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Kawasaki disease (KD) is a febrile, systemic, self-limiting vasculitis affecting children and characterized by inflammation in the medium sized vessels associated with coronary arterial aneurysms (CAA) that may be life threatening when untreated. KD is the most common cause of acquired heart disease in children in developed countries and is a risk factor for ischemic heart disease in adulthood.
Biomarker and diagnostic research for mucocutaneous lymph node syndrome has been reported in the published literature.
No approved treatments are currently available for mucocutaneous lymph node syndrome. An additional 3 compounds hold orphan drug designation.
While no drugs are FDA-approved specifically for mucocutaneous lymph node syndrome, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for mucocutaneous lymph node syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: Unknown (Unknown prevalence).
19 clinical trials registered, 11 recruiting. Interventions under study include other interventions and drug therapy. Pipeline includes 3 PHASE4, 3 PHASE3, 2 PHASE2. Research is primarily sponsored by academic and government institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT04278404](https://clinicaltrials.gov/study/NCT04278404) |
Data assembled from 6 of 12 sources · Last updated Sep 18, 2026, 7:06 AM UTC
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Designated
Exclusivity End |
|---|
Designation Status |
|---|
infliximab | infliximab | ODDIFACT SAS | 2024 | — | Designated |
aspirin for injection (acetylsalicylic acid) | aspirin for injection (acetylsalicylic acid) | Rhoshan Pharmaceuticals Inc. | 2020 | — | Designated |
Ulinastatin | Ulinastatin | RNR BioMedical Inc. | 2020 | — | Designated |
19 trials found
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
— |
Duke University |
RECRUITING |
[NCT04656184](https://clinicaltrials.gov/study/NCT04656184) | A 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 | PHASE3 | Assistance Publique - Hôpitaux de Paris | RECRUITING |
[NCT06978439](https://clinicaltrials.gov/study/NCT06978439) | Model-informed Dose Optimization for Rivaroxaban in Children With Giant Coronary Artery Aneurysm After Kawasaki Disease | PHASE4 | Children's Hospital of Fudan University | RECRUITING |
[NCT07086989](https://clinicaltrials.gov/study/NCT07086989) | Cardiovascular Risk in Children With Chronic Conditions Study | — | Semmelweis University | RECRUITING |
[NCT01793168](https://clinicaltrials.gov/study/NCT01793168) | Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford | — | Sanford Health | RECRUITING |
224 publications have been identified in PubMed for mucocutaneous lymph node syndrome. Kisho has analyzed 29 by research type. Research spans Review / Meta-Analysis (41%), Basic Science / Preclinical (17%), and Case Report / Case Series (14%).
Research Type | Count | % of Total |
|---|---|---|
Research summaries | 12 | 41% |
Laboratory research | 5 | 17% |
Patient case studies | 4 | 14% |
Disease patterns and progression | 4 | 14% |
Testing and diagnosis research | 3 | 10% |
Clinical study results | 1 | 3% |
Murakami F (2026). [PMID: 41583049](https://pubmed.ncbi.nlm.nih.gov/41583049/). *Case Rep Oncol*. [Case Report / Case Series]
Yeh YT (2025). [PMID: 40448518](https://pubmed.ncbi.nlm.nih.gov/40448518/). *Virulence*. [Basic Science / Preclinical]
Fischetti G (2025). [PMID: 40980138](https://pubmed.ncbi.nlm.nih.gov/40980138/). *Front Pediatr*. [Case Report / Case Series]
Wang X (2025). [PMID: 40457357](https://pubmed.ncbi.nlm.nih.gov/40457357/). *Pediatr Rheumatol Online J*. [Diagnostic / Biomarker]
Varol SE (2025). [PMID: 40590520](https://pubmed.ncbi.nlm.nih.gov/40590520/). *Int J Rheum Dis*. [Epidemiology / Natural History]
Khan S (2025). [PMID: 41042754](https://pubmed.ncbi.nlm.nih.gov/41042754/). *PLoS One*. [Diagnostic / Biomarker]
Guan Y (2025). [PMID: 39987982](https://pubmed.ncbi.nlm.nih.gov/39987982/). *Chem Biol Interact*. [Basic Science / Preclinical]
Feng C (2025). [PMID: 40276515](https://pubmed.ncbi.nlm.nih.gov/40276515/). *Front Immunol*. [Basic Science / Preclinical]
Nigrovic PA (2025). [PMID: 39491365](https://pubmed.ncbi.nlm.nih.gov/39491365/). *Arthritis Rheumatol*. [Review / Meta-Analysis]
Kwak JH (2025). [PMID: 39929006](https://pubmed.ncbi.nlm.nih.gov/39929006/). *Semin Arthritis Rheum*. [Epidemiology / Natural History]