An Open-Label Study to Investigate Safety and Preliminary Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
Krystal Biotech, Inc.
Summary
An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease
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 impre…
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
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 treat…
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.
Location
- Pediatric Skin ResearchMiami, Florida