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Safety and Efficacy of Axicabtagene Ciloleucel in Combination With Rituximab in Participants With Refractory Large B-Cell Lymphoma

Phase 2
Completed
Conditions
Refractory Large B-cell Lymphoma
Interventions
Registration Number
NCT04002401
Lead Sponsor
Kite, A Gilead Company
Brief Summary

The primary objective of this study is to estimate the efficacy of axicabtagene ciloleucel in combination with rituximab, as measured by assessment of response rates in adult participants with relapsed/refractory large B-cell lymphoma.

Detailed Description

Following at least 24 months of assessments after axicabtagene ciloleucel infusion, participants will be asked to rollover to a separate long-term follow-up study (Study KT-US-982-5968). Participants will complete the remainder of the 15-year follow-up assessments in the KT-US-982-5968 study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
27
Inclusion Criteria
  • Histologically confirmed large B-cell lymphoma

  • Chemotherapy-refractory disease, defined as one or more of the following:

    • No response to first-line therapy (primary refractory disease)
    • No response to second or greater lines of therapy OR
    • Refractory after autologous stem cell transplant (ASCT)
  • At least 1 measureable lesion according to the Lugano Classification (Cheson 2014).

  • Individuals must have received adequate prior therapy, including at a minimum:

    • Anti-CD20 monoclonal antibody
    • An anthracycline-containing chemotherapy regimen
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1

  • Adequate renal, hepatic, pulmonary, and cardiac function

Key

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Exclusion Criteria
  • Known CD19 negative or CD20 negative tumor
  • History of Richter's transformation of Chronic Lymphocytic Leukemia (CLL)
  • Prior CAR therapy or other genetically modified T-cell therapy
  • Prior organ transplantation including prior allogeneic stem cell transplant (SCT)
  • Prior CD19 targeted therapy
  • Clinically significant infection or cardiopulmonary disease
  • Presence of any in-dwelling lines or drains (dedicated central venous access catheters allowed)
  • History or presence of central nervous system (CNS) lymphoma or nonmalignant CNS disorder or cerebrospinal fluid (CSF) malignant cells or brain metastases
  • History of autoimmune disease
  • History of deep vein thrombosis (DVT) or pulmonary embolism (PE) within the last 6 months

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Axicabtagene Ciloleucel and Rituximab CombinationAxicabtagene CiloleucelParticipants will receive rituximab 375 mg/m\^2, once on Day -5 along with conditioning chemotherapy (fludarabine 30 mg/m\^2 over 30 minutes and cyclophosphamide 500 mg/m\^2 over 60 minutes) once on Days -5 to -3, followed by axicabtagene ciloleucel 2 x 10\^6 anti-cluster of differentiate 19 (CD19) chimeric antigen receptor (CAR) T cells/kg once on Day 0 and additional rituximab 375 mg/m\^2 of 5 doses, once every 28 days starting from Day 21 up to Day 133.
Axicabtagene Ciloleucel and Rituximab CombinationRituximabParticipants will receive rituximab 375 mg/m\^2, once on Day -5 along with conditioning chemotherapy (fludarabine 30 mg/m\^2 over 30 minutes and cyclophosphamide 500 mg/m\^2 over 60 minutes) once on Days -5 to -3, followed by axicabtagene ciloleucel 2 x 10\^6 anti-cluster of differentiate 19 (CD19) chimeric antigen receptor (CAR) T cells/kg once on Day 0 and additional rituximab 375 mg/m\^2 of 5 doses, once every 28 days starting from Day 21 up to Day 133.
Axicabtagene Ciloleucel and Rituximab CombinationFludarabineParticipants will receive rituximab 375 mg/m\^2, once on Day -5 along with conditioning chemotherapy (fludarabine 30 mg/m\^2 over 30 minutes and cyclophosphamide 500 mg/m\^2 over 60 minutes) once on Days -5 to -3, followed by axicabtagene ciloleucel 2 x 10\^6 anti-cluster of differentiate 19 (CD19) chimeric antigen receptor (CAR) T cells/kg once on Day 0 and additional rituximab 375 mg/m\^2 of 5 doses, once every 28 days starting from Day 21 up to Day 133.
Axicabtagene Ciloleucel and Rituximab CombinationCyclophosphamideParticipants will receive rituximab 375 mg/m\^2, once on Day -5 along with conditioning chemotherapy (fludarabine 30 mg/m\^2 over 30 minutes and cyclophosphamide 500 mg/m\^2 over 60 minutes) once on Days -5 to -3, followed by axicabtagene ciloleucel 2 x 10\^6 anti-cluster of differentiate 19 (CD19) chimeric antigen receptor (CAR) T cells/kg once on Day 0 and additional rituximab 375 mg/m\^2 of 5 doses, once every 28 days starting from Day 21 up to Day 133.
Primary Outcome Measures
NameTimeMethod
Complete Response (CR) Rate Per the International Working Group (IWG) Lugano Classification as Determined by Study InvestigatorsFirst infusion date up to maximum duration of 32.7 months

