Defibrotide in Children With High Risk Kawasaki Disease

Phase 2

Active, not recruiting

• Conditions: Kawasaki Disease
• Interventions: Drug: Defibrotide

• Registration Number: NCT04777422
• Lead Sponsor: New York Medical College

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2021-02-24
• Study First Submitted: 2021-02-26
• Study First Submitted QC: 2021-02-26
• Study First Posted: 2021-03-02
• Status Verified: 2023-10
• Primary Completion: 2023-10-24
• Last Update Submitted: 2023-10-24
• Last Update Posted: 2023-10-26
• Study Completion: 2024-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 2

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Interventional	Defibrotide	Defibrotide 6.25 mg/kg IV q6h

Primary Outcome Measures

Name	Time	Method
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.
Number of patients with grade III/IV hemorrhage attributable to defibrotide	42 days	All patients will be monitored for hemorrhage probably or definitely related to defibrotide.

Secondary Outcome Measures

Name	Time	Method
Number of patients with improvement in clinical progression/signs of Kawasaki disease	42 days	Patients will have pre and post treatment assessments with imaging and blood tests to monitor clinical signs of Kawasaki disease

Trial Locations

Locations (3)

Columbia University; 🇺🇸 New York, New York, United States

New York University; 🇺🇸 New York, New York, United States

Mitchell Cairo; 🇺🇸 Valhalla, New York, United States