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A Phase 2 Multicenter Study of Axicabtagene Ciloleucel in Subjects With Relapsed/Refractory Indolent Non-Hodgkin Lymphoma

Phase 2
Completed
Conditions
Follicular Lymphoma
Marginal Zone Lymphoma
Indolent Non-Hodgkin Lymphoma
Interventions
Registration Number
NCT03105336
Lead Sponsor
Kite, A Gilead Company
Brief Summary

This study will enroll approximately 160 adult participants who have relapsed or refractory (r/r) iNHL to be infused with the study treatment, axicabtagene ciloleucel, to see if their disease responds to this experimental product and if this product is safe. Axicabtagene ciloleucel is made from the participants own white blood cells which are genetically modified and grown to fight cancer. An objective response rate of 70% is targeted.

Detailed Description

All enrolled participants will be screened for eligibility then will undergo leukapheresis to collect white blood cells for manufacturing. In preparation for the infusion with axicabtagene ciloleucel, participants will undergo conditioning chemotherapy with cyclophosphamide and fludarabine for 3 days to help the study treatment be effective. After the product is manufactured and conditioning chemotherapy period is complete, participants will be infused with axicabtagene ciloleucel and then monitored in a hospital for a minimum of 7 days. After completing at least 60 months (FL participants) or at least 24 months (MZL participants) of assessments in this study since the initial axicabtagene ciloleucel infusion and after agreement by the Sponsor, participants will transition to a long-term follow-up (LTFU) study, KT-US-982-5968 where they will complete the remainder of the 15 year follow-up assessments.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
159
Inclusion Criteria
  1. Individual has [follicular lymphoma or marginal zone lymphoma that has progressed after at least 2 lines of treatment with combination chemoimmunotherapy] (e.g. R-bendamustine, R-CHOP).
  2. Individual has [measurable disease].
  3. Individual has no known presence or history of central nervous system (CNS) involvement by lymphoma.
  4. If individual is on conventional systemic therapy or systemic inhibitory/stimulatory immune checkpoint therapy, individual is able to stop conventional therapy 2 weeks or 5 half-lives, whichever is shorter, or immune checkpoint therapy 3 half-lives prior to planned leukapheresis.
  5. Individual has Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 and adequate renal, hepatic, pulmonary, and cardiac function
  6. Individual is not pregnant or breastfeeding (female individuals only) and is willing to use birth control from the time of consent through 12 months following chimeric antigen receptor (CAR) T cell infusion (both male and female individuals).

Key

Exclusion Criteria
  1. Transformed follicular lymphoma (FL) or marginal zone lymphoma (MZL)
  2. Small lymphocytic lymphoma
  3. Histological Grade 3b FL
  4. Individual will have undergone autologous transplant within 6 weeks of planned leukapheresis or has undergone allogeneic transplant.
  5. Individual has evidence of involvement of the heart by lymphoma or requirement for urgent therapy due to ongoing or impending oncologic emergency (e.g. mass effect, tumor lysis syndrome, etc.)

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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
axicabtagene ciloleucelaxicabtagene ciloleucelParticipants will receive a conditioning chemotherapy regimen of fludarabine and cyclophosphamide, followed by a single infusion of CAR transduced autologous T cells.
axicabtagene ciloleucelFludarabineParticipants will receive a conditioning chemotherapy regimen of fludarabine and cyclophosphamide, followed by a single infusion of CAR transduced autologous T cells.
axicabtagene ciloleucelCyclophosphamideParticipants will receive a conditioning chemotherapy regimen of fludarabine and cyclophosphamide, followed by a single infusion of CAR transduced autologous T cells.
Primary Outcome Measures
NameTimeMethod
Objective response rate per central readUp to 15 years

Complete response (CR) + partial response (PR) per the Lugano Classiciation (Cheson et al, 2014).

Secondary Outcome Measures
NameTimeMethod
Overall Survival (OS)Up to 15 years

OS is defined as the time from KTE-C19 infusion to the date of death.

Levels of cytokines in serumAt enrollment, prior to axicabtagene ciloleucel infusion on Day 0, Day 3, Day 7, Week 2, Week 4
Percentage of Participants Experiencing clinically significant changes in lab valuesUp to 5 years
CR Rate per central readUp to 15 years

CRR is defined as the incidence of CR as best response to treatment by the Lugano Classification (Cheson et al, 2014)

Percentage of Participants Experiencing Treatment-Emergent Adverse EventsUp to 15 years
DORUp to 15 years

DOR is defined only for subjects who experience an objective response and is the time from the first objective response to disease progression per (Cheson et al, 2014) or disease-related death, whichever comes first.

PFSUp to 15 years

PFS is defined as the time from the axicabtagene ciloleucel infusion date to the date of disease progression per (Cheson et al, 2014) or death from any cause.

Levels of anti-CD19 CAR T cells in bloodAt enrollment, Day 7, Week 2, Week 4, Month 3, Month 6, Month 12, Month 18, Month 24, annually up to year 5.
Percentage of Participants experiencing anti-axicabtagene ciloleucel antibodiesAt enrollment, Week 4, Month 3, every 3 months up to Month 12
Time to next TherapyUp to 15 years

Time from axi-cel infusion date to the start of the subsequent new lymphoma therapy or death from any cause.

Objective Response Rate as Determined by the Investigator ReadUp to 15 years

ORR per investigator read is defined as the incidence of a CR or a PR by the Lugano Classification.

Best Objective Response per Central Read or Investigator ReadUp to 15 years

Best objective response is defined as the incidence of CR, PR, stable disease (SD), PD, or non-evaluable (NE) as best response to treatment by the Lugano Classification

Complete response rate among those participants with 3 or more lines of prior therapyUp to 15 years

Complete response rate is defined as the incidence of CR as best response to treatment by the Lugano Classification (Cheson et al, 2014) for participants with 3 or more lines of prior therapy.

Objective response rate among participants with 3 or more lines of prior therapyUp to 15 years

Complete response (CR) + partial response (PR) per the Lugano Classiciation (Cheson et al, 2014) for participants with 3 or more lines of prior therapy.

Trial Locations

Locations (19)

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Georgetown Lombardi Comprehensive Cancer Center

🇺🇸

Washington, District of Columbia, United States

Banner MD Anderson Cancer Center

🇺🇸

Gilbert, Arizona, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

H Lee Moffitt Cancer Center

🇺🇸

Tampa, Florida, United States

Sarah Cannon Research Institute

🇺🇸

Nashville, Tennessee, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

University of Rochester Medical Center (URMC)

🇺🇸

Rochester, New York, United States

Centre Hospitalier Régional Universitaire de Lille

🇫🇷

Lille, France

USC Norris Comprehensive Cancer Center

🇺🇸

Los Angeles, California, United States

University of California Los Angeles

🇺🇸

Los Angeles, California, United States

University of Miami Hospital and Clinics

🇺🇸

Miami, Florida, United States

Hackensack University Medical Center - John Theurer Cancer Center

🇺🇸

Hackensack, New Jersey, United States

Fox Chase Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

Ohio State University Medical Center

🇺🇸

Cleveland, Ohio, United States

UPMC Hillman Cancer Center

🇺🇸

Pittsburgh, Pennsylvania, United States

Fred Hutchinson Cancer Research Center

🇺🇸

Seattle, Washington, United States

Centre Hospitalier Lyon Sud

🇫🇷

Pierre Benite, France

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