Clinical Trial: Allogeneic Stem Cell Transplantation for Myelofibrosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Allogeneic Stem Cell Transplantation for Myelofibrosis Using Reduced Intensity Busulfan and Fludarabine Conditioning

Brief Summary: The goal of this clinical research study is to learn if using a combination of fludarabine, busulfan, and antithymocyte globulin (ATG) can help to control myelofibrosis or myelodysplastic syndrome in patients receiving a bone marrow or blood stem cell transplant. The safety of these drugs will also be studied.

Detailed Summary:

Busulfan is a chemotherapy drug that kills cancer cells by binding to DNA, and is commonly used in stem cell transplants. Fludarabine is a drug that has anti-leukemia and immunosuppressive effects. ATG helps to reduce the risk of transplant rejection and to prevent graft versus host disease.

You will receive fludarabine by vein over 1 hour on Days -5 to -2. You will receive busulfan by vein over 3 hours on Day -5 to -2 immediately after completing fludarabine. If you have an unrelated or a mismatched donor, you will receive ATG by vein over 6 hours on Days -3 to -1 to prevent graft versus host disease and to help engraftment.

You will first receive a low-level "test" dose of busulfan, and blood samples (about 1 teaspoon each time) will be drawn for pharmacokinetic (PK) tests. This may be done as an outpatient prior to inpatient admission. PK tests measure the level of the study drug in the blood over different time points. This information will be used to decide the next dose needed to reach the target blood level that matches your body size.

About 11 total samples of blood will be drawn for PK testing after the test dose and before the first high-dose busulfan treatment. A heparin lock will be placed in your vein to lower the number of needle sticks needed for these draws. If it is not possible for these blood level tests to be performed for technical or scheduling reasons or for any other reason, you will receive the standard fixed busulfan dose without the "test dose."

You will receive the donor bone marrow or blood stem cells by vein over about 1 hour on Day 0.

Two (2) days before the stem cell infusion on Day -2, tacrolimus will be started as a non-st
Sponsor: M.D. Anderson Cancer Center

Current Primary Outcome: Rate of Non-Relapse Mortality (NRM) [ Time Frame: 100 Days ]

Non-relapse mortality defined as any death not attributable to disease recurrence.


Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: M.D. Anderson Cancer Center

Dates:
Date Received: May 15, 2007
Date Started: January 2006
Date Completion:
Last Updated: September 12, 2016
Last Verified: September 2016