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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
15
All participants receive SION-719, as specified by their treatment sequence assignment
All participants receive placebo to match SION-719, as specified by their treatment sequence assignment
National Jewish Health
Denver, Colorado, United States
University of Iowa
Iowa City, Iowa, United States
Johns Hopkins
Baltimore, Maryland, United States
To evaluate the incidence of treatment emergent adverse events [Safety and Tolerability] when SION-719 is administered to people with cystic fibrosis (CF) who are taking a standard stable dose of physician-prescribed Trikafta
Adverse events will be assessed by Common Terminology Criteria for Adverse Events (CTCAE), v. 5.0
Time frame: Day 1 through day 57
Change from baseline in sweat chloride levels
Changes from baseline in sweat chloride levels will be analyzed with mixed model repeated measures (MMRM) methods.
Time frame: Baseline to Day 15 in each treatment period
Concentration of SION-719 in plasma and change of concentration of SION-719 in plasma.
Concentrations of plasma SION-719 will be evaluated at each collected timepoint and summarized using descriptive statistics.
Time frame: Day 1 through day 57
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
...and 3 more locations