Recruiting

Protective medication to prevent liver damage during stem cell transplant for sickle cell disease

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

Read our Sickle Cell Disease research guide →

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 called can help protect the liver during a stem cell transplant procedure for patients with sickle cell disease. Stem cell transplants can sometimes cause a serious liver problem called sinusoidal obstructive syndrome, and this study aims to see if giving the medication before and during the transplant can prevent it from happening. Researchers believe this protection could make transplants safer and more successful for this group of patients.

Patients with sickle cell disease who receive stem cell transplants have a high risk of developing a serious liver condition that can affect their recovery. This medication has shown promise in treating this complication, so researchers want to test whether giving it as a preventive measure beforehand could reduce or eliminate this dangerous side effect.

Do you qualify?

You likely qualify if…

you have been diagnosed with sickle cell disease (including hemoglobin S disease, hemoglobin SC disease, or beta thalassemia)
you have experienced serious complications from your sickle cell disease, such as stroke, chest syndrome, frequent pain episodes, or kidney or bone problems
you are between infancy and young adulthood (or for some participants, between 18–35 years old with certain blood or heart markers of disease severity)
you have a family member who is a partial genetic match and is willing to donate stem cells for your transplant
you are in good enough health overall (performance score of 50% or better) to tolerate the transplant procedure

You likely don't qualify if…

you are currently taking blood-thinning or clot-dissolving medications
you have had a previous allergic reaction to defibrotide
you are pregnant or breastfeeding
you have a serious active infection at the time you would start the study
you have already received a stem cell transplant in the past

What participation looks like

If you join this trial, you will receive the protective medication starting before your stem cell transplant conditioning (the intensive treatment that prepares your body) and continuing through your transplant procedure. You will be closely monitored with blood tests and imaging to watch for any side effects and to see how well the transplant takes hold. The exact length of participation depends on your individual transplant timeline and recovery, but you can expect regular hospital visits and follow-up appointments as part of your transplant care.

AI-generated summary from trial data · Jun 6, 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