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

Leukemia Clinical Trial

Official title:

ONTAK (Denileukin Diftitox) in Patients With Systemic Mastocytosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00493129
• Other study ID #: 2004-0142
• Status: Completed
• Phase: Phase 2
• First received: June 26, 2007
• Last updated: February 7, 2012
• Start date: July 2004
• Est. completion date: August 2007

Study information

• Verified date: February 2012
• Source: M.D. Anderson Cancer Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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To collect a bone marrow sample, an area of the hip bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle.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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: August 2007
• Est. primary completion date: August 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

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

Related Conditions & MeSH terms

• Leukemia
• Mastocytosis
• Systemic Mastocytosis

Intervention

Drug:
• Ontak (Denileukin Diftitox)
9 µg/kg by vein Days 1-5 of a 21 day cycle.

Locations

Country	Name	City	State
United States	The University of Texas M.D. Anderson Cancer Center	Houston	Texas

Sponsors (1)

Lead Sponsor	Collaborator
M.D. Anderson Cancer Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Objective Response Rate	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.	3 months	No

External Links

•   M.D. Anderson's website