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PCI-32765 for Special Cases of Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Phase 2
Active, not recruiting
Conditions
Leukemia
Leukemia, Lymphocytyc
CLL (Chronic Lymphocytic Leukemia)
SLL (Small Lymphocytic Lymphoma)
Interventions
Registration Number
NCT01500733
Lead Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Brief Summary

Background:

- Chronic lymphocytic leukemia and small lymphocytic lymphoma (CLL/SLL) are types of blood or lymph node cancers that mostly affect the elderly. CLL/SLL both create abnormal white blood cells that hurt the immune system and make it more difficult to fight infections. These cancers are usually diagnosed after age 50; more than half of the people with CLL/SLL are over age 70. Elderly people often do not respond well to standard chemotherapy for CLL/SLL. They may have other health problems that make chemotherapy difficult. In addition, individuals who have a genetic abnormality called 17p deletion also do not respond well to standard treatments for CLL/SLL. Researchers want to test a new cancer treatment drug, PCI-32765, to see if it can treat CLL/SLL in these hard-to-treat groups.

Objectives:

- To see if PCI-32765 is a safe and effective treatment for CLL/SLL in older people and people with 17p deletion.

Eligibility:

* Individuals over 65 years of age who have CLL/SLL.

* Individuals at least 18 years of age who have CLL/SLL and 17p deletion.

Design:

* Participants will be screened with a medical history, physical exam, and imaging studies. Blood and urine samples will be taken. Optional bone marrow and lymph node biopsies may also be taken.

* Participants will take PCI-32765 capsules every day for 28 days (one cycle of treatment). Treatment will be monitored with frequent blood tests and clinic visits.

* PCI-32765 will be given for six cycles of treatment. Those who benefit from the drug will continue to take it as long as there are no side effects and the disease does not progress. Those who do not benefit will stop treatment and have regular followup exams.

Detailed Description

CLL/SLL is a malignancy of B cells that predominantly affects the elderly population. Diagnosis is typically made in adults over the age of 50 and more than half of the people with CLL/SLL are over the age of 70. Elderly patients in particular have other comorbidities that limit the tolerability of standard CLL/SLL chemoimmunotherapy regimens and they often have inferior response to these particular regimens. In addition, those patients with a 17p deletion also have inferior outcomes due to rapid progression of disease as well as refractoriness to standard treatment regimens.

It is still unclear what leads to the development of CLL/SLL. However, recent studies indicated that B cell receptor (BCR) signaling is an important contributor to the disease and could be a target for therapy. Bruton s Tyrosine Kinase (Btk) is an enzyme required for BCR signaling.

Ibrutinib is a potent and selective inhibitor of Bruton Tyrosine Kinase (Btk). In vitro lymphoma models have demonstrated that ibrutinib inhibits Btk. Inhibition of Btk blocks downstream BCR signaling pathways and thus prevents B cell proliferation. A phase 1 study of ibrutinib shows activity in multiple lymphomas including CLL/SLL.

This study will investigate the efficacy of ibrutinib for patients with CLL/SLL in patients that are older than 65 who are in need of therapy.This protocol is intended both for patients who have been untreated and those who have undergone other therapies.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
86
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Elderly greater than 65PCI 32765-
17p DeletioinPCI 32765-
Primary Outcome Measures
NameTimeMethod
Overall Response Rate at 6 Months6 months

The primary endpoint was response after 6 cycles of therapy. Overall response rate was calculated as complete response plus partial response, based on the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) 2008 criteria.as follows:

Complete response (CR): all group A and group B criteria are met

* Group A criteria: resolution of enlarged lymph nodes, normal size spleen and liver, absolute lymphocyte count \< 4,000/uL, normocellular bone marrow with \< 30% lymphocytes without nodules

* Group B criteria: improved blood count (platelet count \> 100,000/uL, hemoglobin \> 11.0 g/dL, neutrophils \> 1,500/uL)

Partial response (PR): at least 2 of the group A criteria plus one of the group B criteria are met

* Group A criteria: \>=50% decrease in target lymph nodes, \>=50% decrease in spleen size, \>=50% decrease in liver size, 50% reduction in marrow infiltrates

* Group B criteria: platelet count \> 100,000/uL, hemoglobin \> 11.0 g/dL, neutrophils \> 1,500/uL

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

🇺🇸

Bethesda, Maryland, United States

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