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. 2023 May 15;19(1):2210046. doi: 10.1080/21645515.2023.2210046

Table 1.

List of pivotal studies to support registration of tisagenlecleucel.

Study population Pivotal study for registration First patient first visit Protocol-specified dose range for single infusion Response rate/Efficacy outcomes
Pediatric and young adult patients with B-cell ALL who are chemo-refractory, have relapsed after allogeneic SCT, or are otherwise ineligible for allogeneic SCT CTL019B2202 (ELIANA), pivotal; CTL019B2205J (ENSIGN), supportive April 8, 2015

  • 0.2 to 5.0 × 106 cells/kg for patients ≤50 kg

  • 0.1 to 2.5 × 108 cells for patients >50 kg

 N = 79 infused (ELIANA)

  • ORR (CR or CR with incomplete hematologic recovery) within 3 months after infusion, 82% (95% CI, 72–90)

  • 5-year RFS*: 49% (95% CI, 34–62)

  • 5-year OS*: 55% (95% CI, 43–66)


*median not reached
Reference: Rives et al.1
Adult patients with r/r DLBCL after failure of two or more lines of therapy who are not eligible for HSCT CTL019C2201 (JULIET) July 29, 2015

  • 0.1 to 6.0 × 108 cells

 N = 115 infused

  • ORR (CR+PR): 53% (95% CI: 43.5–62.4)

  • CR: 39%

  • Median PFS: 2.9 months* (95% CI: 2.3–5.2)

  • Median OS: 11.1 months* (95% CI: 6.6–23.9)


*median not reached for patients who had CR at 3 months or 6 months
Reference: Schuster et al.2
Adult patients with r/r FL grade 1, 2, or 3A after failure of two or more lines of therapy CTL019E2202 (ELARA) November 12, 2018

  • 0.6 to 6.0 × 108 cells

 N = 97 infused

  • ORR (CR+PR): 86% (95% CI, 78–92)

  • CRR: 68% (95% CI, 58–77))

  • DOR at 24 months: 66% (95% CI, 54–76) for all patients and 78% (95% CI, 65–87) for patients in CR

  • PFS at 24 months for all patients: 57% (95% CI, 46–67)

  • OS at 24 months for all patients: 88% (95% CI, 78–93)


Reference: Dreyling et al.3

ALL, acute lymphoblastic leukemia; CI, confidence interval; CR, complete response; CRR, complete response rate; DLBCL, diffuse large B-cell lymphoma; DOR, duration of response; FL, follicular lymphoma; HSCT, hematopoietic stem cell transplantation; ORR, overall response rate; OS, overall survival; PFS, progression-free survival; PR, partial response; RFS, relapse-free survival; r/r, relapsed or refractory; SCT, stem cell transplantation.