A Study of Lonitoclax (ZE50-0134) in Relapsed or Refractory B-cell Malignancies
Phase 1 Study of Lonitoclax (ZE50-0134) in Relapsed and Refractory Chronic Lymphocytic Leukemia (CLL), Small Lymphocytic Lymphoma (SLL), and Select Low-grade Lymphomas
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests the safety and doses of lonitoclax, a medicine taken by mouth, and looks for early signs that it fights cancer. It is for adults with certain B-cell blood cancers that came back or did not respond to treatment, including chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).
This Phase 1, open-label, multicenter study is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of lonitoclax (ZE50-0134) in adults with relapsed or refractory chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), and select low-grade B-cell lymphomas. The study has two sequential parts. Part 1 uses dose escalation to determine the biologically effective dose and/or maximum tolerated dose of lonitoclax. Participants receive a 3-day step-up regimen followed by continuous once-daily or twice-daily oral dosing in 28-day cycles. Part 2 is a randomized dose-expansion comparison of two selected lonitoclax dose levels in venetoclax-naive participants with relapsed or refractory CLL/SLL. Treatment may continue for up to 12 cycles and, for participants deriving clinical benefit, for up to 24 cycles with approval from the Medical Monitor.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 18 or older and have cancer that the study doctor thinks needs treatment.
- For Part 1 with CLL or SLL: have had at least 2 prior treatments, including a covalent BTK inhibitor (a targeted cancer drug), and have taken or declined venetoclax.
- For Part 1 with certain slow-growing lymphomas: have worsening cancer after at least 1 treatment that included a BTK inhibitor or CD20 antibody treatment.
- For Part 2: have CLL or SLL after at least 1 prior treatment that included a BTK inhibitor, and never have taken venetoclax.
- Meet blood cell, liver, and kidney test limits. Be able to care for yourself and be out of bed or a chair for at least half the day.
- If able to become pregnant, have a negative pregnancy test within 7 days before the first dose and before each treatment cycle.
- If able to have children, agree to use highly effective birth control from signing consent until 90 days after the last dose.
- Understand and sign the consent form, including permission for genetic tests on tissue and blood samples.
Who can't join
- Prior venetoclax use rules out Part 2. Active Richter transformation, when CLL becomes a faster-growing cancer, also rules out joining.
- Allergy to lonitoclax, its ingredients, or another medicine given in the study.
- Certain serious heart problems or a heart rhythm test result above the study limit that cannot be corrected. The test limit does not apply to people with a pacemaker.
- Active CMV, hepatitis B, hepatitis C, or COVID-19 infection; HIV not well controlled with medicine; or a serious infection needing injected treatment. Past COVID-19 is allowed after full recovery more than 14 days earlier.
- Uncontrolled immune attacks on red blood cells or platelets within 28 days; a donor bone marrow transplant within 4 months; or ongoing medicines that weaken the immune system. Some steroid use is allowed.
- Another active cancer that requires treatment during the study or limits expected survival to less than 2 years, with some exceptions. Other findings that make study treatment too risky may also rule out joining.
- Major surgery or a serious injury within 4 weeks before the first dose.
