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RecruitingPhase 2NCT06651489

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

Sponsor

Yale University

Enrollment

10 participants

Start Date

Mar 13, 2025

Study Type

INTERVENTIONAL

Conditions

Hailey Hailey Disease

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.

Eligibility

Min Age: 18 YearsMax Age: 90 Years

Inclusion Criteria4

  • 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 Criteria14

  • 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.

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Interventions

DRUGGuselkumab

Subcutaneous injection of 100 mg at Weeks 0, 4, 12, and 20

Locations(1)

Church Street Research Unit, Yale Center for Clinical Investigation

New Haven, Connecticut, United States

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NCT06651489