NCT06651489

Efficacy of Guselkumab in Treating Hailey Hailey Disease

Efficacy of Guselkumab in the Treatment of Hailey Hailey Disease: An Open-label, Proof of Concept Study

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Yale University · Academic / Other
• Sex: All
• Age: 18 Years – 90 Years
• Healthy volunteers: Accepted

Summary

Hailey-Hailey disease (HHD) is a debilitating genetic skin disorder, affecting mainly body folds with erythema and painful erosions and blisters. Histopathological findings include epidermal hyperplasia, suprabasilar clefting, dyskeratosis and acantholysis of keratinocytes. A final diagnosis of HHD is usually confirmed based on clinical and histopathological findings in line with genetic testing. Several treatment options have been proposed for this chronic and disabling disorder, however, there is no reproducibly effective therapeutic for it. The primary objective is to evaluate the treatment response of guselkumab. Single-center, non-randomized, single-arm, open-label, phase II trial to evaluate the efficacy and safety of guselkumab for the treatment of patients with HHD.

Detailed description

Several treatment options have been proposed for HHD as chronic and disabling disorder, however, there is no reproducibly effective therapeutic for it. The primary objective is to evaluate the treatment response of guselkumab. After the participant has been deemed an appropriate candidate for the study and has signed the consent form, they will be enrolled. All participants will be started on guselkumab 100mg at the FDA-approved psoriasis dose and will be seen for clinical follow-up 4, 12, 24 weeks after initiation of therapy. A 12-week follow-up period will be conducted post-administration of the final dose of guselkumab to monitor safety. Laboratory monitoring including Complete Blood Count, Basic metabolic panel, Liver function tests, and pregnancy test (if applicable) will also be performed at 1 month, 3 months, and 6 months after starting therapy. At each visit except Week 20 a full H\&P, complete Review of Systems and physical examination will be performed. Outcomes will be assessed and full-body photography will be performed at each visit except Week 20. Research tissue samples (skin biopsies) will be obtained at baseline (prior to therapy) after 24 weeks of treatment in all participants. Research blood will be obtained at screening, baseline and after 4, 12, and 24 weeks of guselkumab therapy.

Conditions

• Hailey Hailey Disease

Interventions

Type	Name	Description
DRUG	Guselkumab	Subcutaneous injection of 100 mg at Weeks 0, 4, 12, and 20

Timeline

• Start date: 2025-03-13
• Primary completion: 2026-04-01
• Completion: 2026-04-01
• First posted: 2024-10-21
• Last updated: 2025-12-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study