Actively Recruiting

Phase 2
Age: 18Years - 85Years
All Genders
NCT03986034

Early Clonal Dynamics During Venetoclax Treatment for Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL)

Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-02-24

75

Participants Needed

1

Research Sites

366 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Background: The drug venetoclax treats chronic lymphocytic leukemia (CLL). Researchers want to find better treatments for CLL. To do that, they need to learn how the drug affects CLL cancer cells and the immune system. Objective: To learn about genetic changes that happen during treatment of CLL with venetoclax. Eligibility: Adults ages 18 and older with relapsed or refractory CLL after at least 1 prior therapy Design: Participants will be screened under a separate protocol. In Phase 1, participants will get venetoclax free of charge through the NIH. Venetoclax is started at a low dose. The dose will be increased every week until participants reach their maximum tolerable dose. This usually take about 5 weeks. Participants will visit the NIH at least once per week. Visits will be about 4 hours. They may have to stay in the hospital to be observed. In Phase 2, participants will continue to get the drug through their local cancer doctor and their health insurance. Patients will also visit the NIH every 6 months, or if their disease progresses. At the NIH participants will have regular health assessments. These will include physical exams and a review of the medicines they are taking. They will talk about how they are feeling. The study included the following tests: Blood draws CT scans: Participants will lie in a machine that takes pictures of the body (maximum 3 per year) Bone marrow biopsies: A small amount of marrow will be taken out of the participant s hip bone with a needle. Optional lymph node biopsies: A small piece of the participant s tissue will be taken out with a needle. The study will last at least 2 years.

CONDITIONS

Official Title

Early Clonal Dynamics During Venetoclax Treatment for Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL)

Who Can Participate

Age: 18Years - 85Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) as defined by updated NCI criteria
  • Active disease showing at least one symptom such as significant weight loss, extreme fatigue, fever, night sweats, worsening marrow failure, enlarged spleen or lymph nodes, or rapidly increasing lymphocyte count
  • Have a designated hematologist/oncologist who will assume care and continue venetoclax after ramp-up phase
  • Completed G6PD testing to determine rasburicase suitability
  • Completed HLA testing to assess risk of allopurinol hypersensitivity
  • Age 18 years or older
  • ECOG performance status between 0 and 2
  • Agree to use acceptable contraception methods if sexually active and able to have children during treatment
  • Willing and able to participate in all study evaluations and procedures including swallowing capsules
  • Able to understand the investigational nature of the study and provide informed consent
Not Eligible

You will not qualify if you...

  • Female patients who are pregnant or nursing
  • Any uncontrolled active systemic infection
  • Any life-threatening illness, medical condition, or organ dysfunction that may compromise safety or study outcomes
  • Additional progressing malignancies requiring active treatment, except certain skin cancers and treated in situ cervical cancer
  • Richter's Transformation
  • Previous treatment with BCL-2 inhibitors
  • Use of strong CYP3A4 inhibitors
  • Significant gastrointestinal disease affecting function or absorption
  • Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
  • Concurrent systemic cancer treatments like immunotherapy, chemotherapy, or radiotherapy
  • Absolute neutrophil count less than 1000/microL or platelet count less than 30,000/microL unless related to CLL and expected to improve with treatment
  • Serum bilirubin more than 3 times the upper normal limit
  • Severe psychiatric, social, or cognitive issues limiting ability to comply with study requirements

AI-Screening

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Trial Site Locations

Total: 1 location

1

National Institutes of Health Clinical Center

Bethesda, Maryland, United States, 20892

Actively Recruiting

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Research Team

I

Ingrid C Frey

CONTACT

C

Christine E Gruessner, M.D.

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

BASIC_SCIENCE

Number of Arms

1

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