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

Purpose

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.

Condition

  • Cystic Fibrosis (CF)

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Crossover Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
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.
  • Drug: SION-719
    All participants receive SION-719, as specified by their treatment sequence assignment
  • Drug: Placebo-to-match SION-719
    All participants receive placebo to match SION-719, as specified by their treatment sequence assignment
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.
  • Drug: SION-719
    All participants receive SION-719, as specified by their treatment sequence assignment
  • Drug: Placebo-to-match SION-719
    All participants receive placebo to match SION-719, as specified by their treatment sequence assignment

Recruiting Locations

National Jewish Health
Denver 5419384, Colorado 5417618 80206

University of Iowa
Iowa City 4862034, Iowa 4862182 52242

Boston Children's Hospital
Boston 4930956, Massachusetts 6254926 02115

Washington University
St Louis 4407066, Missouri 4398678 63110

New York Medical College
Hawthorne 5120284, New York 5128638 10532

Columbia University
New York 5128581, New York 5128638 10032

University Hospital of Cleveland
Cleveland 5150529, Ohio 5165418 44106

University of Washington
Seattle 5809844, Washington 5815135 98195

More Details

Status
Recruiting
Sponsor
Sionna Therapeutics Inc.

Study Contact

VP, Head of Medical Affairs & Patient Advocacy
617-819-1389
medinfo@sionnatx.com