Chronic Lymphocytic LeukemiaPhase 2STOP-CLL-DCTNCT07526597
Study on Treatment Outcome Patterns for Patients With CLL After Discontinuation of BTK Inhibitors
A Phase 2 study testing Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapy in people with Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma, run by UNC Lineberger Comprehensive Cancer Center at 1 site in United States.
Trial summary
This Phase II hybrid decentralized multicenter study examines the outcomes of stopping Bruton tyrosine kinase inhibitor (BTKi) therapy in patients with chronic lymphocytic leukemia (CLL) who have remained in remission for at least two years. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
- What is being tested?
- Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2026to Jun 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- In order to participate in this study a subject must meet all of the eligibility criteria outlined below.
- Written informed consent obtained to participate in the study and HIPAA authorization for release of personal health information.
- Subjects is willing and able to comply with study procedures based on the judgement of the investigator.
- Age ≥ 18 years at the time of consent.
- Eastern Cooperative Oncology Group Performance Status of 0-3 or Karnofsky Performance Status of ≥40
- Confirmed of a diagnosis of CLL (Chronic Lymphocytic Leukemia) or SLL (Small Lymphocytic Lymphoma) according to International Workshop on Chronic Lymphocytic Leukemia 2018 Guidelines at any point prior to study enrollment.
- At the time of enrollment, patients must be receiving treatment with a covalent Bruton tyrosine kinase inhibitor in the first or second line setting and have been receiving this treatment for at a minimum 2 years. Patients may have previously received anti-CD20 monoclonal antibodies (such as rituximab or obinutuzumab) in combination with the cBTKi.
Who cannot join
- Patients must not have evidence of progressive CLL as defined by IWCLL 2018 criteria.
- Participants with history of malignancy other than CLL/SLL are allowed
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill
Chapel Hill, North Carolina, 27599
Brice Messenger, 919-984-0000, brice_messenger@med.unc.edu
Deborah Stephens, DO
About this study
This Phase II hybrid decentralized multicenter study examines the outcomes of stopping Bruton tyrosine kinase inhibitor (BTKi) therapy in patients with chronic lymphocytic leukemia (CLL) who have remained in remission for at least two years. The primary objective is to determine how long patients remain free from disease progression or death after discontinuing treatment, measured as event free survival.
The study also evaluates whether stopping BTKi therapy leads to improvements in quality of life and reductions in treatment related side effects. Researchers will follow patients over time to assess if the cancer returns, whether resistance to therapy develops, and how patients feel during the treatment free period.
Read more
In addition, the trial will investigate how discontinuing BTKi therapy affects immune function, including whether immune recovery occurs and infection risk decreases.
Preliminary data indicate that patients may experience a treatment free interval exceeding two years after stopping therapy, with associated improvements in quality of life. By prospectively evaluating a time limited treatment strategy, this trial aims to determine whether durable disease control can be maintained off therapy while also assessing the resolution of BTKi related adverse events and changes in patient reported outcomes.
Overall, the study seeks to determine whether patients can safely discontinue BTKi therapy and potentially restart treatment later if needed, thereby maintaining disease control while reducing the burden of continuous therapy.
- Being tested
- Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapy
- Keywords
- Bruton's tyrosine kinase inhibitors (BTKi)
- Medical subject headings
- Leukemia, Lymphocytic, Chronic, B-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapyOther
Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapy.
| Study group | Role | What they receive |
|---|---|---|
| Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) | Other | Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have been receiving covalent Bruton tyrosine kinase inhibitors for at least 2 years and have achieved a clinical remission. Receives: Other: Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival (EFS)
EFS will be defined as the proportion of patients who remain alive, do not meet International Workshop on Chronic Lymphocytic Leukemia (IWCLL) criteria for initiation of next chronic lymphocytic leukemia (CLL) therapy, and have not started a new CLL - directed treatment.
Measured: 2 years
Secondary outcomes (10)
Progression Free Survival (PFS)
Progression Free Survival (PFS) will be defined as the proportion of patients who remain alive, free of relapse from complete response or partial response, free of progression of chronic lymphocytic leukemia (CLL), and have not started a new CLL treatment at 2 years after discontinuation of Bruton tyrosine kinase inhibitors (BTKi).
