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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05918211




Registration number
NCT05918211
Ethics application status
Date submitted
15/06/2023
Date registered
26/06/2023
Date last updated
21/10/2024

Titles & IDs
Public title
Fontan Udenafil Exercise Longitudinal Assessment Trial - 2
Scientific title
Fontan Udenafil Exercise Longitudinal Assessment Trial (FUEL-2)
Secondary ID [1] 0 0
MZ_Udenafil-05
Universal Trial Number (UTN)
Trial acronym
FUEL-2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Single Ventricle Heart Disease 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Coronary heart disease
Cardiovascular 0 0 0 0
Other cardiovascular diseases
Cardiovascular 0 0 0 0
Other cardiovascular diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Udenafil
Treatment: Drugs - Placebo

Experimental: Drug - Approximately 218 subjects dosed with udenafil will be enrolled at approximately 30 sites.

Experimental: Placebo - Approximately 218 subjects dosed with matching placebo will be enrolled at approximately 30 sites.


Treatment: Drugs: Udenafil
Active drug

Treatment: Drugs: Placebo
Matching Placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Exercise Capacity
Timepoint [1] 0 0
Baseline to 26 Weeks
Secondary outcome [1] 0 0
Change in VO2 at the ventilatory anaerobic threshold (VAT)
Timepoint [1] 0 0
Baseline to 26 weeks
Secondary outcome [2] 0 0
Change in Enhanced Liver Fibrosis (ELF) Score
Timepoint [2] 0 0
Baseline to 26 weeks
Secondary outcome [3] 0 0
Work rate (watts) at ventilatory anaerobic threshold (VAT)
Timepoint [3] 0 0
Baseline to 26 weeks
Secondary outcome [4] 0 0
Change in ventilatory efficiency (VE/VCO2) at ventilatory anaerobic threshold
Timepoint [4] 0 0
Baseline to 26 weeks

Eligibility
Key inclusion criteria
1. Males and females with Fontan physiology who are 12 to less than 19 years of age at enrollment.
2. Participant consent or parental/guardian consent and participant assent.
3. Participant fluency in primary language of country in which study is being conducted.
4. Current antiplatelet or anticoagulant therapy.
Minimum age
12 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Height < 132 cm.
2. Hospitalization for acute decompensated heart failure within the last 12 months.
3. Current intravenous inotropic drugs.
4. Undergoing evaluation for heart transplantation or listed for transplantation.
5. Diagnosis of active protein losing enteropathy or plastic bronchitis within the last 3 years, or a history of liver cirrhosis.
6. Known Fontan baffle obstruction, branch pulmonary artery stenosis, or pulmonary vein stenosis resulting in a mean gradient of > 4 mmHg between the regions proximal and distal to the obstruction as measured by either catheterization or echocardiography, obtained prior to screening for the trial.
7. Single lung physiology with greater than 80% flow to one lung.
8. Peak minute oxygen consumption (VO2) less than 45% or = 80% of predicated for age and gender at enrollment.
9. Severe ventricular dysfunction assessed qualitatively by clinical echocardiography within 6 months prior to enrollment.
10. Severe valvar regurgitation, ventricular outflow obstruction, or severe aortic arch obstruction assessed by clinical echocardiography within six months prior to enrollment.
11. History of significant renal (serum creatinine > 2.0), hepatic (serum AST and/or ALT > 3 times upper limit of normal), gastrointestinal or biliary disorders that could impair absorption, metabolism or excretion of orally administered medications.
12. Inability to complete exercise testing at baseline screening.
13. History of PDE-5 inhibitor use within 12 months prior to enrollment.
14. History of any other medication for treatment of pulmonary hypertension within 3 months before study onset.
15. Known intolerance to oral udenafil.
16. Frequent use of medications or other substances that inhibit or induce CYP3A4.
17. Current use of alpha-blockers or nitrates.
18. Ongoing or planned participation in another research protocol that would either prevent successful completion of planned study testing or invalidate its results.
19. Noncardiac medical, psychiatric, and/or social disorder that would prevent successful completion of planned study testing or would invalidate its results.
20. Cardiac care, ongoing or planned, at a non-study center that would impede study completion.
21. For females: Pregnancy at the time of screening, pregnancy planned before study completion, or refusal to use an acceptable method of contraception for study duration if sexually active.
22. Unable to abstain or limit intake of grapefruit juice and grapefruit containing drinks during the duration of the trial.
23. Refusal to provide written informed consent/assent.
24. In the opinion of the investigator, the subject is likely to be non-compliant with the study protocol.
25. History of clinically significant thromboembolic event, in the option of the site Principal Investigator, that may put the subject at increased risk of a subsequent event while participating in the study.
26. Coronavirus disease 2019 (COVID-19) vaccination or symptoms of COVID-19 infection within 7 days of Visit 1.
27. Not taking antiplatelet or anticoagulant therapy.

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
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/10/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/10/2025
Actual
Sample size
Target
436
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
District of Columbia
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Georgia
Country [8] 0 0
United States of America
State/province [8] 0 0
Illinois
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Michigan
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
Nebraska
Country [13] 0 0
United States of America
State/province [13] 0 0
New York
Country [14] 0 0
United States of America
State/province [14] 0 0
North Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Ohio
Country [16] 0 0
United States of America
State/province [16] 0 0
Pennsylvania
Country [17] 0 0
United States of America
State/province [17] 0 0
South Carolina
Country [18] 0 0
United States of America
State/province [18] 0 0
Tennessee
Country [19] 0 0
United States of America
State/province [19] 0 0
Texas
Country [20] 0 0
United States of America
State/province [20] 0 0
Utah
Country [21] 0 0
United States of America
State/province [21] 0 0
Washington
Country [22] 0 0
United States of America
State/province [22] 0 0
Wisconsin
Country [23] 0 0
Korea, Republic of
State/province [23] 0 0
Gyeonggi
Country [24] 0 0
Korea, Republic of
State/province [24] 0 0
Seoul

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Mezzion Pharma Co. Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure.
Trial website
https://clinicaltrials.gov/study/NCT05918211
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
John Hariadi, MD
Address 0 0
Mezzion Pharma Co. Ltd
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
John Hariadi, MD
Address 0 0
Country 0 0
Phone 0 0
443.699.6746
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05918211