Efficacy of Guselkumab in Treating Hailey Hailey Disease
ConditionHailey Hailey Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-90
SponsorYale University
About this trial
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 criteria
This trial accepts healthy volunteersQualifiers
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
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Guselkumab
DrugSubcutaneous injection of 100 mg at Weeks 0, 4, 12, and 20
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: GuselkumabExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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).
Time frame
baseline and week 242
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.
Time frame
baseline and week 24Secondary outcomes
1
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.
Time frame
week12 and week 242
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.
Time frame
baseline, week12 and week 243
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.
Time frame
baseline and weeks 4,12 and 24.4
Changes in cytokines
Cytokine levels (pg/ml) will be assessed in plasma
Time frame
baseline and weeks 4,12 and 24Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Yale University
Lead sponsor
Janssen Scientific Affairs, LLC
Collaborator
Source ClinicalTrials.gov