Clinical Trial: Everolimus (RAD001) as Therapy for Patients With Systemic Mastocytosis

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

Official Title: Evaluation of RAD001 as Therapy for Patients With Systemic Mastocytosis

Brief Summary: The goal of this clinical research study is to see if RAD001 can help to control the disease in patients with systemic mastocytosis (SM). The safety of this treatment will also be studied.

Detailed Summary:

RAD001 is designed to stop cancer cells from multiplying. It may also stop the growth of new blood vessels that help tumor growth, which may cause the tumor cells to die.

Before you can start treatment on this study, you will have "screening tests." These tests will help the doctor decide if you are eligible to take part in this study. They will be performed within 2 weeks before starting the study, unless listed otherwise. You will have a complete physical exam, including measurement of vital signs (blood pressure, heart rate, temperature, and breathing rate). Blood (about 2 tablespoons) will be drawn for routine tests. This routine blood draw will include a pregnancy test for women who are able to have children. To be eligible to take part in this study, the pregnancy test must be negative. You will need to "fast" for 6 hours before having these blood tests performed. This means you will not be able to have food or drink (except water) during this time.

Also as part of the screening tests, you will have an electrocardiogram (ECG)a test that measures the electrical activity of the heart). You will have a bone marrow biopsy and aspiration within 3 months of starting the study (or within 2 weeks of starting the study, if you have received treatment for SM). To collect a bone marrow biopsy/aspirate, an area of the hip bone is numbed with anesthetic, and a small amount of bone marrow and bone is withdrawn through a large needle. The bone marrow samples will be used not only to check the status of the disease, but also for a routine test to see if there is a mutation (change) in a certain gene. If your doctor feels it is necessary, you may need to have additional screening tests (such as a bone scan) to check the status of the disease.

If you are found to be
Sponsor: M.D. Anderson Cancer Center

Current Primary Outcome: Number of Participants With Objective Response [ Time Frame: Monthly for first 3 months, then every 3 months ]

Efficacy reported as objective response. Objective response defined as change in serum tryptase level or bone marrow mast cell percentage.


Original Primary Outcome:

  • Primary
  • · To assess the response rate of RAD001 in patients with Systemic Mastocytosis (SM).


Current Secondary Outcome:

Original Secondary Outcome:

  • Secondary
  • To assess the safety of RAD001 in SM patients.
  • To evaluate the time to progression and duration of response following treatment with RAD001.
  • To evaluate change in pertinent biological markers during the therapy with RAD001


Information By: M.D. Anderson Cancer Center

Dates:
Date Received: March 19, 2007
Date Started: April 2007
Date Completion:
Last Updated: August 1, 2012
Last Verified: August 2012