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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
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
Dose 1 Undisclosed dose (single administration)
Dose 2 Undisclosed dose (2 administrations)
Dose 3 Undisclosed dose (4 administrations)
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2022-08-15
Completion Date 2024-07
Last Update 2024-04-09

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
Recent Updates Completed dosing in first two cohorts and on track to start third in Q2 2024

Resources/Links