NCT01500733Active, not recruitingPhase 2

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

A Phase II Study of PCI-32765 for Patients With Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) Who Need Therapy and Are Older Than 65 or Have a 17p Deletion

Source: ClinicalTrials.gov · Last updated 28 Apr 2026

Brief summary (registry, in English)

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.

Status (registry): Active, not recruiting
Phase (registry): Phase 2
Study type: Interventional
Enrollment: 86 (actual)
First submitted: 22 Dec 2011
Start date: 5 Jan 2012
Primary completion date: 20 Jun 2014
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Diseases: Chronic lymphocytic leukaemia
NCT number: NCT01500733

What you commit to

Study type: Interventional · Phase 2
Participants enrolled: 86
From 5 Jan 2012 to the primary completion date, 20 Jun 2014
1 site in 1 country
Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

Previous radiotherapy, radioimmunotherapy, biological therapy, chemotherapy, or treatment with an investigational product fpr CCL treatement in the last 4 weeks (i.e. intravenous immunoglobulin).
Concomitant immunotherapy, chemotherapy, radiotherapy, corticosteroids (at dosages equivalent to prednisone \> 20 mg/day), or experimental therapy
Any prior therapy with PCI 32765 or any other BTK inhibitors.
Requires treatment with strong CY3A4/5 and/or CYP2D6 inhibitors (unless no alternative is available).

Other illness

Autoimmune hemolytic anemia or thrombocytopenia requiring steroid therapy
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 PO, or put the study outcomes at undue risk
Active Hepatitis B infection
HIV infection
Psychiatric illness/social situations that would limit the patient s ability to tolerate and/or comply with study requirements.
Known hypersensitivity to any component of PCI-32765

Lab values

Impaired hepatic function: Total bilirubin greater than or equal to 1.5 times upper limit of normal unless dute to Gilbert's disease, AST/ ALT greater than or equal to 2.5 times institutional upper limit of normal unless due to infiltration of the liver.
Impaired renal funtion: Creatinine greater than or equal to 2.0 mg/dL or GFR less than or equal to 50ml/min

Age

Individuals \< 18 yrs old

Other

Transformed CLL
Female patients: Current pregnancy or unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential or currently breastfeeding. Male patients who are unwilling to follow the contraception requirements described in this protocol.
Unable to understand the investigational nature of the study or give informed consent.
Requires anticoagulation with warfarin.

Criteria the registry did not label

-INCLUSION CRITERIA:
Cohort 1: Treated and untreated patients age 65 or older and need for therapy
- Cohort 2: Treated (maximum accrual n=16) and untreated (n=27, evaluable) patients at least 18 years old with 17p deletion or p53 expression by immunohistochemistry or p53 mutation by sequencing analysis.
Men and women with histologically confirmed disease as defined by the following:
- B-lymphocytosis greater than 5000 cells/microL (may be less than 5000 cells/microL if lymphadenopathy is present with histologic confirmation of lymph node involvement by SLL)
- Immunophenotypic profile read by an expert pathologist as consistent with CLL. This will include CD5, CD19, and CD20 expression by the CLL cells typically also with CD23 expression, but CD23 negative cases may be included if there is no t11;14 translocation present.
Active disease as defined by at least one of the following:
- Weight loss greater than or equal to 10% within the previous 6 months
- Extreme fatigue
- Fevers of greater than 100.5 degrees F for greater than or equal to 2 weeks without evidence of infection
- Night sweats for more than one month without evidence of infection
- Evidence of progressive marrow failure as manifested by the development of, or worsening of, anemia and/or thrombocytopenia
- Massive or progressive splenomegaly
- Massive nodes or clusters or progressive lymphadenopathy
- Progressive lymphocytosis with an increase of greater than 50% over a 2 month period, or an anticipated doubling time of less than 6 months
- Compensated autoimmune hemolysis
Eastern Cooperative Oncology Group (ECOG) performance status of less than or equal to 2
ANC greater than 500/microL, platelets greater than 30,000/microL
Agreement to use contraception during the study and for 90 days after the last dose of study drug if sexually active and able to bear children
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)

Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

Sites in registry order. Pick your city on the disease page to sort them by distance.

Site	City	Country	Site status
National Institutes of Health Clinical Center, 9000 Rockville Pike Coordinator of this site? Confirm its status	Bethesda	United States	Unknown status

Related publications

A decade of ibrutinib for CLL with and without TP53 aberration: final report on an investigator-sponsored phase 2 study.Blood · 2026 · PMID 41405491

This page is information, not medical advice. Discuss any trial with your doctor.