Guselkumab treatment for Hailey-Hailey disease

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

This study is testing if a medication called guselkumab, usually used for psoriasis, can help people with Hailey-Hailey disease improve their skin condition.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 18 Years to 90 Years
• Sex: All
• Sponsor: Yale University (Academic / other)
• Drugs / interventions: guselkumab, methotrexate, prednisone
• Locations: 1 site (New Haven, Connecticut)
• Trial ID: NCT06651489 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of guselkumab, a medication typically used for psoriasis, in treating Hailey-Hailey disease, a chronic genetic skin disorder. Participants will receive guselkumab at a standard dosage and will be monitored through clinical follow-ups at 4, 12, and 24 weeks after treatment initiation. The study includes laboratory monitoring and the collection of skin biopsies and blood samples to assess treatment response and safety. This is a single-center, non-randomized, open-label phase II trial.

Who should consider this trial

Good fit: Ideal candidates are individuals with a documented diagnosis of Hailey-Hailey disease affecting multiple body sites with at least moderate severity.

Not a fit: Patients with a history of malignancy or those currently using certain immunosuppressants or biologics may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a reproducibly effective therapeutic option for patients suffering from Hailey-Hailey disease.

How similar studies have performed: While there have been various treatment approaches for Hailey-Hailey disease, the use of guselkumab specifically for this condition is novel and has not been extensively tested in prior studies.

Eligibility criteria

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 cyclosporine.
* Are pregnant, nursing, or planning a pregnancy while enrolled in the study or for 12 weeks after the study agent injection for women or are planning to father a child while enrolled in the study or for 12 weeks after the last study agent injection.
* Prior biologic use within the last 3 months.
* Participant has known allergies, hypersensitivity, or intolerance to guselkumab or its excipients (refer to Investigator's brochure)
* Presence of significant uncontrolled respiratory, hepatic, renal, endocrine, hematologic, neurologic, or neuropsychiatric disorders, or abnormal laboratory screening values that, in the opinion of the investigator, pose an unacceptable risk to the subject if participating in the study or of interfering with the interpretation of the data.
* Active, untreated, acute infection, or immunocompromised to an extent that participation in the study would pose an unacceptable risk to the subject based on the investigator's clinical assessment.
* Symptomatic herpes zoster infection within 12 weeks of screening or recurrent or disseminated (even a single episode) herpes zoster.
* Symptomatic herpes simplex or disseminated (even a single episode) herpes simplex at the Week 0 (baseline) visit.
* Patients with the potential for keloid formation, e.g. patients with a propensity for keloid formation, defined as a personal history of 3 or more keloids.

Where this trial is running

New Haven, Connecticut

• Church Street Research Unit, Yale Center for Clinical Investigation — New Haven, Connecticut, United States (Recruiting)

Study contacts

• Principal investigator: Keith Choate, MD, PhD — Yale University
• Study coordinator: Caroline Echeandia-Francis, BA

Conditions Hailey Hailey Disease