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Defibrotide in Children With High Risk Kawasaki Disease

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: NCT04777422
Recruitment Status : Recruiting
First Posted : March 2, 2021
Last Update Posted : November 3, 2021
Sponsor:
Collaborators:
Johns Hopkins University
Columbia University
New York University
Information provided by (Responsible Party):
New York Medical College

Study Description
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Brief Summary:
This study evaluates the safety of defibrotide with IVIG in children with high risk Kawasaki disease.

Condition or disease Intervention/treatment Phase
Kawasaki Disease Drug: Defibrotide Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study to Determine the Safety of Defibrotide in Children With High Risk Kawasaki Disease
Actual Study Start Date : February 24, 2021
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : June 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kawasaki Disease

Arms and Interventions
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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®


Outcome Measures
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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 attributable to defibrotide [ Time Frame: 42 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 progression/signs of Kawasaki disease [ Time Frame: 42 days ]
    Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of Kawasaki disease


Eligibility Criteria
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Ages Eligible for Study:   0 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Kawasaki disease presumptive diagnosis defined according AHA criteria;
  • Signed informed consent and patient assent (if applicable)
  • Diagnosis of KD and initiation of defibrotide within 96 hours from the conclusion of IVIG treatment
  • Age: 0 - 11 years old
  • High risk category defined as patient meeting ≥2 of the following criteria: male, age <6 months or >8yrs, IVIG-resistance, Fever lasting greater than 10 days, prior to diagnosis, Coronary artery aneurysms, and/or Laboratory tests indicating worse systemic inflammation
  • Patients must be ineligible for or refused to participate in the Kawasaki Disease Comparative Effectiveness (KIDCARE) study.
  • PT and PTT within institutional normal limits
  • Platelet count ≥100,000/mm3

Exclusion Criteria:

  • History of Grade III or IV hemorrhage or active bleeding;
  • Previous Grade II-IV hypersensitivity to defibrotide
  • Current systemic anti-coagulant therapy and/or fibrinolytic therapy, excluding aspirin (5 mg/kg/dose maximum).
  • Patients on an active experimental trial for Kawasaki disease
Contacts and Locations
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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): NCT04777422


Contacts
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Contact: Mitchell Cairo, MD 914-594-2150 mitchell_cairo@nymc.edu
Contact: Erin Morris, RN 914-594-2150 erin_morris@nymc.edu

Locations
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United States, New York
Columbia University Not yet recruiting
New York, New York, United States, 10032
Contact: Anne Ferris, MD    212-305-8509    af240@cumc.columbia.edu   
Contact: Katrina Golub, MPH    (212) 342-1562    kg2697@cumc.columbia.edu   
New York University Not yet recruiting
New York, New York, United States, 10032
Contact: Puneet Bhatla, MD       puneet.bhatla@nyulangone.org   
Mitchell Cairo Recruiting
Valhalla, New York, United States, 10595-1524
Contact: Mitchell Cairo, MD    914-594-2150    mitchell_cairo@nymc.edu   
Contact: Erin Morris, RN    9145942150    erin_morris@nymc.edu   
Sub-Investigator: Marise D'Souza, MD         
Sub-Investigator: Jordan Milner, MD         
Sub-Investigator: Heather Cowles, NP, RN         
Sub-Investigator: Edo Schaefer, MD         
Sponsors and Collaborators
New York Medical College
Johns Hopkins University
Columbia University
New York University
More Information
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Responsible Party: New York Medical College
ClinicalTrials.gov Identifier: NCT04777422    
Other Study ID Numbers: NYMC-204
First Posted: March 2, 2021    Key Record Dates
Last Update Posted: November 3, 2021
Last Verified: October 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by New York Medical College:
Kawasaki
Children
Defibrotide
Additional relevant MeSH terms:
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Mucocutaneous Lymph Node Syndrome
Vasculitis
Vascular Diseases
Cardiovascular Diseases
Lymphatic Diseases
Skin Diseases, Vascular
 Skin Diseases
Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors