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RecruitingNCT07717346HALITE-1Updated Jul 21, 2026

A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease

An Early Phase 1 interventional study of KB111 in Hailey Hailey Disease, Skin Diseases and Lesion Skin, sponsored by Krystal Biotech, Inc.. Recruiting at 1 site in United States. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-07-21.

Sponsored by Krystal Biotech, Inc. · Early Phase 1, Interventional, and Treatment

From the registry’s dates

  • Started May 2026; still recruiting 6 months later.
Phase
Early Phase 1
Study type
Interventional
Enrollment
7
Allocation
Not applicable
Ages
12 Years and older
Sex
All
01

Study summary

An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease

Read the detailed description

This clinical study will investigate the safety, tolerability, and preliminary efficacy of open-label KB111 in pediatric and adult subjects with Hailey-Hailey Disease (HHD). Up to 7 subjects will be administered topical KB111 to treat HHD lesions for 12 weeks. KB111 will be administered in the home setting by a trained designee. The Investigator (or designee) will contact subjects weekly (or their parent/legal guardian) to assess for any adverse events or changes in medications, treatments, or procedures. Preliminary efficacy will be assessed by investigator assessments, patient-reported impressions of severity and molecular analysis of skin biopsies. A safety follow-up visit will occur 4 weeks after treatment stops.

02

Conditions studied

  • Hailey Hailey Disease
  • Skin Diseases
  • Lesion Skin

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03

In context

Skin Diseases

496 studies on the registry are indexed under Skin Diseases; 77 are open to participants now.

This study's planned enrollment of 7 is below the median of 70 across 375 interventional studies indexed under Skin Diseases.

Browse Skin Diseases studies →

Lead sponsor

Krystal Biotech, Inc. is the lead sponsor of 18 studies on the registry; 9 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • The participant must have read, understood, and signed an Institutional Review Board (IRB)- approved 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
  • 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 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

Exclusion Criteria:

  • Participation in another clinical study or treatment with an 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 requirements per protocol (for subjects of )
  • 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
Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
7 participants (estimated)

Study arms

  • Experimental
    KB111

    KB111

    Biological: KB111

Interventions

  • BiologicalKB111

    KB111 is a replication-defective, non-integrating herpes simplex virus (HSV-1)-based vector encoding functional, full-length human ATP2C1 protein combined with a gel for topical application.

06

What researchers measure

Primary outcomes

  1. To evaluate the safety and tolerability of KB111

    Number of subjects with treatment related adverse events as assessed by CTCAE v5

    Time frame: 12 weeks

Secondary outcomes

  1. To evaluate the effect of KB111 on patient-reported lesion severity throughout the treatment period.

    Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4)

    Time frame: 12 weeks

  2. To evaluate the effect of KB111 on patient-reported global impression of severity throughout the treatment period

    Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4)

    Time frame: 12 weeks

  3. To evaluate the effect of KB111 on Investigator-based assessments of lesion severity

    Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe

    Time frame: 12 weeks

  4. To evaluate the effect of KB111 on Investigator-based assessment of global severity

    Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe

    Time frame: 12 weeks

07

Study locations

1 of 1 sites recruiting
  • Pediatric Skin Research
    Miami, Florida 33156, United States
    Recruiting
    • Jean Marco Varanese · Contact+1 786-585-5774
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 21, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT07717346
Lead sponsor
Krystal Biotech, Inc.
Responsible party
Sponsor
First posted
Jul 21, 2026
Start date
May 6, 2026
Primary completion
Nov 2026 (estimated)
Completion
Dec 2026 (estimated)
Last update
Jul 21, 2026

Study contacts

Brittani Agostini
Contact
CNE@krystalbio.com
412-586-5830

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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