Ibrutinib for Hairy Cell Leukemia

Not currently 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of ibrutinib (Imbruvica) for patients with recurrent hairy cell leukemia. Ibrutinib works by blocking specific enzymes that promote cancer cell growth. Suitable candidates for this trial have a diagnosis of hairy cell leukemia, have undergone at least one prior treatment, and experience symptoms such as severe tiredness, unexpected weight loss, or frequent fevers. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

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.

Is there any evidence suggesting that ibrutinib is likely to be safe for humans?

Research has shown that ibrutinib is generally safe and well-tolerated for patients with hairy cell leukemia. Studies have found it to be an effective treatment, particularly for those unable to undergo chemotherapy. The safety profile of ibrutinib in treating hairy cell leukemia aligns with its use in other conditions, offering reassurance to patients. This indicates that side effects are manageable, and the treatment is considered safe for most people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hairy cell leukemia?

Unlike the standard chemotherapy treatments for hairy cell leukemia, Ibrutinib acts on a different target, specifically inhibiting Bruton's tyrosine kinase (BTK). This unique mechanism of action disrupts the signaling pathways crucial for cancer cell survival and proliferation, offering a more targeted approach to treatment. Researchers are excited because it has the potential to be more effective and less toxic than conventional options, providing hope for better outcomes and improved quality of life for patients.

What evidence suggests that ibrutinib might be an effective treatment for hairy cell leukemia?

Research has shown that ibrutinib, a drug that inhibits certain enzymes necessary for cancer cell growth, might help treat hairy cell leukemia that returns after treatment. In this trial, participants will receive ibrutinib. A study found that patients who took ibrutinib showed positive results at both 32 and 48 weeks, with many experiencing either complete or partial remission, meaning the cancer became less active or disappeared. Ibrutinib has also been used successfully in other blood cancers, such as chronic lymphocytic leukemia (CLL), supporting its potential effectiveness in hairy cell leukemia. This treatment aims to stop cancer cells from growing and spreading, offering hope for those with relapsed hairy cell leukemia. ^{1 6 7 8 9}

Who Is on the Research Team?

Kerry A Rogers

Principal Investigator

Ohio State University Comprehensive Cancer Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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 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 (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 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]

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 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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8225920/

Phase 2 study of ibrutinib in classic and variant hairy cell ... Response to ibrutinib is shown at both preplanned response assessments at 32 and 48 weeks. Patients with either a CR or a PR were considered responders.

2. ashpublications.org ashpublications.org/blood/article/140/Supplement%201/6494/493127/Extended-Follow-up-of-a-Phase-2-Study-of-Ibrutinib

Extended Follow up of a Phase 2 Study of Ibrutinib in Hairy ... Efficacy and Safety of the Bruton Tyrosine Kinase Inhibitor Ibrutinib in Patients with Hairy Cell Leukemia: Stage 1 Results of a Phase 2 Study.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT01841723

Ibrutinib in Treating Patients With Relapsed Hairy Cell ... This phase II trial studies how well ibrutinib works in treating patients with hairy cell leukemia that has returned after a period of improvement.

4. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.e19547

A comprehensive systematic review and meta-analysis. The highest efficacy was seen in patients receiving combination therapy of cladribine and rituximab, with an ORR of 97% and CRR of 92%.

5. hairycellleukemia.org hairycellleukemia.org/blog/2024/5/2/btk-inhibitors-for-classic-hcl-and-hcl-variants

Exploring BTK Inhibitors in Classic HCL and HCL Variants Ibrutinib, the pioneering BTK inhibitor, has shown promising results in treating diseases such as chronic lymphocytic leukemia (CLL).

6. hairycellleukemia.org hairycellleukemia.org/published-research

Published Research in Hairy Cell Leukemia Ibrutinib can result in durable disease control in hairy cell leukemia patients who are not expected to benefit from purine analogues. The safety profile of ...

7. pharmacytimes.com pharmacytimes.com/view/study-ibrutinib-effective-treatment-for-difficult-to-treat-forms-of-hairy-cell-leukemia

Study: Ibrutinib Effective Treatment for Difficult to Treat ... “Our study shows that ibrutinib is a safe, effective and well-tolerated option for patients with relapsed or variant forms of hairy cell ...

8. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1002/ajh.27240

Hairy cell leukemia 2024: Update on diagnosis, risk‐ ... Safety data from the clinical trials either with vemurafenib include as adverse events (AES) and serious adverse events (SAES) skin toxicity ...

9. researchgate.net researchgate.net/publication/337318739_Efficacy_and_Safety_of_the_Bruton_Tyrosine_Kinase_Inhibitor_Ibrutinib_in_Patients_with_Hairy_Cell_Leukemia_Stage_1_Results_of_a_Phase_2_Study

Efficacy and Safety of the Bruton Tyrosine Kinase Inhibitor ... Preliminary results from a clinical trial by Jones et al., ibrutinib have shown 25% partial response in patients with HCL-v. 52 Although novel ...