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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study Assessing KB407 for the Treatment of Cystic Fibrosis
NCTID
NCT05504837
(View at clinicaltrials.gov)
Description
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
(Show More)
Indication
Cystic Fibrosis
Compound Name
KB-407
Sponsor
Krystal Biotech, Inc.
Funder Type
Industry
Status
Recruiting
Enrollment Count
12
Therapy Information
Target Gene/Variant
CFTR
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Inhalational
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
HSV-1
Editor Type
none
Dose 1
10E9 PFU (single administration
Dose 2
10E9 PFU (2 administrations)
Dose 3
10E9 PFU (4 administrations)
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2022-08-15
Completion Date
2025-07
Last Update
2025-02-04
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
3
Locations
United States
Regulatory Information
Has US IND
True
Recent Updates
Interim data expected for cohort 3 mid-2025
Resources/Links
Clinical Publications
(Corporate Presentation) Genetic Medicines for High Unmet Medical Needs - May 2024
News and Press Releases
Krystal Biotech Announces Initial Clinical Update for Rare Respiratory Disease Programs KB408 and KB407 Including Early Clinical Evidence of Gene Delivery to the Lung of AATD Patients and Increase in Lung AAT to Therapeutic Levels
Preclinical Publications
(Poster) Evaluation of KB407, an HSV-1-Based Gene Therapy Vector for the Treatment of Cystic Fibrosis, in Healthy and Patient-Derived Airway Cells Including an Apical-Out Diseased Airway Organoid Model - ATS 2024
(Presentation) Respiratory cell-type affinity and absolute CFTR expression in the primate airway upon nebulization of KB407 - NACFC 2022
(Poster) In Vitro Pharmacology of KB407, an HSV-1-Based Gene Therapy Vector, for the Treatment of Cystic Fibrosis - ASGCT 2020