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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT07108153




Registration number
NCT07108153
Ethics application status
Date submitted
18/07/2025
Date registered
6/08/2025
Date last updated
6/08/2025

Titles & IDs
Public title
Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta
Scientific title
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
Secondary ID [1] 0 0
SION-719-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis (CF) 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SION-719
Treatment: Drugs - Placebo-to-match SION-719

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.


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

Treatment: Drugs: Placebo-to-match SION-719
All participants receive placebo to match SION-719, as specified by their treatment sequence assignment
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Day 1 through day 57
Secondary outcome [1] 0 0
Change from baseline in sweat chloride levels
Timepoint [1] 0 0
Baseline to Day 15 in each treatment period
Secondary outcome [2] 0 0
Concentration of SION-719 in plasma and change of concentration of SION-719 in plasma.
Timepoint [2] 0 0
Day 1 through day 57

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/10/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2026
Actual
Sample size
Target
16
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Gold Coast University Hospital - Southport
Recruitment hospital [3] 0 0
Monash University - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
4215 - Southport
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Iowa
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
South Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sionna Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Sionna Therapeutics Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
VP, Head of Medical Affairs & Patient Advocacy
Address 0 0
Country 0 0
Phone 0 0
617-819-1389
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT07108153