National Cancer Institute - U.S. National Institutes of Health - www.cancer.govSkip navigationNational Cancer Institute - U.S. National Institutes of Health - www.cancer.govClinical Trials at NIH - Be part of the cure
National Cancer Institute - U.S. National Institutes of Health - www.cancer.gov
Clinical Trials at NIH Home

Why is this trial important?

Patients who have been treated many times for hairy cell leukemia (HCL) often have poor responses to standard treatment, have cumulative toxicity from chemotherapy, and can die of refractory HCL, usually from infections. HCL cells that are resistant to chemotherapy display high levels of CD22, making them good targets for HA22, a recombinant immunotoxin that binds to CD22 using an antibody fragment, goes inside the cell and has a toxin fragment that kills the cell. HA22 is an improved version of BL22, and it is reported to achieve complete remissions in a high percentage of HCL patients resistant to chemotherapy.

Who is eligible for this trial? (PDQ)

  • Confirmed diagnosis of hairy cell leukemia (HCL)
  • At least one of the following:
    • Neutropenia (ANC < 1,000 cells/μL)
    • Anemia (Hgb < 10 g/dL)
    • Thrombocytopenia (Plt < 100,000/μL)
    • Absolute lymphocyte count of > 20,000 cells/μL or symptomatic splenomegaly
  • At least 2 prior systemic therapies (must have had at least 2 courses of purine analog, or 1 if response lasted < 2 years or unacceptable toxicity to purine analog)
  • ECOG performance 0–2
  • Life expectancy > 6 months
  • ≥ 18 years of age
  • Measurable disease
  • No history of bone marrow transplant
  • Must use approved method of contraception
  • Patients with other cancers who have had < 5 years disease free considered on a case-by-case basis
  • ALT, AST or bilirubin < grade 2 (unless due to Gilbert’s disease)
  • Creatinine clearance > 60 mL/min
  • ANC ≥ 1,000/cmm or platelet ≥ 50,000/cmm (unless due to underlying disease)
  • No baseline coagulopathy ≥ grade 3 (unless due to anticoagulation therapy)
  • FEV ≥ 50%
  • DLCO ≥ 50%
  • Pancytopenia ≥ grade 3 or erythropoietin dependence allowed, if due to disease, based on bone marrow studies
  • No ongoing CNS involvement (prior CNS involvement allowed)
  • Not pregnant or nursing
  • No significant level of antibody to CAT-8015 or antibody that neutralizes the binding of CAT-8015 to CD22
  • No standard or investigational therapy for 3 weeks prior to study entry (patients who have received or are receiving radiation therapy are not excluded, providing that the volume of bone marrow treated is < 10% and the patient has measurable disease outside the radiation port)
  • ≤ 3 months since prior monoclonal therapy
  • No history of pseudomonas-exotoxin immunotoxin (PE)
  • HIV negative
  • No hepatitis B surface antigen positivity
  • No uncontrolled, symptomatic illness including but not limited to:
    • Infections requiring systemic antibodies
    • Congestive heart failure
    • Unstable angina pectoris
    • Cardiac arrhythmia
    • Psychiatric illness
    • Social situations limiting compliance with study requirements

What types of drugs or therapies are being used?

HA22, a recombinant anti-CD22 immunotoxin

What is the study outline? (PDQ)

This is a standard, 3+3 dose-finding study with an expanded maximum tolerated dose (MTD) cohort.

  • Cohorts of 3 patients each will receive escalating doses of CAT-8015 until the maximum tolerated dose (MTD) is determined
  • Once the MTD is determined, the MTD cohort will be expanded to 12 patients; MTD is defined as the dose level at which ≤ 1 of 6 patients experience DLT or the dose level immediately below the level at which > 1 out of 2–6 patients experience DLT
  • Patients receive CAT-8015 every 4 weeks for a total of 10 cycles until progressive disease or until they become otherwise ineligible
  • CAT-8015 will be given intravenously (IV) on Days 1, 3, and 5 of each cycle
  • Dose escalation to a new cohort may not occur until authorized by the medical monitor, which will require that all patients from the prior cohort have reached Cycle 2, Day 10 without DLT if eligible for retreatment
  • Disease assessment will be completed prior to the first cycle of CAT-8015, prior to Cycle 2, and prior to every other subsequent cycle and at post-treatment follow-up visit

What is the frequency and duration of the visits?

Patients who are eligible are treated with HA22 by 30-minute intravenous infusion every other day for 3 doses. Patients not making antibodies or having progressive disease are allowed to receive up to 9 repeat cycles at 4-week intervals. Patients achieving complete remission stop receiving HA22 two cycles after the complete remission was first detected.

What are the costs?

There is no charge for medical care received at the National Institutes of Health (NIH) Clinical Center. Patients will be responsible for travel costs for their initial screening visits. In most cases, once patients are enrolled in a trial, the National Cancer Institute (NCI) will pay the transportation costs for all subsequent trial-related visits for patients who do not live in the local area. In addition, these patients will receive a small per diem to help offset the costs of meals and lodging if they are being treated as outpatients.

It will be important to maintain your current insurance plan to cover all medical care that is provided away from the NIH Clinical Center.

No U.S. citizen or permanent U.S. resident residing in the U.S. who otherwise meets the eligibility requirements will be denied enrollment in clinical research protocols because of their inability to pay the costs of travel and subsistence.

Who is the Principal Investigator?

Dr. Kreitman received his M.D. from Ohio State University in 1985 and obtained his internal medicine residency training at Duke University from 1985 to 1988. He received his medical oncology fellowship training at NIH from 1988 to 1991, has been working in the immunotoxin field since 1989, and has been directing clinical trials with immunotoxins since 1996.

Where is this trial taking place?

NIH Clinical Center
National Institutes of Health
NCI Medical Oncology Branch and Affiliates
10 Center Drive
Bethesda, MD 20892

Who are the contacts for this trial?

Robert J. Kreitman, M.D.
Principal Investigator
Phone: 301-496-6947
kreitmar@mail.nih.gov

Referrals:

Natasha Kormanik, R.N., B.S.N., O.C.N.
Research Nurse
Phone: 301-496-9458
Fax: 301-451-5765
kormanikn@mail.nih.gov

Elizabeth Maestri, R.N.
Research Nurse
Phone: 301-402-5633
Fax: 301-451-5765
maestrie@mail.nih.gov

Joan Aaron, R.N., B.S.N., O.C.N.
Research Nurse
Phone: 301-594-1778
Fax: 301-451-5765
aaronj@cc.nih.gov

Where can additional information be found?

Back to Top
Health and Human Services National Institutes of Health National Cancer Institute USA.gov