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

NCT ID: NCT07108153

Last Updated: 2025-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-11-03

Study Completion Date

2026-07-31

Brief Summary

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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.

Detailed Description

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Conditions

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Cystic Fibrosis (CF)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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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.

Group Type EXPERIMENTAL

SION-719

Intervention Type DRUG

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

Placebo-to-match SION-719

Intervention Type DRUG

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

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.

Group Type EXPERIMENTAL

SION-719

Intervention Type DRUG

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

Placebo-to-match SION-719

Intervention Type DRUG

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

Interventions

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SION-719

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

Intervention Type DRUG

Placebo-to-match SION-719

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

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sionna Therapeutics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Sionna Therapeutics Inc.

Locations

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National Jewish Health

Denver, Colorado, United States

Site Status RECRUITING

University of Iowa

Iowa City, Iowa, United States

Site Status RECRUITING

Johns Hopkins

Baltimore, Maryland, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Washington University

St Louis, Missouri, United States

Site Status RECRUITING

New York Medical College

Hawthorne, New York, United States

Site Status RECRUITING

Columbia University

New York, New York, United States

Site Status RECRUITING

University Hospital of Cleveland

Cleveland, Ohio, United States

Site Status RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status RECRUITING

University of Washington

Seattle, Washington, United States

Site Status RECRUITING

Royal Prince Alfred Hospital

Camperdown, New South Wales, Australia

Site Status RECRUITING

Gold Coast University Hospital

Southport, Queensland, Australia

Site Status RECRUITING

Monash University

Melbourne, Victoria, Australia

Site Status NOT_YET_RECRUITING

Countries

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United States Australia

Central Contacts

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VP, Head of Medical Affairs & Patient Advocacy

Role: CONTACT

Phone: 617-819-1389

Email: [email protected]

Other Identifiers

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SION-719-201

Identifier Type: -

Identifier Source: org_study_id