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Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

Sponsor
New York Medical College (Other)
Overall Status
Recruiting
CT.gov ID
NCT03805581
Collaborator
Johns Hopkins University (Other)
20
Enrollment
1
Location
1
Arm
65.6
Anticipated Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
Actual Study Start Date :
Jan 12, 2018
Anticipated Primary Completion Date :
Jul 1, 2022
Anticipated Study Completion Date :
Jul 1, 2023

Arms and Interventions

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®

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number of patients with grade III/IV allergic reaction to defibrotide [30 days]

      All patients will be monitored for allergic reaction probably or definitely related to defibrotide administration.

    2. Number of patients with grade III/IV hemorrhage [30 days]

      All patients will be monitored for hemorrhage probably or definitely related to defibrotide.

    Secondary Outcome Measures

    1. Number of patients with improvement in clinical signs of Acute Chest Syndrome [30 days]

      Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of ACS

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 40 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 New York Medical College Valhalla New York United States 10595

    Sponsors and Collaborators

    • New York Medical College
    • Johns Hopkins University

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    New York Medical College
    ClinicalTrials.gov Identifier:
    NCT03805581
    Other Study ID Numbers:
    • NYMC-192
    First Posted:
    Jan 15, 2019
    Last Update Posted:
    Nov 22, 2021
    Last Verified:
    Nov 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Acute Chest Syndrome
    Anemia, Sickle Cell
    Syndrome
    Defibrotide

    Study Results

    No Results Posted as of Nov 22, 2021