Collection of Human Samples to Study Hairy Cell and Other Leukemias, and to Develop Recombinant Immunotoxins for Cancer Treatment

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.

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.

Study Overview

• Status: Recruiting
• Conditions: Chronic Lymphocytic Leukemia (CLL); Hairy Cell Leukemia (HCL); Cutaneous T Cell Lymphoma (CTCL); Non-Hodgkins Lymphoma (NHL); Adult T Cell Lymphoma (ATL)

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.

• Study Type: Observational
• Enrollment (Estimated): 1263

Contacts and Locations

• Study Contact: Name: Julie C Feurtado, R.N.; Phone Number: (301) 480-6186; Email: julie.feurtado@nih.gov
• Study Contact Backup: Name: Robert J Kreitman, M.D.; Phone Number: (301) 648-7375; Email: kreitmar@mail.nih.gov

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • Recruiting
      • National Institutes of Health Clinical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Primary clinical population; normal donors

Description

• INCLUSION CRITERIA:

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.

EXCLUSION CRITERIA:

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Control
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
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 the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tissue Acquisition	Collection of a variety of clinical samples, including blood, urine, lymphapheresis samples, and other tissues and associated data	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
HCL follow-up	Assess long-term treatment complications and disease outcomes in participants with HCL/HCLv	about every 2 years

Collaborators and Investigators

• Sponsor: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Robert J Kreitman, M.D., National Cancer Institute (NCI)

Publications and helpful links

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): March 2, 2010

Study Registration Dates

• First Submitted: March 13, 2010
• First Submitted That Met QC Criteria: March 13, 2010
• First Posted (Estimated): March 16, 2010

Study Record Updates

• Last Update Posted (Actual): March 22, 2024
• Last Update Submitted That Met QC Criteria: March 21, 2024
• Last Verified: December 14, 2023

More Information

Terms related to this study

• Keywords: Natural History; Apheresis; Flow Cytometry; Neutralizing Antibodies; Cytotoxicity Assay; Hemolytic Uremic Syndrome (HUS)
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Disease Attributes; Hematologic Diseases; Leukemia, Lymphoid; Leukemia, B-Cell; Leukemia, T-Cell; Chronic Disease; Lymphoma; Leukemia; Lymphoma, Non-Hodgkin; Leukemia, Lymphocytic, Chronic, B-Cell; Lymphoma, T-Cell; Lymphoma, T-Cell, Peripheral; Lymphoma, T-Cell, Cutaneous; Leukemia-Lymphoma, Adult T-Cell; Leukemia, Hairy Cell
• Other Study ID Numbers: 100066; 10-C-0066

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: .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.
• IPD Sharing Time Frame: Clinical data available during the study and indefinitely. Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.
• IPD Sharing Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI. Genomic data are made available via dbGaP through requests to the data custodians.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No