CR rate is defined as the incidence of a CR per the IWG Lugano Classification as determined by study investigators. CR rate: percentage of participants with CR \[complete metabolic response (CMR); complete radiological response (CRR)\]. CMR: positron emission tomography (PET) 5-point scale (5-PS) scores of 1 (no uptake above background), 2 (uptake ≤ mediastinum), 3 (uptake \> mediastinum but ≤ liver) with/without a residual mass); no new lesions; and no evidence of fluorodeoxyglucose (FDG)-avid disease in bone marrow (BM). CRR: target nodes/nodal masses regressed to ≤ 1.5 cm in longest transverse diameter of lesion (LDi); no extralymphatic sites of disease; absent non-measured lesion (NMLs); organ enlargement regress to normal; no new sites; and bone marrow normal by morphology. 95% confidence interval (CI) was calculated by Clopper-Pearson method.

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants Who Experienced Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter ValueFirst infusion date up to maximum duration of 27 months

Grading categories were determined by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Laboratory parameters with only non-zero values are presented.

Peak Level of Anti-CD19 CAR T Cells in BloodBaseline (Day 0), post-infusion on Days 7, 14, 21, 28, 49, 105, 180, Months 9, 12, 15, 18, and 24

Peak was defined as the maximum number of CAR T cells in blood measured after infusion.

Percentage of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs)First infusion date up to maximum duration of 27 months

An AE is defined as any untoward medical occurrence in a clinical trial participant that does not necessarily have a relationship with study treatment or worsening of a pre-existing medical condition. TEAEs are any AEs with onset on or after axicabtagene ciloleucel infusion or worsening of a pre-existing medical condition that occurs on or after axicabtagene ciloleucel infusion.

Duration of Response (DOR) Per the IWG Lugano Classification as Determined by Study InvestigatorsFrom the date of first confirmed objective response (CR or PR) to disease progression or death regardless of cause (up to approximately 32.7 months)

DOR is defined only for participants who experience an objective response and is the time from the first objective response to disease progression \[progressive metabolic disease (PMD); progressive radiologic disease (PRD)\] or death from any cause. Objective response is defined in outcome measure (OM) 4. PMD: scores 4 (uptake moderately \> liver), 5 (uptake markedly \> liver, new lesions) with increased uptake compared with baseline; new fluorodeoxyglucose (FDG)-avid foci consistent with lymphoma rather than another etiology; new or recurrent FDG-avid foci in bone marrow. PRD: LDi \>1.5 cm; ≥ 50% increase from cross product of LDi and perpendicular diameter (PPD); increase in LDi or shortest axis perpendicular to the LDi (SDi) of 0.5 cm for lesions ≤1.5 cm and 1 cm for lesions \>2 cm; spleen increased by \>50% in length beyond normal; new or recurrent splenomegaly, bone marrow involvement; new lesions; progression of pre-existing lesions. Kaplan-Meier (KM) estimate of median was reported.

Overall Survival (OS)First infusion date up to death regardless of cause (up to approximately 32.7 months)

OS is defined as the time from axicabtagene ciloleucel infusion to the date of death from any cause. KM estimate of median was reported.

