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

NCT07717346 | Recruiting Early Phase 1 FDA Drug

• 1 other identifier: KB111-A
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Hailey Hailey Disease; Skin Diseases; Lesion Skin

Outcome Measures

Primary Outcomes (1)

• To evaluate the safety and tolerability of KB111

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

  12 weeks

Secondary Outcomes (4)

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

  12 weeks

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

  12 weeks

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

  12 weeks

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

  12 weeks

Study Arms (1)

KB111

EXPERIMENTAL

KB111

Biological: KB111

Interventions

KB111 BIOLOGICAL

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.

KB111

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Krystal Biotech, Inc.: lead

Study Sites (1)

Pediatric Skin Research

Miami, Florida, 33156, United States

RECRUITING

MeSH Terms

Conditions

Pemphigus, Benign Familial; Skin Diseases

Condition Hierarchy (Ancestors)

Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Central Study Contacts

Brittani Agostini

CONTACT

412-586-5830; CNE@krystalbio.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2026
• First Posted: July 21, 2026
• Study Start: May 6, 2026
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: July 21, 2026

Record last verified: 2026-07

Locations

US (1)