Autosomal Recessive IchthyosisPhase 2NCT05735158
Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
A Phase 2 study testing KB105 in people with Autosomal Recessive Ichthyosis, run by Krystal Biotech, Inc. at 1 site in United States.
Trial summary
KB105-02 is an intrasubject randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of KB105 in children and adults with autosomal recessive congenital ichthyosis (ARCI). Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Autosomal Recessive Ichthyosis
- What is being tested?
- KB105
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2023to Apr 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject, or legally authorized representative, must be willing and able to give informed consent/assent
- Aged ≥6 months
- A genetically confirmed diagnosis of TGM1-deficient ARCI
- Clinical diagnosis of lamellar ichthyosis
- Two target areas located on similar anatomical regions with the same scaling severity by IGA and scaling severity ≥3
- Subjects taking systemic retinoids must be on a stable regimen for at least 4 weeks and agree to continue to take the medication consistently throughout the study. If the subject prefers to withhold systemic retinoids during the study, then a minimum 4-week washout period is required
- Clinically stable and in good general health
Who cannot join
- Subject has a physical condition or other dermatological disorder (e.g., atopic, seborrheic, or contact dermatitis, psoriasis, tinea infections, etc.) which, in the Investigator's opinion, might impair evaluation of the target areas or which exposes the subject to unacceptable risk by study participation
- Participation in another clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer
- Any condition or illness (including a history or current evidence of substance abuse or dependence) that, in the opinion of the Investigator, would impact a subject's ability to complete all study-related procedures and/or poses an additional risk to the assessment of safety of KB105
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Mission Dermatology
Rancho Santa Margarita, California, 92688
About this study
Official title: A Phase 2, Randomized, Placebo-controlled Study of Topical KB105, a Replication-defective, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
KB105-02 is an intrasubject randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of KB105 in children and adults with autosomal recessive congenital ichthyosis (ARCI).
- Conditions
- Autosomal Recessive Ichthyosis
- Being tested
- KB105, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB105Biological
Replication-defective, non-integrating HSV-1 vector expressing TGM1 formulated as a topical gel
PlaceboOther
Visually matched excipient gel
| Study group | Role | What they receive |
|---|---|---|
| KB105 | Experimental | Weekly topical application Receives: Biological: KB105 |
| Placebo | Placebo comparator | Weekly topical application Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite Investigator's Global Assessment (IGA) Responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
Responder target areas are defined as:
Target areas with ≥2 point reduction from baseline on IGA scaling severity at Week 9 or Target areas with ≥1 point reduction from baseline on IGA scaling severity for 2 consecutive weeks (Week 8 and Week 9)
Measured: Week 9
Secondary outcomes (7)
Composite regional Visual Index of Ichthyosis Severity (VIIS) responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
Responder target areas are defined as:
Target areas with ≥2 point reduction from baseline on VIIS scaling score at Week 9 or Target areas with ≥1 point reduction from baseline on VIIS scaling score for 2 consecutive weeks (Week 8 and Week 9)
Measured: Week 9
Visual Index of Ichthyosis Severity score change from baseline
Change from baseline in regional Visual Index of Ichthyosis Severity (VIIS) scaling scores, where 0 is normal and 4 is severe
Measured: Up to 11 weeks
Investigator's Global Assessment change from baseline
Change from baseline in Investigator's Global Assessment (IGA) scaling severity scores, where 0 is clear and 4 is severe
Measured: Up to 11 weeks
Investigator's Global Assessment (IGA) 2 point responder
Comparison of proportion of KB105 and placebo target areas with ≥2 point reduction from baseline on IGA scaling severity at Week 9
Measured: Week 9
Investigator's Global Assessment (IGA) 1 point responder
Comparison of proportion of KB105 and placebo target areas with ≥1 point reduction from baseline on IGA scaling severity for 2 consecutive weeks (Week 8 and Week 9)
Measured: Week 9
Visual Index of Ichthyosis Severity (VIIS) 2 point responder
Comparison of proportion of KB105 and placebo target areas with ≥2 point reduction from baseline on regional VIIS scaling severity at Week 9
Measured: Week 9
Visual Index of Ichthyosis Severity (VIIS) 2 point responder
Comparison of proportion of KB105 and placebo target areas with ≥1 point reduction from baseline on regional VIIS scaling severity for 2 consecutive weeks (Week 8 and Week 9)
Measured: Week 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2023
- Study startMar 2023 (estimated)
- Primary completionMar 2024 (estimated)
- Study completionApr 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Recessive IchthyosisStudies of the same condition, open and under way.