Area Under the Curve of CAR T Cells From Day 0 to Day 28 (AUC0-28)Baseline (Day 0), post-infusion on Days 7, 14, 21, and 28

AUC0-28 is defined as the area under curve in a plot of number of CAR T cells against scheduled visit from Day 0 to Day 28.

Time to Peak Level of Anti-CD19 CAR T Cells in BloodBaseline (Day 0), post-infusion on Days 7, 14, 21, 28, 49, 105, 180, Months 9, 12, 15, 18, and 24

Time to peak was defined as the number of days from the date of the axicabtagene ciloleucel infusion to the date of peak level defined as the maximum number of CAR T cells in blood measured after infusion.

Objective Response Rate (ORR) Per the IWG Lugano Classification as Determined by Study InvestigatorsFirst infusion date up to maximum duration of 32.7 months

ORR: percentage of participants with CR \[CMR;CRR\] or PR \[partial metabolic response (PMR); partial radiologic response (PRR)\].CMR: PET 5PS scores of 1 (no uptake above background, 2 (uptake ≤ mediastinum), 3 (uptake \> mediastinum but ≤ liver) with/without a residual mass; no new lesions; no evidence of FDG-avid disease in BM. CRR: target nodes/nodal masses regressed to ≤ 1.5 cm in LDi;no extralymphatic sites of disease;absent NMLs;organ enlargement regress to normal;no new sites;bone marrow morphology normal. PMR: scores 4 (uptake moderately \> liver), 5 (uptake markedly \> liver, new lesions) with reduced uptake compared with baseline and residual mass; no new lesions; responding disease at interim/residual disease at end of treatment (EOT). PRR: ≥ 50% decrease in sum of the product of perpendicular diameters (SPD) of up to 6 target measurable nodes and extra-nodal sites; absent/normal, regressed, but no increase of NMLs; spleen regressed by \> 50% in length beyond normal; no new sites.

Progression-Free Survival (PFS) Per the IWG Lugano Classification as Determined by Study InvestigatorsFirst infusion date up to disease progression or death regardless of cause (up to approximately 32.7 months)

PFS is defined as the time from the axicabtagene ciloleucel infusion date to the date of disease progression (PMD; PRD) or death from any cause. PMD: scores 4 (uptake moderately \> liver), 5 (uptake markedly \> liver, new lesions) with increased uptake compared with baseline; new FDG-avid foci consistent with lymphoma rather than another etiology; new or recurrent FDG-avid foci in bone marrow. PRD: LDi \>1.5 cm; ≥ 50% increase from cross product of LDi and PPD; increase in LDi or SDi of 0.5 cm for lesions ≤1.5 cm and 1 cm for lesions \>2 cm; spleen increased by \>50% in length beyond normal; new or recurrent splenomegaly, bone marrow involvement; new lesions; progression of pre-existing lesions. KM estimate of median was reported.

Level of Anti-CD19 CAR T Cells in Blood by VisitBaseline (Day 0), post-infusion on Days 7, 14, 21, 28, 49, 105, 180, Months 9, 12, 15, 18, and 24

Trial Locations

Locations (14)

Swedish Cancer Institute

🇺🇸

Seattle, Washington, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

John Theurer Cancer Center at Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

City of Hope National Medical Center

🇺🇸

Duarte, California, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

Banner MD Anderson Cancer Center

🇺🇸

Gilbert, Arizona, United States

UCLA Hematology/Oncology

🇺🇸

Santa Monica, California, United States

Mayo Clinic Florida

🇺🇸

Jacksonville, Florida, United States

University of Chicago Medical Center

🇺🇸

Chicago, Illinois, United States

Stanford Cancer Institute

🇺🇸

Palo Alto, California, United States

Columbia University Medical Center, New York Presbyterian Hospital

🇺🇸

New York, New York, United States

Loyola University Medical Center

🇺🇸

Maywood, Illinois, United States

St. David's South Austin Medical Center

🇺🇸

Austin, Texas, United States

The University of Texas MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

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