An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease
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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
7
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.
Pediatric Skin Research
Miami, Florida, United States
RECRUITINGTo evaluate the safety and tolerability of KB111
Number of subjects with treatment related adverse events as assessed by CTCAE v5
Time frame: 12 weeks
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
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
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
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
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