Cystic FibrosisPhase 1NCT05504837
A Study Assessing KB407 for the Treatment of Cystic Fibrosis
A Phase 1 study testing KB407 (Nebulization) in people with Cystic Fibrosis, run by Krystal Biotech, Inc. at 6 sites in United States.
Trial summary
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- KB407 (Nebulization)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 6 sitesUnited States
- Study dates
- 30 Jun 2023to Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
St. Lukes CF Center of Idaho
Boise, Idaho, 83712
Dixie Durham, 208-381-7092, durhamd@slhs.org
Karen Miller, MD
- Recruiting
University of Florida, Gainesville
Gainesville, Florida, 32610
Alexa Smith, 352-273-5085, alexa.howell@ufl.edu
Jorge Lascano, MD
- Recruiting
New York Medical College/ Boston Children's Health Physicians
Hawthorne, New York, 10532
Armando Ramirez, CRC, 914-504-0152, armando.ramirez@bchphysicians.org
John Welter, MD
- Recruiting
Northwell Health Physicians
New York, New York, 10028
Teresa Demarco, 212-434-4790, tdemarco3@northwell.edu
Maria Berdella, MD
- Recruiting
The Cystic Fibrosis Institute
Northfield, Illinois, 60093
Karolina Roszko, research@wecare4lungs.com
Steven Boas, MD
- Recruiting
Atrium Health Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
Christian Smith, 336-173-8550, chnsmith@wakehealth.edu
Chad R Marion, DO, PhD
About this study
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
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
- Conditions
- Cystic Fibrosis
- Being tested
- KB407 (Nebulization)
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB407 (Nebulization)Biological
Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (open label) | Experimental | A single administration of KB407 Receives: Biological: KB407 (Nebulization) |
| Cohort 2 (open label) | Experimental | Two administrations of KB407 Receives: Biological: KB407 (Nebulization) |
| Cohort 3 (open label) | Experimental | Four administrations of KB407 Receives: Biological: KB407 (Nebulization) |
| Cohort 4 (open label) | Experimental | Four consecutive administrations of KB407 followed by weekly administration for up to 6 months Receives: Biological: KB407 (Nebulization) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2022
- Study start30 Jun 2023
- Primary completionDec 2026 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Krystal Biotech, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05504837
- Sponsor's protocol ID
- KB407-02
- Status last verified
- May 2026
- First submitted
- 15 Aug 2022
- Last updated
- 7 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05504837
- Acronym / sponsor ID
- KB407-02
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Jun 2023 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 17 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (open label) | Experimental | Biological: KB407 (Nebulization)A single administration of KB407 |
| Cohort 2 (open label) | Experimental | Biological: KB407 (Nebulization)Two administrations of KB407 |
| Cohort 3 (open label) | Experimental | Biological: KB407 (Nebulization)Four administrations of KB407 |
| Cohort 4 (open label) | Experimental | Biological: KB407 (Nebulization)Four consecutive administrations of KB407 followed by weekly administration for up to 6 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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:
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KB407 (Nebulization)Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR | Biological | Cohort 1 (open label), Cohort 2 (open label), Cohort 3 (open label), Cohort 4 (open label) |
Primary outcome
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: 6 months
Number of adult subjects with treatment related adverse events as assessed by NCI-CTCAE v5
1 secondary outcome
To evaluate the effects of KB407 on pulmonary function, as measured by change from baseline in absolute and percent predicted FEV1
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing KB407 (Nebulization) in people with Cystic Fibrosis, run by Krystal Biotech, Inc. at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact David Sweet, MD, PhD (412-586-5830, dsweet@krystalbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2026; AzTrials last refreshed its copy on 8 Nov 2026.