Ignite Creation Date:
2025-12-24 @ 9:47 PM
Ignite Modification Date:
2026-01-02 @ 3:24 AM
Study NCT ID:
NCT04047732
Status:
UNKNOWN
Last Update Posted:
2021-09-13
First Post:
2019-07-29
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
Sponsor:
Krystal Biotech, Inc.
Organization:
Study Overview
Official Title:
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
Status Verified Date:
2021-09
Last Known Status:
ACTIVE_NOT_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: