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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03805581
Recruitment Status : Recruiting
First Posted : January 15, 2019
Last Update Posted : January 15, 2019
Sponsor:
Collaborator:
Johns Hopkins University
Information provided by (Responsible Party):
New York Medical College

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
Sickle Cell Disease Acute Chest Syndrome Drug: Defibrotide Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
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 : January 12, 2018
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : January 2022

Resource links provided by the National Library of Medicine


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 Name: Defitelio®




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03805581


Contacts
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Contact: Mitchell S Cairo, MD 9145972150 mitchell_cairo@nymc.edu
Contact: Erin Morris, RN 7149645359 erin_morris@nymc.edu

Locations
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United States, New York
New York Medical College Recruiting
Valhalla, New York, United States, 10595
Contact: Mitchell S Cairo, MD    914-594-2150    mitchell_cairo@nymc.edu   
Principal Investigator: Mitchell S. Cairo, MD         
Sponsors and Collaborators
New York Medical College
Johns Hopkins University
Investigators
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Principal Investigator: Mitchell S Cairo, MD New York Medical College

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Responsible Party: New York Medical College
ClinicalTrials.gov Identifier: NCT03805581     History of Changes
Other Study ID Numbers: NYMC-192
First Posted: January 15, 2019    Key Record Dates
Last Update Posted: January 15, 2019
Last Verified: December 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Acute Chest Syndrome
Anemia, Sickle Cell
Syndrome
Disease
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Lung Diseases
Respiratory Tract Diseases
Respiration Disorders
Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors