A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: KB111.
- Who it may be relevant to
- Registry conditions: Hailey Hailey Disease, Skin Diseases, Lesion Skin. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-Label Study to Investigate Safety and Preliminary Efficacy of KB111 for the Treatment of Hailey-Hailey Disease
Overview
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
- Biological KB111
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.
Primary outcome measures
- To evaluate the safety and tolerability of KB111 [Time frame: 12 weeks]
Secondary outcome measures (4)
- 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]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Pediatric Skin Research — Miami
Identifiers
NCT: NCT07717346 · KB111-A