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A Study of PCI-32765 (Ibrutinib) in Combination With Either Bendamustine and Rituximab or Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone in Participants With Previously Treated Indolent Non-Hodgkin Lymphoma

Phase 3
Completed
Conditions
Lymphoma
Interventions
Registration Number
NCT01974440
Lead Sponsor
Janssen Research & Development, LLC
Brief Summary

The purpose of this study is to evaluate the efficacy and safety of PCI-32765 (ibrutinib) administered in combination with either bendamustine and rituximab (BR) or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) in adult participants with previously treated indolent Non-Hodgkin lymphoma.

Detailed Description

This is a randomized (individuals assigned to study treatment by chance), double-blind (individuals and study personnel will not know the identity of study treatments), placebo (an inactive substance that is compared with a drug to test whether the drug has a real effect in a clinical trial)-controlled study in approximately 400 adult participants with follicular lymphoma or marginal zone lymphoma. The study will include the following phases: Screening, Treatment, and a Post-treatment Follow-up. Eligible participants will be randomly assigned in a 1:1 ratio to either treatment Arm A (background immune-chemotherapy + placebo) or treatment Arm B (background immune-chemotherapy + 560 milligram \[mg\] of ibrutinib). All participants will receive 6 cycles of background immune-chemotherapy with either BR or R-CHOP in combination with either placebo (Arm A) or ibrutinib (Arm B). Selection of background immune-chemotherapy will be based on prior treatment history and cardiac function. After completion of background immune-chemotherapy, study drug (ibrutinib or placebo) will continue until disease progression, unacceptable toxicity, or study end, whichever comes first. Assessment of tumor response and progression will be conducted in accordance with the Revised Response Criteria for Malignant Lymphoma. Serial pharmacokinetic (study of what a drug does to the body) blood samples will be collected. Safety will be assessed throughout the study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
403
Inclusion Criteria
  • Histologically confirmed diagnosis of B-cell indolent Non-Hodgkin lymphoma with histological subtype limited to follicular lymphoma or marginal zone lymphoma, at initial diagnosis and without evidence of pathological transformation or clinical signs suggesting transformation
  • At least 1 prior treatment with a CD20 antibody combination chemo-immunotherapy regimen
  • Disease that has relapsed or was refractory after prior chemo-immunotherapy
  • At least 1 measurable site of disease according to Revised Response Criteria for Malignant Lymphoma 2007
  • Eastern Cooperative Oncology Group performance status grade 0 or 1
  • Laboratory values within protocol-defined parameters
  • Agrees to protocol-defined use of effective contraception
  • Men must agree not to donate sperm during and after the study for 6 months after the last dose of bendamustine, 12 months after the last dose of rituximab, or 3 months after the last dose of study medication, whichever is later
  • Women of childbearing potential must have a negative serum or urine pregnancy test at Screening
Exclusion Criteria
  • Prior treatment according to protocol-defined criteria
  • Unable to receive background chemotherapy based on prior treatment history and cardiac function
  • Known central nervous system lymphoma
  • Diagnosed or treated for malignancy other than indolent Non-Hodgkin lymphoma
  • History of stroke or intracranial hemorrhage within 6 months prior to randomization
  • Requires anticoagulation with warfarin or equivalent Vitamin K antagonists
  • Requires treatment with strong CYP3A inhibitors
  • Clinically significant cardiovascular disease
  • Known history of human immunodeficiency virus or active hepatitis C virus (HCV; ribonucleic acid [RNA] polymerase chain reaction [PCR]-positive) or active hepatitis B virus (HBV; DNA PCR-positive) infection or any uncontrolled active systemic infection requiring intravenous antibiotics
  • Any life-threatening illness, medical condition, or organ system dysfunction which, in the Investigator's opinion, could compromise the participant's safety, interfere with the absorption or metabolism of ibrutinib capsules, or put the study outcomes at undue risk
  • Women who are pregnant or breastfeeding

