40	NYR
R/R NHL	Adult	Pembrolizum ab to subjects failing or relapsing post-tisagenlecleu cel	[133]	UPENN			Single center	ACC/UP	I/II	12	NA
R/R NHL	Adult	FDG-PET response post-CART19		UPENN			Single center	ACC/UP ENN	I	9	Completed
R/R DLBCL	Adult	tisagenlecleu cel-pembrolizum ab combination therapy		Novartis		PORTIA	Multicenter	United States & Austria	Ib	32	R
R/R FL	Adult			Novartis		ELARA	Multicenter	United States & Australia	II	113	R
CLL
R/R CLL	Adult	CTL119 + ibrutinib	[131]	UPENN			Single center	ACC/UPENN	Pilot	20	ANR
R/R CLL/SLL	Adult	Randomization between two different doses	[128, 171]	UPENN			Single center	ACC/UPENN	II		Completed
B cell malignancies (all kinds)
R/R	All ages	Determine safety and maximum tolerated dose of tisagenlecleu cel		MDA/NCI			Single center	MDA	I	26	ANR
	All ages	Long term follow up All ages CD19-directed CART		Novartis			Multicenter	Global	NA	620	R
Multiple myeloma
	Adult	CART19 day 2 post-ASCT following early relapse	[172, 173]	UPENN			Single center	ACC/UPENN	I	13	Completed
	Adult	CART19 day 60 post-ASCT		UPENN			Single center	UPENN/ACC	II	5	Terminated
Relapsed	Adult	Up-front BCMA-CART alone or with CTL119		UPENN			Single center	UPENN	I	39	R
Solid cancer
Pancreatic cancer	Adult	tisagenlecleu cel to prolong meso-specific CART response		UPENN/UCSF			Single center	UCSF	I	4	Completed

R/R, relapsed/refractory. CLL, chronic lymphocytic leukemia. B-ALL, B-cell acute lymphoblastic leukemia. DLBCL, diffuse large B cell lymphoma. FL, follicular lymphoma. MM, multiple myeloma. NHL, non-Hodgkin lymphoma. MRD, minimal residual disease. UPENN, University of Pennsylvania. CHOP, Childrens Hospital of Philadelphia. ACC, Abramson Cancer Center. ASCT, autologous stem cell transplant. MCC, Masonic Cancer Center, University of Minnesota. MDA, MD Anderson Cancer center. NCI, National Cancer Institute. UCSF, University of California - San Francisco. NA, not available/not applicable. NYR, not yet recruiting. R, recruiting, ANR, active, not recruiting. CART, chimeric antigen receptor T cell.

Novel information added