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Safety and Efficacy of PCI-32765 in Participants With Relapsed/Refractory Mantle Cell Lymphoma (MCL)

Phase 2
Completed
Conditions
Mantle Cell Lymphoma
Interventions
Registration Number
NCT01236391
Lead Sponsor
Pharmacyclics LLC.
Brief Summary

The primary objective of this study was to evaluate the efficacy of ibrutinib in participants with relapsed or refractory MCL.

The secondary objective was to evaluate the safety of a fixed daily dosing regimen (560 mg daily) of PCI-32765 in this population.

Detailed Description

This is a Phase 2, open-label, nonrandomized, multicenter, monotherapy study in subjects with histologically documented MCL who have relapsed after ≥ 1 (but not \> 5) prior treatment regimens. All subjects meeting eligibility criteria will receive PCI-32765 capsules at a dosage of 560 mg/day once daily for a 28-day cycle until disease progression, unacceptable toxicity, or enrollment in a long-term extension study, whichever occurs earlier.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
115
Inclusion Criteria
  • Men and women ≥ 18 years of age
  • ECOG performance status of ≤ 2
  • Pathologically confirmed MCL, with documentation of either overexpression of cyclin D1 or t(11;14), and measurable disease on cross sectional imaging that is ≥ 2 cm in the longest diameter and measurable in 2 perpendicular dimensions
  • Documented failure to achieve at least partial response (PR) with, or documented disease progression disease after, the most recent treatment regimen
  • At least 1, but no more than 5, prior treatment regimens for MCL (Note: Subjects having received ≥2 cycles of prior treatment with bortezomib, either as a single agent or as part of a combination therapy regimen, will be considered to be bortezomib-exposed.)
  • Willing and able to participate in all required evaluations and procedures in this study protocol including swallowing capsules without difficulty
  • Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (in accordance with national and local subject privacy regulations)

Major exclusion criteria:

  • Prior chemotherapy within 3 weeks, nitrosoureas within 6 weeks, therapeutic anticancer antibodies within 4 weeks, radio- or toxin-immunoconjugates within 10 weeks, radiation therapy within 3 weeks, or major surgery within 2 weeks of first dose of study drug

  • Any life-threatening illness, medical condition or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety, interfere with the absorption or metabolism of PCI-32765 capsules, or put the study outcomes at undue risk

  • Clinically significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of screening, or any Class 3 or 4 cardiac disease as defined by the New York Heart Association Functional Classification

  • Malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel or ulcerative colitis, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction

  • Any of the following laboratory abnormalities:

    1. Absolute neutrophil count (ANC) < 750 cells/mm3 (0.75 x 109/L) unless there is documented bone marrow involvement
    2. Platelet count < 50,000 cells/mm3 (50 x 109/L) independent of transfusion support unless there is documented bone marrow involvement
    3. Serum aspartate transaminase (AST/SGOT) or alanine transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN)
    4. Creatinine > 2.0 x ULN
Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Participants received PCI-32765 560 mg dailyPCI-32765Participants were enrolled and received 560 mg/day dose, stratified into 2 groups based on prior bortezomib exposure.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants Achieving ResponseThe median follow-up time on study for all treated participants is 15.3 (range 1.9 - 22.3) months

The primary endpoint of the study was overall response rate (ORR), defined as the proportion of participants who achieved a best overall response of complete response (CR) or partial response (PR), according to the revised International Working Group Criteria for non-Hodgkin's lymphoma (Cheson et al, 2007), as assessed by the investigator. CR is a complete disappearance of all disease, no new lesions, lymph nodes must have regressed and be PET negative, spleen and liver should not be palpable and without nodules, and bone marrow must be negative. PR is a \>/= 50% decrease in the sum of the product of diameters of the target lesions, and \>/= 50% decrease of splenic and hepatic nodules from baseline, no new lesions and no increase in the size of liver, spleen or non-target lesions.

Secondary Outcome Measures
NameTimeMethod
Number of Participants With Treatment Emergent Adverse Events (AEs)From first dose of PCI-32765 to within 30 days of last dose for each participant or until study closure

Number of participants who had experienced at least one treatment emergent AE

Mean Change From Baseline to Cycle 5 in EORTC QLQ-C30 Global Health Status ScoreFrom Baseline to Cycle 5 (Week 20)

Mean change from baseline to Cycle 5 in the European Organisation for Research and Treatment of Cancer quality of life questionnaire (EORTC QLQ-C30) Global Health Status Score according to EORTC QLQ-C30 Scoring Manual (3rd Edition, 2001). For global health status, positive changes indicated better health status or functioning, and negative changes indicated worsening of health status or functioning. Scale scores range from 0 to 100. A change in 5 to 10 points in either direction represents a small change; 10 to 20 points represents a moderate change and greater than 20 points represents a large change.

PCI-32765 and Its Metabolite (PCI-45227) AUC0-24h After Repeat Dosing of PCI-32765Performed During the First Month of Receiving PCI-32765

Area under the plasma concentration-time curve using data collected at 0, 1, 2, 4, 6-8, and 24 hours post dose (AUC0-24h)

Trial Locations

Locations (18)

New York Presbyterian Hospital/Cornell Medical Center

🇺🇸

New York, New York, United States

Cll Research and Treatment Program

🇺🇸

New Hyde Park, New York, United States

Stanford University School of Medicine

🇺🇸

Stanford, California, United States

The Ohio Sate university

🇺🇸

Columbus, Ohio, United States

University of Virginia School of Medicine Hospital

🇺🇸

Charlottesville, Virginia, United States

Oregon Health & Science University

🇺🇸

Portland, Oregon, United States

Universitatsklinikum Ulm, Klinik fur Innere Medizin II

🇩🇪

ULM, Germany

University of Wisconsin

🇺🇸

Madison, Wisconsin, United States

Oddzail Kliniczny Onkologil

🇵🇱

Bydgoszcz, Poland

Klinikum der Universitat Munchen - Campus Grosshadern

🇩🇪

Munchen, Germany

Derriford Hospital

🇬🇧

Plymouth, United Kingdom

Southampton General Hospital

🇬🇧

Southampton, United Kingdom

Christie Hospital

🇬🇧

Manchester, United Kingdom

Centre for Experimental Cancer Medicine

🇬🇧

London, United Kingdom

Malopolskie Centrum Medyczne

🇵🇱

Krakow, Poland

Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

MTZ Clinical Research Sp. z o.o.

🇵🇱

Warsaw, Poland

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

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