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
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• 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.
Locations
United States
Colorado
National Jewish Health
RECRUITING
Denver
Iowa
University of Iowa
RECRUITING
Iowa City
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Maryland
Johns Hopkins
NOT_YET_RECRUITING
Baltimore
Missouri
Washington University
RECRUITING
St Louis
New York
New York Medical College
RECRUITING
Hawthorne
Columbia University
RECRUITING
New York
Ohio
University Hospital of Cleveland
RECRUITING
Cleveland
South Carolina
Medical University of South Carolina
NOT_YET_RECRUITING
Charleston
Washington
University of Washington
RECRUITING
Seattle
Other Locations
Australia
Royal Prince Alfred Hospital
NOT_YET_RECRUITING
Camperdown
Monash University
NOT_YET_RECRUITING
Melbourne
Gold Coast University Hospital
NOT_YET_RECRUITING
Southport
Contact Information
Primary
VP, Head of Medical Affairs & Patient Advocacy
medinfo@sionnatx.com
617-819-1389
Time Frame
Start Date: 2025-11-03
Estimated Completion Date: 2026-07
Participants
Target number of participants: 16
Treatments
Experimental: Crossover Treatment Sequence 1: SION-719 followed by 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.
Experimental: Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by 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.
Related Therapeutic Areas
Sponsors
Leads: Sionna Therapeutics Inc.