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Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03805581
Recruitment Status : Active, not recruiting
First Posted : January 15, 2019
Last Update Posted : October 25, 2022
Sponsor:
Collaborator:
Johns Hopkins University
Information provided by (Responsible Party):
New York Medical College

Tracking Information
First Submitted Date  ICMJE December 18, 2018
First Posted Date  ICMJE January 15, 2019
Last Update Posted Date October 25, 2022
Actual Study Start Date  ICMJE January 12, 2018
Actual Primary Completion Date July 1, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 14, 2019)
  • 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.
  • 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 14, 2019)		 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
Official Title  ICMJE A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
Brief Summary This study evaluates the safety of defibrotide in subjects with sickle cell disease (SCD)-associated acute chest syndrome (ACS).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Disease
  • Acute Chest Syndrome
Intervention  ICMJE Drug: Defibrotide
Defibrotide 6.25 mg/kg IV q6h up to 7 days
Other Name: Defitelio®
Study Arms  ICMJE Experimental: Interventional
Defibrotide 6.25 mg/kg IV q6h
Intervention: Drug: Defibrotide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 14, 2019)		 20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2023
Actual Primary Completion Date July 1, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 40 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03805581
Other Study ID Numbers  ICMJE NYMC-192
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party New York Medical College
Original Responsible Party Same as current
Current Study Sponsor  ICMJE New York Medical College
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Johns Hopkins University
Investigators  ICMJE
Principal Investigator: Mitchell S Cairo, MD New York Medical College
PRS Account New York Medical College
Verification Date October 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP