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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05095246




Registration number
NCT05095246
Ethics application status
Date submitted
27/08/2021
Date registered
27/10/2021
Date last updated
14/08/2023

Titles & IDs
Public title
A Study of Inhaled KB407 for the Treatment of Cystic Fibrosis
Scientific 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
Secondary ID [1] 0 0
KB407-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - KB407 (Nebulization)

Experimental: Cohort 1 (KB407) - A single dose of KB407 administered on Day 0

Experimental: Cohort 2 (KB407) - Two (2) doses of KB407 administered at Day 0 and Day 14

Experimental: Cohort 3 (KB407) - Four (4) doses of KB407 administered at Day 0, Day 7, Day 14, and Day 21


Other interventions: KB407 (Nebulization)
Nebulized solution of KB407, a replication-incompetent HSV-1 expressing full length human CFTR
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To evaluate the safety and tolerability of KB407 in subjects with Cystic Fibrosis through adverse events as assessed by NCI-CTCAE v5.0
Timepoint [1] 0 0
Baseline to End of the treatment assessed up to an average of 60 days
Secondary outcome [1] 0 0
To measure the difference in lung function over the course of the study, by change from baseline in forced expiratory volume (FEV1).
Timepoint [1] 0 0
Baseline to End of the treatment up to an average of 60 days

Eligibility
Key inclusion criteria
1. 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.

2. Male or female subject aged 18 years old or older at the time of Informed Consent.

3. A confirmed diagnosis of cystic fibrosis (CF) that is clinically stable, in the
opinion of the Investigator.

4. FEV1 =50% and =100% of the predicted normal for age, gender, and height at Visit 1
(Screening).
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. 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).

2. 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.

3. 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).

4. 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).

5. History of lung transplantation.

6. 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).

7. An active oral herpes infection within 30 Days of Visit 2 (Day 0).

8. Women who are pregnant or nursing.

9. Subject who is unwilling to comply with contraception requirements per-protocol.

10. 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.

11. Subject is known to be noncompliant or is unlikely to comply with the requirements of
the study protocol, in the opinion of the Investigator.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/10/2024
Actual
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Hunter Medical Research Institute - Newcastle
Recruitment postcode(s) [1] 0 0
2305 - Newcastle

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Krystal Biotech, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Novotech (Australia) Pty Limited
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief 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. 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05095246
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05095246