Efficacy of Guselkumab in Treating Hailey Hailey Disease

NCT06651489 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 2000038224
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hailey Hailey Disease

Outcome Measures

Primary Outcomes (2)

• Change in Physician's Global Assessment (PGA) score

  The PGA is a widely recognized and frequently utilized scale for evaluating treatment outcomes in clinical trials, spanning across both adult and pediatric populations. It is a straightforward tool with easily interpretable results. . In order to conduct this assessment, a healthcare provider or physician relies on a comprehensive analysis of data gathered from all aspects of the patient's medical history and condition. Scoring is based on a 9 point Likert scale ranging from Markedly Improved (+4) to Markedly Worse (-4).

  baseline and week 24

• Change in Ichthyosis Scoring System (ISS) score

  ISS is a reliable system to measure global ichthyosis severity in adults and children. The body is divided into 10 regions, and Likert scales (0-4) are used to quantify scale and erythema, with extensive descriptors and photographic standards. The ISS score takes values from 0 to 75. Higher scores indicate more severe injury.

  baseline and week 24

Secondary Outcomes (4)

• Change in Dermatology Life Quality Index (DLQI) score

  week12 and week 24

• Changes in Health-Related Quality of Life (HRQoL) score

  baseline, week12 and week 24

• Physician's Global Assessment (PGA) scores

  baseline and weeks 4,12 and 24.

• Changes in cytokines

  baseline and weeks 4,12 and 24

Study Arms (1)

Guselkumab

EXPERIMENTAL

Participants with HHD with be given guselkumab

Drug: Guselkumab

Interventions

Guselkumab DRUG

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

Also known as: Tremfya

Guselkumab

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Yale University: lead
• Janssen Scientific Affairs, LLC: collaborator

Study Sites (1)

Church Street Research Unit, Yale Center for Clinical Investigation

New Haven, Connecticut, 06520, United States

RECRUITING

MeSH Terms

Conditions

Pemphigus, Benign Familial

Interventions

guselkumab

Condition Hierarchy (Ancestors)

Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Study Officials

• Keith Choate, MD, PhD

  Yale University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Caroline Echeandia-Francis, BA

CONTACT

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

Sheila Garcia, BS

CONTACT

475-321-0545; Sheila.garcia@yale.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2024
• First Posted: October 21, 2024
• Study Start: March 13, 2025
• Primary Completion: April 1, 2026
• Study Completion: April 1, 2026
• Last Updated: December 4, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)