Skin RoughnessPhase 1NCT04540900
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
A Phase 1 study testing KB301 in people with Skin Roughness and Wrinkle, run by Krystal Biotech, Inc. at 4 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Skin Roughness, Wrinkle, Fine Lines
- What is being tested?
- KB301
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 85 participants
- Where
- 4 sitesUnited States
- Study dates
- 7 Oct 2020to 31 May 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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
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- 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)
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Skin and Cancer Associates, LLP. D/B/A Center for Clinical and Cosmetic Research
Aventura, Florida, 33180
Aesthetx
Campbell, California, 95008
Steve Yoelin, MD Medical Associates, Inc.
Newport Beach, California, 92663
Mission Dermatology
Rancho Santa Margarita, California, 92688
About this study
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
Read the 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
- Being tested
- KB301, Placebo
- Keywords
- KB301, Wrinkles, Krystal Biotech, Décolletage, Lateral Canthal Lines, Décolleté, Pigmentation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB301Biological
non-integrating HSV-1 vector expressing human type III collagen
PlaceboOther
sterile isotonic saline
| Study group | Role | What they receive |
|---|---|---|
| KB301 | Active comparator | non-integrating HSV-1 vector expressing human type III collagen Receives: Biological: KB301 |
| Placebo | Placebo comparator | sterile isotonic saline Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 4 months
Secondary outcomes (10)
Evaluation of COL3A1 transgene expression 2-days post-dose.
The secondary outcome measure in Cohorts 1 and 1a will compare a KB301 injection site biopsy, to an area of untreated healthy buttock skin biopsy (at least 6 cm away from the sites of KB301 injection), via qRT-PCR analysis.
Measured: 12 months
Assessment of skin roughness as compared to baseline, using the Skin Roughness Scale (SRS).
The secondary outcome measure in Cohort 2 will look at the mean change in the assessment of skin roughness as compared to baseline, using the Skin Roughness Scale (SRS). The SRS ranges from no fine lines (0) to extreme fine lines (4). A third-party independent blinded evaluator will perform the evaluations.
Measured: 65 Days
Assessment of fine line improvement as compared to baseline, using the Fine Lines Scale (FLS).
The secondary outcome measure in Cohort 2, will look at the mean change in the assessment of fine lines as compared to baseline, using the Fine Lines Scale (FLS). The FLS ranges from no texture (0) to diffuse texture (4). A third-party independent blinded evaluator will perform the evaluations.
Measured: 65 Days
Subject assessment of aesthetic improvement over baseline, using the Subject Satisfaction Scale (SSS).
The secondary outcome measure in Cohort 2, will look at the mean change in Subject's Satisfaction with the treatment per target area, as compared to baseline, using the Subject Satisfaction Scale (SSS), which is scored on a scale from 0-4, with 0 indicating not satisfied and 4 indicating excellent.
Measured: 65 Days
Assessment of skin fold thickness improvement over baseline, using a Caliper.
The secondary outcome measure in Cohort 2, will look at the mean change in skinfold thickness of the non-facial target areas above the knee, using a Caliper to measure, as compared to baseline.
Measured: 65 Days
Subject assessment of aesthetic improvement over baseline, using the Subject Satisfaction Scale (SSS).
The secondary outcome measure in Cohort 2 Extension, will look at the mean change in Subject's Satisfaction with the treatment per target area, as compared to baseline, using the Subject Satisfaction Scale (SSS), which is scored on a scale from 0-4, with 0 indicating not satisfied and 4 indicating excellent.
Measured: 3 Months
Investigator assessment of durability of high dose KB301, as compared to baseline.
The secondary outcome measure in Cohort 2 Extension, will look at the durability of high dose KB301, as compared to baseline through an Investigator assessment.
Measured: 3 Months
Investigator assessment of global aesthetic improvement, as compared to baseline.
The secondary outcome measure in Cohort 3 and Cohort 4 will look at the mean change in global aesthetic improvement, which is scored on a 5-point scale, ranging from much improved (2) to much worse (-2).
Measured: 4 Months
Subject assessment of global aesthetic improvement, as compared to baseline.
The secondary outcome measure in Cohort 3 and Cohort 4 will look at the mean change in global aesthetic improvement, which is scored on a 5-point scale, ranging from much improved (2) to much worse (-2).
Measured: 4 Months
Subject treatment satisfaction as compared to baseline, using the Subject Satisfaction Questionnaire (SSQ).
The secondary outcome measure in Cohort 3 and Cohort 4 will look at mean change in subject treatment satisfaction over baseline, using a 5-point Subject Satisfaction Questionnaire, ranging from very satisfied (2) to very dissatisfied (-2).
Measured: 4 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2020
- Study start7 Oct 2020
- Primary completion16 Oct 2024
- Study completion31 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Skin RoughnessStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 85 participants (actual)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04540900
- Sponsor's protocol ID
- PEARL-1
- Status last verified
- May 2025
- First submitted
- 19 Aug 2020
- Last updated
- 13 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04540900
- Acronym / sponsor ID
- PEARL-1
- Conditions
- Skin Roughness, Wrinkle, Fine Lines, Skin Thickness
- Keywords
- KB301, Wrinkles, Krystal Biotech, Décolletage, Lateral Canthal Lines, Décolleté, Pigmentation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 85 participants (actual)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Oct 2020 (actual)
- Primary completion
- 16 Oct 2024 (actual)
- Study completion
- 31 May 2029 (estimated)
- First posted
- 7 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| KB301 | Active comparator | Biological: KB301non-integrating HSV-1 vector expressing human type III collagen |
| Placebo | Placebo comparator | Other: Placebosterile isotonic saline |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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:
- 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.
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KB301non-integrating HSV-1 vector expressing human type III collagen | Biological | KB301 |
| Placebosterile isotonic saline | Other | Placebo |
Primary outcome
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.
Time frame: 4 months
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.
10 secondary outcomes
Evaluation of COL3A1 transgene expression 2-days post-dose.
Time frame: 12 months
Assessment of skin roughness as compared to baseline, using the Skin Roughness Scale (SRS).
Time frame: 65 Days
Assessment of fine line improvement as compared to baseline, using the Fine Lines Scale (FLS).
Time frame: 65 Days
Subject assessment of aesthetic improvement over baseline, using the Subject Satisfaction Scale (SSS).
Time frame: 65 Days
Assessment of skin fold thickness improvement over baseline, using a Caliper.
Time frame: 65 Days
Subject assessment of aesthetic improvement over baseline, using the Subject Satisfaction Scale (SSS).
Time frame: 3 Months
Investigator assessment of durability of high dose KB301, as compared to baseline.
Time frame: 3 Months
Investigator assessment of global aesthetic improvement, as compared to baseline.
Time frame: 4 Months
Subject assessment of global aesthetic improvement, as compared to baseline.
Time frame: 4 Months
Subject treatment satisfaction as compared to baseline, using the Subject Satisfaction Questionnaire (SSQ).
Time frame: 4 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2025.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing KB301 in people with Skin Roughness and Wrinkle, run by Krystal Biotech, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 13 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 4 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2025; AzTrials last refreshed its copy on 31 Oct 2026.