Ibrutinib for Hairy Cell Leukemia

Recruiting at 4 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: National Cancer Institute (NCI)

Must not be taking: Strong CYP3A inhibitors, Warfarin

Disqualifiers: Brain metastases, Uncontrolled illness, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 32 weeks

44 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stop taking any strong CYP3A4/5 inhibitors at least 7 days before starting ibrutinib. If you are on warfarin or require daily corticosteroids at a high dose, you may need to stop or adjust these medications as well. It's important to discuss your current medications with the study team to ensure they don't interfere with the trial.

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

In a study, Ibrutinib showed a 54% best overall response rate in patients with relapsed or variant hairy cell leukemia, and 73% of patients had no disease progression after 36 months, suggesting it may help control the disease. ^{1 2 3 4 5}

Is ibrutinib safe for humans?

Ibrutinib has been studied in various conditions and is generally considered safe, but it can cause side effects like diarrhea, fatigue, muscle pain, and nausea. Serious risks include bleeding, heart problems, and infections, so patients should be monitored closely. ^{1 2 4 6 7}

How is the drug ibrutinib unique in treating hairy cell leukemia?

Ibrutinib is unique because it is an oral medication that targets a specific protein called Bruton's tyrosine kinase, which is involved in the growth of cancerous B-cells. This makes it different from traditional chemotherapy, as it specifically disrupts cancer cell signaling, offering a novel approach for patients who do not respond well to standard treatments. ^{1 7 8 9 10}

What is the purpose of this trial?

This phase II trial studies how well ibrutinib works in treating patients with hairy cell leukemia that has returned after a period of improvement. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Research Team

Kerry A Rogers

Principal Investigator

Ohio State University Comprehensive Cancer Center

Eligibility Criteria

This trial is for adults with relapsed Hairy Cell Leukemia who've had prior treatment. They should have certain blood count levels, be fit enough for daily activities (ECOG <=2), and not be pregnant or breastfeeding. Contraception is required during the study. Exclusions include uncontrolled illnesses, recent major surgery, other cancers within 3 years (some exceptions apply), known allergies to similar drugs, active hepatitis B/C infection, strong CYP3A inhibitors use recently, and certain cardiovascular diseases.

Inclusion Criteria

Agreement to use adequate contraception for women of child-bearing potential and men

Specific laboratory criteria including creatinine, bilirubin, AST, PT/INR, PTT, and baseline peripheral blood cell counts

I have been diagnosed with hairy cell leukemia that needs treatment.

Exclusion Criteria

Pregnant or breastfeeding women

Incarceration at the time of enrollment

I have lingering side effects from previous cancer treatments.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ibrutinib orally once daily on days 1-28. Cycles repeat every 28 days for up to 8 cycles if lack of response, up to 12 cycles if failure to achieve an objective response, or continually at physician discretion.

32 weeks

Regular visits for bone marrow aspiration, biopsy, blood sample collection, and imaging

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

Up to 3 years

Follow-up every 3 months

Treatment Details

Interventions

• Ibrutinib

Trial Overview The trial tests Ibrutinib's effectiveness in patients whose Hairy Cell Leukemia has returned after initial improvement. It explores whether Ibrutinib can halt cancer cell growth by inhibiting enzymes necessary for their proliferation.

Participant Groups

Group I: Treatment (ibrutinib) Experimental Treatment 6 Interventions

Patients receive ibrutinib PO QD on days 1-28. Cycles repeat every 28 days for up to 8 cycles if lack of response to therapy, up to 12 cycles if failure to achieve an objective response (CR/PR), or continually at per physician discretion in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo bone marrow aspiration and biopsy, blood sample collection, and CT or ultrasound throughout the study.

Ibrutinib is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Imbruvica for:

• Chronic lymphocytic leukemia
• Mantle cell lymphoma
• Waldenström's macroglobulinemia
• Marginal zone lymphoma
• Graft-versus-host disease

Approved in United States as Imbruvica for:

• Chronic lymphocytic leukemia/small lymphocytic lymphoma
• Mantle cell lymphoma
• Waldenström's macroglobulinemia
• Marginal zone lymphoma
• Graft-versus-host disease

Approved in Canada as Imbruvica for:

• Chronic lymphocytic leukemia
• Mantle cell lymphoma
• Waldenström's macroglobulinemia
• Marginal zone lymphoma

Approved in Japan as Imbruvica for:

• Chronic lymphocytic leukemia
• Mantle cell lymphoma
• Waldenström's macroglobulinemia

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Findings from Research

Hairy cell leukemia (HCL) is effectively treated with purine analogs, but about half of patients relapse and may become resistant to these treatments, necessitating alternative therapies.

