Ontak (Denileukin Diftitox) in Patients With SM
Information source: M.D. Anderson Cancer Center
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Leukemia; Systemic Mastocytosis
Intervention: Ontak (Denileukin Diftitox) (Drug)
Phase: Phase 2
Sponsored by: M.D. Anderson Cancer Center
Official(s) and/or principal investigator(s):
Srdan Verstovsek, MD, Principal Investigator, Affiliation: U.T.M.D. Anderson Cancer Center
1. To assess the response rate of ONTAK in Systemic Mastocytosis (SM) patients.
1. To assess the safety of ONTAK in SM patients.
2. To evaluate the time to progression and duration of response following treatment with
Official title: ONTAK (Denileukin Diftitox) in Patients With Systemic Mastocytosis
Study design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
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amount of bone marrow is withdrawn through a large needle.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.
Minimum age: N/A.
Maximum age: N/A.
- Patients with SM, including mast cell leukemia (MCL).
- ECOG Performance Status (PS) 0-3
- Adequate renal function (indicated by serum creatinine = 2. 5 mg/dL); adequate
hepatic function (indicated by ALT = 3 x upper limit of normal; total bilirubin =
3 x 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.
- History of hypersensitivity to diphtheria toxin.
- Active cardiovascular disease as defined by New York Heart Association (NYHA) Class
- Serious intercurrent medical illnesses or active infections requiring parenteral
antibiotics that would interfere with the ability of the patient to carry out the
- 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
- 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.
Locations and Contacts
The University of Texas M.D. Anderson Cancer Center, Houston, Texas 77030, United States
M.D. Anderson's website
Starting date: July 2004
Last updated: August 31, 2007