RecruitingPhase 2

Efficacy of Guselkumab in Treating Hailey Hailey Disease

United States10 participantsStarted 2025-03-13

Plain-language 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.

Who can participate

Age range

18 Years – 90 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 biologics other than TNF-α inhibitors will not be permitted. * Patients must be willing to have skin biopsies, blood collection, and total body photography and to comply with clinic visits Exclusion Criteria: * Patients with a history of malignancy (except history of successfully treated basal cell or squamous cell carcinoma of the skin over six months prior to first study drug administration). * Patients known to be HIV or hepatitis B or C positive. * Patients planning to receive live vaccines during the duration of the study. * Patients with a positive tuberculin skin test or positive QuantiFERON TB test. * Patients with significant hepatic impairment (Child-Pugh class B and C). * Patients taking immunosuppressive and biologic medications, except for methotrexate, low-dose prednisone, and TNF-α inhibitors, including but not limited to mycophenolate mofetil, azathioprine, tacrolimus, and c…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in Physician's Global Assessment (PGA) score

Timeframe: baseline and week 24

Change in Ichthyosis Scoring System (ISS) score

Timeframe: baseline and week 24

Trial details

NCT IDNCT06651489

SponsorYale University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-12

Contact for this trial

Caroline Echeandia-Francis, BA

203-785-2789 caroline.francis@yale.edu

View on ClinicalTrials.gov More Hailey Hailey Disease trials