Hailey Hailey DiseaseEarly Phase 1HALITE-1NCT07717346
A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
A Early Phase 1 study testing KB111 in people with Hailey Hailey Disease and Skin Diseases, run by Krystal Biotech, Inc. at 1 site in United States.
Trial summary
An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hailey Hailey Disease, Skin Diseases, Lesion Skin
- What is being tested?
- KB111
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 6 May 2026to Dec 2026
- Phase
- Early Phase 1
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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
Pediatric Skin Research
Miami, Florida, 33156
Jean Marco Varanese, +1 786-585-5774
About this study
Official title: An Open-Label Study to Investigate Safety and Preliminary Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
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.
- Conditions
- Hailey Hailey Disease, Skin Diseases, Lesion Skin
- Being tested
- KB111
- Medical subject headings
- Pemphigus, Benign Familial, Skin Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB111Biological
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.
| Study group | Role | What they receive |
|---|---|---|
| KB111 | Experimental | KB111 Receives: Biological: KB111 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of KB111
Number of subjects with treatment related adverse events as assessed by CTCAE v5
Measured: 12 weeks
Secondary outcomes (4)
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)
Measured: 12 weeks
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)
Measured: 12 weeks
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
Measured: 12 weeks
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
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2026
- Study start6 May 2026
- Primary completionNov 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 Krystal Biotech, Inc.The 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07717346
- Sponsor's protocol ID
- KB111-A
- Acronym
- HALITE-1
- Status last verified
- Jul 2026
- First submitted
- 8 Jul 2026
- Last updated
- 21 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Investigate Safety and Preliminary Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
- ClinicalTrials.gov ID
- NCT07717346
- Acronym / sponsor ID
- HALITE-1 / KB111-A
- Conditions
- Hailey Hailey Disease, Skin Diseases, Lesion Skin
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 7 participants (estimated)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 May 2026 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 21 Jul 2026 (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.
An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KB111 | Experimental | Biological: KB111KB111 |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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
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 |
|---|---|---|
| KB111KB111 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. | Biological | KB111 |
Primary outcome
To evaluate the safety and tolerability of KB111
Time frame: 12 weeks
Number of subjects with treatment related adverse events as assessed by CTCAE v5
4 secondary outcomes
To evaluate the effect of KB111 on patient-reported lesion severity throughout the treatment period.
Time frame: 12 weeks
To evaluate the effect of KB111 on patient-reported global impression of severity throughout the treatment period
Time frame: 12 weeks
To evaluate the effect of KB111 on Investigator-based assessments of lesion severity
Time frame: 12 weeks
To evaluate the effect of KB111 on Investigator-based assessment of global severity
Time frame: 12 weeks
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 21 Jul 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing KB111 in people with Hailey Hailey Disease and Skin Diseases, run by Krystal Biotech, Inc. 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 6 inclusion and 5 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 21 Jul 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Brittani Agostini (412-586-5830, CNE@krystalbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of KB111 (time frame: 12 weeks). 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 21 Jul 2026.