Phase 2 Sickle Cell Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with one of the following blood disorders: Homozygous Sickle Cell Disease (HbSS), Hemoglobin S Beta Thalassemia, Hemoglobin SC Disease, or Beta Thalassemia Intermedia/Major.
- You have had a stroke or a neurological problem lasting more than 24 hours that showed up on a brain scan.
- You have had a serious lung complication called Acute Chest Syndrome in the past two years, and either did not respond to, did not take, or chose not to take a medicine called hydroxyurea, or this happened before you started regular blood transfusion treatment.
- You have had at least 3 painful sickle cell episodes in the two years before joining, or before starting regular blood transfusion treatment.
- A special blood flow test of your brain (called a TCD study) showed abnormal results, leading doctors to recommend regular blood transfusions.
- A brain scan showed at least one silent (symptom-free) area of damage, or you have a kidney problem, spleen crisis, aplastic crisis, hip bone damage, two or more leg ulcers, repeated painful erections (priapism), or hand/foot swelling in infancy — all related to your sickle cell disease.
- For a specific group (Cohort 2) only: You are between 18 and 34 years old AND have at least two of the following — a very high white blood cell count when not ill, an abnormal heart valve reading on ultrasound, a need for more than 12 blood transfusions per year, a history of serious blood infection (sepsis), or an elevated heart-stress protein level in your blood when not acutely ill (confirm with trial site).
- You meet the trial's requirements related to your age, organ function, and having a matching stem cell donor available.
Who may not be able to join:
- You are currently taking blood-thinning or clot-dissolving medicines.
- You have previously had a serious allergic reaction to a drug called defibrotide.
- You are currently pregnant or breastfeeding.
- You have an active infection that is not under control at the time of joining.
- You have a fully matched, healthy family member who is willing to donate stem cells to you.
- Your general health and ability to perform daily activities is rated below 50% on a standard medical scale (note: having weakness or paralysis from a previous stroke alone does not automatically disqualify you).
- You have a history of not following through with medical care or treatment plans.
- You have significant scarring or serious damage to your liver.
- You have previously had a stem cell transplant.
- You have any medical reason that would make it unsafe for you to take defibrotide (confirm with trial site).
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Mitchell Cairo, MD, New York Medical College
Phone: 914-594-2150
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
All patients will be monitored for known and unknown side effects of defibrotide with daily physical exams while in the hospital and then as needed in addition to daily laboratory values including chemistries, hematology labs as needed; All patients will be monitored for the development of SOS.
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.