Clinical trial NCT01087333: Collection of Human Samples to Study Hairy Cell and Other Leukemias, and to Develop Recombinant Immunotoxins for Cancer Treatment
Official title: A Study of Hairy Cell and Other Leukemias With a Focus on Recombinant Immunotoxins for Cancer Treatment
Study type: OBSERVATIONAL
Overall status: RECRUITING
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
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2010-03-16
Enrollment: 1263
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Primary clinical population; normal donors

Brief 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. 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.

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.

Eligibility criteria: * 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.

Primary outcomes:
measure: Tissue Acquisition
timeFrame: 4 weeks
Secondary outcomes:
measure: HCL follow-up
timeFrame: about every 2 years
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
Study officials:
affiliation: National Cancer Institute (NCI)
name: Robert J Kreitman, M.D.
role: PRINCIPAL_INVESTIGATOR
References: