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Evaluating safety of tisagenlecleucel that doesn't meet specifications

A Phase IIIb Study of the Safety and Efficacy of Tisagenlecleucel Out of Specification for Commercial Release in Patients Who Are Consistent With the Label Indication

Phase 3 Interventional Novartis · NCT04094311

This study is testing a special treatment called tisagenlecleucel to see if it is safe and works for kids and young adults with a type of leukemia and adults with a type of lymphoma, even if the treatment doesn't meet standard quality checks.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment200 (estimated)
Ages0 Years to 100 Years
SexAll
SponsorNovartis Industry-sponsored
Locations53 sites (Hamilton, Ontario and 52 other locations)
Trial IDNCT04094311 on ClinicalTrials.gov

What this trial studies

This Phase IIIb study aims to assess the safety and efficacy of tisagenlecleucel (CTL019) in pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia and adult patients with relapsed/refractory diffuse large B-cell lymphoma. The study is single-arm and open-label, focusing on patients whose manufactured tisagenlecleucel product does not meet commercial release specifications. Participants will receive a single infusion of CTL019 and will be monitored for safety and efficacy over specified follow-up periods. The study is conducted across multiple Novartis investigative sites in Ontario, Canada.

Who should consider this trial

Good fit: Ideal candidates include pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia and adult patients with relapsed/refractory diffuse large B-cell lymphoma who have received tisagenlecleucel that is out of specification.

Not a fit: Patients with active viral infections such as HIV, HBV, or HCV, or those with primary central nervous system lymphoma may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients with relapsed or refractory B-cell malignancies using tisagenlecleucel that would otherwise be discarded.

How similar studies have performed: While this approach is novel in evaluating out of specification tisagenlecleucel, similar studies have shown promise in the use of CAR T-cell therapies for treating B-cell malignancies.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key inclusion criteria:

* Signed informed consent/assent must be obtained for this study prior to participation in the study.
* Patients for whom the final manufactured tisagenlecleucel product does not meet the commercial release specifications.
* Not excluded from commercial manufacturing under the Health Authority-approved tisagenlecleucel prescribing information for their respective country/region.
* OOS material has not been deemed to pose an undue safety risk to the patient.
* Patient is suffering from a serious or life-threatening disease or condition.
* Repeat leukapheresis is not clinically appropriate per the investigator assessment.

Key exclusion criteria:

For part 1, patients meeting any of the following criteria are not eligible for inclusion in this study:

* Human immunodeficience virus (HIV) positive patients.
* Patients with active replication of Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
* Patients with primary central nervous system (CNS) lymphoma.
* History of hypersensitivity to any drugs or metabolites of similar chemical classes as tisagenlecleucel.
* Uncontrolled active infection or inflammation.
* Any medical condition identified by the investigator that may impact the assessment of the safety or efficacy outcomes in relation to study treatment.
* Pregnant or nursing (lactating) women. For part 2, exclusion criteria are not set; however, administration should be performed in accordance with the latest versions of the package insert of CTL019.

Where this trial is running

Hamilton, Ontario and 52 other locations

+3 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions B-cell Acute Lymphoblastic LeukemiaDiffuse Large B-cell Lymphomaout of specificationtisagenlecleucelacute lymphoblastic leukemialarge B-cell lymphomarelapsed/refractorypediatric
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.