Cystic FibrosisPhase 1NCT05095246
A Study of Inhaled 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 1 site in Australia.
Trial summary
The Sponsor is developing KB407, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) to the airways of people with cystic fibrosis via nebulization. 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
- 0 participants
- Where
- 1 siteAustralia
- Study dates
- 8 Mar 2022to 30 Oct 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 or legally appointed and authorized representative must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent Form and must be able to and willing to follow study procedures and instructions.
- Male or female subject aged 18 years old or older at the time of Informed Consent.
- A confirmed diagnosis of cystic fibrosis (CF) that is clinically stable, in the opinion of the Investigator.
- FEV1 ≥50% and ≤100% of the predicted normal for age, gender, and height at Visit 1 (Screening).
Who cannot join
- Initiation of any new chronic therapy (e.g., ibuprofen, hypertonic saline, azithromycin, Pulmozyme®, Cayston®, TOBI®) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days of Visit 2 (Day 0).
- Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness within 14 days of Visit 2 (Day 0) that, in the opinion of the Investigator, may confound study results.
- A positive culture (saliva or sputum) indicating infection with highly virulent bacteria associated with accelerated decline in pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia, Burkholderia dolosa, Mycobacterium abscessus) within 6-months of Visit 2 (Day 0).
- Participation in another clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, of Visit 2 (Day 0).
- History of lung transplantation.
- Any condition (including a history or current evidence of substance abuse or dependence) 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 the Investigational Product (IP).
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- An active oral herpes infection within 30 Days of Visit 2 (Day 0).
- 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 Visit 1 (Screening) that the Investigator believes may interfere with the assessment of safety and/or efficacy of the study treatment.
- Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator.
Trial locations and contacts
1 site in 1 country.
Australia1 site
Hunter Medical Research Institute
Newcastle, New South Wales, 2305
About this study
Official title: A Phase I Study of Inhaled KB407, a Replication-Incompetent, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), for the Treatment of Cystic Fibrosis
The Sponsor is developing KB407, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) to the airways of people with cystic fibrosis via nebulization. This study is designed to evaluate safety and tolerability of KB407 in people with cystic fibrosis. This study will enroll 4 participants into each of the first two cohorts and will enroll five subjects into the last cohort. Cohort 1 will receive a single dose of KB407 and be followed for 60 days. Subjects in Cohort 1 may rollover into Cohort 2 at the Day 28 Visit. A Data Safety Monitoring Board (DSMB) will meet to determine study progress from Cohort 2 into Cohort 3. In Cohort 2, subjects will be dosed bi-weekly at Day 0 and Day 14. In Cohort 3 subjects will be dosed weekly at Day 0, Day 7, Day 14 and Day 21. All subjects will be followed for a year after the last dose of KB407.
- 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-incompetent HSV-1 expressing full length human CFTR
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (KB407) | Experimental | A single dose of KB407 administered on Day 0 Receives: Biological: KB407 (Nebulization) |
| Cohort 2 (KB407) | Experimental | Two (2) doses of KB407 administered at Day 0 and Day 14 Receives: Biological: KB407 (Nebulization) |
| Cohort 3 (KB407) | Experimental | Four (4) doses of KB407 administered at Day 0, Day 7, Day 14, and Day 21 Receives: Biological: KB407 (Nebulization) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of KB407 in subjects with Cystic Fibrosis through adverse events as assessed by NCI-CTCAE v5.0
Number of participants with treatment related adverse events as assessed by NCI-CTCAE v5.0
Measured: Baseline to End of the treatment assessed up to an average of 60 days
Secondary outcomes (1)
To measure the difference in lung function over the course of the study, by change from baseline in forced expiratory volume (FEV1).
Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by pulmonary function test (PFT) as compared to baseline.
