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cryopyrin-associated periodic syndrome (caps) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

cryopyrin-associated periodic syndrome

MONDO:0016168

Also known as:capsCryopyrinopathy

Find Clinical TrialsTrials

Overview

Cryopyrin associated periodic syndrome (CAPS) defines a group of autoinflammatory diseases, characterized by recurrent episodes of systemic inflammatory attacks in the absence of infection or autoimmune disease. CAPS comprises 3 disorders on a continuum of severity: severe CINCA syndrome, intermediate Muckle-Wells syndrome (MWS) and milder familial cold urticaria (FCAS).

OnsetADULT, LATE, CHILDHOOD, CONGENITAL

TreatmentsNo approved treatments · 2 in pipeline

Clinical Trials3 registered · 2 recruiting

Diagnosis

Biomarker and diagnostic research for cryopyrin-associated periodic syndrome has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for cryopyrin-associated periodic syndrome. An additional 2 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for cryopyrin-associated periodic 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 cryopyrin-associated periodic syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

3 clinical trials registered, 2 recruiting. Interventions under study include drug therapy and biologic therapy. Pipeline includes 2 PHASE1. Research is sponsored by a mix of industry and academic institutions.

57 publications have been identified in PubMed for cryopyrin-associated periodic syndrome. Research spans Case Report / Case Series (33%), Basic Science / Preclinical (25%), and Epidemiology / Natural History (12%).

Research Type	Count	% of Total
Patient case studies	19

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 12:16 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:208650

GARD

Genetic and Rare Diseases Info Center

GARD:10927

Questions for Your Care Team

→Are any of the 3 active clinical trials a good fit for my situation?
→What is the current development status of ATI-450, an orally available small molecule inhibitor of the p38alpha mitogen activated protein kinase (MAPK)/MAPK-activated protein kinase 2 (MK2) inflammatory signaling pathway, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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cryopyrin-associated periodic syndrome

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

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