Defibrotide Prophylaxis + Stem Cell Transplant for Sickle Cell Disease
(NYMC-571 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests if Defibrotide can safely and effectively prevent liver damage in high-risk sickle cell or beta thalassemia patients undergoing a special stem cell transplant. The medication works by improving blood flow in the liver to prevent blockages.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking systemic anticoagulants or fibrinolytic therapies.
Is defibrotide safe for use in humans?
Defibrotide has been used safely in humans, particularly for treating a liver condition called sinusoidal obstruction syndrome (SOS) after stem cell transplants. It is generally well tolerated, with common side effects including bleeding and low blood pressure, but it has not been linked to a higher risk of serious bleeding events.12345
How is the drug Defibrotide unique in treating sickle cell disease with stem cell transplant?
Defibrotide is unique because it is primarily used to prevent and treat veno-occlusive disease (a liver condition) in patients undergoing stem cell transplants, and it works by protecting blood vessel cells and restoring balance in blood clotting and breakdown. Its use in sickle cell disease with stem cell transplant is novel, as it is not a standard treatment for this condition.12367
What data supports the effectiveness of the drug Defibrotide in combination with stem cell transplant for sickle cell disease?
Who Is on the Research Team?
Mitchell S Cairo, MD
Principal Investigator
New York Medical College
Are You a Good Fit for This Trial?
This trial is for high-risk sickle cell or beta thalassemia patients who have had severe complications like acute chest syndrome, stroke, or organ damage. It's open to those with specific genetic forms of the disease and certain health indicators like elevated NT-proBNP levels. Adults up to age 34.99 can join if they meet additional criteria such as a history of frequent blood transfusions or leg ulcers.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Prophylactic Treatment
Defibrotide prophylaxis is administered starting 10 days before stem cell infusion and continues through Day +21
Stem Cell Transplantation
Familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback
Follow-up
Participants are monitored for safety and effectiveness after treatment, including monitoring for the development of SOS
What Are the Treatments Tested in This Trial?
Interventions
- Defibrotide
Defibrotide is already approved in United States, European Union for the following indications:
- Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT)
- Severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT)
Find a Clinic Near You
Who Is Running the Clinical Trial?
New York Medical College
Lead Sponsor
Baylor College of Medicine
Collaborator
Johns Hopkins University
Collaborator
Children's Hospital Los Angeles
Collaborator
Dana-Farber Cancer Institute
Collaborator
Medical College of Wisconsin
Collaborator
Tufts Medical Center
Collaborator
University of California, Los Angeles
Collaborator