Measured: Baseline to End of the treatment up to an average of 60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2021
- Study start8 Mar 2022
- Primary completion1 Jul 2024 (estimated)
- Study completion30 Oct 2024 (estimated)
Why it stopped: Pandemic, logistical, and recruitment challenges
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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Krystal Biotech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05095246
- Sponsor's protocol ID
- KB407-01
- Status last verified
- Aug 2023
- First submitted
- 27 Aug 2021
- Last updated
- 14 Aug 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Inhaled KB407, a Replication-Incompetent, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), for the Treatment of Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT05095246
- Acronym / sponsor ID
- KB407-01
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Pandemic, logistical, and recruitment challenges
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Krystal Biotech, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Study start
- 8 Mar 2022 (actual)
- Primary completion
- 1 Jul 2024 (estimated)
- Study completion
- 30 Oct 2024 (estimated)
- First posted
- 27 Oct 2021 (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.
The Sponsor is developing KB407, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) to the airways of people with cystic fibrosis via nebulization. This study is designed to evaluate safety and tolerability of KB407 in people with cystic fibrosis. This study will enroll 4 participants into each of the first two cohorts and will enroll five subjects into the last cohort. Cohort 1 will receive a single dose of KB407 and be followed for 60 days. Subjects in Cohort 1 may rollover into Cohort 2 at the Day 28 Visit. A Data Safety Monitoring Board (DSMB) will meet to determine study progress from Cohort 2 into Cohort 3. In Cohort 2, subjects will be dosed bi-weekly at Day 0 and Day 14. In Cohort 3 subjects will be dosed weekly at Day 0, Day 7, Day 14 and Day 21. All subjects will be followed for a year after the last dose of KB407.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (KB407) | Experimental | Biological: KB407 (Nebulization)A single dose of KB407 administered on Day 0 |
| Cohort 2 (KB407) | Experimental | Biological: KB407 (Nebulization)Two (2) doses of KB407 administered at Day 0 and Day 14 |
| Cohort 3 (KB407) | Experimental | Biological: KB407 (Nebulization)Four (4) doses of KB407 administered at Day 0, Day 7, Day 14, and Day 21 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- The subject or legally appointed and authorized representative must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent Form and must be able to and willing to follow study procedures and instructions.
- Male or female subject aged 18 years old or older at the time of Informed Consent.
- A confirmed diagnosis of cystic fibrosis (CF) that is clinically stable, in the opinion of the Investigator.
- FEV1 ≥50% and ≤100% of the predicted normal for age, gender, and height at Visit 1 (Screening).
Key exclusion criteria (11 listed)
- Initiation of any new chronic therapy (e.g., ibuprofen, hypertonic saline, azithromycin, Pulmozyme®, Cayston®, TOBI®) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days of Visit 2 (Day 0).
- Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness within 14 days of Visit 2 (Day 0) that, in the opinion of the Investigator, may confound study results.
- A positive culture (saliva or sputum) indicating infection with highly virulent bacteria associated with accelerated decline in pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia, Burkholderia dolosa, Mycobacterium abscessus) within 6-months of Visit 2 (Day 0).
- Participation in another clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, of Visit 2 (Day 0).
- History of lung 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-incompetent HSV-1 expressing full length human CFTR | Biological | Cohort 1 (KB407), Cohort 2 (KB407), Cohort 3 (KB407) |
Primary outcome
To evaluate the safety and tolerability of KB407 in subjects with Cystic Fibrosis through adverse events as assessed by NCI-CTCAE v5.0
Time frame: Baseline to End of the treatment assessed up to an average of 60 days
Number of participants with treatment related adverse events as assessed by NCI-CTCAE v5.0
1 secondary outcome
To measure the difference in lung function over the course of the study, by change from baseline in forced expiratory volume (FEV1).
Time frame: Baseline to End of the treatment up to an average of 60 days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2023.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site 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 1 site in Australia.
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 4 inclusion and 11 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: withdrawn, as of the sponsor's last update on 14 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. 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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of KB407 in subjects with Cystic Fibrosis through adverse events as assessed by NCI-CTCAE v5.0 (time frame: Baseline to End of the treatment assessed up to an average of 60 days). 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 14 Aug 2023.