Actively Recruiting
KB407-02 Phase 1 Study of Inhaled KB407 for Treating Cystic Fibrosis
Led by Krystal Biotech, Inc. · Updated on 2026-05-07
20
Participants Needed
6
Research Sites
12 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of different doses of a nebulized treatment called KB407 in adults with cystic fibrosis, a genetic condition affecting the lungs. This is a Phase 1 study sponsored by Krystal Biotech, Inc., focusing on how well adults with cystic fibrosis tolerate this new inhaled therapy designed to deliver a functional version of the CFTR protein. Participants will receive KB407 through inhalation in one of several dosing schedules: a single dose, two doses, four doses, or four consecutive doses followed by weekly doses up to six months. The study includes four open-label groups, each receiving different administration frequencies to assess safety and potential effects over time. During the study, participants will undergo evaluations of adverse events, physical exams, vital signs, ECGs, and laboratory tests over six months to monitor safety and tolerability. Researchers will also measure lung function changes using forced expiratory volume in one second (FEV1). Participants will be closely monitored to track any side effects and changes in lung health throughout the treatment period and follow-up.
CONDITIONS
Brief Title
A Study Assessing KB407 for the Treatment of Cystic Fibrosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must have read, understood, and signed an IRB/IEC approved informed consent form and be willing to follow study procedures
- Age 18 years or older at the time of consent
- Confirmed diagnosis of cystic fibrosis by clinical signs and either a sweat chloride over 60 mmol/L or two copies of a disease-causing CFTR gene mutation
- Clinically stable as judged by the Investigator
- Percent predicted FEV1 between 40% and 90% at screening
- Resting oxygen saturation of at least 92% on room air at screening
You will not qualify if you...
- Started or changed any chronic therapy (except pancreatic enzymes) within 28 days before the first dose
- Hospitalization, lung infection, cystic fibrosis exacerbation, or significant illness within 14 days before the first dose
- Treatment for Mycobacterium abscessus within 3 months before the first dose or more than two lung exacerbations with Burkholderia cenocepacia infection within 6 months prior
- Participation in another clinical study or investigational treatment within 30 days or 5 half-lives before the first dose
- History of or listed for solid organ transplantation
- Any condition, including substance abuse, uncontrolled asthma, or immunocompromised status, that may impact ability to complete the study or safety assessment
- Active oral herpes infection within 30 days before the first dose
- Pregnant or nursing women
- Unwillingness to comply with contraception requirements
- Significant abnormal blood tests that may interfere with safety or efficacy assessment
- Known allergy to inhaled glycerol
- Known or suspected noncompliance with study protocol
- For bronchoscopy participants: inability to tolerate bronchoscopy and airway sampling
- For cohort 4 participants: ineligibility for or intolerance or lack of benefit from modulator therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 6 months depending on cohort assignment
Participants receive nebulized KB407 administrations according to their assigned cohort, ranging from a single administration to multiple administrations over several months.
1 to 4 administrations plus weekly visits for up to 6 months for some participants
Duration - Up to 6 months
Participants are monitored for safety and pulmonary function changes following KB407 treatment.
Regular visits during the follow-up period
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
Research Team
D
David Sweet, MD, PhD
B
Brittani Agostini, RN, CCRC
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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