Clinical trial NCT05735158: Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
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)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Autosomal Recessive Ichthyosis
Lead sponsor: Krystal Biotech, Inc.
Sponsor class: INDUSTRY
First posted: 2023-02-21
Enrollment: 15
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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.
Primary purpose: TREATMENT

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).


Eligibility criteria: Key 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 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

Primary outcomes:
measure: Composite Investigator's Global Assessment (IGA) Responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
timeFrame: Week 9
Secondary outcomes:
measure: Composite regional Visual Index of Ichthyosis Severity (VIIS) responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
timeFrame: Week 9
measure: Visual Index of Ichthyosis Severity score change from baseline
timeFrame: Up to 11 weeks
measure: Investigator's Global Assessment change from baseline
timeFrame: Up to 11 weeks
measure: Investigator's Global Assessment (IGA) 2 point responder
timeFrame: Week 9
measure: Investigator's Global Assessment (IGA) 1 point responder
timeFrame: Week 9
measure: Visual Index of Ichthyosis Severity (VIIS) 2 point responder
timeFrame: Week 9
measure: Visual Index of Ichthyosis Severity (VIIS) 2 point responder
timeFrame: Week 9
Other outcomes:
Locations:
city: Rancho Santa Margarita
country: United States
facility: Mission Dermatology
state: California
Study officials:
References: