NCT01841723

Brief Summary

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
44

participants targeted

Target at P25-P50 for phase_2

Timeline
44mo left

Started Apr 2013

Longer than P75 for phase_2

Geographic Reach
1 country

5 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress78%
Apr 2013Dec 2029

First Submitted

Initial submission to the registry

April 24, 2013

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 26, 2013

Completed
4 days until next milestone

Study Start

First participant enrolled

April 30, 2013

Completed
16.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2029

Last Updated

April 13, 2026

Status Verified

March 1, 2026

Enrollment Period

16.7 years

First QC Date

April 24, 2013

Last Update Submit

April 9, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • Overall response rate (complete response [CR] and partial response [PR])

    Calculated as the proportion of patients who achieve a PR or CR to therapy within the first 32 weeks of therapy divided by the total number of evaluable patients.

    32 weeks

Secondary Outcomes (9)

  • Incidence of adverse events

    Up to 30 days after treatment

  • Progression-free survival

    From the date of study registration to the date of event or the date of last follow-up if no event has occurred, assessed up to 3 years

  • Overall survival

    From the date of study registration to the date of event or the date of last follow-up if no event has occurred, assessed up to 3 years

  • Rate of molecular remission (minimal residual disease [MRD]-negative CR)

    Up to 32 weeks

  • Immunologic outcomes during single agent ibrutinib administration

    Up to 12 months

  • +4 more secondary outcomes

Other Outcomes (1)

  • Pharmacokinetic (PK) parameters

    Predose, 0.5, 1, 2, 4, 6, and 24 hours post-dose on days 1 and 8 of cycle 1 and predose and 2 hours on days 15 and 22 of cycle 1 and day 1 of cycle 2

Study Arms (1)

Treatment (ibrutinib)

EXPERIMENTAL

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.

Procedure: Biospecimen CollectionProcedure: Bone Marrow AspirationProcedure: Bone Marrow BiopsyProcedure: Computed TomographyDrug: IbrutinibProcedure: Ultrasound Imaging

Interventions

Undergo bone marrow aspiration and biopsy

Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow
Treatment (ibrutinib)

Undergo CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography
Treatment (ibrutinib)

Given PO

Also known as: BTK Inhibitor PCI-32765, CRA 032765, CRA-032765, CRA032765, Imbruvica, PCI 32765, PCI-32765, PCI32765
Treatment (ibrutinib)

Undergo ultrasound

Also known as: 2-Dimensional Grayscale Ultrasound Imaging, 2-Dimensional Ultrasound Imaging, 2D-US, Ultrasonography, Ultrasound, Ultrasound Test, Ultrasound, Medical, US
Treatment (ibrutinib)

Undergo bone marrow aspiration and biopsy

Treatment (ibrutinib)

Undergo blood sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection
Treatment (ibrutinib)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Histologically confirmed diagnosis of hairy cell leukemia or variant according to World Health Organization (WHO) criteria with any of the following indications for therapy:
  • Hemoglobin \< 11 g/dL
  • Platelet count \< 100,000/mL
  • Absolute neutrophil count \< 1,000/mL
  • Progressive or symptomatic splenomegaly or hepatomegaly
  • Enlarging lymphadenopathy \>= 2 cm
  • Absolute lymphocyte count \> 5,000/mL
  • Disease related constitutional symptoms consisting of unexplained weight loss exceeding 10% of body weight over the preceding 6 months, Cancer Therapy Evaluation Program (CTEP) active version of the Common Terminology Criteria for Adverse Events (CTCAE) grade 2 or 3 fatigue, fevers \> 100.5 degrees Fahrenheit (F) or night sweats for greater than 2 weeks without evidence of infection
  • Patients with classic hairy cell leukemia may receive therapy under the following conditions:
  • After at least 1 prior purine nucleoside analog-containing regimen (fludarabine, pentostatin, or cladribine), or
  • Relapsed or de novo disease if deemed medically unfit for therapy with a purine nucleoside analog
  • Because there is no recognized standard of care for patients with variant hairy cell leukemia, both previously treated and previously untreated patients with this diagnosis will be eligible
  • Age ≥ 18 years. Because no dosing or adverse event data are currently available on the use of ibrutinib in patients \< 18 years of age, children are excluded from this study, but may be eligible for future pediatric trials
  • Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (Karnofsky \>= 60%)
  • Life expectancy of greater than 12 months
  • +7 more criteria