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Treatment Arm APlaceboTreatment Arm A = background immune-chemotherapy (bendamustine and rituximab \[BR\] or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone \[R-CHOP\]) for 6 cycles + placebo.
Treatment Arm BPCI-32765 (Ibrutinib)Treatment Arm B = background immune-chemotherapy (BR or R-CHOP) for 6 cycles + PCI-32765 (Ibrutinib).
Treatment Arm BCyclophosphamideTreatment Arm B = background immune-chemotherapy (BR or R-CHOP) for 6 cycles + PCI-32765 (Ibrutinib).
Treatment Arm ABendamustineTreatment Arm A = background immune-chemotherapy (bendamustine and rituximab \[BR\] or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone \[R-CHOP\]) for 6 cycles + placebo.
Treatment Arm ARituximabTreatment Arm A = background immune-chemotherapy (bendamustine and rituximab \[BR\] or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone \[R-CHOP\]) for 6 cycles + placebo.
Treatment Arm ADoxorubicinTreatment Arm A = background immune-chemotherapy (bendamustine and rituximab \[BR\] or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone \[R-CHOP\]) for 6 cycles + placebo.
Treatment Arm ACyclophosphamideTreatment Arm A = background immune-chemotherapy (bendamustine and rituximab \[BR\] or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone \[R-CHOP\]) for 6 cycles + placebo.
Treatment Arm AVincristineTreatment Arm A = background immune-chemotherapy (bendamustine and rituximab \[BR\] or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone \[R-CHOP\]) for 6 cycles + placebo.
Treatment Arm BBendamustineTreatment Arm B = background immune-chemotherapy (BR or R-CHOP) for 6 cycles + PCI-32765 (Ibrutinib).
Treatment Arm APrednisoneTreatment Arm A = background immune-chemotherapy (bendamustine and rituximab \[BR\] or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone \[R-CHOP\]) for 6 cycles + placebo.
Treatment Arm BDoxorubicinTreatment Arm B = background immune-chemotherapy (BR or R-CHOP) for 6 cycles + PCI-32765 (Ibrutinib).
Treatment Arm BRituximabTreatment Arm B = background immune-chemotherapy (BR or R-CHOP) for 6 cycles + PCI-32765 (Ibrutinib).
Treatment Arm BPrednisoneTreatment Arm B = background immune-chemotherapy (BR or R-CHOP) for 6 cycles + PCI-32765 (Ibrutinib).
Treatment Arm BVincristineTreatment Arm B = background immune-chemotherapy (BR or R-CHOP) for 6 cycles + PCI-32765 (Ibrutinib).
Primary Outcome Measures
NameTimeMethod
Supplementary Analysis: Progression Free Survival: Unstratified Analysis - Participants With Marginal Zone Lymphoma (MZL)Up to 8 years

PFS in MZL participants was defined as duration (in months) from the date of randomization to the date of disease progression or relapse from CR or death, whichever was first reported. PFS was assessed by the investigator based on the 2007 Revised Response Criteria for Malignant Lymphoma. Disease progression was defined as any new lesion or increase by \>=50% of previously involved sites from nadir disease progression criteria: Appearance of new nodal lesion 1.5 cm in any axis, 50% increase in SPD of \>1 node or 50% increase in longest diameter of previously identified node 1 cm in short axis. Participants who were progression-free and alive or had unknown status were censored at the last tumor assessment. Kaplan-Meier method was used for the analysis. For this outcome measure, unstratified analysis was performed on participants with MZL.

Primary Analysis: Progression Free Survival (PFS): Stratified AnalysisUp to 8 years

PFS was defined as duration (in months) from the date of randomization to the date of disease progression or relapse from complete response (CR) or death, whichever was first reported. PFS was assessed by the investigator based on the 2007 Revised Response Criteria for Malignant Lymphoma. Disease progression was defined as any new lesion or increase by greater than or equal to (\>=) 50 percent (%) of previously involved sites from nadir disease progression criteria: Appearance of new nodal lesion 1.5 centimeters (cm) in any axis, 50% increase in sum of product of diameters (SPD) of greater than (\>) 1 node or 50% increase in longest diameter of previously identified node 1 cm in short axis. Participants who were progression-free and alive or had unknown status were censored at the last tumor assessment. Kaplan-Meier method was used for the analysis. Stratification factors were used for the analysis.

