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

NCT04540900 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: PEARL-1
• Study Type: interventional
• Target: 85
• Locations: 1 country
• Sites: 4

Brief 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

Conditions

Skin Roughness; Wrinkle; Fine Lines; Skin Thickness

Keywords

KB301; Wrinkles; Krystal Biotech; Décolletage; Lateral Canthal Lines; Décolleté; Pigmentation

Outcome Measures

Primary Outcomes (1)

• Assessment of the safety profile of KB301 by recording adverse events using MedDRA System Organ Class and Preferred Term and collection of other clinical study procedures.

  The primary outcome will look at the safety and tolerability of KB301 through the collection and quantification of adverse events, physical examinations, vital signs and clinical laboratory tests.

  4 months

Secondary Outcomes (10)

• Evaluation of COL3A1 transgene expression 2-days post-dose.

  12 months

• Assessment of skin roughness as compared to baseline, using the Skin Roughness Scale (SRS).

  65 Days

• Assessment of fine line improvement as compared to baseline, using the Fine Lines Scale (FLS).

  65 Days

• Subject assessment of aesthetic improvement over baseline, using the Subject Satisfaction Scale (SSS).

  65 Days

• Assessment of skin fold thickness improvement over baseline, using a Caliper.

  65 Days

Study Arms (2)

KB301

ACTIVE COMPARATOR

non-integrating HSV-1 vector expressing human type III collagen

Biological: KB301

Placebo

PLACEBO COMPARATOR

sterile isotonic saline

Other: Placebo

Interventions

KB301 BIOLOGICAL

non-integrating HSV-1 vector expressing human type III collagen

KB301

Placebo OTHER

sterile isotonic saline

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female in good general health, as determined by the investigator, aged ≥18 and ≤75 at the time of consent (only females for Cohort 4)
• Regions of suitable skin to be selected as Target Area(s), as determined by a physical examination conducted by the investigator during the initial visit:
• Cohort 1 - region of healthy buttock skin
• Cohort 1a - bilaterally symmetric regions of healthy buttock skin at least 6 cm apart
• Cohort 2 - bilaterally symmetric regions of moderate to severe fine lines and skin roughness (score of 2 or 3), as determined by investigator using the 5-point FLS and SRS on the cheeks above and below the zygomatic arch
• Cohort 2 Extension- A subject who received a high dose regimen of KB301 to Target Areas below the zygomatic arch
• Treatment group- Unilateral region of the cheek below the zygomatic arch, which was randomized to received placebo under Cohort 2, that per investigator assessment and consultation with the sponsor at Visit 1, is determined to be uneven as compared to the Target Area which received KB301
• Observational group - Bilateral region of the cheeks below the zygomatic arch which were randomized to receive high dose 301 under Cohort 2 that show durability of KB301 to at least one of the two Target Areas
• Cohort 3- Lateral canthal lines of mild to moderate severity at rest, per the Investigator's clinical assessment
• Cohort 4- Dynamic décolleté wrinkles and /or pigmentation
• A Fitzpatrick skin phototype score of I-IV
• A negative pregnancy test at the Study Day 0 Visit for subjects of child-bearing potential and
• Signed and dated informed consent and willingness to attend all study visits and complete all procedures required by this protocol.

You may not qualify if:

• Any transient or chronic skin condition, disorder, or infection within 20 cm of the Target Areas at Day 0 that, in the opinion of the investigator, may confound study results
• History of laser treatment or chemical peels to the Target Areas within six (6) months of the Study Day 0 Visit
• History of surgical procedures to Target Areas, including removal of benign or malignant skin cancers that, in the opinion of the investigator, may confound study results
• Administration of a cosmetic and/or clinical research investigational agent, at the location of the Target Areas within 6 months of the Study Day 0 Visit, that, in the opinion of the investigator, may confound study results
• Scars, tattoos or tanned skin (defined as skin that is tanned from sun exposure or an applied skin color such as a spray tan) within the Target Area
• Any condition (including a history or current evidence of substance abuse or dependence) that, in the investigator's opinion, would impact subject's ability to complete all study-related procedures and/or poses an additional risk to the assessment of safety of the Investigational Product
• Women who are pregnant or nursing
• Subject who is unwilling to comply with contraception requirements per-protocol
• Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the investigator.
• Previously treated with KB301 under Cohorts 2, 2-extension, 3a, or 3b (Cohort 3c only)

Sponsors & Collaborators

• Krystal Biotech, Inc.: lead

Study Sites (4)

Aesthetx

Campbell, California, 95008, United States

Location

Steve Yoelin, MD Medical Associates, Inc.

Newport Beach, California, 92663, United States

Location

Mission Dermatology

Rancho Santa Margarita, California, 92688, United States

Location

Skin and Cancer Associates, LLP. D/B/A Center for Clinical and Cosmetic Research

Aventura, Florida, 33180, United States

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 19, 2020
• First Posted: September 7, 2020
• Study Start: October 7, 2020
• Primary Completion: October 16, 2024
• Study Completion (Estimated): May 31, 2029
• Last Updated: May 13, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)