Hailey Hailey DiseasePhase 2NCT06651489
Efficacy of Guselkumab in Treating Hailey Hailey Disease
A Phase 2 study testing Guselkumab in people with Hailey Hailey Disease, run by Yale University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 90 yearsHealthy volunteers can take part
- Conditions the study is for
- Hailey Hailey Disease
- What is being tested?
- Guselkumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Mar 2025to Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Church Street Research Unit, Yale Center for Clinical Investigation
New Haven, Connecticut, 06520
About this study
Official title: Efficacy of Guselkumab in the Treatment of Hailey Hailey Disease: An Open-label, Proof of Concept Study
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.
Read the detailed 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 of the final dose of guselkumab to monitor safety. Laboratory monitoring including Complete Blood Count, Basic metabolic panel, Liver function tests, and pregnancy test (if applicable) will also be performed at 1 month, 3 months, and 6 months after starting therapy. At each visit except Week 20 a full H\&P, complete Review of Systems and physical examination will be performed. Outcomes will be assessed and full-body photography will be performed at each visit except Week 20. Research tissue samples (skin biopsies) will be obtained at baseline (prior to therapy) after 24 weeks of treatment in all participants. Research blood will be obtained at screening, baseline and after 4, 12, and 24 weeks of guselkumab therapy.
- Conditions
- Hailey Hailey Disease
- Being tested
- Guselkumab
- Medical subject headings
- Pemphigus, Benign Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GuselkumabDrug
Also called Tremfya
Subcutaneous injection of 100 mg at Weeks 0, 4, 12, and 20
| Study group | Role | What they receive |
|---|---|---|
| Guselkumab | Experimental | Participants with HHD with be given guselkumab Receives: Drug: Guselkumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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.
Measured: baseline and week 24
Secondary outcomes (4)
Change in Dermatology Life Quality Index (DLQI) score
The Dermatology Life Quality Index (DLQI) is a questionnaire consisting of ten questions used to gauge how a skin disease affects an individual's quality of life. Each question evaluates the influence of the skin condition on the patient's life during the preceding week. Each question is rated on a scale from 0 to 3, resulting in a potential score range from 0 (indicating no impact of the skin disease on quality of life) to 30 (representing the maximum impact on quality of life). A decrease in DLQI scores indicates improved well-being.
Measured: week12 and week 24
Changes in Health-Related Quality of Life (HRQoL) score
HRQoL is an evaluation of how an individual's overall well-being could be influenced over a period of time due to a medical condition, disability, or disorder. The scale comprises three components. The first module which will be used in this study, known as the Core Healthy Days module, includes a single item prompting respondents to assess their overall health using a 5-point scale (1 = excellent; 3 = good; 5 = poor). Within this module, there are also three additional items that inquire about the number of days when respondents experienced poor physical health, poor mental health, and days when their health disrupted their daily activities. To calculate an unhealthy day's score, researchers sum the number of days marked as physically unhealthy and mentally unhealthy. The highest possible score is 30 unhealthy days. Lowest score is 0 unhealthy days.
Measured: baseline, week12 and week 24
Physician's Global Assessment (PGA) scores
Physicians evaluate the overall severity of ichthyosis based on clinical judgment, considering factors such as scaling, erythema, and patient-reported symptoms. Scoring is based on a 9 point Likert scale ranging from Markedly Improved (+4) to Markedly Worse (-4). Improvement in PGA involves a decrease in overall severity as judged by the physician.
Measured: baseline and weeks 4,12 and 24.
Changes in cytokines
Cytokine levels (pg/ml) will be assessed in plasma
Measured: baseline and weeks 4,12 and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2024
- Study start13 Mar 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hailey Hailey DiseaseStudies of the same condition, open and under way.
- More trials run by Yale UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yale University
- Sponsor type
- University, hospital or other
- Collaborators
- Janssen Scientific Affairs, LLC
- Responsible party
- Sponsor
- Investigators
- Keith Choate, MD, PhD, Principal investigator, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06651489
- Sponsor's protocol ID
- 2000038224
- Status last verified
- Jun 2026
- First submitted
- 17 Oct 2024
- Last updated
- 22 Jun 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Guselkumab in the Treatment of Hailey Hailey Disease: An Open-label, Proof of Concept Study
- ClinicalTrials.gov ID
- NCT06651489
- Acronym / sponsor ID
- 2000038224
- Conditions
- Hailey Hailey Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Yale UniversityUniversity, hospital or other
- Collaborators
- Janssen Scientific Affairs, LLC
- Study start
- 13 Mar 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 21 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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 of the final dose of guselkumab to monitor safety. Laboratory monitoring including Complete Blood Count, Basic metabolic panel, Liver function tests, and pregnancy test (if applicable) will also be performed at 1 month, 3 months, and 6 months after starting therapy. At each visit except Week 20 a full H\&P, complete Review of Systems and physical examination will be performed. Outcomes will be assessed and full-body photography will be performed at each visit except Week 20. Research tissue samples (skin biopsies) will be obtained at baseline (prior to therapy) after 24 weeks of treatment in all participants. Research blood will be obtained at screening, baseline and after 4, 12, and 24 weeks of guselkumab therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Guselkumab | Experimental | Drug: GuselkumabParticipants with HHD with be given guselkumab |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (14 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GuselkumabAlso: TremfyaSubcutaneous injection of 100 mg at Weeks 0, 4, 12, and 20 | Drug | Guselkumab |
Primary outcome
Change in Physician's Global Assessment (PGA) score
Time frame: baseline and week 24
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).
Change in Ichthyosis Scoring System (ISS) score
Time frame: baseline and week 24
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.
4 secondary outcomes
Change in Dermatology Life Quality Index (DLQI) score
Time frame: week12 and week 24
Changes in Health-Related Quality of Life (HRQoL) score
Time frame: baseline, week12 and week 24
Physician's Global Assessment (PGA) scores
Time frame: baseline and weeks 4,12 and 24.
Changes in cytokines
Time frame: baseline and weeks 4,12 and 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Keith Choate, MD, PhD principal investigator
Yale University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Guselkumab in people with Hailey Hailey Disease, run by Yale University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Caroline Echeandia-Francis, BA (203-785-2789, caroline.francis@yale.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Physician's Global Assessment (PGA) score (time frame: baseline and week 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.