Recruiting

Testing a preventive medication to protect the liver during stem cell transplant for sickle cell disease

Plain-English translation of NCT02675959 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated August 2026 · How we translate trials

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Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.

What is this trial?

This trial is testing whether , a medication given before and during a stem cell transplant, can prevent a serious liver condition called sinusoidal obstructive syndrome (SOS) in people with sickle cell disease. SOS is a known risk after the intense chemotherapy used to prepare the body for a stem cell transplant. The medication has already shown promise in treating SOS, and this study will see if giving it beforehand can stop it from happening in the first place.

Patients with sickle cell disease who receive a stem cell transplant face a high risk of developing a serious liver injury. This treatment aims to prevent that complication from occurring, which could make transplants safer and more successful for these patients.

Do you qualify?

You likely qualify if…

you have been diagnosed with sickle cell disease or beta thalassemia
you have experienced serious complications from your sickle cell disease, such as stroke, severe pain episodes, chest syndrome, or organ damage
you are between infancy and young adulthood
you have a family member or matched unrelated donor who is willing to provide stem cells
your heart, lungs, kidneys, and liver are healthy enough to handle the transplant procedure

You likely don't qualify if…

you are currently taking blood-thinning medications or clot-dissolving drugs
you have had a previous allergic reaction to defibrotide
you are pregnant, planning to become pregnant, or breastfeeding
you have a serious active infection that is not controlled
you have advanced liver disease, scarring, or cirrhosis

What participation looks like

You would receive intravenously (through an IV) starting before your stem cell transplant conditioning begins and continuing during the transplant process. You'll also undergo the standard stem cell transplant procedure, which includes intensive chemotherapy to prepare your body, followed by infusion of stem cells from your donor. You would stay in the hospital for several weeks during the initial phase and then have follow-up visits to monitor your recovery and check for any complications.

AI-generated summary from trial data · Aug 2, 2026 · Not medical advice

Trial locations(4 sites)

United States

University of California Los Angeles, Los Angeles, California

University of Florida, Gainesville, Florida

New York Medical College, Valhalla, New York

Medical College of Wisconsin, Milwaukee, Wisconsin