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Gene therapy gel targets rare blistering skin disease

NCT ID NCT07717346

Disease control Sponsor: Krystal Biotech, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 21, 2026 · Last updated Jul 22, 2026 · Updated 1 time

Summary

This early study tests a new gene therapy gel called KB111 for Hailey-Hailey disease, a rare genetic condition that causes painful blisters and skin lesions. The gel uses a modified virus to deliver a working copy of the faulty gene directly to the skin. The trial enrolls about 7 people aged 12 and older to see if the treatment is safe and whether it can reduce symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
a topical gel containing a modified herpes simplex virus that delivers a working copy of the ATP2C1 gene
What this could lead to
If successful, this could point toward a treatment that reduces painful blisters and improves quality of life for people with Hailey-Hailey disease.
What could go wrong
This is a very early, small study (7 people) focused mainly on safety. The treatment may not work or could cause side effects, and larger trials are needed.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 7 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * The participant must have read, understood, and signed an Institutional Review Board (IRB)- approved Informed Consent Form and must be willing and able to comply with study procedures and instructions * Aged ≥ 12 years of age at the time of informed consent/assent * Documented clinical diagnosis of HHD with confirmed ATP2C1 variant by genetic testing * A negative pregnancy test prior to initial dose for subjects of child-bearing potential * If taking systemic therapies, subject must be on a stable dosing regimen 1 month prior to Screening and throughout the 12-week treatment period * An acceptable form of contraception must be taken from the first dose (Week 1) through 3 months after the last dose of IP for subjects of childbearing potential Exclusion Criteria: * Participation in another clinical study or treatment with an investigational agent 30 days or 5 half-lives, whichever is longer, prior to initial dosing in Week 1 * Participant who is pregnant or nursing * Participant who is unwilling to comply with contraception requirements per protocol (for subjects of child-bearing potential) * Participant who is known to be non-compliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator * Presence of any dermatologic or systemic condition that might hinder the efficacy of the study treatment or its evaluation, could possibly interfere with the interpretation of study results, or could be judged by the Investigator to be incompatible with the study visit schedule or conduct

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Conditions

The condition(s) this trial relates to.

Hailey-Hailey disease skin disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hailey hailey disease Lesion skin Skin diseases

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Pediatric Skin Research

    RECRUITING

    Miami, Florida, 33156, United States

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