Actively Recruiting

Phase 1
Age: 18Years +
All Genders
NCT05504837

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

Led by Krystal Biotech, Inc. · Updated on 2026-05-07

20

Participants Needed

6

Research Sites

191 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

CONDITIONS

Official Title

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Must have signed an approved consent form and be willing to follow study procedures
  • Age 18 years or older at consent
  • Confirmed cystic fibrosis diagnosis by clinical signs and either a sweat chloride value over 60 mmol/L or two disease-causing CFTR gene mutations
  • Clinically stable as judged by the Investigator
  • Percent predicted FEV1 between 40% and 90% at screening
  • Resting oxygen saturation of 92% or higher on room air at screening
Not Eligible

You will not qualify if you...

  • Started any new chronic therapy or changed chronic therapy (except pancreatic enzyme replacement) within 28 days before first dose
  • Hospitalized, had sinopulmonary infection, CF exacerbation, or significant illness within 14 days before first dose
  • Treated for Mycobacterium abscessus within 3 months prior or had more than two lung exacerbations with Burkholderia cenocepacia infection within 6 months prior to first dose
  • Participated in another clinical study or used an investigational agent within 30 days or 5 half-lives before first dose
  • History of or listed for solid organ transplant
  • Any condition or history (including substance abuse, uncontrolled asthma, immunocompromised) that may affect ability to complete study or safety assessment
  • Active oral herpes infection within 30 days before first dose
  • Pregnant or nursing women
  • Unwilling to follow contraception requirements
  • Clinically significant blood or chemistry abnormalities at screening
  • Known allergy to inhaled glycerol
  • Known or expected noncompliance with study protocol
  • For bronchoscopy participants: inability to tolerate bronchoscopy and airway sampling
  • For Cohort 4: ineligible for, intolerant of, or not benefiting from modulator therapy

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 6 locations

1

University of Florida, Gainesville

Gainesville, Florida, United States, 32610

Actively Recruiting

2

St. Lukes CF Center of Idaho

Boise, Idaho, United States, 83712

Actively Recruiting

3

The Cystic Fibrosis Institute

Northfield, Illinois, United States, 60093

Actively Recruiting

4

New York Medical College/ Boston Children's Health Physicians

Hawthorne, New York, United States, 10532

Actively Recruiting

5

Northwell Health Physicians

New York, New York, United States, 10028

Actively Recruiting

6

Atrium Health Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States, 27157

Actively Recruiting

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Research Team

D

David Sweet, MD, PhD

CONTACT

B

Brittani Agostini, RN, CCRC

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

4

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