NCT04540900

A Phase I Study of KB301, a Replication-Incompetent, Non-Integrating Vector Expressing Human Type III Collagen (COL3) for the Treatment of Superficial Skin Depressions

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 85 (actual)
• Sponsor: Krystal Biotech, Inc. · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Accepted

Summary

A Phase I Study of KB301, a Replication-Incompetent, Non-Integrating Vector Expressing Human Type III Collagen (COL3) for the Treatment of Superficial Skin Depressions

Detailed description

Subjects enrolled in Cohort 1 will have a region of healthy buttock skin as a non-facial target area to receive two (2) injectable doses of KB301 (low or high). Cohort 1a will also present two regions of healthy buttock skin as a non-facial target area to receive two (2) injectable doses of KB301 (low or high) as well as a placebo injection. The objective of this cohort is to evaluate COL3A1 transgene expression (as measured by qRT-PCR of skin biopsies) and determine the optimal dose. Subjects enrolled in Cohort 2 will present with bilaterally symmetrical regions of moderate to severe fine lines and skin roughness as the target areas; these areas will be randomly assigned to receive either KB301 or a placebo to determine the extent of skin improvement in the KB301 arm. Throughout the study subjects will complete questionnaires, have images captured of the target areas, undergo physical examinations, and have safety labs monitored. Cohort 2 subjects may enroll in Cohort 2 extension, if they have not reverted back to baseline on the KB301 treated target area of the cheek. If subjects received KB301 to both cheek areas they will not be eligible for additional treatment and will be followed for durability. Subjects who received KB301 treatment to one cheek area, who have not reverted to baseline on the KB301 treated target area will be treated with KB301 on the cheek which previously received placebo. Subjects will be followed for durability, captured through investigator assessments, and questionnaires, while continuing to monitor safety. Cohort 3 will be open label. Subjects will present with mild to moderate lateral canthal lines. The cohort will be split into two (3) subsets, Cohort 3a, Cohort 3b and Cohort 3c. Subjects in Cohort 3a and Cohort 3c will receive microinjections of KB301 and subjects in Cohort 3b will receive microneedling prior to topical application of KB301. Throughout the study, subjects will complete questionnaires, have images captured of the target areas, undergo physical examinations, and have assessment of improvement completed by the investigator. At Visit 5 the subject may begin retreatment with KB301 once weekly for three weeks and then will be followed for an additional 3 months, at the discretion of the investigator. Cohort 4 will be open label. Subjects will present with dynamic décolleté wrinkles and/or pigmentation. Subjects in Cohort 4 will receive microinjections of KB301 to the décolleté area. Throughout the study, subjects will complete questionnaires, have images captured of the target areas, undergo physical examinations, and have assessment of improvement completed by the investigator. At Visit 5 the subject may begin retreatment with KB301 once weekly for three weeks and then will be followed for an additional 3 months, at the discretion of the investigator.

Conditions

• Skin Roughness
• Wrinkle
• Fine Lines
• Skin Thickness

Interventions

Type	Name	Description
BIOLOGICAL	KB301	non-integrating HSV-1 vector expressing human type III collagen
OTHER	Placebo	sterile isotonic saline

Timeline

• Start date: 2020-10-07
• Primary completion: 2024-10-16
• Completion: 2029-05-31
• First posted: 2020-09-07
• Last updated: 2025-05-13

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study