- Breastfeeding must stop before treatment, throughout treatment, and for at least 3 months afterward.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Men and women aged 18 years or older. 2. Disease as defined below: * Part 1: Relapsed or refractory CLL or SLL, as defined by iwCLL, after at least 2 prior therapies that included a covalent Bruton tyrosine kinase inhibitor (BTKi) and venetoclax, or after the participant declined venetoclax; or progressive low-grade lymphoma, including marginal zone lymphoma or lymphoplasmacytic lymphoma (including Waldenstrom macroglobulinemia), after at least 1 prior therapy that included either a BTKi or CD20 antibody-based therapy. * Part 2: Relapsed or refractory CLL or SLL, as defined by iwCLL, after at least 1 prior therapy that included a BTKi; participants must be venetoclax naive. 3. Disease requiring therapy in the investigator's opinion. 4. Adequate bone marrow, liver, and renal function during screening: * Absolute neutrophil count greater than 0.75 x 10\^9/L; for participants with documented bone marrow involvement, at least 0.5 x 10\^9/L. * Platelet count greater than 50 x 10\^9/L; for participants with documented bone marrow involvement, at least 30 x 10\^9/L. * AST and ALT no greater than 3.0 times the upper limit of normal. * Total bilirubin no greater than 1.5 times the upper limit of normal. Participants with suspected or known Gilbert disease may have total bilirubin up to 3 times the upper limit of normal if predominantly unconjugated. * Creatinine- or cystatin C-based glomerular filtration rate at least 60 mL/min, or at least 40 mL/min with a normal urine neutrophil gelatinase-associated lipocalin level. Estimated GFR is calculated using the Modification of Diet in Renal Disease formula. 5. Eastern Cooperative Oncology Group performance status of 0, 1, or 2. 6. For women of childbearing potential, a negative serum or urine pregnancy test within 7 days before the first dose and a negative result before each treatment cycle. Pregnancy testing is not required for women older than 50 years with at least 12 months of amenorrhea; women aged 50 years or younger with at least 6 months of spontaneous amenorrhea and follicle-stimulating hormone greater than 40 mIU/mL; or permanently sterilized women, including hysterectomy, bilateral salpingectomy, or uterine ablation. 7. Women and men of reproductive potential must agree to use highly effective contraception from signing informed consent until 90 days after the last dose of study drug. 8. Ability to understand and willingness to sign written informed consent, including consent for genetic biomarker analyses from tissue and plasma, before study-specific procedures. Exclusion Criteria: 1. Part 2 only: Prior venetoclax treatment. 2. Known active Richter transformation. Participants previously treated for Richter transformation may be eligible if they have been in remission for more than 2 years, have no evidence of Richter transformation, and have CLL only. 3. Known hypersensitivity to lonitoclax, its excipients, or an agent administered in association with the study. 4. Clinically significant cardiac disease, including congestive heart failure greater than New York Heart Association Class II; uncontrolled coronary artery disease; unstable angina; new-onset angina or myocardial infarction within 6 months before first dose; major regional wall-motion abnormalities on baseline echocardiography; or cardiac arrhythmias requiring antiarrhythmic treatment other than beta-blockers or digoxin. 5. Known active cytomegalovirus, hepatitis B virus, or hepatitis C virus infection. 6. HIV-positive disease that is not adequately controlled by antiviral therapy. Participants with adequately controlled HIV may enroll. 7. Known active SARS-CoV-2 infection. Prior infection is allowed if the participant completely recovered more than 14 days previously. 8. Active clinically serious infection of Grade greater than 2 requiring parenteral therapy. Participants may be eligible after the infection resolves. 9. Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura within 28 days before enrollment. 10. Allogeneic bone marrow transplant within 4 months before first dose. Immunosuppressive therapy related to the transplant must be completed before enrollment. 11. Active cancer that limits expected survival to less than 2 years or requires anticancer therapy concomitantly with study treatment. Exceptions may include resected localized skin, breast, or prostate cancer and malignancies treated with hormonal or immune therapies alone; secondary cancers should be discussed with the Medical Monitor. 12. Physical examination or laboratory finding that contraindicates investigational therapy or otherwise places the participant at excessively high treatment risk in the investigator's opinion. 13. Requirement for ongoing immunosuppressive therapy, including systemic corticosteroids, for cancer or another condition. Topical or inhaled corticosteroids and low-dose systemic steroids of no more than 20 mg prednisone equivalent per day are permitted for comorbid conditions. Short courses above this dose before first dose and during Week 1 may be used for tumor flare. 14. Major surgery or significant trauma within 4 weeks before first dose. 15. Breastfeeding. Breastfeeding must be discontinued before and during treatment and for at least 3 months after treatment ends. 16. QT interval corrected using Fridericia's formula greater than 470 milliseconds that cannot be corrected with electrolyte replacement, hydration, or medication modification. This criterion does not apply to participants with a pacemaker.
Study sites (5)
KY
- Norton Cancer Institute, St. Matthews Campus · Louisville, KY 40207More trials in Louisville
NC
- University of North Carolina at Chapel Hill · Chapel Hill, NC 27599More trials in Chapel Hill
OH
- University of Cincinnati · Cincinnati, OH 45221More trials in Cincinnati
- The Ohio State University · Columbus, OH 43210More trials in Columbus
TX
- UT Southwestern Medical Center · Dallas, TX 75390More trials in Dallas