RecruitingEarly phase 1NCT07717346
Dermatological
Hailey-Hailey Disease
Also known as benign familial pemphigus, Gougerot-Hailey-Hailey, ATP2C1 mutation
Hailey-Hailey disease is an autosomal dominant acantholytic disorder caused by loss-of-function mutations in ATP2C1, encoding the secretory pathway calcium/manganese ATPase SPCA1. Defective calcium signalling within keratinocytes impairs ce
ORPHA:407 ↗Gene ATP2C1Prevalence 1 in 50,000Onset Early adulthood (typically 2nd–3rd decade)Autosomal dominant
2
studies recruiting now
as of 7 Sept 2026
7
studies registered in total
as of 7 Sept 2026
1
countries with a recruiting site
as of 7 Sept 2026
21 Jul 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2NCT06651489
Efficacy of Guselkumab in Treating Hailey Hailey Disease
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 Hailey-Hailey Disease
Hailey-Hailey disease is an autosomal dominant acantholytic disorder caused by loss-of-function mutations in ATP2C1, encoding the secretory pathway calcium/manganese ATPase SPCA1. Defective calcium signalling within keratinocytes impairs cell-cell adhesion, leading to acantholysis — the separation of epidermal cells — particularly in friction-prone, intertriginous areas. The resulting erosions, maceration, and fissuring have a relapsing-remitting course that is significantly worsened by heat, sweating, and secondary bacterial or candidal infection.
Common clinical features
Painful, erythematous erosions and fissures in intertriginous areas including the neck, axillae, groin, and submammary foldsRecurrent blistering that ruptures rapidly to form moist, macerated plaquesCharacteristic "wet gravel" or cobblestone surface texture of established plaquesSecondary bacterial superinfection (Staphylococcus aureus, Pseudomonas) causing malodour and exacerbating inflammationSecondary candidal infection compounding skin breakdown in warm, moist sitesLongitudinal white bands of leukonychia on fingernailsSignificant psychosocial burden and impaired quality of life due to chronic, malodorous, visible skin lesions
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 2Guselkumab (Tremfya)
Before you apply
Things trial teams commonly ask about for Hailey-Hailey Disease. Not eligibility rules; those are set by each study.
- Trials targeting ATP2C1/SPCA1 pathway or keratinocyte calcium signalling require genetic confirmation; obtain sequencing results documenting the specific ATP2C1 pathogenic variant.
- Exclusion criteria frequently include active secondary infections — ensure bacterial and fungal cultures are negative and superinfection is treated before the screening visit.
- Concomitant use of topical or systemic antibiotics and antifungals is common in this population; document all current medications as they may affect eligibility or require washout.
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).