Hairy Cell Leukemia (HCL)NCT01087333
Collection of Human Samples to Study Hairy Cell and Other Leukemias, and to Develop Recombinant Immunotoxins for Cancer Treatment
An observational study of Hairy Cell Leukemia (HCL) and Chronic Lymphocytic Leukemia (CLL), run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: Researchers who are studying hairy cell leukemia, and how the disease compares with other disorders, are interested in obtaining additional samples from leukemia patients and healthy volunteers. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Hairy Cell Leukemia (HCL), Chronic Lymphocytic Leukemia (CLL), Non-Hodgkins Lymphoma (NHL)
- How many people
- 1,263 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Mar 2010to not given
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Primary clinical population; normal donors
Who may be able to join
- All participants
- 18 years of age and older
- Desire of the individual to submit data and samples for research
- Ability to understand and the willingness to sign a written informed consent document.
- Individuals with cancer
- -Participants may have a diagnosis of hematologic malignancy or solid tumor. These participants would not be excluded if they were in long-term complete remission.
- Normal donors (Individuals without cancer)
- -Individuals must not have a current or prior diagnosis of a hematologic malignancy or solid tumor.
Who cannot join
- All participants
- -Known pregnancy at enrollment. NOTE: A pregnancy test will be required in individuals on study prior to any procedure done for research purposes that is greater than minimal risk.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Study of Hairy Cell and Other Leukemias With a Focus on Recombinant Immunotoxins for Cancer Treatment
Background:
- Researchers who are studying hairy cell leukemia, and how the disease compares with other disorders, are interested in obtaining additional samples from leukemia patients and healthy volunteers. The investigators are particularly interested in samples from individuals who have diseases that can be treated with a new type of drug called immunotoxin, in which an antibody carrying a toxin binds to a cancer cell and allows the toxin to kill the cell.
Read more
Objectives:
- To collect a variety of clinical samples, including blood, urine, lymph samples, and other tissues, in order to study the samples and develop new treatments for leukemia.
Eligibility:
- Individuals 18 years of age and older who have been diagnosed with leukemia or other kinds of blood and lymphatic system cancers, or who are healthy volunteers.
Design:
- Individuals who have leukemia will be asked to provide blood, bone marrow, urine, and tumor tissue samples as requested by the researchers. Healthy volunteers will provide only blood and urine samples.
- No treatment will be given as part of this protocol.
Read the detailed description
Background
- Hairy cell leukemia (HCL) is highly responsive to but not curable by standard chemotherapy, and also responds well to investigational agents called recombinant immunotoxins which have been developed by the Laboratory of Molecular Biology (LMB).
- HCL variants often resemble classic HCL but are more aggressive and less responsive to treatments, such as HCLv and IGHV4-34+ HCL that are immunophenotypically indistinguishable from classic HCL and highly aggressive and resistant like HCLv.
- The investigators on this protocol are studying molecular and clinical aspects of HCL, and how they compare to normal or to other disorders, including other hematologic malignancies and solid tumors.
- The LMB are also studying agents for HCL/HCLv, including recombinant immunotoxins developed in the LMB. Specific targets and agents include BL22 and a high affinity variant, HA22 or Moxetumomab Pasudotox (Moxe), targeting CD22, LMB-2, targeting CD25, and SS1P, targeting Mesothelin as well as single agents and combinations of purine analogs (e.g., cladribine, pentostatin, and bendamustine), anti-CD20 monoclonal antibodies (e.g., rituximab), and small molecule inhibitors (e.g., BRAF V600E inhibitors dabrafenib and encorafenib, MEK inhibitors trametinib and binimetinib, and Bruton s tyrosine kinase (BTK) inhibitor ibrutinib).
- Longitudinal evaluation of HCL is needed as a basis to identify more effective treatments.