Secondary Outcome Measures
NameTimeMethod
Primary Analysis: Duration of Response (DOR): Stratified AnalysisUp to 8 years

DOR was defined as the interval (in months) between the date of initial documentation of response (CR or PR) and the date of first documented evidence of progressive disease (or relapse for participants who experienced CR during the study) or death, whichever occurred first. Criteria for CR: disappearance of all evidence of disease; mass of any size permitted if PET negative; regression to normal size on CT; spleen and liver: not palpable, nodules disappeared; bone marrow: infiltrate cleared on repeat biopsy and no new sites of disease detected during assessment. Criteria for PR: \>=50% decrease in sum of the diameter of all target lesions compared with baseline, in absence of new lesions or unequivocal progression of non-target lesions. Kaplan-Meier method was used for the analysis. Stratification factors were used for the analysis.

Supplementary Analysis: Duration of Response: Unstratified Analysis - Participants With MZLUp to 8 years

DOR in MZL participants was defined as the interval (in months) between the date of initial documentation of response (CR or PR) and the date of first documented evidence of progressive disease (or relapse for participants who experienced CR during the study) or death, whichever occurred first. Criteria for CR: disappearance of all evidence of disease; mass of any size permitted if PET negative; regression to normal size on CT; spleen and liver: not palpable, nodules disappeared; bone marrow: infiltrate cleared on repeat biopsy and no new sites of disease detected during assessment. Criteria for PR: \>=50% decrease in sum of the diameter of all target lesions compared with baseline, in absence of new lesions or unequivocal progression of non-target lesions. Kaplan-Meier method was used for the analysis. For this outcome measure, unstratified analysis was performed on participants with MZL.

Primary Analysis: Overall Survival (OS): Stratified AnalysisUp to 8 years

OS was defined as the interval (in months) between the date of randomization and the date of the participant's death due to any cause. Kaplan-Meier method was used for the analysis. Stratification factors were used for the analysis.

Supplementary Analysis: Overall Response Rate: Unstratified Analysis - Participants With MZLUp to 8 years

ORR in MZL participants was defined as the percentage of participants who achieved a CR or PR. Criteria for CR: disappearance of all evidence of disease; mass of any size permitted if PET negative; regression to normal size on CT; spleen and liver: not palpable, nodules disappeared; bone marrow: infiltrate cleared on repeat biopsy and no new sites of disease detected during assessment. Criteria for PR: \>=50% decrease in sum of the diameter of all target lesions compared with baseline, in absence of new lesions or unequivocal progression of non-target lesions. Kaplan-Meier method was used for the analysis. For this outcome measure, unstratified analysis was performed on participants with MZL.

Primary Analysis: Complete Response Rate (CRR): Stratified AnalysisUp to 8 years

CRR was defined as the percentage of participants who achieved a complete response (CR); (based on investigator assessment) on or prior to the initiation of subsequent antilymphoma therapy. Criteria for CR: disappearance of all evidence of disease; mass of any size permitted if positron emission tomography (PET) negative; regression to normal size on CT; spleen and liver: not palpable, nodules disappeared; bone marrow: infiltrate cleared on repeat biopsy and no new sites of disease detected during assessment. Kaplan-Meier method was used for the analysis. Stratification factors were used for the analysis.

Supplementary Analysis: Complete Response Rate: Unstratified Analysis - Participants With MZLUp to 8 years

CRR in MZL participants was defined as the percentage of participants who achieved a CR (based on investigator assessment) on or prior to the initiation of subsequent antilymphoma therapy. Criteria for CR: disappearance of all evidence of disease; mass of any size permitted if PET negative; regression to normal size on CT; spleen and liver: not palpable, nodules disappeared; bone marrow: infiltrate cleared on repeat biopsy and no new sites of disease detected during assessment. Kaplan-Meier method was used for the analysis. For this outcome measure, unstratified analysis was performed on participants with MZL.