- More trials run by Krystal Biotech, Inc.The sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- An intra-participant parallel study. Matched target areas will be randomized within the participant, such that one target area receives KB105 and the other target area will receive placebo. An additional target area may be selected to receive open-label treatment with KB105.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05735158
- Sponsor's protocol ID
- KB105-02
- Status last verified
- Feb 2023
- First submitted
- 9 Feb 2023
- Last updated
- 23 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Placebo-controlled Study of Topical KB105, a Replication-defective, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
- ClinicalTrials.gov ID
- NCT05735158
- Acronym / sponsor ID
- KB105-02
- Conditions
- Autosomal Recessive Ichthyosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2023 (estimated)
- Primary completion
- Mar 2024 (estimated)
- Study completion
- Apr 2024 (estimated)
- First posted
- 21 Feb 2023 (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.
KB105-02 is an intrasubject randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of KB105 in children and adults with autosomal recessive congenital ichthyosis (ARCI).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- An intra-participant parallel study. Matched target areas will be randomized within the participant, such that one target area receives KB105 and the other target area will receive placebo. An additional target area may be selected to receive open-label treatment with KB105.
| Arm | Type | Receives |
|---|---|---|
| KB105 | Experimental | Biological: KB105Weekly topical application |
| Placebo | Placebo comparator | Other: PlaceboWeekly topical application |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Subject, or legally authorized representative, must be willing and able to give informed consent/assent
- Aged ≥6 months
- A genetically confirmed diagnosis of TGM1-deficient ARCI
- Clinical diagnosis of lamellar ichthyosis
- Two target areas located on similar anatomical regions with the same scaling severity by IGA and scaling severity ≥3
Key exclusion criteria (6 listed)
- Subject has a physical condition or other dermatological disorder (e.g., atopic, seborrheic, or contact dermatitis, psoriasis, tinea infections, etc.) which, in the Investigator's opinion, might impair evaluation of the target areas or which exposes the subject to unacceptable risk by study participation
- Participation in another clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer
- Any condition or illness (including a history or current evidence of substance abuse or dependence) that, in the opinion of the Investigator, would impact a subject's ability to complete all study-related procedures and/or poses an additional risk to the assessment of safety of KB105
- Women who are pregnant or nursing
- Subject who is unwilling to comply with contraception requirements per protocol
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 |
|---|---|---|
| KB105Replication-defective, non-integrating HSV-1 vector expressing TGM1 formulated as a topical gel | Biological | KB105 |
| PlaceboVisually matched excipient gel | Other | Placebo |
Primary outcome
Composite Investigator's Global Assessment (IGA) Responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
Time frame: Week 9
Responder target areas are defined as: Target areas with ≥2 point reduction from baseline on IGA scaling severity at Week 9 or Target areas with ≥1 point reduction from baseline on IGA scaling severity for 2 consecutive weeks (Week 8 and Week 9)
7 secondary outcomes
Composite regional Visual Index of Ichthyosis Severity (VIIS) responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
Time frame: Week 9
Visual Index of Ichthyosis Severity score change from baseline
Time frame: Up to 11 weeks
Investigator's Global Assessment change from baseline
Time frame: Up to 11 weeks
Investigator's Global Assessment (IGA) 2 point responder
Time frame: Week 9
Investigator's Global Assessment (IGA) 1 point responder
Time frame: Week 9
Visual Index of Ichthyosis Severity (VIIS) 2 point responder
Time frame: Week 9
Visual Index of Ichthyosis Severity (VIIS) 2 point responder
Time frame: Week 9
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2023.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing KB105 in people with Autosomal Recessive Ichthyosis, run by Krystal Biotech, Inc. at 1 site 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 7 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 23 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Brittani Agostini, RN, CCRC (412-586-5830, bagostini@krystalbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Composite Investigator's Global Assessment (IGA) Responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas) (time frame: Week 9). 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 23 Feb 2023.