Objective
-To allow the collection and analysis of a variety of samples, including blood, tumor and other tissues from individuals with and without cancer to better understand the disease processes which are being studied, particularly hairy cell leukemia, or to determine eligibility and/or optimal timing for clinical testing
Eligibility
- Greater than or equal to 18 years of age
- Diagnosis of a hematologic malignancy or solid tumor; or normal donors (i.e., individuals without a known malignancy).
Design
- Collection of data and samples for research, including blood, tumor, and other tissues from participants and normal volunteers.
- Samples may be obtained prior to/after treatment, during disease assessments, and at the time of response/relapse. This protocol does not involve treatment, although participants may receive treatment as standard of care or as part of another research protocol during participation.
- Samples can be obtained at NIH or at local providers (and sent to NIH).
- Systematic follow-up of participants with HCL, in particular those who have completed prior treatment.
- We anticipate accruing 1263 participants on this protocol.
- Conditions
- Hairy Cell Leukemia (HCL), Chronic Lymphocytic Leukemia (CLL), Non-Hodgkins Lymphoma (NHL), Cutaneous T Cell Lymphoma (CTCL), Adult T Cell Lymphoma (ATL)
- Keywords
- Cytotoxicity Assay, Neutralizing Antibodies, Apheresis, Flow Cytometry, Hemolytic Uremic Syndrome (HUS), Natural History
- Medical subject headings
- Leukemia, Hairy Cell, Leukemia, Lymphocytic, Chronic, B-Cell, Lymphoma, Non-Hodgkin, Lymphoma, T-Cell, Cutaneous, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Hemolytic-Uremic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Patients with hematologic malignancies or solid tumors. | |
| 2 | Normal Donors who are defined as individuals without a diagnosis of or history of any cancer. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tissue Acquisition
Collection of a variety of clinical samples, including blood, urine, lymphapheresis samples, and other tissues and associated data
Measured: 4 weeks
Secondary outcomes (1)
HCL follow-up
Assess long-term treatment complications and disease outcomes in participants with HCL/HCLv
Measured: about every 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2010 (estimated)
- Study start2 Mar 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Hairy Cell Leukemia (HCL)Studies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 1,263 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Robert J Kreitman, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01087333
- Sponsor's protocol ID
- 100066
- Other IDs
- 10-C-0066
- Status last verified
- 13 Oct 2026
- First submitted
- 13 Mar 2010
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
Publications
Kreitman RJ, James L, Feurtado J, Eager H, Sierra Ortiz O, Gould M, Shpilman I, Zhou H, Burbelo PD, Cohen JI, Wang HW, Yuan CM, Arons E. COVID-19 vaccination in patients with classic and variant hairy cell leukemia. Blood Neoplasia. 2024 Aug 28;1(4):100035. doi: 10.1016/j.bneo.2024.100035. eCollection 2024 Dec.
PubMed 40552139Arons E, Tai CH, Suraj J, Liu Y, Day CP, Raffeld M, Xi L, Zhou H, Gould M, Shpilman I, Oakes CC, Bhat S, Grever M, Jones D, Rogers K, Wang HW, Yuan CM, Sahinalp C, Kreitman RJ. Non-V600E BRAF mutations and treatment for hairy cell leukemia. Blood. 2025 Apr 24;145(17):1957-1961. doi: 10.1182/blood.2024026894.
PubMed 39719040Arons E, Henry K, Haas C, Gould M, Tsintolas J, Mauter J, Zhou H, Burbelo PD, Cohen JI, Kreitman RJ. Characterization of B-cell receptor clonality and immunoglobulin gene usage at multiple time points during active SARS-CoV-2 infection. J Med Virol. 2023 Oct;95(10):e29179. doi: 10.1002/jmv.29179.
PubMed 37877800Kreitman RJ, Yu T, James L, Feurtado J, Eager H, Ortiz OS, Gould M, Mauter J, Zhou H, Burbelo PD, Cohen JI, Wang HW, Yuan CM, Arons E. COVID-19 in patients with classic and variant hairy cell leukemia. Blood Adv. 2023 Dec 12;7(23):7161-7168. doi: 10.1182/bloodadvances.2023011147.
PubMed 37729613