Cystic Fibrosis (CF)
Conditions
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.
Interventions
DRUGSION-719
All participants receive SION-719, as specified by their treatment sequence assignment
DRUGPlacebo-to-match SION-719
All participants receive placebo to match SION-719, as specified by their treatment sequence assignment
Sponsors
Sionna Therapeutics Inc.
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Australia, United States
Contacts
STUDY_DIRECTORMedical Director
Sionna Therapeutics Inc.
Outcome results
None listed