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

NCT07108153 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: SION-719-201
• Study Type: interventional
• Target: 16
• Locations: 2 countries
• Sites: 13

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.

Conditions

Cystic Fibrosis (CF)

Outcome Measures

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

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

  Day 1 through day 57

Secondary Outcomes (2)

• Change from baseline in sweat chloride levels

  Baseline to Day 15 in each treatment period

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

  Day 1 through day 57

Study Arms (2)

Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719

EXPERIMENTAL

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.

Drug: SION-719; Drug: Placebo-to-match SION-719

Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719

EXPERIMENTAL

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.

Drug: SION-719; Drug: Placebo-to-match SION-719

Interventions

SION-719 DRUG

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

Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719; Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719

Placebo-to-match SION-719 DRUG

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

Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719; Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sionna Therapeutics Inc.: lead

Study Sites (13)

National Jewish Health

Denver, Colorado, 80206, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Johns Hopkins

Baltimore, Maryland, 21205, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

New York Medical College

Hawthorne, New York, 10532, United States

Location

Columbia University

New York, New York, 10032, United States

Location

University Hospital of Cleveland

Cleveland, Ohio, 44106, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

Location

Gold Coast University Hospital

Southport, Queensland, 4215, Australia

Location

Monash University

Melbourne, Victoria, 3004, Australia

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Medical Director

  Sionna Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 18, 2025
• First Posted: August 6, 2025
• Study Start: November 3, 2025
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: May 6, 2026

Record last verified: 2026-05

Locations

AU (3); US (10)