A Study Assessing KB407 for the Treatment of Cystic Fibrosis

Sponsor
Krystal Biotech, Inc. (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05504837
Collaborator
(none)
20
1
3
16
1.3

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
  • Biological: KB407 (Nebulization)
Phase 1

Study Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Allocation:
Randomized
Intervention Model:
Sequential Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
Official Title:
KB407-02 A Phase 1 Study of Inhaled KB407, a Replication-Defective, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator, for the Treatment of Cystic Fibrosis
Anticipated Study Start Date :
Nov 1, 2022
Anticipated Primary Completion Date :
Mar 1, 2024
Anticipated Study Completion Date :
Mar 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Cohort 1 (open label)

A single administration of KB407

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

Experimental: Cohort 2 (open label)

Two administrations of KB407

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

Experimental: Cohort 3 (randomized, placebo-controlled)

Four administrations of KB407 or placebo

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

Outcome Measures

Primary Outcome Measures

  1. To evaluate safety and tolerability of KB407 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in physical examinations, vital signs, ECG, and clinical laboratory test results [2 months]

    Number of adult subjects with treatment related adverse events as assessed by NCI-CTCAE v5

Secondary Outcome Measures

  1. To evaluate the effects of KB407 on pulmonary function, as measured by change from baseline in absolute and percent predicted FEV1 [2 months]

    Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by spirometry as compared to baseline

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

  1. Clinically stable in the opinion of the Investigator

  2. Percent predicted FEV1 ≥50% and ≤100% of the predicted normal for age, gender, and height at Screening

  3. 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. Cohort 3 only: Receiving treatment with a CFTR modulator (eg, ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor, elexacaftor/tezacaftor/ivacaftor). Patients with a prior history of treatment with CFTR modulator therapy are eligible to participate if treatment has been discontinued for medically indicated reasons, at least 28 days prior to the first dose

  3. 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

  4. Treatment for Burkholderia cenocepacia, Burkholderia dolosa, or Nontuberculosis Mycobacteria infection within 3 months prior to the first dose

  5. 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

  6. History of or listed for solid organ transplantation

  7. 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

  8. An active oral herpes infection 30 days prior to the first dose

  9. Has received a vaccine within 72 hours prior to the first dose or has a planned vaccination during the treatment period

  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 Screening that the Investigator believes may interfere with the assessment of safety and/or efficacy of the study treatment

  13. Subject has a known hypersensitivity to inhaled glycerol

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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Yale University School of Medicine New Haven Connecticut United States 06520

Sponsors and Collaborators

  • Krystal Biotech, Inc.

Investigators

  • Study Director: Brittani Agostini, RN, CCRC, Associate Director, Clinical Operations, Krystal Biotech

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Krystal Biotech, Inc.
ClinicalTrials.gov Identifier:
NCT05504837
Other Study ID Numbers:
  • KB407-02
First Posted:
Aug 17, 2022
Last Update Posted:
Aug 17, 2022
Last Verified:
Aug 1, 2022
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Aug 17, 2022