NCT05735158尚未招募2 期
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)
适应症
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Composite Investigator's Global Assessment (IGA) Responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Composite Investigator's Global Assessment (IGA) Responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)
时间窗: 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)
次要结局
- Composite regional Visual Index of Ichthyosis Severity (VIIS) responder (Comparison of proportion of responder KB105 target areas to responder placebo target areas)(Week 9)
- Investigator's Global Assessment change from baseline(Up to 11 weeks)
- Investigator's Global Assessment (IGA) 1 point responder(Week 9)
- Visual Index of Ichthyosis Severity (VIIS) 2 point responder(Week 9)
- Investigator's Global Assessment (IGA) 2 point responder(Week 9)
- Visual Index of Ichthyosis Severity score change from baseline(Up to 11 weeks)
研究者
研究点 (2)
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