Efficacy of Phosphodiesterase-type 5 Inhibitors in Patients With Univentricular Congenital Heart … (NCT03997097) | Clinical Trial Compass
WithdrawnPhase 3
Efficacy of Phosphodiesterase-type 5 Inhibitors in Patients With Univentricular Congenital Heart Disease
Stopped: New sponsor
0Started 2023-06-01
Plain-language summary
In univentricular hearts, selective lung vasodilators such as phosphodiesterase type 5 (PDE5) inhibitors would decrease pulmonary resistance and improve exercise tolerance. However, the level of evidence for the use of PDE5 inhibitors in patients with a single ventricle (SV) remains limited. the investigators present the SV-INHIBITION study rationale, design and methods.The SV-INHIBITION trial is a nationwide multicentre, randomised, double blind, placebo-controlled, phase III study, aiming to evaluate the efficacy of sildenafil on the ventilatory efficiency during exercise, in teenagers and adult patients (\>15 y.o.) with a SV. Patients with pulmonary arterial hypertension (mean pulmonary arterial pressure (mPAP) \> 15 mmHg and trans-pulmonary gradient \> 5 mmHg) measured by cardiac catheterisation, will be eligible. The primary outcome is the variation of the VE/VCO2 slope, measured by a cardiopulmonary exercise test, between baseline and 6 months of treatment. A total of 50 patients are required to observe a decrease of 5 ± 5 points in the VE/VCO2 slope, with a power of 90% power and an alpha risk of 5%. The secondary outcomes are: clinical outcomes, 6 minute walk test, SV function, NT Pro BNP, VO2max, stroke volume, mPAP, trans-pulmonary gradient, SF36 quality of life score, safety and acceptability. This study aims to answer the question whether PDE5 inhibitors should be prescribed in patients with a SV. This trial has been built focusing on the 3 levels of research defined by the WHO: disability (exercise tolerance), deficit (SV function), and handicap (quality of life).
Who can participate
Age range15 Years – 80 Years
SexALL
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Inclusion criteria
✓. 15 years of age and over.
✓. Patient's weight over 20 kg
✓. Patients with CHD with a single ventricular type defined by the classification of congenital heart diseases in Orphanet (53).
✓. PAH defined by diagnostic catheterization with mean PAP \> 15 mmHg and a trans-pulmonary gradient \> 5 mmHg, performed as part of the usual follow-up. No definition of PAH in SV is available as a result of a particular physiology. Therefore, we chose the 15mmHg cut-off, which is used in clinical routine to allow or contra-indicate the Fontan procedure \[50,51\].
✓. Appropriate written informed consent (adult patients, legal parents for teenagers), and formal assent (teenagers), should to be provided.
✓. Beneficiary of a health insurance.
Exclusion criteria
✕. Patient who is unable to perform a cardio-pulmonary exercise test.
. Patient treated by any pulmonary arterial vasodilator drug, as defined in the 2015 PH guidelines (52), within 6 months before inclusion, regardless the duration and the type(s) (oral, intravenous, subcutaneous, inhaled) of administration.
✕. Patient treated by Sildenafil or any other type of phosphodiesterase-type 5 inhibitor (such as tadalafil) within 6 months before inclusion, regardless the duration of administration.
✕. Interventional cardiac catheterization planned during the trial (collateral occlusion, fenestration occlusion, stenting, angioplasty, ablation of rhythm disorder), other than during the screening.
✕. Participation in another clinical trial or administration of an off-label drug in the 4 weeks preceding the screening.
✕. Pregnancy, desire for pregnancy, absence of contraception during the study period.
✕. Severe hepatic insufficiency (Child-Pugh C class).