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Dose-escalation, Dose-expansion Study of Safety of Azer-cel (PBCAR0191) in Patients With r/r NHL and r/r B-cell ALL

I

Imugene

Status and phase

Enrolling
Phase 1

Conditions

B-cell Acute Lymphoblastic Leukemia
Non-Hodgkin Lymphoma

Treatments

Drug: Cyclophosphamide
Drug: Fludarabine
Drug: IL-2
Biological: Azer-cel
Drug: Bendamustine

Study type

Interventional

Funder types

Industry

Identifiers

NCT03666000
PBCAR0191-01

Details and patient eligibility

About

This is a Phase 1/1b, nonrandomized, open-label, parallel assignment, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of azer-cel, an allogeneic anti-CD19 CAR T, in adults with r/r B ALL and r/r B-cell NHL.

Full description

This is a multicenter, nonrandomized, open-label, parallel assignment, dose-escalation, and dose-expansion study to evaluate the safety and tolerability, find an appropriate dose to optimize safety and efficacy, and evaluate clinical activity of azer-cel in participants with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma (NHL). Before initiating azer-cel, participants will be administered lymphodepletion (LD). At Day 0 of the Treatment Period, participants will receive an intravenous (IV) infusion of azer-cel. All participants will be monitored through D720 or progression. All participants who receive a dose of azer-cel will be followed in a separate long-term follow-up (LTFU) study for up to 15 years after exiting this study.

Enrollment

129 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria

Criteria for B-ALL:

  • Participant has unequivocal r/r CD19+ B-ALL that has been confirmed by morphology, flow cytometry, or a validated minimal residual disease (MRD) assay.
  • Participants with Philadelphia chromosome positive disease can be eligible if they are intolerant to tyrosine kinase inhibitor therapy or if they have r/r disease.

Criteria for NHL:

  • Participant has unequivocal aggressive CD19+ r/r B-cell NHL that is confirmed by archived tumor biopsy tissue from last relapse after CD19-directed therapy and corresponding pathology report. Alternatively, if at least 1 tumor involved site is accessible at time of Screening, the participant's diagnosis is confirmed by pretreatment biopsy (excisional when possible) or by flow cytometry of fine needle aspirate (FNA). If a participant never had a CR, a sample from the most recent biopsy is acceptable. NHL subtypes included but are not limited to:

  • For Phase 1 Dose Escalation:

    • Diffuse large B-cell lymphoma (DLBCL) including Richter's transformation
    • FL including Grade 3 or transformed FL
    • High-grade B-cell lymphoma
    • Primary mediastinal lymphoma
  • For Phase 1b Dose Expansion:

    • DLBCL not otherwise specified (NOS)
    • High grade B-Cell Lymphoma
    • DLBCL transformed from the following indolent lymphoma subtypes (Follicular lymphoma, Marginal Zone lymphoma and Waldenstrom's Macroglobulinemia)
  • Participant has measurable or detectable (for example positron emission tomography-positive) disease according to the Lugano Classification.

  • Participant must have received at least 2 lines of prior anti-cancer therapy for the disease under study, including at least 1 chemoimmunotherapy regimen (e.g., anti-CD20 monoclonal antibody plus chemotherapy), consistent with standard of care treatment guidance (e.g., National Comprehensive Cancer Network [NCCN]), unless no second line therapy of known benefit exists for a given subject. For Richter's transformation, only 1 prior line of therapy is required for the DLBCL component.

  • Participant has received no more than 7 systemic lines of anti-cancer therapy for the disease under study.

  • 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.

  • Expansion 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.

Criteria for both B-ALL and NHL:

  • 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 (i.e., intact immune function).

  • Participant has adequate bone marrow, renal, hepatic, pulmonary, and cardiac function defined as:

    1. Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 (calculated using the CKD-EPI equation [Levey et al, 2009]). For participants receiving an LD regimen that contains 4 days of Fludarabine, an eGFR >=60 mL/min/1.73 m2 (calculated using the CKD-EPI equation) is required. If there is a concern that eGFR calculation is not an accurate reflection of renal function, a 24-hour urine collection for creatinine clearance may be used at the investigator's discretion.
    2. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels both ≤3 times of upper limit of normal (ULN), unless there is suspected disease in the liver.
    3. Total bilirubin <2.0 mg/dL, except in participants with Gilbert's syndrome.
    4. Platelet count ≥50,000/μL and absolute neutrophil count of ≥1000/ μL. Platelet transfusions within 14 days of screening are not allowed except for participants in B-ALL disease cohort with extensive bone marrow disease burden, in which case adequate bone marrow recovery after prior treatment is required to be documented. NHL participants with a platelet count <50,000/μL and absolute neutrophil count (ANC) of <1000/ μL may be enrolled with Medical Monitor approval if there is documentation of significant bone marrow involvement by disease and in the Investigator's assessment, no other reasonable etiology for the low counts.
    5. Left ventricular ejection fraction >45% as assessed by echocardiogram (ECHO) or multiple gated acquisition scan performed within 1 month before starting lymphodepleting chemotherapy. ECHO results performed within 6 months before Screening and at least 28 days after the last cancer treatment may be acceptable if the participant has not received any treatment with cardiotoxicity risks.
    6. No clinically significant evidence of pericardial effusion or pleural effusion causing clinical symptoms and needing immediate intervention, based on the investigator's opinion. Any known effusion must be stable without need for drainage within 2 weeks of enrollment.
    7. No clinically significant renal/pulmonary comorbidities.
    8. Baseline oxygen saturation >92% on room air.

