RECRUITINGPhase 2INTERVENTIONAL
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
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
- 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.
Who Should NOT Join This Trial:
- 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.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.
Treatments Being Tested
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
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