You may not qualify if:

  • Chemotherapy =\< 21 days prior to first administration of study treatment and/or monoclonal antibody =\< 6 weeks prior to first administration of study treatment
  • Patients who are receiving any other investigational agents
  • Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition as ibrutinib
  • Ibrutinib is extensively metabolized by CYP3A4/5; patients who received a strong cytochrome P450 (CYP) 3A inhibitor within 7 days prior to the first dose of ibrutinib or patients who require continuous treatment with a strong CYP3A inhibitor; therefore, any medications or substances that are strong inhibitors of CYP3A4/5 should be discontinued; patients unable to change these medications must be excluded from participation; because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated list; medical reference texts such as the Physicians' Desk Reference may also provide this information; as part of the enrollment/informed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements; recent infections requiring systemic treatment need to have completed therapy \> 14 days before the first dose of study drug
  • Pregnant women are excluded from this study because ibrutinib is a tyrosine kinase inhibitor with the potential for teratogenic or abortifacient effects; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ibrutinib, breastfeeding should be discontinued if the mother is treated with ibrutinib
  • Human immunodeficiency virus (HIV)-positive patients will be eligible unless they have been previously diagnosed with an acquired immune deficiency syndrome (AIDS)-defining illness
  • Patients who require anticoagulation with warfarin (Coumadin) or who have taken warfarin within 28 days prior to enrollment are not eligible due to a potential increased risk of hemorrhage; patients who are currently taking vitamin K antagonists are also ineligible for this study
  • Patients requiring daily corticosteroids at a prednisone equivalent of \>= 20 mg daily should not be enrolled; if corticosteroids can be discontinued (or reduced to \< 20 mg per day of prednisone or equivalent), the discontinuation or dose reduction should be done at least 7 days prior to first dose
  • Prior exposure to a Bruton's tyrosine kinase (BTK) inhibitor
  • Major surgery within 4 weeks of first dose of study drug
  • A history of prior malignancy, with the exception of the following:
  • Malignancy treated with curative intent and with no evidence of active disease present for more than 3 years prior to screening, and felt to be at low risk for recurrence by the treating physician
  • Adequately treated non-melanomatous skin cancer or lentigo maligna melanoma without current evidence of disease
  • +11 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Wayne State University/Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

Location

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (1)

  • Rogers KA, Andritsos LA, Wei L, McLaughlin EM, Ruppert AS, Anghelina M, Blachly JS, Call T, Chihara D, Dauki A, Guo L, Ivy SP, James LR, Jones D, Kreitman RJ, Lozanski G, Lucas DM, Ngankeu A, Phelps M, Ravandi F, Schiffer CA, Carson WE, Jones JA, Grever MR. Phase 2 study of ibrutinib in classic and variant hairy cell leukemia. Blood. 2021 Jun 24;137(25):3473-3483. doi: 10.1182/blood.2020009688.

MeSH Terms

Conditions

Leukemia, Hairy Cell

Interventions

Specimen HandlingBiopsyibrutinibHigh-Energy Shock Waves

Condition Hierarchy (Ancestors)

LeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisInvestigative TechniquesCytodiagnosisCytological TechniquesDiagnostic Techniques, SurgicalSurgical Procedures, OperativeUltrasonic WavesSoundRadiation, NonionizingRadiationPhysical Phenomena

Study Officials

  • Kerry A Rogers

    Ohio State University Comprehensive Cancer Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 24, 2013

First Posted

April 26, 2013

Study Start

April 30, 2013

Primary Completion (Estimated)

December 31, 2029

Study Completion (Estimated)

December 31, 2029

Last Updated

April 13, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will share

NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

More information

Locations