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Venetoclax

Clinical trials investigating Venetoclax are studying how well it works in different diseases, along with safety and tolerability. These trials include patients with leukemia, lymphoma, multiple myeloma, and some other conditions, in both newly diagnosed and relapsed or refractory settings. Many studies compare Venetoclax combinations with other treatments or standard care.

Table of Contents

Overview of Venetoclax trials

These clinical trials study Venetoclax in many cancer settings and in a few other diseases.[1] Most studies test Venetoclax as part of a combination, not as a single treatment.[2] The main goals are to learn about safety, tolerability, and how well the treatment works in specific patient groups.[1]

The trial data show a wide range of study sizes, from small early studies to large Phase 3 trials with hundreds of patients.[2] Some studies are completed, while others are authorised or suspended.[1]

Conditions being studied

Many trials focus on acute myeloid leukemia (AML), including newly diagnosed AML, relapsed or refractory AML, and AML with specific genetic changes such as NPM1, KMT2A, FLT3, IDH1, or RARA-related disease.[3] Several studies also include chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), including first-line, relapsed, and high-risk disease.[4]

Other blood cancers in the trial set include mantle cell lymphoma, multiple myeloma, Waldenström’s macroglobulinemia, chronic myelomonocytic leukemia (CMML), and myelodysplastic syndrome (MDS).[5] There are also studies in blastic plasmacytoid dendritic cell neoplasm, Richter syndrome, and acute graft-versus-host disease of the skin.[6]

A few trials look beyond cancer, including studies in HIV-1 disease and HIV-1 infection, where the aim is to study safety and effects on the HIV reservoir, which means the hidden pool of virus in the body.[7]

Trial designs and phases

The trials include Phase 1, Phase 2, Phase 3, and some Phase 4 studies.[1] Phase 1 studies mainly check safety, dose limits, and the recommended dose for later testing.[8] Phase 2 studies often look at early signs of benefit, such as remission or MRD negativity.[5]

Phase 3 studies are larger and usually compare Venetoclax combinations with another treatment, placebo, or standard care.[4] Some studies are open-label, which means both the researchers and participants know which treatment is being given.[4] Other studies are randomized, meaning patients are assigned by chance to different treatment groups.[4]

Main endpoints being measured

The most common endpoints are overall survival (OS), progression-free survival (PFS), event-free survival (EFS), and response rates such as overall response rate (ORR) or complete remission (CR).[9] These endpoints help show whether a treatment helps people live longer, stay well longer, or have a better cancer response.[9]

Many trials also use minimal residual disease (MRD) or undetectable MRD (uMRD) as important endpoints.[10] MRD testing looks for very small amounts of cancer that may remain after treatment, even when the patient seems to be in remission.[10] Some studies also measure dose-limiting toxicities, adverse events, and pharmacokinetics, which is how the body handles a treatment.[11]

Who may be able to participate

Eligibility depends on the trial and the disease being studied.[3] Some studies are for people who have never been treated before, while others are for people whose cancer has returned or did not respond to earlier therapy.[1]

Several studies focus on special groups, such as older adults, people who are not fit for intensive chemotherapy, patients with specific genetic changes, or patients who already reached remission but still have MRD.[12] Some trials also look at people after stem cell transplantation or after previous Venetoclax treatment.[13]

Important trial examples

NCT02993523 studied Venetoclax plus azacitidine versus azacitidine alone in previously untreated AML, with outcomes of CR/CRi and overall survival.[9] NCT03069352 tested Venetoclax with low-dose cytarabine in untreated AML patients who were not eligible for intensive chemotherapy, with overall survival as the main endpoint.[14]

NCT04608318, NCT06073821, and NCT05057494 are large Phase 3 studies in previously untreated CLL or CLL/SLL that compare Venetoclax-based regimens with other treatment approaches and measure PFS.[4] NCT06943872 and NCT04965493 study relapsed or refractory CLL/SLL and compare Venetoclax-based retreatment or combinations against other options using PFS or response-based outcomes.[2]

NCT02899052 and NCT03539744 focus on multiple myeloma, especially t(11;14)-positive disease, and measure response rates and PFS.[15] NCT05099471 studies Venetoclax with rituximab in Waldenström’s macroglobulinemia and looks at complete or very good partial remission rates.[5]

NCT04581512, NCT06156579, NCT04763928, and NCT05431257 are examples of AML and related myeloid disorder studies that test Venetoclax with other agents and measure remission, EFS, or response rates.[1] NCT05807932 and NCT2025-521372-62-00 study Venetoclax around allogeneic stem cell transplantation and measure safety or survival after transplant.[13]

Patient-friendly explanation of key terms

Combination therapy means Venetoclax is given with one or more other drugs in the same study.[2] Maintenance therapy means treatment given after the main treatment phase to help keep the disease under control.[16]

Relapsed means the disease came back after treatment, and refractory means it did not respond well to treatment.[2] Randomized means patients are assigned by chance to different study groups, which helps compare treatments fairly.[4]

