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Trial record 1 of 1 for:    NCT05095246
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A Study of Inhaled KB407 for the Treatment of Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT05095246
Recruitment Status : Recruiting
First Posted : October 27, 2021
Last Update Posted : June 8, 2022
Sponsor:
Collaborator:
Novotech (Australia) Pty Limited
Information provided by (Responsible Party):
Krystal Biotech, Inc.

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Biological: KB407 (Nebulization) Biological: KB407 (Intradermal) Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 13 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : March 8, 2022
Estimated Primary Completion Date : July 1, 2024
Estimated Study Completion Date : October 30, 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Cohort 1 (KB407)

A single dose of KB407 administered on Day 0

A single intradermal injection of KB407 at Day 0 (Optional)

Biological: KB407 (Nebulization)
Nebulized solution of KB407, a replication-incompetent HSV-1 expressing full length human CFTR

Biological: KB407 (Intradermal)
Intradermal injection of KB407, a replication-incompetent HSV-1 expressing full length human CFTR (Cohort 1 only)

Experimental: Cohort 2 (KB407)
Two (2) doses of KB407 administered at Day 0 and Day 14
Biological: KB407 (Nebulization)
Nebulized solution of KB407, a replication-incompetent HSV-1 expressing full length human CFTR

Experimental: Cohort 3 (KB407)
Four (4) doses of KB407 administered at Day 0, Day 7, Day 14, and Day 21
Biological: KB407 (Nebulization)
Nebulized solution of KB407, a replication-incompetent HSV-1 expressing full length human CFTR




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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 ]
    Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by pulmonary function test (PFT) as compared to baseline.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 ≤90% of the predicted normal for age, gender, and height at Visit 1 (Screening).

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. Currently taking or has taken a CFTR modulator within seven (7) days of Visit 2 (Day 0).
  5. 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)
  6. History of lung transplantation.
  7. 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.
  8. An active oral herpes infection within 30 Days of Visit 2 (Day 0).
  9. HSV-1 wild type seropositive subjects (Screening Visit)
  10. Women who are pregnant or nursing.
  11. Subject who is unwilling to comply with contraception requirements per-protocol.
  12. 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.
  13. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05095246


Contacts
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Contact: Brittani Agostini, Senior Manager, Clin Ops. 412-586-5830 bagostini@krystalbio.com

Locations
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Australia, New South Wales
Hunter Medical Research Institute Recruiting
Newcastle, New South Wales, Australia, 2305
Contact: Peter Wark, MD    61 2 4042 0133    peter.wark@newcastle.edu.au   
Contact: Douglas Dorahy Douglas Dorahy, PhD    61 2 4042 0133    douglas.dorahy@newcastle.edu.au   
Sponsors and Collaborators
Krystal Biotech, Inc.
Novotech (Australia) Pty Limited
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Responsible Party: Krystal Biotech, Inc.
ClinicalTrials.gov Identifier: NCT05095246    
Other Study ID Numbers: KB407-01
First Posted: October 27, 2021    Key Record Dates
Last Update Posted: June 8, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases