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Ontak (Denileukin Diftitox) in Patients With Systemic Mastocytosis (SM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00493129
Recruitment Status : Completed
First Posted : June 28, 2007
Last Update Posted : February 8, 2012
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Tracking Information
First Submitted Date  ICMJE June 26, 2007
First Posted Date  ICMJE June 28, 2007
Last Update Posted Date February 8, 2012
Study Start Date  ICMJE July 2004
Actual Primary Completion Date August 2007   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 7, 2012)
Objective Response Rate [ Time Frame: 3 months ]
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.
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00493129 on Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Ontak (Denileukin Diftitox) in Patients With Systemic Mastocytosis (SM)
Official Title  ICMJE ONTAK (Denileukin Diftitox) in Patients With Systemic Mastocytosis
Brief Summary

Primary Objective:

1. To assess the response rate of ONTAK in Systemic Mastocytosis (SM) patients.

Secondary Objectives:

  1. To assess the safety of ONTAK in SM patients.
  2. To evaluate the time to progression and duration of response following treatment with ONTAK.
Detailed Description

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 treatment.

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. Anderson.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Leukemia
  • Systemic Mastocytosis
Intervention  ICMJE Drug: Ontak (Denileukin Diftitox)
9 µg/kg by vein Days 1-5 of a 21 day cycle.
Other Name: Denileukin Diftitox
Study Arms  ICMJE Experimental: Ontak
Ontak administered intravenously on Days 1-5 at the dose of 9 µg/kg/day, with a rest period from Days 6-21.
Intervention: Drug: Ontak (Denileukin Diftitox)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 19, 2012)
Original Estimated Enrollment  ICMJE
 (submitted: June 26, 2007)
Actual Study Completion Date  ICMJE August 2007
Actual Primary Completion Date August 2007   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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 * upper limit of normal; total bilirubin </= 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00493129
Other Study ID Numbers  ICMJE 2004-0142
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party M.D. Anderson Cancer Center
Study Sponsor  ICMJE M.D. Anderson Cancer Center
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Srdan Verstovsek, MD M.D. Anderson Cancer Center
PRS Account M.D. Anderson Cancer Center
Verification Date February 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP