Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta
- Conditions
- Cystic Fibrosis (CF)
- Interventions
- Drug: Placebo-to-match SION-719
- Registration Number
- NCT07108153
- Lead Sponsor
- Sionna Therapeutics Inc.
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 16
- Diagnosis of CF with F508del homozygous genotype based on documented CFTR genotype laboratory report.
- Adherent to physician-prescribed Trikafta, as assessed by the Investigator, for at least 3 months prior to the Screening visit, taken at the recommended dose without modifications and on the regimen described in the current prescribing information.
- Stable CF disease without pulmonary exacerbation within 28 days before Baseline or acute non-CF-related illness within 14 days before Baseline.
- Participant has clinically significant current or recurrent illness, other than CF
- Participant has a history of malignancy, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of recurrence for at least 1 year.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719 SION-719 Participants receive SION-719 for one treatment period, and placebo to match SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study. Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719 Placebo-to-match SION-719 Participants receive SION-719 for one treatment period, and placebo to match SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study. Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719 SION-719 Participants receive placebo to match SION-719 for one treatment period and SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study. Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719 Placebo-to-match SION-719 Participants receive placebo to match SION-719 for one treatment period and SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.
- Primary Outcome Measures
Name Time Method 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 Day 1 through day 57 Adverse events will be assessed by Common Terminology Criteria for Adverse Events (CTCAE), v. 5.0
- Secondary Outcome Measures
Name Time Method Change from baseline in sweat chloride levels Baseline to Day 15 in each treatment period Changes from baseline in sweat chloride levels will be analyzed with mixed model repeated measures (MMRM) methods.
Concentration of SION-719 in plasma and change of concentration of SION-719 in plasma. Day 1 through day 57 Concentrations of plasma SION-719 will be evaluated at each collected timepoint and summarized using descriptive statistics.
Trial Locations
- 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
Scroll for more (3 remaining)National Jewish Health🇺🇸Denver, Colorado, United States