20 Participants Needed

Venetoclax for Relapsed Hairy Cell Leukemia

Recruiting at 11 trial locations
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: National Cancer Institute (NCI)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This phase II trial tests how well venetoclax works in treating patients with hairy cell leukemia that has come back after a period of improvement (relapsed). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot be on other investigational agents, and live vaccines should not be given within 4 weeks before, during, or 30 days after the study treatment.

Is Venetoclax generally safe for humans?

In a study involving patients with a specific type of blood disorder, Venetoclax was generally well-tolerated, with serious side effects occurring in 19% of patients, including 7% due to infections. This suggests that while Venetoclax can cause some serious side effects, it is generally considered safe for use in humans.12345

How is the drug Venetoclax unique for treating relapsed hairy cell leukemia?

Venetoclax is unique because it is an oral drug that selectively inhibits the BCL-2 protein, which helps cancer cells survive. This mechanism is different from traditional chemotherapy, offering a targeted approach that has shown effectiveness in other blood cancers like chronic lymphocytic leukemia.678910

What data supports the effectiveness of the drug Venetoclax for treating relapsed Hairy Cell Leukemia?

Venetoclax has shown effectiveness in treating other blood-related conditions, such as relapsed/refractory multiple myeloma and light-chain amyloidosis, with a high response rate in patients with specific genetic markers. This suggests potential effectiveness in similar blood cancers like Hairy Cell Leukemia.12111213

Who Is on the Research Team?

RJ

Robert J Kreitman

Principal Investigator

National Cancer Institute LAO

Are You a Good Fit for This Trial?

This trial is for adults over 18 with relapsed hairy cell leukemia after prior treatment, who can't have BRAF therapy and haven't tried Venetoclax. They need functioning kidneys, acceptable liver tests, and a decent performance status. People with treated hepatitis C or controlled HIV are eligible; those with chronic hepatitis B must be on suppressive therapy.

Inclusion Criteria

Total bilirubin ≤ 3 x institutional upper limit of normal (ULN) unless consistent with Gilbert's (ratio between total and direct bilirubin > 5)
I can take care of myself but may not be able to do heavy physical work.
My hepatitis B virus load is undetectable with treatment.
See 12 more

Exclusion Criteria

I have previously been treated with venetoclax.
I do not have conditions that affect how my body absorbs nutrients.
Patients who are receiving any other investigational agents
See 4 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive venetoclax orally once daily on days 1-28 of each cycle, repeating every 28 days for up to 19 cycles

Up to 19 months
Monthly visits for each cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

4 weeks
1 visit (in-person) at 30 days post-treatment

What Are the Treatments Tested in This Trial?

Interventions

  • Venetoclax
Trial Overview The study is testing Venetoclax's effectiveness in patients whose hairy cell leukemia has returned. It involves taking the drug and undergoing various assessments like blood tests, bone marrow procedures, CT scans, and MRIs to see how well it works against cancer cells.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment (venetoclax)Experimental Treatment7 Interventions

Venetoclax is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Venclexta for:
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Approved in European Union as Venclyxto for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Published Research Related to This Trial

In a study of 43 patients with light-chain amyloidosis (AL) treated with venetoclax, the overall hematologic response rate was 68%, with 63% achieving very good partial response (VGPR) or complete response (CR), indicating that venetoclax is an effective treatment option for AL.
Patients with the t(11;14) genetic alteration had significantly better outcomes, with an 81% hematologic response rate and a median progression-free survival (PFS) that was not reached, compared to 40% response and a median PFS of 6.7 months for those without this alteration.
Venetoclax induces deep hematologic remissions in t(11;14) relapsed/refractory AL amyloidosis.Premkumar, VJ., Lentzsch, S., Pan, S., et al.[2021]
A 71-year-old patient with recurrent cervical cancer who is a compound heterozygote for UGT1A1*6 and UGT1A1*28 experienced significant hematotoxicity (grade 4 neutropenia, grade 3 anemia, and grade 4 thrombocytopenia) while receiving irinotecan hydrochloride (CPT-11) at a dose of 60 mg/m², indicating potential risks associated with this treatment in similar patients.
Despite the absence of previous case reports linking serious adverse events to CPT-11 in this genetic context, the observed severe side effects suggest that careful monitoring and dose adjustments are necessary for patients with these UGT1A1 variants undergoing combined chemotherapy.
Recurrent cervical cancer in a patient who was compound heterozygous for UGT1A1*6 and UGT1A1*28 presenting with serious adverse events during irinotecan hydrochloride/nedaplatin therapy.Takatori, E., Shoji, T., Miura, Y., et al.[2018]
The maximum tolerated dose (MTD) of irinotecan for patients with the UGT1A1 *1/*1 genotype is 310 mg/m2, while for those with the *1/*28 genotype, it is 260 mg/m2, indicating that genetic differences can influence drug tolerance in metastatic colorectal cancer treatment.
Bevacizumab does not significantly affect the pharmacokinetics of irinotecan, suggesting that the dosing adjustments based on genotype can be made without concern for altered drug metabolism due to this additional treatment.
Genotype-Guided Dosing Study of FOLFIRI plus Bevacizumab in Patients with Metastatic Colorectal Cancer.Toffoli, G., Sharma, MR., Marangon, E., et al.[2022]

Citations

Venetoclax induces deep hematologic remissions in t(11;14) relapsed/refractory AL amyloidosis. [2021]
Recurrent cervical cancer in a patient who was compound heterozygous for UGT1A1*6 and UGT1A1*28 presenting with serious adverse events during irinotecan hydrochloride/nedaplatin therapy. [2018]
Genotype-Guided Dosing Study of FOLFIRI plus Bevacizumab in Patients with Metastatic Colorectal Cancer. [2022]
Studies of the efficacy and pharmacology of irinotecan against human colon tumor xenograft models. [2018]
Chemo-sensitivity in a panel of B-cell precursor acute lymphoblastic leukemia cell lines, YCUB series, derived from children. [2013]
First-line liposomal irinotecan with oxaliplatin, 5-fluorouracil and leucovorin (NALIRIFOX) in pancreatic ductal adenocarcinoma: A phase I/II study. [2021]
Intermittent, repetitive administrations of irinotecan (CPT-11) reduces its side-effects. [2019]
Irinotecan hydrochloride for the treatment of recurrent and refractory non-Hodgkin lymphoma: a single institution experience. [2019]
Venetoclax: First Global Approval. [2018]
Single-Center Retrospective Clinical Evaluation of Venetoclax Combined with HMAs and Half-Dose CAG for Unfit or Refractory/Relapsed AML. [2023]
Venetoclax: A Review in Relapsed/Refractory Chronic Lymphocytic Leukemia. [2020]
12.United Statespubmed.ncbi.nlm.nih.gov
Venetoclax: Management and Care for Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia . [2018]
Venetoclax: A Review in Previously Untreated Chronic Lymphocytic Leukaemia. [2021]
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