Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

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

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

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease; Acute Chest Syndrome
• Intervention / Treatment: Drug: Defibrotide

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 40 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Interventional; Defibrotide 6.25 mg/kg IV q6h	Drug: Defibrotide; Defibrotide 6.25 mg/kg IV q6h up to 7 days; Other Names: Defitelio®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Number of patients with grade III/IV hemorrhage	All patients will be monitored for hemorrhage probably or definitely related to defibrotide.	30 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: New York Medical College
• Collaborators: Johns Hopkins University
• Investigators: Principal Investigator: Mitchell S Cairo, MD, New York Medical College

Study record dates

Study Major Dates

• Study Start (Actual): January 12, 2018
• Primary Completion (Actual): July 1, 2022
• Study Completion (Actual): November 7, 2023

Study Registration Dates

• First Submitted: December 18, 2018
• First Submitted That Met QC Criteria: January 14, 2019
• First Posted (Actual): January 15, 2019

Study Record Updates

• Last Update Posted (Actual): December 8, 2023
• Last Update Submitted That Met QC Criteria: December 6, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Respiration Disorders; Lung Diseases; Disease; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Syndrome; Anemia, Sickle Cell; Acute Chest Syndrome; Molecular Mechanisms of Pharmacological Action; Fibrinolytic Agents; Fibrin Modulating Agents; Platelet Aggregation Inhibitors; Defibrotide
• Other Study ID Numbers: NYMC-192

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No