Supplementary Analysis: Time to Worsening (TTW) in the Lymphoma (Lym) Subscale of the Functional Assessment of Cancer Therapy - Lymphoma Subscale (FACT-LymS) Questionnaire: Participants With MZLUp to 8 years

TTW in MZL participants in the Lymphoma subscale of the FACT-Lym was defined as the time (in months) from the date of randomization to the start date of the worsening of participant symptoms. Worsening was defined by a 5-point decrease from baseline in participant symptoms. FACT-Lym Lymphoma subscale contains 15 questions, scores from 0 to 4 for each question (0 = not at all, 1 = a little bit, 2 = some what, 3 = quite a bit and 4 = very much, where the higher score indicated worse condition). Lymphoma subscale score is the total of reverse scores, range 0 to 60. Higher scores indicate a better quality of life.

Number of Participants With Treatment-emergent Adverse Events (TEAEs)Placebo + Chemoimmunotherapy (CIT) arm: From Day 1 up to 30 days after date of last dose of study medication (up to 8 years); Ibrutinib + CIT arm: From Day 1 up to 30 days after date of last dose of study medication (up to 8 years 8 months)

Number of participants with TEAEs were reported. Adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE did not necessarily have a causal relationship with the pharmaceutical/biological agent under study. TEAEs were defined as adverse events with onset or worsening on or after date of first dose of study treatment up to and including 30 days after date of last dose of study medication.

Supplementary Analysis: Overall Survival: Unstratified Analysis - Participants With MZLUp to 8 years

OS in MZL participants was defined as the interval (in months) between the date of randomization and the date of the participant's death due to any cause. Kaplan-Meier method was used for the analysis. For this outcome measure, unstratified analysis was performed on participants with MZL.

Primary Analysis: Overall Response Rate (ORR): Stratified AnalysisUp to 8 years

ORR was defined as the percentage of participants who achieved a CR or partial response (PR). Criteria for CR: disappearance of all evidence of disease; mass of any size permitted if PET negative; regression to normal size on CT; spleen and liver: not palpable, nodules disappeared; bone marrow: infiltrate cleared on repeat biopsy and no new sites of disease detected during assessment. Criteria for PR: \>=50% decrease in sum of the diameter of all target lesions compared with baseline, in absence of new lesions or unequivocal progression of non-target lesions. Kaplan-Meier method was used for the analysis. Stratification factors were used for the analysis.

Primary Analysis: Time to Worsening (TTW) in the Lymphoma (Lym) Subscale of the Functional Assessment of Cancer Therapy - Lymphoma Subscale (FACT-LymS) QuestionnaireUp to 8 years

Time-to-worsening in the Lymphoma subscale of the FACT-Lym was defined as the time (in months) from the date of randomization to the start date of the worsening of participant symptoms. Worsening was defined by a 5-point decrease from baseline in participant symptoms. FACT-Lym Lymphoma subscale contains 15 questions, scores from 0 to 4 for each question (0 = not at all, 1 = a little bit, 2 = some what, 3 = quite a bit and 4 = very much, where the higher score indicated worse condition). Lymphoma subscale score is the total of reverse scores, range 0 to 60. Higher scores indicate a better quality of life.

Number of Participants With TEAEs: Participants With MZLPlacebo + Chemoimmunotherapy (CIT) arm: From Day 1 up to 30 days after date of last dose of study medication (up to 8 years); Ibrutinib + CIT arm: From Day 1 up to 30 days after date of last dose of study medication (up to 8 years 8 months)

Number of MZL participants with TEAEs were reported. AE was defined as any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE did not necessarily have a causal relationship with the pharmaceutical/biological agent under study. TEAEs were defined as adverse events with onset or worsening on or after date of first dose of study treatment up to and including 30 days after date of last dose of study medication.

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