Back to resultsDefibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
Primary Purpose
Sickle Cell Disease, Acute Chest Syndrome
Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Defibrotide
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease
Eligibility Criteria
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
Sites / Locations
- New York Medical College
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Interventional
Arm Description
Defibrotide 6.25 mg/kg IV q6h
Outcomes
Primary Outcome Measures
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.
Number of patients with grade III/IV hemorrhage
All patients will be monitored for hemorrhage probably or definitely related to defibrotide.
Secondary Outcome Measures
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
Full Information
NCT ID
NCT03805581
First Posted
December 18, 2018
Last Updated
October 24, 2022
Sponsor
New York Medical College
Collaborators
Johns Hopkins University
1. Study Identification
Unique Protocol Identification Number
NCT03805581
Brief Title
Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
Official Title
A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 12, 2018 (Actual)
Primary Completion Date
July 1, 2022 (Actual)
Study Completion Date
July 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
New York Medical College
Collaborators
Johns Hopkins University
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study evaluates the safety of defibrotide in subjects with sickle cell disease (SCD)-associated acute chest syndrome (ACS).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease, Acute Chest Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Interventional
Arm Type
Experimental
Arm Description
Defibrotide 6.25 mg/kg IV q6h
Intervention Type
Drug
Intervention Name(s)
Defibrotide
Other Intervention Name(s)
Defitelio®
Intervention Description
Defibrotide 6.25 mg/kg IV q6h up to 7 days
Primary Outcome Measure Information:
Title
Number of patients with grade III/IV allergic reaction to defibrotide
Description
All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.
Time Frame
30 days
Title
Number of patients with grade III/IV hemorrhage
Description
All patients will be monitored for hemorrhage probably or definitely related to defibrotide.
Time Frame
30 days
Secondary Outcome Measure Information:
Title
Number of patients with improvement in clinical signs of Acute Chest Syndrome
Description
Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS
Time Frame
30 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mitchell S Cairo, MD
Organizational Affiliation
New York Medical College
Official's Role
Principal Investigator
Facility Information:
Facility Name
New York Medical College
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
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