Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

Phase 2

Completed

• Conditions: Acute Chest Syndrome; Sickle Cell Disease
• Interventions: Drug: Defibrotide

• Registration Number: NCT03805581
• Lead Sponsor: New York Medical College

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2018-01-12
• Study First Submitted: 2018-12-18
• Study First Submitted QC: 2019-01-14
• Study First Posted: 2019-01-15
• Primary Completion: 2022-07-01
• Study Completion: 2023-11-07
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-06
• Last Update Posted: 2023-12-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Interventional	Defibrotide	Defibrotide 6.25 mg/kg IV q6h

Primary Outcome Measures

Name	Time	Method
Number of patients with grade III/IV allergic reaction to defibrotide	30 days	All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.
Number of patients with grade III/IV hemorrhage	30 days	All patients will be monitored for hemorrhage probably or definitely related to defibrotide.

Secondary Outcome Measures

Name	Time	Method
Number of patients with improvement in clinical signs of Acute Chest Syndrome	30 days	Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS

Trial Locations

Locations (1)

New York Medical College; 🇺🇸 Valhalla, New York, United States