Study to Evaluate the Safety and Efficacy of Tapinarof in Adults With Palmoplantar Keratoderma
An Open-label, Phase II Study to Evaluate the Clinical Efficacy and Safety of Tapinarof for Adult Patients With Palmoplantar Keratoderma
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
This study aims to assess the benefits of the treatment. Should the treatment demonstrate benefits (as defined by the primary outcome measure) without any observed serious adverse events (SAEs), further clinical trials would be justified. If at least 2 out of 6 subjects benefits from the treatment (as defined by our primary outcome measure) and there are no SAEs, future clinical trials would be warranted. This initial data will determine whether it is worthwhile to proceed with a phase 2 trial, that will be both blinded and well-controlled.
The study is designed to gather preliminary experiences that can be used to design subsequent, well-controlled studies that are appropriately powered for statistical analysis. Currently, there is not enough experience with this medication in treating this specific condition, which is necessary to design successful randomized controlled trials (RCTs) for regulatory approval by the FDA. In dermatology, conducting studies in this manner is standard practice.
Study Type
Enrollment (Estimated)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
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Indiana
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Indianapolis, Indiana, United States, 46202
- Indiana University Department of Dermatology
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Ability to discern and provide written informed consent and voluntarily adhere to all of the protocol requirements
- Male or female subjects in their adult years (≥ 18 years of age), inclusive, from the time of providing written subject-investigator agreement.
- Confirmed clinical diagnosis of palmoplantar keratoderma.
Exclusion Criteria:
Treatment with any of the following medications and therapies during the duration of the study*:
- Topical corticosteroids
- Keratolytic agents (eg, 6% salicylic acid, 70% propylene glycol, 30% water)
- Topical retinoids
- Topical vitamin D ointment (calcipotriol)
Oral retinoids (acitretin)
- If on any of these then a washout period of at least 2 weeks will be enforced before the study begins. This washout period is meant to ensure that any effects or residues from previous treatments are minimized before starting the study.
- Prior exposure to Tapinarof treatment
- Known history or suspicion of hypersensitivity reactions, or contraindications to any or all components of the drug or drugs of similar properties.
- Currently participating in another clinical study for the same purpose.
- Potential or confirmed pregnancy: Individuals who are currently pregnant or have confirmed pregnancy at the time of screening will be excluded from participation in the study. This criterion aims to ensure the safety of participants and adhere to ethical considerations regarding the potential risks associated with the study interventions during pregnancy.
- Any other factors specifically not listed above but deemed patient unsuitable by the discretion of the investigators.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: Tapinarof Drug Treatment
Subjects in this arm will apply Tapinarof 1% cream topically once daily for a 16-week open-label treatment period.
Participants who achieve disease clearance after 16 weeks may discontinue treatment and will be monitored for sustained response up to 52 weeks.
Those who do not achieve clearance will continue the treatment during an extension period for up to 36 additional weeks or until clearance is achieved, whichever comes first.
Regular follow-up visits will assess the duration of disease clearance and monitor for any potential recurrence of keratoderma.
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Daily topical Tapinarof use will stop disease advancement and improve the quality of life in patients with palmoplantar keratoderma, through its AhR & NRF2 agonism properties.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Physician's Global Assessment (PGA)
Time Frame: Week 16 (Visit 4)
|
To evaluate the treatment benefit of Tapinarof applied topically to patients affected with palmoplantar keratoderma. Either an overall change in the PGA score to 0 (clear) or 1 (almost clear) or a decrease from baseline of at least 2 points. Higher scores indicate a worse outcome. Title: PHYSICIAN GLOBAL ASSESSMENT Minimum value: 0 Maximum value: 5 |
Week 16 (Visit 4)
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Treatment Emergent Adverse Event (TEAEs)
Time Frame: through study completion, 52 weeks
|
To assess the safety of Tapinarof applied topically to patients affected with palmoplantar keratoderma. Frequency and severity of treatment emergent adverse events (TAEs). |
through study completion, 52 weeks
|
|
Skin assessment every visit
Time Frame: through study completion, 52 weeks
|
To assess additional measures of efficacy of Tapinarof applied topically to patients affected with palmoplantar keratoderma, through qualitative analysis done by the investigator. Skin assessment of the affected areas, palms and/or soles, that are being managed with topical Tapinarof. |
through study completion, 52 weeks
|
|
Skin pictures at baseline to 52 weeks
Time Frame: through study completion, 52 weeks
|
To assess additional measures of efficacy of Tapinarof applied topically to patients affected with palmoplantar keratoderma, through qualitative analysis done by the investigator. To identify and note any visible differences due to the application of Tapinarof, throughout the treatment phase from baseline, 4 weeks, end of primary endpoint and lastly at the end of the monitoring phase at 52 weeks. |
through study completion, 52 weeks
|
|
Patient Global Assessment (PtGA)
Time Frame: through study completion, 52 weeks
|
To assess additional measures of efficacy of Tapinarof applied topically to patients affected with palmoplantar keratoderma, through qualitative analysis done by the patient. Note any patient reported outcomes and patient satisfaction of the treatment through a standardized Patient Global Assessment (PtGA) questionnaire. Higher scores indicate a worse outcome. Title: PATIENT GLOBAL ASSESSMENT Minimum value: 0 Maximum value: 5 |
through study completion, 52 weeks
|
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Dermatology Life Quality Index (DLQI)
Time Frame: through study completion, 52 weeks
|
To assess additional measures of efficacy of Tapinarof applied topically to patients affected with palmoplantar keratoderma, through qualitative analysis done by the patient. Done to monitor patient's observation and impact of Tapinarof use to their quality of life. Higher scores indicate a worse outcome. Title: DERMATOLOGY LIFE QUALITY INDEX Minimum value: 0 Maximum value: 30 |
through study completion, 52 weeks
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: David Rosmarin, MD, Dermatology
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 21197
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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