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Gene Therapy Trial Report

Summary

A Study Assessing KB407 for the Treatment of Cystic Fibrosis


NCTID NCT05504837 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Cystic Fibrosis
Disease Ontology Term DOID:1485
Compound Name KB407
Sponsor Krystal Biotech, Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

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
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)

Study Record Dates


Current Stage Phase1
Submit Date 2022-08-15
Completion Date 2026-01
Last Update 2025-08-21

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. The subject must have read, understood, and signed an Institutional Review Board/Ethics Committee (IRB/IEC) approved Informed Consent Form and must be able to and willing to follow study procedures and instructions 2. Subjects aged 18 years or older at the time of Informed Consent 3. A confirmed diagnosis of CF as defined by clinical signs and symptoms of CF and at least one of the following: * A historical sweat chloride value \>60 mmol/L * Two copies of a disease causing mutation in the CFTR gene 4. Clinically stable in the opinion of the Investigator 5. Percent predicted FEV1 ≥40% and ≤100% of the predicted normal for age, gender, and height at Screening 6. Resting oxygen saturation ≥92% on room air at Screening Exclusion Criteria: 1. Initiation of any new chronic therapy (eg, CFTR modulator, hypertonic saline, inhaled antibiotic) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days prior to the first dose 2. Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness 14 days prior to the first dose that, in the opinion of the Investigator, may confound study results 3. Treatment for Burkholderia cenocepacia, Burkholderia dolosa, or Nontuberculosis Mycobacteria infection within 3 months prior to the first dose 4. Participation in another clinical study or treatment with an investigational agent 30 days or 5 half-lives, whichever is longer, prior to the first dose 5. History of or listed for solid organ transplantation 6. Any condition (including a history or current evidence of substance abuse or dependence, uncontrolled asthma, or is considered to be immunocompromised) 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 KB407 7. An active oral herpes infection 30 days prior to the first dose 8. Has received a vaccine within 72 hours prior to the first dose or has a planned vaccination during the treatment period 9. Women who are pregnant or nursing 10. Subject who is unwilling to comply with contraception requirements per protocol 11. Clinically significant abnormalities of hematology or chemistry testing at Screening that the Investigator believes may interfere with the assessment of safety and/or efficacy of the study treatment 12. Subject has a known hypersensitivity to inhaled glycerol 13. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator 14. Bronchoscopy participants only: Unable to tolerate bronchoscopy procedure and airway sampling, in the opinion of the Investigator
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 5
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Interim data for cohort 3 (highest dose) presented in January 2026

Resources/Links