1 United States site
2 to 40 Years
SC
Phase 2
Interventional
All
Drug
Unknown
This study evaluates the safety of defibrotide in subjects with sickle cell disease
(SCD)-associated acute chest syndrome (ACS).
– SCD-associated ACS with the presence of any two or more of the following signs not
explained by other etiologies: Fever, Chest pain, Cough, Dyspnea, Tachypnea for age,
Pulmonary infiltrate on CXR and/or Chest CT scan, Decreased O2 saturation with or
without oxygen supplement;
– Age 2 to 40 years of age;
– Homozygous Hemoglobin S Disease, Hemoglobin SC Disease or Hemoglobin S 0/+
thalassemia;
– Informed consent/assent;
– Consent of patient/parent within ≤72 hours after inpatient admission for
SCD-associated ACS.
– Females of childbearing age will have a negative pregnancy test.
– Current Grade III or IV hemorrhage;
– Previous hypersensitivity reaction to defibrotide;
– Current systemic anti-coagulant therapy and/or fibrinolytic therapy;
– Consent of patient/parent greater than 72 hours of inpatient admission for
SCD-associated ACS;
– No signed informed consent