- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03805581
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
Conditions
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
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-
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:
|
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.
Sponsor
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
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
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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