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Defibrotide in Children With High Risk Kawasaki Disease

Primary Purpose

Kawasaki Disease

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Defibrotide

About this trial

This is an interventional treatment trial for Kawasaki Disease focused on measuring Kawasaki, Children, Defibrotide

Eligibility Criteria

0 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Columbia UniversityRecruiting
New York UniversityRecruiting
Mitchell CairoRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Interventional

Arm Description

Defibrotide 6.25 mg/kg IV q6h

Outcomes

Primary Outcome Measures

Number of patients with grade III/IV allergic reaction to defibrotide

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

Number of patients with grade III/IV hemorrhage attributable to defibrotide

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

Secondary Outcome Measures

Number of patients with improvement in clinical progression/signs of Kawasaki disease

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

Full Information

NCT ID

NCT04777422

First Posted

February 26, 2021

Last Updated

March 24, 2023

Sponsor

New York Medical College

Collaborators

Johns Hopkins University, Columbia University, New York University

1. Study Identification

Unique Protocol Identification Number

NCT04777422

Brief Title

Defibrotide in Children With High Risk Kawasaki Disease

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Recruiting

Study Start Date

February 24, 2021 (Actual)

Primary Completion Date

March 2024 (Anticipated)

Study Completion Date

June 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

New York Medical College

Collaborators

Johns Hopkins University, Columbia University, New York University

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Kawasaki Disease

Keywords

Kawasaki, Children, Defibrotide

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Interventional

Arm Type

Experimental

Arm Description

Defibrotide 6.25 mg/kg IV q6h

Intervention Type

Drug

Intervention Name(s)

Defibrotide

Other Intervention Name(s)

Defitelio®

Intervention Description

Defibrotide 6.25 mg/kg IV q6h up to 7 days

Primary Outcome Measure Information:

Title

Number of patients with grade III/IV allergic reaction to defibrotide

Description

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

Time Frame

30 days

Title

Number of patients with grade III/IV hemorrhage attributable to defibrotide

Description

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

Time Frame

42 days

Secondary Outcome Measure Information:

Title

Number of patients with improvement in clinical progression/signs of Kawasaki disease

Description

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

Time Frame

42 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

0 Years

Maximum Age & Unit of Time

11 Years

Accepts Healthy Volunteers

Eligibility 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 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Mitchell Cairo, MD

Phone

914-594-2150

mitchell_cairo@nymc.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Erin Morris, RN

Phone

914-594-2150

erin_morris@nymc.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Mitchell S. Cairo, MD

Organizational Affiliation

New York Medical College

Official's Role

Principal Investigator

Facility Information:

Facility Name

Columbia University

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Anne Ferris, MD

Phone

212-305-8509

af240@cumc.columbia.edu

Facility Name

New York University

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Puneet Bhatla, MD

puneet.bhatla@nyulangone.org

Facility Name

Mitchell Cairo

City

Valhalla

State/Province

New York

ZIP/Postal Code

10595-1524

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Mitchell Cairo, MD

Phone

914-594-2150

mitchell_cairo@nymc.edu

First Name & Middle Initial & Last Name & Degree

Erin Morris, RN

Phone

9145942150

erin_morris@nymc.edu

First Name & Middle Initial & Last Name & Degree

Heather Cowles, NP, RN

First Name & Middle Initial & Last Name & Degree

Edo Schaefer, MD

12. IPD Sharing Statement

Learn more about this trial

Defibrotide in Children With High Risk Kawasaki Disease

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