Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

• Status: Active, not recruiting
• Trial end date: 2023-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: New York Medical College
• Collaborator: Johns Hopkins University
• Treatments: Defibrotide

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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