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Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta
Sponsor: Sionna Therapeutics Inc.
Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SION-719 when given to people with CF who are already taking Trikafta.
Official title: A Phase 2a Randomized, Double-blind, Placebo-controlled Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Physician-prescribed Trikafta® in People With Cystic Fibrosis Who Are Homozygous for the F508del Mutation
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
16
Start Date
2025-11-03
Completion Date
2026-07
Last Updated
2026-03-02
Healthy Volunteers
No
Conditions
Interventions
SION-719
All participants receive SION-719, as specified by their treatment sequence assignment
Placebo-to-match SION-719
All participants receive placebo to match SION-719, as specified by their treatment sequence assignment
Locations (13)
National Jewish Health
Denver, Colorado, United States
University of Iowa
Iowa City, Iowa, United States
Johns Hopkins
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University
St Louis, Missouri, United States
New York Medical College
Hawthorne, New York, United States
Columbia University
New York, New York, United States
University Hospital of Cleveland
Cleveland, Ohio, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Washington
Seattle, Washington, United States
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia
Gold Coast University Hospital
Southport, Queensland, Australia
Monash University
Melbourne, Victoria, Australia