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Phase 2 N=15 Treatment

LMB-2 to Treat Hairy Cell Leukemia

Hairy Cell Leukemia

Enrolled (actual)
15
Serious AEs
26.7%
Results posted
Apr 2019
Primary outcome: Primary: Number of Patients Who Obtain Partial Response/Complete Remission — 1; 5 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
National Cancer Institute (NCI)
Primary completion
Aug 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Patients Who Obtain Partial Response/Complete Remission
1; 5
SECONDARY
Duration of Response
23.5
SECONDARY
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
13

Summary

Background: * About 80% of patients with hairy cell leukemia (HCL) have tumor cells that have a protein on their surface called cluster of differentiation 25 (CD25). * The experimental drug LMB-2 is a recombinant immunotoxin that has been shown to kill leukemia and lymphoma cells with the CD25 protein. (A recombinant immunotoxin is a genetically engineered drug that has two parts - a protein that binds or targets a cancer cell, and a toxin that kills the cancer cell to which it binds.) Objectives: * To evaluate the safety and effectiveness of LMB-2 in patients with HCL whose cancer cells contain the CD25 protein. * To evaluate the effects of LMB-2 on the immune system, determine how the drug is metabolized by the body and examine its side effects. Eligibility: -Adults with hairy cell leukemia whose tumor cells have CD25 on their surface Design: * Up to 27 patients may be included in the study. * Patients receive an infusion of LMB-2 through a vein every other day for three doses (days 1, 3, 5), constituting one treatment cycle. * Patients may receive up to six treatment cycles every 4 weeks unless their cancer worsens or they develop unacceptable side effects. * Blood is drawn weekly for various tests. * Before each cycle and in follow-up visits, disease status is evaluated with a physical examination, blood tests, chest x-ray and electrocardiogram. * Before the first cycle, patients may have a computed tomography (CT) scan, echocardiogram (heart ultrasound test) and bone marrow biopsy. With the patient's permission, these tests may be repeated before other cycles also.

Eligibility Criteria

  • INCLUSION CRITERIA:
  • Histopathological evidence of cluster of differentiation 25 (CD25)+ Hairy Cell Leukemia (HCL) confirmed by the National Institutes of Health (NIH) pathology department. This will require a monoclonal population of peripheral malignant lymphocytes that are CD25 positive by fluorescence activated cell sorting (FACS) with anti-CD25 antibody. Positive expression in a FACS assay is defined as more than 2 times the mean fluorescence intensity (MFI) of the control antibody by FACS. HCLv (HCL variant) is usually CD25 negative, and eligibility would require CD25+ HCLv.
  • At least one of the following indications for treatment: neutropenia (absolute neutrophil count (ANC) less than 1000 cells/ microL), anemia (hemoglobin (Hgb) less than 10g/dL), thrombocytopenia (platelet (Plt) less than 100,000/ microL), an absolute lymphocyte count of greater than 20,000 cells/microL or symptomatic splenomegaly.
  • Previous treatment with or inability to receive BL22 or HA22 recombinant immunotoxin. Patients must have had at least 2 prior systemic therapies, including 2 courses of a purine nucleoside analog (PNA), or 1 course of either rituximab or BRAF inhibitor following a single prior course of PNA.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2.
  • At least 18 years old.
  • Understand and give informed consent.
  • A negative pregnancy test in female patients of childbearing potential. Women must not be breast-feeding.
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) less than or equal to 5-times the upper limits of normal. Albumin greater than or equal to 3.0 gm/dL. Total bilirubin less than or equal to 2.2 mg/dL.
  • Creatinine less than or equal to 1.4 mg/dL or creatinine clearance greater than or equal to 50 ml/min.
  • Serum that neutralizes less than or equal to 75% of the activity of 1 microg/mL of LMB-2 using a bioassay.
  • No systemic cytotoxic chemotherapy within 4 weeks of enrollment or systemic steroids (except stable doses of Prednisone less than or equal to 20 mg/day, or up to 4 doses of steroid given for non-therapy reasons) within 4 weeks of enrollment.
  • No anti-cluster of differentiation 25 (CD25) monoclonal antibody therapy within 12 weeks of enrollment.
  • No prior treatment with LMB-2.
  • Patients may not be receiving any other investigational agents.
  • Patients should not have uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.

EXCLUSION CRITERIA

  • Patients who have human immunodeficiency virus (HIV) or hepatitis C. Patients would not be excluded for hepatitis B surface antigen positivity if on Lamivudine.
  • Patients receiving coumadin.
  • Patients with a left ventricular ejection fraction of less than 45%.
  • Patients with a diffusing capacity of the lungs for carbon monoxide (DLCO) less than 55% of normal or an forced expiratory volume 1 (FEV1) less than 60% of normal based on either National Institutes of Health (NIH) or United States of America (USA) normal ranges.
  • Patients who have an active 2nd malignancy requiring systemic treatment.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00321555). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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