Kawasaki DiseasePhase 2NCT04777422
Defibrotide in Children With High Risk Kawasaki Disease
A Phase 2 study testing Defibrotide in people with Kawasaki Disease, run by New York Medical College at 3 sites in United States.
Trial summary
This study evaluates the safety of defibrotide with IVIG in children with high risk Kawasaki disease. Read more about this study
- Who is it for?
- Ages 0 to 11 years
- Conditions the study is for
- Kawasaki Disease
- What is being tested?
- Defibrotide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 3 sitesUnited States
- Study dates
- 24 Feb 2021to Jun 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Columbia University
New York, New York, 10032
New York University
New York, New York, 10032
Mitchell Cairo
Valhalla, New York, 10595-1524
About this study
Official title: 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.
- Conditions
- Kawasaki Disease
- Being tested
- Defibrotide
- Keywords
- Kawasaki, Children, Defibrotide
- Medical subject headings
- Mucocutaneous Lymph Node Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DefibrotideDrug
Also called Defitelio®
Defibrotide 6.25 mg/kg IV q6h up to 7 days
| Study group | Role | What they receive |
|---|---|---|
| Interventional | Experimental | Defibrotide 6.25 mg/kg IV q6h Receives: Drug: Defibrotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 42 days
Secondary outcomes (1)
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
Measured: 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2021
- Study start24 Feb 2021
- Primary completion24 Oct 2023
- Study completionJun 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Kawasaki DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins University, Columbia University, New York University
- Responsible party
- Sponsor
- Investigators
- Mitchell S. Cairo, MD, Principal investigator, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04777422
- Sponsor's protocol ID
- NYMC-204
- Status last verified
- Oct 2023
- First submitted
- 26 Feb 2021
- Last updated
- 26 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Determine the Safety of Defibrotide in Children With High Risk Kawasaki Disease
- ClinicalTrials.gov ID
- NCT04777422
- Acronym / sponsor ID
- NYMC-204
- Conditions
- Kawasaki Disease
- Keywords
- Kawasaki, Children, Defibrotide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 2 participants (actual)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Collaborators
- Johns Hopkins University, Columbia University, New York University
- Study start
- 24 Feb 2021 (actual)
- Primary completion
- 24 Oct 2023 (actual)
- Study completion
- Jun 2024 (estimated)
- First posted
- 2 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates the safety of defibrotide with IVIG in children with high risk Kawasaki disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Interventional | Experimental | Drug: DefibrotideDefibrotide 6.25 mg/kg IV q6h |
Who may qualify
- Ages
- 0 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DefibrotideAlso: Defitelio®Defibrotide 6.25 mg/kg IV q6h up to 7 days | Drug | Interventional |
Primary outcome
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.
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.
1 secondary outcome
Number of patients with improvement in clinical progression/signs of Kawasaki disease
Time frame: 42 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mitchell S. Cairo, MD principal investigator
New York Medical College
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Defibrotide in people with Kawasaki Disease, run by New York Medical College at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with grade III/IV allergic reaction to defibrotide (time frame: 30 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2023.