RECRUITING

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

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

Quick Facts

Study Start:2023-06-30

Study Completion:2024-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05504837

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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	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

Inclusion Criteria

Exclusion 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

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

Contacts and Locations

Study Contact

David Chien, MD

CONTACT

412-586-5830

dchien@krystalbio.com

Brittani Agostini, RN, CCRC

CONTACT

412-586-5830

bagostini@krystalbio.com

Principal Investigator

David Chien, MD

STUDY_DIRECTOR

Senior Vice President of Clinical Development

Study Locations (Sites)

Yale University School of Medicine

New Haven, Connecticut, 06520

United States

The Cystic Fibrosis Institute

Northfield, Illinois, 60093

United States

Collaborators and Investigators

Sponsor: Krystal Biotech, Inc.

David Chien, MD, STUDY_DIRECTOR, Senior Vice President of Clinical Development

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-06-30

Study Completion Date2024-07

Study Record Updates

Study Start Date2023-06-30

Study Completion Date2024-07

Terms related to this study

Additional Relevant MeSH Terms

Cystic Fibrosis