Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

Phase 2

Not yet recruiting

• Conditions: Autosomal Recessive Ichthyosis
• Interventions: Other: Placebo; Biological: KB105

• Registration Number: NCT05735158
• Lead Sponsor: Krystal Biotech, Inc.

Brief 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).

Detailed Description

Not available

Study Timeline

• Status Verified: 2023-02
• Study First Submitted: 2023-02-09
• Study First Submitted QC: 2023-02-09
• Last Update Submitted: 2023-02-20
• Study First Posted: 2023-02-21
• Last Update Posted: 2023-02-23
• Study Start: 2023-03
• Primary Completion: 2024-03
• Study Completion: 2024-04

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Subject, or legally authorized representative, must be willing and able to give informed consent/assent
2. Aged ≥6 months
3. A genetically confirmed diagnosis of TGM1-deficient ARCI
4. Clinical diagnosis of lamellar ichthyosis
5. Two target areas located on similar anatomical regions with the same scaling severity by IGA and scaling severity ≥3
6. 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
7. Clinically stable and in good general health

Key

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Exclusion Criteria

1. 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
2. Participation in another clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer
3. 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
4. Women who are pregnant or nursing
5. Subject who is unwilling to comply with contraception requirements per protocol
6. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Weekly topical application
KB105	KB105	Weekly topical application

Primary Outcome Measures

Name	Time	Method
Composite Investigator's Global Assessment (IGA) Responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)	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)

Secondary Outcome Measures

Name	Time	Method
Composite regional Visual Index of Ichthyosis Severity (VIIS) responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)	Week 9	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)
Investigator's Global Assessment change from baseline	Up to 11 weeks	Change from baseline in Investigator's Global Assessment (IGA) scaling severity scores, where 0 is clear and 4 is severe
Investigator's Global Assessment (IGA) 1 point responder	Week 9	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)
Investigator's Global Assessment (IGA) 2 point responder	Week 9	Comparison of proportion of KB105 and placebo target areas with ≥2 point reduction from baseline on IGA scaling severity at Week 9
Visual Index of Ichthyosis Severity (VIIS) 2 point responder	Week 9	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)
Visual Index of Ichthyosis Severity score change from baseline	Up to 11 weeks	Change from baseline in regional Visual Index of Ichthyosis Severity (VIIS) scaling scores, where 0 is normal and 4 is severe

Trial Locations

Locations (1)

Mission Dermatology; 🇺🇸 Rancho Santa Margarita, California, United States