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NCT Number: NCT07108153

Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Cystic Fibrosis (CF) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Royal Prince Alfred Hospital, Camperdown, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SION-719

Drug

All participants receive SION-719, as specified by their treatment sequence assignment

Placebo-to-match SION-719

Drug

All participants receive placebo to match SION-719, as specified by their treatment sequence assignment

Primary outcomes

  1. 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

    Time frame: Day 1 through day 57

    Adverse events will be assessed by Common Terminology Criteria for Adverse Events (CTCAE), v. 5.0

Secondary outcomes

  1. Change from baseline in sweat chloride levels

    Time frame: 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.

  2. Concentration of SION-719 in plasma and change of concentration of SION-719 in plasma.

    Time frame: Day 1 through day 57

    Concentrations of plasma SION-719 will be evaluated at each collected timepoint and summarized using descriptive statistics.

Sponsors and collaborators

Lead sponsor

Sionna Therapeutics Inc.

Industry

Registry information

Official study 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

Acronym: PreciSION CF

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Aug 6, 2025
Registry last updated
May 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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