Clinical Trial: Ontak (Denileukin Diftitox) in Patients With Systemic Mastocytosis (SM)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: 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 Summary:

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To collect a bone marrow sample, an area of the hip bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle.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.<
Sponsor: M.D. Anderson Cancer Center

Current Primary Outcome: 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:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: M.D. Anderson Cancer Center

Dates:
Date Received: June 26, 2007
Date Started: July 2004
Date Completion:
Last Updated: February 7, 2012
Last Verified: February 2012