Assessing KB407 for treating cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the safety and tolerability of ascending doses of nebulized KB407 in adults diagnosed with cystic fibrosis. Participants will receive different doses of the treatment to determine the optimal dosage that is both safe and effective. The study aims to gather data on how well the medication is tolerated by patients and its potential impact on their condition. The trial is designed for adults aged 18 and older who meet specific health criteria related to cystic fibrosis.

Why it matters

Who can join: the full rules

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 ≤90% 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 Mycobacterium abscessus within 3 months prior to the first dose or more than two pulmonary exacerbations with a history of Burkholderia cenocepacia infection within 6 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. Women who are pregnant or nursing
9. Subject who is unwilling to comply with contraception requirements per protocol
10. 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
11. Subject has a known hypersensitivity to inhaled glycerol
12. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator
13. Bronchoscopy participants only: Unable to tolerate bronchoscopy procedure and airway sampling, in the opinion of the Investigator
14. Cohort 4 participants only: Subjects who are ineligible for, do not tolerate, or do not benefit from modulator therapy

Where it's running

• University of Florida, Gainesville
  Gainesville, Florida, United States Recruiting
• St. Lukes CF Center of Idaho
  Boise, Idaho, United States Recruiting
• The Cystic Fibrosis Institute
  Northfield, Illinois, United States Recruiting
• New York Medical College/ Boston Children's Health Physicians
  Hawthorne, New York, United States Recruiting
• Northwell Health Physicians
  New York, New York, United States Recruiting
• Atrium Health Wake Forest Baptist Medical Center
  Winston-Salem, North Carolina, United States Recruiting

Study contacts

• Study coordinator: David Sweet, MD, PhD
• Email: dsweet@krystalbio.com
• Phone: 412-586-5830

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Collecting sputum samples at home for cystic fibrosis patients

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.