Defibrotide in Children With High Risk Kawasaki Disease

NCT04777422 | Unknown Phase 2 DMC FDA Drug

• 1 other identifier: NYMC-204
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 3

Brief Summary

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

Conditions

Kawasaki Disease

Keywords

Kawasaki; Children; Defibrotide

Outcome Measures

Primary Outcomes (2)

• 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.

  30 days

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

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

  42 days

Secondary Outcomes (1)

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

  42 days

Study Arms (1)

Interventional

EXPERIMENTAL

Defibrotide 6.25 mg/kg IV q6h

Drug: Defibrotide

Interventions

Defibrotide DRUG

Defibrotide 6.25 mg/kg IV q6h up to 7 days

Also known as: Defitelio®

Interventional

Eligibility Criteria

• Age: 0 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• New York Medical College: lead
• Johns Hopkins University: collaborator
• Columbia University: collaborator
• New York University: collaborator

Study Sites (3)

Columbia University

New York, New York, 10032, United States

Location

New York University

New York, New York, 10032, United States

Location

Mitchell Cairo

Valhalla, New York, 10595-1524, United States

Location

MeSH Terms

Conditions

Mucocutaneous Lymph Node Syndrome

Interventions

defibrotide

Condition Hierarchy (Ancestors)

Vasculitis; Vascular Diseases; Cardiovascular Diseases; Lymphatic Diseases; Hemic and Lymphatic Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Mitchell S. Cairo, MD

  New York Medical College

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2021
• First Posted: March 2, 2021
• Study Start: February 24, 2021
• Primary Completion: October 24, 2023
• Study Completion: June 1, 2024
• Last Updated: October 26, 2023

Record last verified: 2023-10

Locations

US (3)