A Study Assessing KB407 for the Treatment of Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:

KB407-02 A Phase 1 Study of Inhaled KB407, a Replication-Defective, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator, for the Treatment of Cystic Fibrosis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05504837
• Other study ID #: KB407-02
• Status: Recruiting
• Phase: Phase 1
• Start date: June 30, 2023
• Est. completion date: July 2024

Study information

• Verified date: April 2024
• Source: Krystal Biotech, Inc.
• Contact: David Chien, MD
• Phone: 412-586-5830
• Email: dchien[@]krystalbio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: July 2024
• Est. primary completion date: July 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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 =50% 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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Biological:
• KB407 (Nebulization)
Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR

Locations

Country	Name	City	State
United States	Yale University School of Medicine	New Haven	Connecticut
United States	The Cystic Fibrosis Institute	Northfield	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
Krystal Biotech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate safety and tolerability of KB407 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in physical examinations, vital signs, ECG, and clinical laboratory test results	Number of adult subjects with treatment related adverse events as assessed by NCI-CTCAE v5	2 months
Secondary	To evaluate the effects of KB407 on pulmonary function, as measured by change from baseline in absolute and percent predicted FEV1	Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by spirometry as compared to baseline	2 months