Measured: 2 years
Overall Survival (OS)
Overall Survival (OS) will be defined as the proportion of patients who remain alive after discontinuation of Bruton tyrosine kinase inhibitors (BTKi).
Measured: Up to 60 months
Time to initial detection of measurable residual disease (MRD)
Time to initial detection of measurable residual disease (MRD) or clinical evidence of progression will respectively be defined as the time from discontinuation of BTKi to initial detection of MRD (via flow cytometry) or progression of CLL that meets IWCLL criteria and/or treating physician requirement for initiation of next CLL therapy.
Measured: Up to 60 months
Overall and complete response rates
Overall and complete response rates will be defined as the percentage of patients who respectively achieve overall and complete response to next CLL therapy, based on IWCLL criteria.
Measured: 2 years
Rate of resolution of BTKi-related adverse events
Rate of resolution of BTKi-related adverse events will be defined as the percentage of patients whose adverse events attributed to BTKi treatment have improved, subsided, or completely disappeared after discontinuation of BTKi, to the extent that the patient's condition returns to baseline or to a clinically acceptable grade (typically Grade ≤ 1 per CTCAE) as graded by NCI CTCAE v5.0 and Quality of Life assessments.
Measured: 2 years
Incidence of new infections
Incidence of new infections will be defined as the percentage of patients who respectively have new infections after discontinuation of BTKi
Measured: 1 year
Incidence of second cancers
Incidence of second cancers will be defined as the percentage of patients who respectively have second cancers after discontinuation of BTKi.
Measured: 5 years
Association between biomarkers and disease outcomes
Association between biomarkers and disease outcomes as defined by EFS, PFS, OS, PFS, duration of response, duration of MRD undetectable disease state, and Richter's transformation (transforms into a fast-growing lymphoma).
Measured: 5 years
Change in Total Score of Functional Assessment of Cancer Therapy (FACT) -Leukemia
Change in FACT -Leukemia Total Score from prior to discontinuation of BTKi to 6 months after discontinuation of BTKi.
The FACT-Leukemia is a patient-reported outcome questionnaire designed to assess quality of life in patients with leukemia. It includes 27 general items covering physical, social/family, emotional, and functional well-being, along with 17 leukemia-specific items addressing symptoms, treatment concerns, and side effects, for a total of 44 items. Each item is rated on a 5-point Likert scale from 0 ("Not at all") to 4 ("Very much"), with some items reverse-scored for consistency. Scores are summed to create subscale scores and a total score, with higher scores indicating better quality of life. The instrument is commonly used in clinical trials and routine care to monitor patient well-being, evaluate treatment impact, and assess the effects of therapy from the patient's perspective.
Measured: Baseline and up to 6 months
Change in Functional Assessment of Cancer Therapy (FACT) -Leukemia physical, social, emotional, and functional well-being components
Change in FACT-Leukemia physical, social, emotional, and functional well-being component scores from prior to discontinuation of BTKi to 6 months after discontinuation of BTKi FACT-Leu evaluates patient-reported quality of life across four domains: physical, social/family, emotional, and functional well-being, capturing how a patient's condition evolves over time during or after treatment. Each domain consists of multiple items scored on a 5-point Likert scale ranging from 0 ("not at all") to 4 ("very much"), with domain scores calculated by summing item responses within each domain. A total score may be derived by summing the four core domains (FACT-G) and, when applicable, including leukemia-specific items for the FACT-Leu total score. Higher scores indicate better quality of life.
Measured: Baseline and up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2026
- Study startSep 2026 (estimated)
- Primary completionJun 2032 (estimated)
- Study completionJun 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Lymphocytic LeukemiaStudies of the same condition, open and under way.