Targeting the BRAF-V600E mutation with specific inhibitors like vemurafenib or dabrafenib offers a promising chemotherapy-free strategy, especially for patients at risk of severe infections or those with relapsed HCL, and can be combined with rituximab for enhanced efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

How I treat refractory/relapsed hairy cell leukemia with BRAF inhibitors. Falini, B., De Carolis, L., Tiacci, E.[2022]

Ibrutinib is a targeted therapy specifically designed for treating chronic lymphocytic leukemia (CLL), which is a type of cancer that affects the blood and bone marrow.

This novel treatment works by inhibiting Bruton's tyrosine kinase (BTK), a key enzyme that helps cancer cells survive and proliferate, leading to improved patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ibrutinib (imbruvica): a novel targeted therapy for chronic lymphocytic leukemia. Parmar, S., Patel, K., Pinilla-Ibarz, J.[2021]

Ibrutinib (Imbruvica) is now approved for treating graft-versus-host disease in pediatric patients aged one year and older who have not responded to other systemic therapies, highlighting its efficacy in a challenging patient population.

The treatment carries significant risks, including severe side effects like hemorrhage, infections, and cardiac issues, necessitating careful monitoring and education for patients and caregivers about these potential adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Receives New Indication for Pediatric Graft-Versus-Host Disease. Aschenbrenner, DS.[2023]

Ibrutinib is an effective treatment for chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL), showing better outcomes than traditional therapies like chlorambucil and ofatumumab in phase III trials involving elderly and previously-treated patients.

The drug has a good safety profile, although it can cause some adverse effects like bleeding and atrial fibrillation; it is recommended in treatment guidelines for patients with high-risk disease, including those with specific genetic mutations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ibrutinib: A Review in Chronic Lymphocytic Leukaemia. Deeks, ED.[2021]

Ibrutinib is a first-in-class oral Bruton's tyrosine kinase inhibitor that effectively blocks B-cell signaling, leading to reduced proliferation and increased cell death in B cell malignancies, including chronic lymphocytic leukaemia (CLL) and mantle cell lymphoma (MCL).

It has received FDA approval for treating MCL in previously treated patients based on phase Ib/II study results and is currently undergoing further phase III trials for CLL, diffuse large B cell lymphoma (DLBCL), and other B cell cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ibrutinib: first global approval. Cameron, F., Sanford, M.[2022]

Ibrutinib (Imbruvica) is an effective treatment for chronic lymphocytic leukemia, targeting specific pathways to inhibit cancer cell growth.

Tasimelteon (Hetlioz) is beneficial for individuals with non-24-hour sleep-wake disorder, helping to regulate sleep patterns, while anti-inhibitor coagulant complex (Feiba) is used to prevent or reduce bleeding episodes in patients with hemophilia A or B, demonstrating its efficacy in managing bleeding risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmaceutical approval update. Goldenberg, MM.[2021]

Ibrutinib is a powerful oral medication that effectively treats relapsed/refractory mantle cell lymphoma (MCL) and chronic lymphocytic leukaemia (CLL), showing high response rates and significantly improving survival outcomes, especially in patients with poor prognostic factors like chromosome 17 deletion (del 17p).

The drug has a good safety profile, with less than 10% of patients discontinuing treatment due to side effects, making it a promising option for patients with these types of blood cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ibrutinib: a review of its use in patients with mantle cell lymphoma or chronic lymphocytic leukaemia. Kim, ES., Dhillon, S.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Phase 2 study of ibrutinib in classic and variant hairy cell leukemia. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

How I treat refractory/relapsed hairy cell leukemia with BRAF inhibitors. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Variant form of hairy cell leukemia. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Ibrutinib (imbruvica): a novel targeted therapy for chronic lymphocytic leukemia. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Hairy cell leukemia: treatment prospects. [2007]

6. United States pubmed.ncbi.nlm.nih.gov

Drug Receives New Indication for Pediatric Graft-Versus-Host Disease. [2023]

7. New Zealand pubmed.ncbi.nlm.nih.gov

Ibrutinib: A Review in Chronic Lymphocytic Leukaemia. [2021]

8. New Zealand pubmed.ncbi.nlm.nih.gov

Ibrutinib: first global approval. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Pharmaceutical approval update. [2021]

10. New Zealand pubmed.ncbi.nlm.nih.gov

Ibrutinib: a review of its use in patients with mantle cell lymphoma or chronic lymphocytic leukaemia. [2022]