A Study Assessing KB407 for the Treatment of Cystic Fibrosis
ConditionCystic Fibrosis
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorKrystal Biotech, Inc.
About this trial
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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 historical sweat chloride value >60 mmol/L
Two copies of a disease causing mutation in the CFTR gene
Disqualifiers
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
Trial design
Design model
Sequential
Treatments tested in this trial
KB407 (Nebulization)
Biological/VaccineNebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR
Treatment groups
20 Participants
are divided into 4 treatment groupsGroup A: Cohort 1 (open label)Experimental treatment 1 intervention
Group B: Cohort 2 (open label)Experimental treatment 1 intervention
Group C: Cohort 3 (open label)Experimental treatment 1 intervention
Group D: Cohort 4 (open label)Experimental treatment 1 intervention
Trial outcomes
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
Time frame
6 monthsSecondary outcomes
1
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
Time frame
6 monthsSponsors and contacts
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Source ClinicalTrials.gov