- More trials run by UNC Lineberger Comprehensive Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- UNC Lineberger Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Deborah Stephens, DO, Principal investigator, UNC Lineberger Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07526597
- Sponsor's protocol ID
- LCCC2522-DCT
- Acronym
- STOP-CLL-DCT
- Status last verified
- Aug 2026
- First submitted
- 6 Apr 2026
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study on Treatment Outcome Patterns for Patients With CLL After Discontinuation of BTK Inhibitors
- ClinicalTrials.gov ID
- NCT07526597
- Acronym / sponsor ID
- STOP-CLL-DCT / LCCC2522-DCT
- Keywords
- Bruton's tyrosine kinase inhibitors (BTKi)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- UNC Lineberger Comprehensive Cancer CenterUniversity, hospital or other
- Study start
- Sep 2026 (estimated)
- Primary completion
- Jun 2032 (estimated)
- Study completion
- Jun 2032 (estimated)
- First posted
- 13 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase II hybrid decentralized multicenter study examines the outcomes of stopping Bruton tyrosine kinase inhibitor (BTKi) therapy in patients with chronic lymphocytic leukemia (CLL) who have remained in remission for at least two years. The primary objective is to determine how long patients remain free from disease progression or death after discontinuing treatment, measured as event free survival.
The study also evaluates whether stopping BTKi therapy leads to improvements in quality of life and reductions in treatment related side effects. Researchers will follow patients over time to assess if the cancer returns, whether resistance to therapy develops, and how patients feel during the treatment free period.
In addition, the trial will investigate how discontinuing BTKi therapy affects immune function, including whether immune recovery occurs and infection risk decreases.
Preliminary data indicate that patients may experience a treatment free interval exceeding two years after stopping therapy, with associated improvements in quality of life. By prospectively evaluating a time limited treatment strategy, this trial aims to determine whether durable disease control can be maintained off therapy while also assessing the resolution of BTKi related adverse events and changes in patient reported outcomes.
Overall, the study seeks to determine whether patients can safely discontinue BTKi therapy and potentially restart treatment later if needed, thereby maintaining disease control while reducing the burden of continuous therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) | Other | Other: Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapyPatients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have been receiving covalent Bruton tyrosine kinase inhibitors for at least 2 years and have achieved a clinical remission. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- In order to participate in this study a subject must meet all of the eligibility criteria outlined below.
- Written informed consent obtained to participate in the study and HIPAA authorization for release of personal health information.
- Subjects is willing and able to comply with study procedures based on the judgement of the investigator.
- Age ≥ 18 years at the time of consent.
- Eastern Cooperative Oncology Group Performance Status of 0-3 or Karnofsky Performance Status of ≥40
Key exclusion criteria (2 listed)
- Patients must not have evidence of progressive CLL as defined by IWCLL 2018 criteria.
- Participants with history of malignancy other than CLL/SLL are allowed
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapyDiscontinuing Bruton tyrosine kinase inhibitors (BTKi) therapy. | Other | Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) |
Primary outcome
Event-free survival (EFS)
Time frame: 2 years
EFS will be defined as the proportion of patients who remain alive, do not meet International Workshop on Chronic Lymphocytic Leukemia (IWCLL) criteria for initiation of next chronic lymphocytic leukemia (CLL) therapy, and have not started a new CLL - directed treatment.
10 secondary outcomes
Progression Free Survival (PFS)
Time frame: 2 years
Overall Survival (OS)
Time frame: Up to 60 months
Time to initial detection of measurable residual disease (MRD)
Time frame: Up to 60 months
Overall and complete response rates
Time frame: 2 years
Rate of resolution of BTKi-related adverse events
Time frame: 2 years
Incidence of new infections
Time frame: 1 year
Incidence of second cancers
Time frame: 5 years
Association between biomarkers and disease outcomes
Time frame: 5 years
Change in Total Score of Functional Assessment of Cancer Therapy (FACT) -Leukemia
Time frame: Baseline and up to 6 months
Change in Functional Assessment of Cancer Therapy (FACT) -Leukemia physical, social, emotional, and functional well-being components
Time frame: Baseline and up to 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2026.
- University of North Carolina Lineberger Comprehensive Cancer Center Clinical TrialsLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Deborah Stephens, DO principal investigator
UNC Lineberger Comprehensive Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Discontinuing Bruton tyrosine kinase inhibitors (BTKi) therapy in people with Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma, run by UNC Lineberger Comprehensive Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Brice Messenger (919-984-0000, brice_messenger@med.unc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free survival (EFS) (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2026.