Efficacy of Guselkumab in the Treatment of Hailey Hailey Disease: An Open-label, Proof of Concept Study
Yale University
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.
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 o…
Eligibility
- Age range
- 18–90 years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: * A documented diagnosis of Hailey-Hailey disease confirmed with clinical, and histopathologic findings * Disease affecting more than one body site with at least moderate severity * If patients are taking other systemic therapies for their HHD (antibiotics, prednisone), they must be taking a stable dose of the other medication(s) for at least 3 months with no plans to change the regimen in the next 6 months. With the exception of antibiotics, methotrexate or low dose prednisone (less than 5 mg daily), use of concomitant immunosuppressants, e.g. azathioprine, etc. and biolo…
Interventions
- DrugGuselkumab
Subcutaneous injection of 100 mg at Weeks 0, 4, 12, and 20
Location
- Church Street Research Unit, Yale Center for Clinical InvestigationNew Haven, Connecticut