A Study of Inhaled KB407 for the Treatment of Cystic Fibrosis
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ClinicalTrials.gov Identifier: NCT05095246 |
Recruitment Status :
Withdrawn
(Pandemic, logistical, and recruitment challenges)
First Posted : October 27, 2021
Last Update Posted : August 14, 2023
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cystic Fibrosis | Biological: KB407 (Nebulization) | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 0 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 |
Arm | Intervention/treatment |
---|---|
Experimental: Cohort 1 (KB407)
A single dose of KB407 administered on Day 0
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Biological: KB407 (Nebulization)
Nebulized solution of KB407, a replication-incompetent HSV-1 expressing full length human CFTR |
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 |
- 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
- 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.
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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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).
Exclusion Criteria:
- 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).
- 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.
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
Australia, New South Wales | |
Hunter Medical Research Institute | |
Newcastle, New South Wales, Australia, 2305 |
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: | August 14, 2023 |
Last Verified: | August 2023 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |