RecruitingNCT05656365
Immune
PFAPA Syndrome
Also known as periodic fever aphthous stomatitis pharyngitis adenitis, Marshall syndrome
PFAPA Syndrome is the most common periodic fever syndrome in children, characterized by remarkably regular febrile episodes recurring every 3-6 weeks and lasting 3-6 days, accompanied by at least one of three clinical features: aphthous sto
ORPHA:42642 ↗Prevalence Estimated 1 in 10,000-20,000 children; exact prevalence uncertainOnset Early childhood (typically before age 5)Autoinflammatory periodic fever syndrome
2
studies recruiting now
as of 7 Sept 2026
6
studies registered in total
as of 7 Sept 2026
23
countries with a recruiting site
as of 7 Sept 2026
19 Dec 2022
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT05200715
AutoInflammatory Disease Alliance Registry (AIDA)
Showing the 2 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
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Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About PFAPA Syndrome
PFAPA Syndrome is the most common periodic fever syndrome in children, characterized by remarkably regular febrile episodes recurring every 3-6 weeks and lasting 3-6 days, accompanied by at least one of three clinical features: aphthous stomatitis, cervical lymphadenopathy, and pharyngitis, in the absence of upper respiratory infection. The etiology remains unclear, though dysregulated innate immune activation and inflammasome involvement are implicated given the dramatic response to single-dose corticosteroids. Tonsillectomy is curative in the majority of cases, and spontaneous resolution typically occurs in adolescence.
Common clinical features
Regular febrile episodes every 3-6 weeks with clockwork periodicityAphthous mouth ulcers during attacksExudative or non-exudative pharyngitisCervical lymphadenopathyComplete well-being and normal growth between attacksAbrupt fever resolution (often with night sweat)Dramatic response to single-dose prednisolone (diagnostic clue)
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Before you apply
Things trial teams commonly ask about for PFAPA Syndrome. Not eligibility rules; those are set by each study.
- PFAPA clinical criteria require excluding other periodic fever syndromes (FMF, HIDS, TRAPS) through genetic testing; documentation of negative genetic panel results strengthens eligibility for PFAPA-specific natural history studies
- Attack diary with dates, duration, and symptom checklist for at least 6 months is essential for trial enrollment; prospective data is far more useful than recalled history
- Tonsillectomy history affects eligibility: surgical trials are limited to tonsil-intact patients, while natural history studies often enroll post-tonsillectomy patients to track recurrence
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).