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ONTAK (Denileukin Diftitox) in Patients With Systemic Mastocytosis

Phase 2
Not Enrolling
Leukemia, Systemic Mastocytosis

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Trial Information

ONTAK (Denileukin Diftitox) in Patients With Systemic Mastocytosis

Denileukin diftitox has been used for the treatment of a variety of disorders, in
particular, malignant lymphoma, another blood-related disease. Denileukin diftitox is
believed to be able to specifically attach to and kill malignant mast cells.

Before you can start treatment on this study, you will have what are called "screening
tests". These tests will help the doctor decide if you are eligible to take part in the
study. You will have blood (around 2 teaspoons) and bone marrow samples collected. To
collect a bone marrow sample, an area of the hip bone is numbed with anesthetic and a small
amount of bone marrow is withdrawn through a large needle. These samples will be used for
tests to confirm the diagnosis of the disease. Women who are able to have children must
have a negative blood pregnancy test.

If you are found to be eligible, you will receive denileukin diftitox as an injection by
vein once a day for 5 days in a row. This will be repeated every 3 weeks (1 cycle). You
will receive treatment on an outpatient basis. Treatment will continue as long as there is
evidence that therapy is affecting the disease and is beneficial to you. If the disease
gets worse or you experience any intolerable side effects, you will be taken off the study
and your doctor will discuss other treatment options with you.

During treatment you will have blood (around 1 teaspoon) collected twice during every 3-week
period. You will also have bone marrow samples collected every 3 months during the

After the end of treatment, blood and bone marrow samples will be collected every 3 months
until the disease gets worse or you start a different therapy. The blood and bone marrow
samples will be used to look at response to therapy.

This is an investigational study. Denileukin diftitox has been approved by the FDA for the
treatment of skin T-cell lymphoma and is commercially available. The use of denileukin
diftitox in this study is investigational. You will be provided denileukin diftitox free of
charge. Up to 25 participants will take part in this study. All be enrolled at M. D.

Inclusion Criteria:

- Patients with SM, including mast cell leukemia (MCL).

- ECOG Performance Status (PS) 0-3

- Adequate renal function (indicated by serum creatinine hepatic function (indicated by ALT 3 * upper limit of normal; and albumin >/= 2.8 g/dL).

- Provide written informed consent.

- Female patients of childbearing potential must have a negative pregnancy test within
14 days prior to first dose of study drug, and must agree to use an effective means
of contraception following the pregnancy test, throughout the study and for at least
three weeks after their last treatment on protocol.

Exclusion Criteria:

- History of hypersensitivity to diphtheria toxin.

- Active cardiovascular disease as defined by New York Heart Association (NYHA) Class
III-IV categorization.

- Serious intercurrent medical illnesses or active infections requiring parenteral
antibiotics that would interfere with the ability of the patient to carry out the
treatment program.

- Concurrent malignancy (other than resected basal or squamous cell skin cancers or
in-situ cervical cancer). Unless, patient has SM-associated clonal hematologic
disease that does not require therapy, as judged by treating physician and approved
by principal investigator.

- Female patients who are pregnant or breastfeeding.

- No chemotherapy, radiotherapy, immunotherapy, hormonal anticancer therapy, or
experimental medications (including approved drugs tested in an investigational
setting) may be administered while a patient is a participant in this protocol.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Objective Response Rate

Outcome Description:

Bone marrow samples used to look at response to therapy collected every 3 months until disease progression or different therapy started. Clinical efficacy assessed as objective response (major and partial response) for responding patients analyzed by the Kaplan-Meier method.

Outcome Time Frame:

3 months

Safety Issue:


Principal Investigator

Srdan Verstovsek, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

July 2004

Completion Date:

August 2007

Related Keywords:

  • Leukemia
  • Systemic Mastocytosis
  • Systemic Mastocytosis
  • SM
  • Mast Cell Leukemia
  • Leukemia
  • Denileukin Diftitox
  • Ontak
  • Leukemia
  • Mastocytosis
  • Urticaria Pigmentosa
  • Mastocytoma
  • Mastocytosis, Systemic



The University of Texas M.D. Anderson Cancer Center Houston, Texas