A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

Locations

1 United States site

Age

2 to 40 Years

Genotypes

SC

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

This study evaluates the safety of defibrotide in subjects with sickle cell disease
(SCD)-associated acute chest syndrome (ACS).

participation requirements

– 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.

participation restrictions

– 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

Locations

  • Valhalla, New York, United States, New York Medical College, 10595