Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

New York Medical College

Status and phase: Completed; Phase 2

Conditions: Sickle Cell Disease; Acute Chest Syndrome

Treatments: Drug: Defibrotide

Study type: Interventional

Funder types: Other

Identifiers

NCT03805581

NYMC-192

Details and patient eligibility

About

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

Enrollment: 20 patients

Sex: All

Ages: 2 to 40 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

20 participants in 1 patient group

Interventional

Experimental group

Description:

Defibrotide 6.25 mg/kg IV q6h

Treatment:

Drug: Defibrotide