ClinicalTrials.gg
RecruitingNCT05504837Updated May 7, 2026

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

A Phase 1 interventional study of KB407 (Nebulization) in Cystic Fibrosis, sponsored by Krystal Biotech, Inc.. Recruiting at 6 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-05-07.

Sponsored by Krystal Biotech, Inc. · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Started Jun 2023; still recruiting 3 years 4 months later.
Phase
Phase 1
Study type
Interventional
Enrollment
20
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

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

02

Conditions studied

  • Cystic Fibrosis

Browse trials for

03

In context

Cystic Fibrosis

1,619 studies on the registry are indexed under Cystic Fibrosis; 199 are open to participants now.

This study's planned enrollment of 20 is below the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Krystal Biotech, Inc. is the lead sponsor of 18 studies on the registry; 9 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. The subject must have read, understood, and signed an Institutional Review Board/Ethics Committee (IRB/IEC) approved 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 ≥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

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 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 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 ) 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 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 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
Terms in these criteria, in plain language(7)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

FEV1

From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
20 participants (estimated)

Study arms

  • Experimental
    Cohort 1 (open label)

    A single administration of KB407

    Biological: KB407 (Nebulization)

  • Experimental
    Cohort 2 (open label)

    Two administrations of KB407

    Biological: KB407 (Nebulization)

  • Experimental
    Cohort 3 (open label)

    Four administrations of KB407

    Biological: KB407 (Nebulization)

  • Experimental
    Cohort 4 (open label)

    Four consecutive administrations of KB407 followed by weekly administration for up to 6 months

    Biological: KB407 (Nebulization)

Interventions

  • BiologicalKB407 (Nebulization)

    Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR

06

What researchers measure

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 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

    Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by spirometry as compared to baseline

    Time frame: 6 months

07

Study locations

6 of 6 sites recruiting
  • University of Florida, Gainesville
    Gainesville, Florida 32610, United States
    Recruiting
    • Alexa Smith · Contactalexa.howell@ufl.edu · 352-273-5085
    • Jorge Lascano, MD · Principal investigator
  • St. Lukes CF Center of Idaho
    Boise, Idaho 83712, United States
    Recruiting
    • Dixie Durham · Contactdurhamd@slhs.org · 208-381-7092
    • Karen Miller, MD · Principal investigator
  • The Cystic Fibrosis Institute
    Northfield, Illinois 60093, United States
    Recruiting
  • New York Medical College/ Boston Children's Health Physicians
    Hawthorne, New York 10532, United States
    Recruiting
  • Northwell Health Physicians
    New York, New York 10028, United States
    Recruiting
  • Atrium Health Wake Forest Baptist Medical Center
    Winston-Salem, North Carolina 27157, United States
    Recruiting
    • Christian Smith · Contactchnsmith@wakehealth.edu · 336-173-8550
    • Chad R Marion, DO, PhD · Principal investigator
08

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Jun 2023 (actual). Primary completion is estimated for Dec 2026, 42 months after the start.

Of 39 industry-sponsored phase 1 interventional studies under Cystic Fibrosis, half reached primary completion within 8 months of starting (the median), counting those still running; a quarter within 2 and three quarters within 16. This study's planned 42 months is above that.

Counting every sponsor, half of 62 studies reached it within 10 months; a quarter within 5 and three quarters within 34.

How these figures are worked out, and which studies are left out of them.

Registered
Aug 15, 2022
First posted
Aug 17, 2022
StartActual
Jun 30, 2023
Primary completionEst.
Dec 2026
CompletionEst.
Mar 2027
Last update
May 7, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on May 7, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT05504837
Lead sponsor
Krystal Biotech, Inc.
Responsible party
Sponsor
First posted
Aug 17, 2022
Start date
Jun 30, 2023
Primary completion
Dec 2026 (estimated)
Completion
Mar 2027 (estimated)
Last update
May 7, 2026

Study contacts

David Sweet, MD, PhD
Contact
dsweet@krystalbio.com
412-586-5830
Brittani Agostini, RN, CCRC
Contact
bagostini@krystalbio.com
412-586-5830

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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