A Randomized Phase III Multicenter Trial Comparing the Efficacy and Safety of Anakinra Versus Intravenous Immunoglobulin (IVIG) Retreatment, in Patients With Kawasaki Disease Who Failed to Respond to Initial Standard IVIG Treatment

Who is this study for? Patients with Kawasaki disease who failed to respond to initial standard IVIG treatment
What treatments are being studied? Anakinra
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

Kawasaki disease (KD) is the most frequent vasculitis in younger children \<5years, and the first cause of acquired ischemic myocardiopathy in childhood. Exceptionally, KD may cause early death during the acute phase by myocardial infarction, but may compromise the long-term cardiovascular outcome by accelerating atherosclerotic disease. The incidence of KD is high in far-Eastern countries and Hawaii but KD is relatively rare in other regions (10/100000 children \<5years in northern Europe) which makes it difficult to develop research on these rare population. Early recognition and treatment by intravenous immunoglobulins (IVIG) influences the prognosis positively. IVIG are the standard of care and decrease significantly the risk of coronary aneurysms. However, despite a first infusion of IVIG, 20% of KD patients remain febrile and have high risk of coronary vasculitis. Recent Japanese research group assessed additional cyclosporine treatment in first line KD treatment but failed preventing relapse. To date there is no agreement for a more effective second line treatment. Based on the auto-inflammatory pattern of KD, the investigators hypothesize that anti IL-1 blocking agents could bring a rapid and sustained effect on systemic and coronary inflammation in patients with KD. Our hypotheses are: 1. Anakinra treatment may reduce the early and long-term mortality of patients with Kawasaki Disease (KD), by a rapid and sustained effect on vascular inflammation. 2. The safety of anakinra is good, as the drug has a very short half-life, which allows its rapid withdrawal in case of serious adverse event. The use of anakinra is not associated with the risk of contamination by infectious agents, which remain even minimal, a possibility with the use of IVIG.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3 months
Maximum Age: 17
Healthy Volunteers: f
View:

• Children, male and female, from 3 months to \<18 years old

• Patient ≥ 5 kg

• Patient with KD according to the American Heart Association definition for complete or incomplete KD. (Fever ≥ 5 days (or at least 3 days if KD with American Heart Association criteria since the third days of fever) and ≥ 4 of 5 main clinical signs: modification of the extremities, polymorphic exanthema, and bilateral bulbar not exudative conjunctivitis, erythema of the lips or oral cavity, and cervical lymph nodes usually unilateral \> 1.5 cm in diameter.

• Patients who failed to respond to the standard therapy of KD, e.g. Persistence or recrudescence of fever ≥ 38°C, 48 hours after the infusion of 2g/kg of IV Ig. Patients may be screened 24h after the end of the first infusion if they remain febrile 24h after the end of the first infusion.

• Patient, parents or legal guardian's written informed consent is required

• Patient with health insurance (SS or CMU).

• Efficient contraception for the duration of participation in the research for childbearing aged women

Locations
Other Locations
France
CHU de Bicêtre
RECRUITING
Le Kremlin-bicêtre
Contact Information
Primary
Isabelle Koné-Paut, Pr
isabelle.kone-paut@aphp.fr
00 33 1 45 21 32 46
Time Frame
Start Date: 2023-10-20
Estimated Completion Date: 2027-03
Participants
Target number of participants: 84
Treatments
Experimental: KINERET
The patients will receive anakinra, an analogue of the IL-1 receptor antagonist, at a starting dose of 4 mg/kg. If patients are still febrile with 12 hours (H12) of treatment, they will receive a supplementary dose of 2 mg/Kg; otherwise, they will remain at a starting dose of 4 mg/kg. If they are still febrile at H24, they will receive a dose of 8mg/kg; otherwise, they will maintain their dose of 6 mg/kg. Patients with temperature \<38°C at any point between initiation and day 14, but who develop secondary fever due to KD could have further escalation dose of anakinra until a maximum dose of 8mg/Kg. Patients will receive anakinra during 14 days independently of the period of escalation dose if any. After the last escalation dose, if any necessary, the primary criteria will be measured. Patients not responding to anakinra will follow usual standard care and will complete information related to all the study visits
Active_comparator: Intravenous Immunoglobulin
The patients will receive a standard therapy, IVIG infusion of 2g/kg, and their treatment will follow usual standard care. Patients in the IVIG treatment will complete information related to the study visits.
Sponsors
Collaborators: Swedish Orphan Biovitrum
Leads: Assistance Publique - Hôpitaux de Paris

This content was sourced from clinicaltrials.gov

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