Hailey Hailey Disease, Lesion Skin, Skin Diseases
Conditions
Brief summary
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.
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.
Sponsors
Krystal Biotech, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
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 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the safety and tolerability of KB111 | 12 weeks | Number of subjects with treatment related adverse events as assessed by CTCAE v5 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the effect of KB111 on patient-reported lesion severity throughout the treatment period. | 12 weeks | Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4) |
| To evaluate the effect of KB111 on patient-reported global impression of severity throughout the treatment period | 12 weeks | Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4) |
| To evaluate the effect of KB111 on Investigator-based assessments of lesion severity | 12 weeks | Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe |
| To evaluate the effect of KB111 on Investigator-based assessment of global severity | 12 weeks | Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe |
Countries
United States
Contacts
CONTACTBrittani Agostini
Outcome results
None listed