SILDENAFIL for Univentricular heart | Fontan procedure

Inclusion criteria

• {"criterion_text":"- Functionally univentricular heart"}
• {"criterion_text":"- Post-Fontan operation condition with the creation of a total cavo-pulmonary connection (TCPC) either extracardiac [extracardiac conduit] or intracardiac [lateral intra-atrial tunnel]"}
• {"criterion_text":"- No clinical indications for cardiac catheterization"}
• {"criterion_text":"- Age > 18 years"}
• {"criterion_text":"- Informed consent to participate in the study"}

Exclusion criteria

• {"criterion_text":"- Patients with a functionally univentricular heart within 6 months of their last cardiac operation or intravascular procedure"}
• {"criterion_text":"- Patients with concomitant diagnoses of: a) cardiovascular events within the last 3 months (acute coronary syndrome, sudden cardiac arrest, ventricular arrhythmias, intracranial hemorrhage, stroke), b) pulmonary embolism, c) infections, d) active bleeding complications, e) severe liver failure (Child-Pugh class C), f) active gastric and/or duodenal peptic ulcer disease, g) malignancy, h) primary muscle and nervous system disease, i) systemic connective tissue disease, j) myocarditis and/or pericarditis, k) predisposition to priapism, sclerosis of the corpus cavernosum or Peyronie's disease, l) sickle cell anemia, leukemia, or multiple myeloma, m) loss of vision in one eye due to non-arteritic anterior ischemic optic neuropathy (NAION), n) enteropathy with protein loss"}
• {"criterion_text":"- Pregnancy (positive pregnancy test result), planning pregnancy, breastfeeding"}
• {"criterion_text":"- Non-use of contraception during the study - applies to women of reproductive age"}
• {"criterion_text":"- Severe central nervous system damage"}
• {"criterion_text":"- Taking products containing nitric oxide or nitrates in any form (e.g., nitroglycerin, isosorbide mononitrate, isosorbide dinitrate, amyl nitrate, pentaerythritol tetranitrate); use in combination with the most potent CYP3A4 inhibitors (e.g., ketoconazole, itraconazole, ritonavir)"}
• {"criterion_text":"- Patients who were taking pulmonary arterial dilators (sildenafil, tadalafil, riociguat, bosentan, macitentan, ambrisentan, epoprostenol, iloprost, selixipag) in the 6 month period preceding study entry, regardless of the duration of therapy and route of administration (oral, intravenous, subcutaneous, inhalated)"}
• {"criterion_text":"- Patients unable to undergo egrospirometric examination"}
• {"criterion_text":"- Advanced heart failure (NYHA IV)"}
• {"criterion_text":"- Resting arterial oxygen saturation <85%"}
• {"criterion_text":"- Chronic kidney disease (creatinine > 150 µmol/l)"}
• {"criterion_text":"- Liver damage manifested by a twofold increase in transaminases above the norm"}
• {"criterion_text":"- Uncontrolled hypotension (blood pressure < 90/50 mmHg) or risk of hypotension (dehydration, systemic ventricular outflow tract obstruction, autonomic nervous system dysfunction, patients taking alpha-blockers)"}
• {"criterion_text":"- Atrial arrhythmia preventing the execution/interpretation of the above-mentioned tests or a life-threatening exercise-induced arrhythmia history"}

Primary endpoints

• {"endpoint_text":"- Increase in peak oxygen uptake (V02max) in ergospirometry test after 24 weeks of treatment","definition_or_measurement_approach":"Measured as peak oxygen uptake (VO2max) using ergospirometry at 24 weeks of treatment."}

Secondary endpoints

• {"endpoint_text":"- Increase in the amount of exercise performed, measured in metabolic equivalents (METS) during cardiopulmonary exercise testing (CPET) after 24 weeks of treatment","definition_or_measurement_approach":"Measured as change in METS during CPET at 24 weeks."}
• {"endpoint_text":"- Increase in the distance covered in the 6-minute walk test (6MWT)","definition_or_measurement_approach":"Measured as change in 6MWT distance (meters); timing/visit after 24 weeks of treatment implied."}
• {"endpoint_text":"- Increase in the absolute value of global longitudinal strain (GLS) in echocardiography after 24 weeks of treatment","definition_or_measurement_approach":"Measured as change in GLS by echocardiography at 24 weeks."}
• {"endpoint_text":"- Reduction in serum Nt-proBNP levels after 24 weeks of treatment","definition_or_measurement_approach":"Measured as change in serum NT-proBNP concentration at 24 weeks."}
• {"endpoint_text":"- Reduction in fibrosis score on elastography (abdominal ultrasound) after 24 weeks of treatment","definition_or_measurement_approach":"Measured as change in liver fibrosis score by abdominal elastography at 24 weeks."}
• {"endpoint_text":"- Increase in the quality of life index as assessed by the SF-36 questionnaire after 24 weeks of treatment","definition_or_measurement_approach":"Measured as change in SF-36 quality of life index at 24 weeks."}

Poland

Earliest CTIS Part Ii Submission Date

23-08-2024

Latest Decision Or Authorization Date

22-09-2024

Processing Time Days

30

Number Of Sites

1

Number Of Participants

60

Primary sponsor

Full Name

National Institute Of Cardiology

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Poland

Third parties

• {"country":"Poland","full_name":"Zakłady Farmaceutyczne \"POLPHARMA\" Spółka Akcyjna","duties_or_roles":"Monetary support; authorisation holder for Remidia product used in the trial","organisation_type":""}