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A Study Assessing KB407 for the Treatment of Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05504837
Recruitment Status : Recruiting
First Posted : August 17, 2022
Last Update Posted : April 9, 2024
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Study Description
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Brief Summary:
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Biological: KB407 (Nebulization) Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : June 30, 2023
Estimated Primary Completion Date : July 2024
Estimated Study Completion Date : July 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1 (open label)
A single administration of KB407
 Biological: KB407 (Nebulization)
Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR
Experimental: Cohort 2 (open label)
Two administrations of KB407
 Biological: KB407 (Nebulization)
Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR
Experimental: Cohort 3 (open label)
Four administrations of KB407
 Biological: KB407 (Nebulization)
Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR


Outcome Measures
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Primary Outcome Measures :
  1. 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 [ Time Frame: 2 months ]
    Number of adult subjects with treatment related adverse events as assessed by NCI-CTCAE v5


Secondary Outcome Measures :
  1. To evaluate the effects of KB407 on pulmonary function, as measured by change from baseline in absolute and percent predicted FEV1 [ Time Frame: 2 months ]
    Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by spirometry as compared to baseline


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 ≥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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05504837


Contacts
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Contact: David Chien, MD 412-586-5830 dchien@krystalbio.com
Contact: Brittani Agostini, RN, CCRC 412-586-5830 bagostini@krystalbio.com

Locations
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United States, Connecticut
Yale University School of Medicine Not yet recruiting
New Haven, Connecticut, United States, 06520
Contact: Claire Cochrane         
Principal Investigator: Jonathan Koff, MD         
United States, Illinois
The Cystic Fibrosis Institute Recruiting
Northfield, Illinois, United States, 60093
Contact: Karolina Roszko       research@wecare4lungs.com   
Principal Investigator: Steven Boas, MD         
Sponsors and Collaborators
Krystal Biotech, Inc.
Investigators
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Study Director: David Chien, MD Senior Vice President of Clinical Development
More Information
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Responsible Party: Krystal Biotech, Inc.
ClinicalTrials.gov Identifier: NCT05504837    
Other Study ID Numbers: KB407-02
First Posted: August 17, 2022    Key Record Dates
Last Update Posted: April 9, 2024
Last Verified: April 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
 Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases