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Evaluating the safety of azer-cel in patients with relapsed/refractory B-cell cancers

A Phase 1/1b, Open-label, Dose-escalation, Dose-expansion, Parallel Assignment Study to Evaluate Safety and Clinical Activity of PBCAR0191 (Azercabtagene Zapreleucel or "Azer-cel") in Subjects With Relapsed/Refractory (r/r) Non-Hodgkin Lymphoma (NHL) and r/r B-cell Acute Lymphoblastic Leukemia (B-ALL)

PHASE1 · Imugene Limited · NCT03666000

This study is testing a new treatment called azer-cel to see if it is safe and effective for adults with certain types of blood cancers that haven't responded to other therapies.

Quick facts

PhasePHASE1
Study typeInterventional
Enrollment135 (estimated)
Ages18 Years and up
SexAll
SponsorImugene Limited (industry)
Drugs / interventionsCAR T
Locations23 sites (Gilbert, Arizona and 22 other locations)
Trial IDNCT03666000 on ClinicalTrials.gov

What this trial studies

This Phase 1/1b study is designed to assess the safety and clinical activity of azer-cel, an allogeneic anti-CD19 CAR T-cell therapy, in adults with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma (NHL). Participants will undergo lymphodepletion before receiving an intravenous infusion of azer-cel. The study will monitor participants for safety and efficacy, with follow-up extending up to 15 years after treatment. The goal is to find an optimal dose that maximizes safety and therapeutic benefit.

Who should consider this trial

Good fit: Ideal candidates include adults with relapsed or refractory CD19+ B-ALL or aggressive B-cell NHL.

Not a fit: Patients with non-CD19+ malignancies or those who have not previously received CD19-directed therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat B-cell malignancies.

How similar studies have performed: Previous studies of CAR T-cell therapies have shown promising results in similar patient populations, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria

Criteria for B-ALL:

• Participant has confirmed unequivocal r/r CD19+ B-ALL.

Criteria for NHL and CLL/SLL:

• Participant has unequivocal aggressive CD19+ r/r B-cell NHL that is confirmed by tumor biopsy tissue from last relapse after CD19-directed therapy.

For Phase 1 Dose Escalation:

* Diffuse large B-cell lymphoma (DLBCL) including Richter's transformation
* Follicular lymphoma (FL) including Grade 3 or transformed FL
* High-grade B-cell lymphoma (HGBCL)
* Primary mediastinal lymphoma

For Phase 1b Dose Expansion (CAR T-relapsed cohort):

* DLBCL not otherwise specified (NOS)
* HGBCL
* DLBCL transformed from the following indolent lymphoma subtypes (FL, Marginal Zone lymphoma \[MZL\], and Waldenstrom's Macroglobulinemia \[WM\])
* Other large B-cell lymphoma (LBCL) subtypes may be enrolled with approval from the Medical Monitor.
* Participants previously treated with CD19-directed autologous CAR T therapies have received no more than 2 lines of therapy after administration of their previous CAR T product.
* For the expansion CAR T-relapsed cohort only: Participants must have received autologous CD19-directed CAR T therapy and demonstrated clinical response to the treatment at Day 28 or later, followed by relapse or progression.

For Phase 1b dose expansion (CAR T-naive cohort):

* DLBCL NOS
* DLBCL transformed from the following indolent lymphoma subtypes (FL, MZL, and WM)
* HGBCL
* FL (Grade 1-3a)
* MZL that is fluorodeoxyglucose (FDG)-avid on positron emission tomography (PET) scan
* WM
* CLL/SLL
* Primary central nervous system (CNS) lymphoma (PCNSL)
* Other LBCL subtypes may be enrolled with approval from the Medical Monitor.
* Participant must have received at least 1-2 prior lines of therapy, depending on histological subtype but no more than 7 systemic lines of anti-cancer therapy.

Criteria for both B-ALL, NHL, and CLL/SLL:

* Eastern Cooperative Oncology Group performance status score of 0 or 1.
* An estimated life expectancy of at least 12 weeks according to the investigator's judgment.
* Seronegative for human immunodeficiency virus antibody.
* Participant has adequate bone marrow, renal, hepatic, pulmonary, and cardiac function.

Key Exclusion Criteria

Criteria for B-ALL:

• Burkitt cell (L3 ALL) or mixed-lineage acute leukemia.

Criteria for NHL:

* Requirement for urgent therapy due to tumor mass effects such as bowel obstruction or blood vessel compression.
* Active hemolytic anemia.

Criteria for B-ALL and NHL:

* No active CNS disease, excluding PCNSL
* History of another primary malignancy
* Any form of primary immunodeficiency (for example, severe combined immunodeficiency disease).
* History of hepatitis B or hepatitis C currently receiving ongoing antiviral therapy.

Any known uncontrolled cardiovascular disease at the time of Screening that, in the investigator's opinion, renders the participant ineligible

* History of hypertension crisis or hypertensive encephalopathy within 3 months prior to Screening.
* History of severe immediate hypersensitivity reaction to any of the agents used in this study.
* Presence of a CNS disorder that, in the opinion of the investigator, renders the participant ineligible for treatment.
* History of concomitant genetic syndrome such as Fanconi anemia, Kostmann syndrome, Shwachman-Diamond syndrome, or any other known bone marrow failure syndrome.
* Active uncontrolled autoimmune disease requiring active immunosuppression at the time of Screening (excluding participants needing steroids for physiologic replacement).
* Participant has received stem cell transplant within 90 days before Screening.
* Participant has active graft-versus-host disease (GvHD) symptoms.
* Participant has received a systemic biologic agent for treatment of the disease under study within 28 days of LD, other systemic anti-cancer therapy within 10 days or 5 half-lives (whichever is shorter) of LD, and no pulse steroid for disease control within 3 days of LD.
* Radiotherapy within 4 weeks before Screening.
* Presence of pleural/peritoneal/pericardial catheter, as well as permeant biliary and ureteral stents (does not apply to intravenous lines).
* Participant has received live vaccine within 4 weeks before Screening. Note: Non-live virus vaccines are not excluded.
* Participant has received CD19-directed therapy other than autologous CD19-directed CAR T therapy within 90 days of the anticipated start date of LD.

Additional criteria apply.

Where this trial is running

Gilbert, Arizona and 22 other locations

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 →

Conditions: Non-Hodgkin Lymphoma, B-cell Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma

Last reviewed 2026-05-15 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.