Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03805581
• Other study ID #: NYMC-192
• Status: Completed
• Phase: Phase 2
• Start date: January 12, 2018
• Est. completion date: November 7, 2023

Study information

• Verified date: December 2023
• Source: New York Medical College
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: November 7, 2023
• Est. primary completion date: July 1, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 40 Years

Eligibility

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

Related Conditions & MeSH terms

• Acute Chest Syndrome
• Anemia, Sickle Cell
• Sickle Cell Disease
• Syndrome

Intervention

Drug:
• Defibrotide
Defibrotide 6.25 mg/kg IV q6h up to 7 days

Locations

Country	Name	City	State
United States	New York Medical College	Valhalla	New York

Sponsors (2)

Lead Sponsor	Collaborator
New York Medical College	Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of patients with grade III/IV allergic reaction to defibrotide	All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.	30 days
Primary	Number of patients with grade III/IV hemorrhage	All patients will be monitored for hemorrhage probably or definitely related to defibrotide.	30 days
Secondary	Number of patients with improvement in clinical signs of Acute Chest Syndrome	Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS	30 days