Defibrotide in Children With High Risk Kawasaki Disease

October 24, 2023 updated by: New York Medical College

A Pilot Study to Determine the Safety of Defibrotide in Children With High Risk Kawasaki Disease

This study evaluates the safety of defibrotide with IVIG in children with high risk Kawasaki disease.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

2

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

  • United States
    • New York
      • New York, New York, United States, 10032
        • Columbia University
      • New York, New York, United States, 10032
        • New York University
      • Valhalla, New York, United States, 10595-1524
        • Mitchell Cairo

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

1 second to 11 years (Child)

Accepts Healthy Volunteers

No

Description

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
  • 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

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
Drug: Defibrotide
Defibrotide 6.25 mg/kg IV q6h up to 7 days
Other Names:
  • Defitelio®

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 attributable to defibrotide
Time Frame: 42 days
All patients will be monitored for hemorrhage probably or definitely related to defibrotide.
42 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
42 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

New York Medical College

Collaborators

Johns Hopkins University
New York University
Columbia University

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)

February 24, 2021

Primary Completion (Actual)

October 24, 2023

Study Completion (Estimated)

June 1, 2024

Study Registration Dates

First Submitted

February 26, 2021

First Submitted That Met QC Criteria

February 26, 2021

First Posted (Actual)

March 2, 2021

Study Record Updates

Last Update Posted (Actual)

October 26, 2023

Last Update Submitted That Met QC Criteria

October 24, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NYMC-204

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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