Open-label means the treatment is known to everyone in the study.[4] Blinded independent review or central review means outside experts check the results in a standard way, which helps reduce bias.[2]

Trial ID Phase Condition studied Status Enrollment
NCT06943872Phase 3Relapsed/Refractory CLL/SLLAuthorised629
NCT06073821Phase 3Previously untreated CLLAuthorised672
NCT04608318Phase 3Previously untreated CLLAuthorised909
NCT05057494Phase 3Previously untreated CLL/SLLAuthorised758
NCT07007312Phase 3Newly diagnosed AML with NPM1 or KMT2A changesAuthorised1320
NCT04401748Phase 3Higher-risk MDSAuthorised526
NCT02993523Phase 3Previously untreated AMLCompleted350
NCT03069352Phase 3Untreated AML ineligible for intensive chemotherapyCompleted129
NCT04628026Phase 3Newly diagnosed AML or MDS-EB2Authorised508
NCT04581512Phase 1/2AML, MDS, CMMLSuspended75
NCT05791409Phase 1Relapsed/Refractory CLL/SLLAuthorised112
NCT02899052Phase 2Relapsed/Refractory multiple myelomaAuthorised128
NCT05099471Phase 2Waldenström’s macroglobulinemiaAuthorised55
NCT05226455Phase 1MDS or AML after transplant relapseAuthorised55
NCT05768711Phase 2Higher-risk CMMLAuthorised44

FAQ

  • What kinds of conditions are being studied with Venetoclax? Most trials focus on blood cancers such as acute myeloid leukemia, chronic lymphocytic leukemia, small lymphocytic lymphoma, mantle cell lymphoma, and multiple myeloma. Some studies also include myelodysplastic syndrome, chronic myelomonocytic leukemia, and a few non-cancer conditions.
  • Who can join Venetoclax trials? It depends on the study. Some trials are for newly diagnosed patients, while others are for people whose disease has come back or did not respond to earlier treatment. Many studies also choose patients based on age, genetic changes, or whether they are fit for intensive treatment.
  • What are these trials trying to measure? Common goals include overall survival, progression-free survival, response rates, and minimal residual disease. Minimal residual disease means a very small number of cancer cells that may still remain after treatment.
  • What phases are Venetoclax trials in? The trials range from Phase 1 to Phase 4. Early phases usually focus on safety, dose finding, and tolerability, while later phases compare how well treatments work and whether they improve patient outcomes.
  • Do all studies use Venetoclax alone? No. Many studies test Venetoclax in combinations with other drugs such as azacitidine, obinutuzumab, rituximab, ibrutinib, or other study drugs. Some trials compare Venetoclax combinations with standard treatment or other new regimens.

Ongoing Clinical Trials on Venetoclax

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Glossary

  • Acute myeloid leukemia (AML): A fast-growing blood cancer that starts in the bone marrow, where blood cells are made.
  • Chronic lymphocytic leukemia (CLL): A slow-growing blood cancer that affects a type of white blood cell called lymphocytes.
  • Small lymphocytic lymphoma (SLL): A lymphoma that is closely related to CLL and involves the same type of cancer cells.
  • Relapsed disease: Cancer that has returned after treatment.
  • Refractory disease: Cancer that does not respond well to treatment.
  • Progression-free survival (PFS): The length of time a patient lives without the cancer getting worse.
  • Overall survival (OS): The length of time patients are alive after starting treatment or joining a study.
  • Minimal residual disease (MRD): A very small number of cancer cells that may remain after treatment and can be hard to detect.
  • Undetectable MRD (uMRD): No cancer cells are found with the test used in the study, down to the stated detection level.
  • Complete remission (CR): No clear signs of cancer are found after treatment.
  • Dose-limiting toxicity (DLT): A side effect that is serious enough to limit how much of a study treatment can be given.
  • Phase: A stage of a clinical trial. Early phases focus more on safety and dose, while later phases focus more on how well the treatment works.

References

  1. https://clinicaltrials.gov/study/NCT04581512
  2. https://clinicaltrials.gov/study/NCT06943872
  3. https://clinicaltrials.gov/study/NCT07007312
  4. https://clinicaltrials.gov/study/NCT04608318
  5. https://clinicaltrials.gov/study/NCT05099471
  6. https://clinicaltrials.gov/study/2024-520154-38-00
  7. https://clinicaltrials.gov/study/2024-521841-26-00
  8. https://clinicaltrials.gov/study/NCT05791409
  9. https://clinicaltrials.gov/study/NCT02993523
  10. https://clinicaltrials.gov/study/NCT04867928
  11. https://clinicaltrials.gov/study/NCT06014489
  12. https://clinicaltrials.gov/study/NCT06696183
  13. https://clinicaltrials.gov/study/NCT05807932
  14. https://clinicaltrials.gov/study/NCT03069352
  15. https://clinicaltrials.gov/study/NCT02899052
  16. https://clinicaltrials.gov/study/NCT04102020