A Study Assessing KB407 for the Treatment of Cystic Fibrosis

NCT ID: NCT05504837

Last Updated: 2025-08-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-30

Study Completion Date

2026-01-31

Brief Summary

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This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1 (open label)

A single administration of KB407

Group Type EXPERIMENTAL

KB407 (Nebulization)

Intervention Type BIOLOGICAL

Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR

Cohort 2 (open label)

Two administrations of KB407

Group Type EXPERIMENTAL

KB407 (Nebulization)

Intervention Type BIOLOGICAL

Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR

Cohort 3 (open label)

Four administrations of KB407

Group Type EXPERIMENTAL

KB407 (Nebulization)

Intervention Type BIOLOGICAL

Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR

Interventions

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KB407 (Nebulization)

Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. 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
2. Subjects aged 18 years or older at the time of Informed Consent
3. 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
4. Clinically stable in the opinion of the Investigator
5. Percent predicted FEV1 ≥40% and ≤100% of the predicted normal for age, gender, and height at Screening
6. Resting oxygen saturation ≥92% on room air at Screening

Exclusion Criteria

1. 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
2. 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
3. Treatment for Burkholderia cenocepacia, Burkholderia dolosa, or Nontuberculosis Mycobacteria infection within 3 months prior to the first dose
4. 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
5. History of or listed for solid organ transplantation
6. 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
7. An active oral herpes infection 30 days prior to the first dose
8. Has received a vaccine within 72 hours prior to the first dose or has a planned vaccination during the treatment period
9. Women who are pregnant or nursing
10. Subject who is unwilling to comply with contraception requirements per protocol
11. 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
12. Subject has a known hypersensitivity to inhaled glycerol
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
14. Bronchoscopy participants only: Unable to tolerate bronchoscopy procedure and airway sampling, in the opinion of the Investigator
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Krystal Biotech, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Florida, Gainesville

Gainesville, Florida, United States

Site Status RECRUITING

The Cystic Fibrosis Institute

Northfield, Illinois, United States

Site Status RECRUITING

New York Medical College/ Boston Children's Health Physicians

Hawthorne, New York, United States

Site Status RECRUITING

Northwell Health Physicians

New York, New York, United States

Site Status RECRUITING

Atrium Health Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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David Sweet, MD, PhD

Role: CONTACT

412-586-5830

Brittani Agostini, RN, CCRC

Role: CONTACT

412-586-5830

Facility Contacts

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Alexa Smith

Role: primary

(352)-273-5085

Karolina Roszko

Role: primary

Armando Ramirez, CRC

Role: primary

914-504-0152

Teresa Demarco

Role: primary

212-434-4790

Christian Smith

Role: primary

+1(336) 173-8550

Other Identifiers

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KB407-02

Identifier Type: -

Identifier Source: org_study_id

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