Treosulfan-Based Conditioning for Bone Marrow Failure
Trial Summary
What is the purpose of this trial?
This trial tests if a combination of three drugs can reduce complications for patients with bone marrow failure diseases. The drugs work by killing harmful cells, stopping their growth, and reducing immune reactions.
Do I need to stop my current medications for this trial?
The trial protocol does not specify if you need to stop taking your current medications. It's best to discuss your specific medications with the trial team to get a clear answer.
What data supports the idea that Treosulfan-Based Conditioning for Bone Marrow Failure is an effective treatment?
The available research shows that Treosulfan-Based Conditioning is effective for treating bone marrow failure disorders. In a study with 14 patients who had various marrow failure disorders, all patients successfully received donor cells, and 13 out of 14 were alive with their diseases corrected after three years. This suggests that the treatment is effective in establishing donor cell acceptance with low side effects and good long-term outcomes. Compared to other treatments, Treosulfan-Based Conditioning has a lower risk of serious complications and high survival rates, making it a promising option for patients with marrow failure disorders.12345
What safety data is available for Treosulfan-based conditioning in bone marrow failure treatment?
Treosulfan-based conditioning, often combined with fludarabine and thymoglobulin, has been studied for its safety and efficacy in hematopoietic cell transplantation. Studies show promising results with good engraftment and low toxicity. In a multicenter trial, 31 patients with nonmalignant diseases received treosulfan, fludarabine, and thymoglobulin, resulting in a 0% transplant-related mortality at day 100 and a 90% survival rate at 2 years. The combination was effective in establishing donor engraftment with low toxicity, although some patients experienced graft-versus-host disease (GVHD). The use of thymoglobulin was associated with a lower incidence of severe acute GVHD. These findings support the safety and potential of treosulfan-based conditioning for bone marrow failure treatment.12346
Is the drug combination of Fludarabine, Thymoglobulin, and Treosulfan a promising treatment for bone marrow failure?
Yes, the combination of Fludarabine, Thymoglobulin, and Treosulfan is promising for treating bone marrow failure. It has shown to be effective in helping patients recover with low toxicity and high survival rates. This drug combination helps establish donor cells in the patient's body, which is crucial for recovery, and has a good safety profile.14578
Research Team
Lauri Burroughs, MD
Principal Investigator
Fred Hutch/University of Washington Cancer Consortium
Margaret MacMillan, MD
Principal Investigator
University of Minnesota
Eligibility Criteria
This trial is for people aged 1 to under 50 with bone marrow failure diseases treatable by transplant, who have specific genetic mutations or meet diagnostic criteria. Excluded are those with certain other conditions, previous transplants, severe lung function impairment, liver issues, uncontrolled infections, HIV positive status or unwillingness to use contraception.Inclusion Criteria
Exclusion Criteria
Treatment Details
Interventions
- Fludarabine (Anti-metabolites)
- Thymoglobulin (Rabbit Anti-thymocyte Globulin; rATG) (Immunosuppressant)
- Treosulfan (Alkylating agents)
Fludarabine is already approved in Canada for the following indications:
- Chronic lymphocytic leukemia
- Non-Hodgkin's lymphoma
Find a Clinic Near You
Who Is Running the Clinical Trial?
Fred Hutchinson Cancer Research Center
Lead Sponsor
Dr. Nancy Davidson
Fred Hutchinson Cancer Research Center
Chief Medical Officer since 2020
MD from Harvard Medical School
Dr. Thomas J. Lynch Jr.
Fred Hutchinson Cancer Research Center
Chief Executive Officer since 2020
MD from Yale University, BA from Yale College
Fred Hutchinson Cancer Center
Lead Sponsor
Dr. W. Thomas Purcell
Fred Hutchinson Cancer Center
Chief Medical Officer since 2022
MD from Emory University School of Medicine, MBA from University of Chicago
Dr. Thomas J. Lynch Jr.
Fred Hutchinson Cancer Center
Chief Executive Officer since 2020
MD from Yale School of Medicine
National Cancer Institute (NCI)
Collaborator
Dr. Douglas R. Lowy
National Cancer Institute (NCI)
Chief Executive Officer since 2023
MD from New York University School of Medicine
Dr. Monica Bertagnolli
National Cancer Institute (NCI)
Chief Medical Officer since 2022
MD from Harvard Medical School
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
Dr. Gary H. Gibbons
National Heart, Lung, and Blood Institute (NHLBI)
Chief Executive Officer since 2012
MD from Harvard Medical School
Dr. James P. Kiley
National Heart, Lung, and Blood Institute (NHLBI)
Chief Medical Officer since 2011
MD from University of California, San Francisco
National Marrow Donor Program
Collaborator
Amy Ronneberg
National Marrow Donor Program
Chief Executive Officer since 2020
MBA from Capella University, BBA in Accounting from University of Wisconsin-Eau Claire
Steven Devine
National Marrow Donor Program
Chief Medical Officer
MD
Blood and Marrow Transplant Clinical Trials Network
Collaborator