A drug that protects the liver may make stem cell transplants safer for children with sickle cell disease
NCT ID NCT02675959
First seen Jul 27, 2026 · Last updated Jul 27, 2026
Summary
This trial tests whether giving defibrotide before and after a stem cell transplant can prevent a serious liver condition called sinusoidal obstructive syndrome (SOS) in children and young adults with sickle cell disease or beta thalassemia. Participants receive defibrotide starting 10 days before the transplant and continuing for 21 days after. The goal is to see if this drug, already used to treat SOS, can safely prevent it in this high-risk group.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- defibrotide
- What this could lead to
- If successful, this approach could make stem cell transplants safer for people with sickle cell disease or beta thalassemia, potentially offering a cure with fewer complications.
- What could go wrong
- This is a small, early-phase trial, so results may not apply to all patients. Defibrotide may not prevent liver damage, and stem cell transplant carries serious risks like infection or graft-versus-host disease.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Medical College of Wisconsin
RECRUITINGMilwaukee, Wisconsin, 53226, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Email: •••••@•••••
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New York Medical College
RECRUITINGValhalla, New York, 10595, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of California Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of Florida
RECRUITINGGainesville, Florida, 32610-0278, United States
Contact Email: •••••@•••••
Contact Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can Eye-Tracking reveal hidden brain risks in babies with sickle cell disease?
- Can a peer coach help young people take control of their chronic illness?
- Could a stronger dose of an old drug protect children with sickle cell from organ damage?
- Gene editing could cure sickle cell by boosting fetal hemoglobin
- New stem cell processing method offers hope for patients without a perfect donor match