RecruitingPhase 2NCT07108153

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

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


Sponsor

Sionna Therapeutics Inc.

Enrollment

16 participants

Start Date

Nov 3, 2025

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 18 YearsMax Age: 65 Years

Inclusion Criteria3

  • 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 Criteria2

  • 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.

Interested in this trial?

Get notified about updates and connect with the research team.

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


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

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT07108153


Related Trials