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

NCT05735158 | Unknown Phase 2 FDA Drug

• 1 other identifier: KB105-02
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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).

Conditions

Autosomal Recessive Ichthyosis

Outcome Measures

Primary Outcomes (1)

• 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)

  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)

  Week 9

• Visual Index of Ichthyosis Severity score change from baseline

  Up to 11 weeks

• Investigator's Global Assessment change from baseline

  Up to 11 weeks

• Investigator's Global Assessment (IGA) 2 point responder

  Week 9

• Investigator's Global Assessment (IGA) 1 point responder

  Week 9

Study Arms (2)

KB105

EXPERIMENTAL

Weekly topical application

Biological: KB105

Placebo

PLACEBO COMPARATOR

Weekly topical application

Other: Placebo

Interventions

KB105 BIOLOGICAL

Replication-defective, non-integrating HSV-1 vector expressing TGM1 formulated as a topical gel

KB105

Placebo OTHER

Visually matched excipient gel

Placebo

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Krystal Biotech, Inc.: lead

Study Sites (1)

Mission Dermatology

Rancho Santa Margarita, California, 92688, United States

Location

Central Study Contacts

Brittani Agostini, RN, CCRC

CONTACT

412-586-5830; bagostini@krystalbio.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: February 9, 2023
• First Posted: February 21, 2023
• Study Start: March 1, 2023
• Primary Completion: March 1, 2024
• Study Completion: April 1, 2024
• Last Updated: February 23, 2023

Record last verified: 2023-02

Locations

US (1)