NCT04047732

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

A Phase I/II Clinical Trial of Topical KB105, a Replication-incompetent, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Krystal Biotech, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. The primary objectives of this study are to evaluate safety and Investigator Global Assessment (IGA) scale improvement of topically administered KB105.

Detailed description

Up to six adult subjects are planned for the Phase I portion of this study. Subjects are enrolled upon obtaining consent and meeting entry criteria. This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. Patients will be evaluated for safety, and Target Areas will be assessed individually with the IGA and VIIS scales. Target Areas will be imaged and evaluated for safety and efficacy. Subjects will be on-trial for approximately 3.5 months.

Conditions

• TGM-1 Related Autosomal Recessive Congenital Ichthyosis

Interventions

Type	Name	Description
BIOLOGICAL	KB105	KB105, a replication-incompetent, non-integrating HSV-1 vector expressing human transglutaminase 1 (TGM1) formulated as a topical gel

Timeline

• Start date: 2019-08-27
• Primary completion: 2022-10-01
• Completion: 2025-03-01
• First posted: 2019-08-07
• Last updated: 2021-09-13

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study