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 central nervous system (CNS) disease. Subjects with a prior history of CNS involvement that has been adequately treated ≥3 months prior to study consent and without symptoms or clinical suspicion of relapsed CNS disease may be enrolled.

  • History of another primary malignancy that has not been in remission for at least 2 years with the following exceptions:

  • Curatively treated basal or squamous cell carcinoma or melanoma of the skin or in situ carcinoma of the breast or cervix

  • Low-grade, early-stage prostate cancer (Gleason score 6 or below, Stage 1 or 2) with no requirement for therapy at any time

  • Completely resected Stage 1 solid tumor with low risk for recurrence within 2 years

  • In the case of Richter's transformation, participants may be enrolled with ongoing chronic lymphocytic leukemia/small lymphocytic lymphoma.

    • Any form of primary immunodeficiency (e.g., 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, including but not limited to:

      1. Active ventricular or atrial dysrhythmia ≥ Grade 2, bradycardia ≥ Grade 2.
      2. Myocardial infarction within 6 months before Screening.
      3. Pulmonary embolism, deep vein thrombosis, or any other significant coagulopathy including disseminated intravascular coagulation.
    • History of hypertension crisis or hypertensive encephalopathy within 3 months prior to Screening. In case of hypertensive crisis caused by omission of well-established treatment regimen, transient and promptly stabilized, enrollment must be discussed and agreed upon with sponsor and medical monitor.

    • 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.

    • Abnormal findings during the Screening Period or any other medical condition(s) or laboratory findings that, in the opinion of the investigator, might jeopardize the participant's safety.

    • 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 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. Note: This criterion does not apply if the subject has clear evidence of disease progression after such an agent has been administered and all AEs have resolved to a Grade 2 or less in severity. This should be discussed with the Medical Monitor for confirmation

    • Radiotherapy within 4 weeks before Screening should be discussed with monitor and determined on a case-by-case basis.

    • 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. 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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Parallel Assignment

Masking

None (Open label)

129 participants in 6 patient groups

Dose Level 1
Experimental group
Description:
Azer-cel 3 x 10\^5 CAR T cells per kilogram (kg) body weight. Route of Administration: Intravenous infusion.
Treatment:
Biological: Azer-cel
Drug: Cyclophosphamide
Drug: Fludarabine
Dose Level 2
Experimental group
Description:
Azer-cel, 1 x 10\^6 CAR T cells per kg body weight
Treatment:
Biological: Azer-cel
Drug: Cyclophosphamide
Drug: Fludarabine
Dose Level 3a
Experimental group
Description:
Azer-cel, 3 x 10\^6 CAR T cells per kg body weight.
Treatment:
Biological: Azer-cel
Drug: Cyclophosphamide
Drug: Fludarabine
Dose Level 4
Experimental group
Description:
Azer-cel, 6 x 10\^6 CAR T cells per kg body weight as 2 administrations of 3 x 10\^6 CAR T cells per kg body weight administered after a single lymphodepletion.
Treatment:
Biological: Azer-cel
Drug: Cyclophosphamide
Drug: Fludarabine
Dose Level 4b
Experimental group
Description:
Azer-cel, 500 x 10\^6 CAR T cells (flat dose)
Treatment:
Drug: Bendamustine
Biological: Azer-cel
Drug: IL-2
Drug: Cyclophosphamide
Drug: Fludarabine
Dose level 4c
Experimental group
Description:
Azer-cel, 1000 x 10\^6 CAR T cells (flat dose) given as 2 administrations of 500 × 106 cells/per dose on Day 0 and Day 5.
Treatment:
Biological: Azer-cel
Drug: Cyclophosphamide
Drug: Fludarabine

Trial contacts and locations

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Central trial contact

Imugene Clinical Team

Data sourced from clinicaltrials.gov

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