New transplant cocktail aims to cut complications in bone marrow failure
NCT ID NCT04965597
First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 40 times
Summary
This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioning regimen could reduce severe complications such as graft-versus-host disease and transplant failure. The study has completed, and results will show whether this approach improves outcomes compared to standard treatments.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
-
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
-
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
-
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
-
Cohen Children's Hospital of NY
Queens, New York, 11040, United States
-
Duke University Medical Center
Durham, North Carolina, 27705, United States
-
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109, United States
-
Johns Hopkins University
Baltimore, Maryland, 21231, United States
-
MD Anderson Cancer Center
Houston, Texas, 77030, United States
-
Medical College of Wisconsin/Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
-
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
-
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
-
Oregon Health & Science University
Portland, Oregon, 97239, United States
-
Primary Children's/University of Utah
Salt Lake City, Utah, 84113, United States
-
Rady Children's Hospital/UCSD
San Diego, California, 92123, United States
-
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14203, United States
-
St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
-
Texas Children's Hospital
Houston, Texas, 77030, United States
-
University of California San Francisco
San Francisco, California, 94143, United States
-
University of Michigan Medical Center
Ann Arbor, Michigan, 48109, United States
-
University of Minnesota
Minneapolis, Minnesota, 55455, United States
-
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Treosulfan, fludarabine, rabbit antithymocyte globulin, tacrolimus, methotrexate
What this could lead to
If successful, this approach could reduce severe complications like graft-versus-host disease after bone marrow transplant for bone marrow failure diseases.
What could go wrong
This is a small, completed phase 2 trial with only 40 participants, so results may not apply to everyone. There are still risks of transplant failure, rejection, or serious side effects from the drugs.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.