Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

December 6, 2023 updated by: New York Medical College

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

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Interventional
Defibrotide 6.25 mg/kg IV q6h
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
Time Frame: 30 days
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
Time Frame: 30 days
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
Time Frame: 30 days
Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS
30 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Collaborators

Investigators

  • Principal Investigator: Mitchell S Cairo, MD, New York Medical College

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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