A Study Assessing KB407 for the Treatment of Cystic Fibrosis
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
1 other identifier
interventional
20
1 country
6
Brief Summary
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Jun 2023
Typical duration for phase_1
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 15, 2022
CompletedFirst Posted
Study publicly available on registry
August 17, 2022
CompletedStudy Start
First participant enrolled
June 30, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 1, 2027
May 7, 2026
May 1, 2026
3.4 years
August 15, 2022
May 1, 2026
Conditions
Outcome Measures
Primary Outcomes (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
Number of adult subjects with treatment related adverse events as assessed by NCI-CTCAE v5
6 months
Secondary Outcomes (1)
To evaluate the effects of KB407 on pulmonary function, as measured by change from baseline in absolute and percent predicted FEV1
6 months
Study Arms (4)
Cohort 1 (open label)
EXPERIMENTALA single administration of KB407
Cohort 2 (open label)
EXPERIMENTALTwo administrations of KB407
Cohort 3 (open label)
EXPERIMENTALFour administrations of KB407
Cohort 4 (open label)
EXPERIMENTALFour consecutive administrations of KB407 followed by weekly administration for up to 6 months
Interventions
Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR
Eligibility Criteria
You may qualify if:
- 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
- Subjects aged 18 years or older at the time of Informed Consent
- 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
- Clinically stable in the opinion of the Investigator
- Percent predicted FEV1 ≥40% and ≤90% of the predicted normal for age, gender, and height at Screening
- Resting oxygen saturation ≥92% on room air at Screening
You may not qualify if:
- 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
- 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
- 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
- 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
- History of or listed for solid organ transplantation
- 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
- An active oral herpes infection 30 days prior to the first dose
- Women who are pregnant or nursing
- Subject who is unwilling to comply with contraception requirements per protocol
- 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
- Subject has a known hypersensitivity to inhaled glycerol
- Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator
- Bronchoscopy participants only: Unable to tolerate bronchoscopy procedure and airway sampling, in the opinion of the Investigator
- Cohort 4 participants only: Subjects who are ineligible for, do not tolerate, or do not benefit from modulator therapy
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
University of Florida, Gainesville
Gainesville, Florida, 32610, United States
St. Lukes CF Center of Idaho
Boise, Idaho, 83712, United States
The Cystic Fibrosis Institute
Northfield, Illinois, 60093, United States
New York Medical College/ Boston Children's Health Physicians
Hawthorne, New York, 10532, United States
Northwell Health Physicians
New York, New York, 10028, United States
Atrium Health Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 15, 2022
First Posted
August 17, 2022
Study Start
June 30, 2023
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
March 1, 2027
Last Updated
May 7, 2026
Record last verified: 2026-05