Effect of Sildenafil on Left Ventricular Function in Pediatric With Primary Dilated Cardiomyopathy (AssiutU)

April 12, 2026 updated by: kholoudali, Assiut University

Effect of Sildenafil on Left Ventricular Function in Pediatric Patients With Primary Dilated Cardiomyopathy Prospective Cohort Study

Dilated cardiomyopathy (DCM) is a clinical diagnosis characterized by left ventricular or biventricular dilation and impaired contraction that is not explained by abnormal loading conditions.(1) Both inherited predisposition and environmental factors play an important part in the natural history of disease.(1) Cardiomyopathies are group of heart diseases that influence cardiac muscles directly and are not related to hypertension, congenital, valvular and pericardial diseases. The most common type of cardiomyopathy is dilated cardiomyopathy (DCM).(2) Definitions of DCM provided by two major professional organizations (American Heart Association (AHA) and European Society of Cardiology (ESC)) Both organizations agreed that DCM could be clinically defined by the presence of left ventricular dilation and contractile dysfunction in the absence of abnormal loading conditions and severe coronary artery disease.(3) Dilated cardiomyopathy usually manifests as chronic systolic heart failure, which is detected by echocardiography as impaired left ventricle fraction shortening less than 28%, with left ventricular end diastolic dimension Z-score>2 ,leading to arrhythmias and sudden death.(4,5) idiopathic dilated cardiomyopathy (DCM) is characterized by dilatation and impaired contraction of the left ventricle or both ventricles, in the absence of underlying causes such as CAD, valve disease, congenital heart disease, or pericardial disease. Most patients present with symptoms of heart failure or arrhythmias, or even sudden cardiac death. An extensive family history (pedigree covering three or four generations), in combination with cardiological screening of first-degree relatives, results in a diagnosis of a familial form of DCM in up to 35% of cases.(11) In a multisite study in the USA and Canada, DCM was the most common form of cardiomyopathy among children (individuals of <18 years of age):66% had idiopathic DCM, whereas of those with DCM due to known causes, 46% had myocarditis and 26% had neuromuscular disease. The annual incidence of DCM in children ranges from 0.18 to 0.73 per 100,000 person-years.(1) Oral PDE5 inhibitors, which primarily include sildenafil, vardenafil ,and tadalafil. Owing to its vasodilation effect and its impact on the endothelial function of blood vessels in the body, it is possible to use them to treat cardiovascular disorders such as pulmonary arterial hypertension and dilated cardiomyopathy. The use of PDE5 inhibitors in HF patients is backed by various theoretical evidence and clinical trials have begun to investigate their potential as an adjunct in the pharmacological management of HF.(6) PDE5 inhibition is an intriguing pharmacological strategy that enhances in vivo NO signaling by increasing the cyclic guanosine monophosphate (cGMP) availability. A number of theoretical backgrounds support the use of PDE5 inhibitors in HF, and several recent clinical studies have tested its clinical viability as a potential adjunct in the pharmacological management of HF.(7) In chronic heart failure improvement in exercise ventilation and aerobic efficiency with sildenafil is sustained and is significantly related with an endothelium-mediated attenuation of exercising muscle over signaling. Chronic sildenafil seems to be a remedy based on CHF pathophysiology and devoid of remarkable adverse effects.(8) Sildenafil is a specific inhibitor of type 5 phosphodiesterase (PDE5) that increases nitric oxide availability and nitric oxide-mediated vasodilation in CHF patients). Interest has therefore been focused on the potential of sildenafil to be beneficial in CHF. In acute studies, sildenafil increased myocardial contractility ,blunted adrenergic stimulation ,reduced left ventricular afterload), and improved lung diffusion capacity ,pulmonary hemodynamics at rest) and on exertion ,and exercise ventilation efficiency and aerobic performance.(8)

Study Overview

Status

Not yet recruiting

Conditions

Detailed Description

Studies with sildenafil in the mdx mouse model support the concept that PDE5 inhibition may have favorable effects to prevent or ameliorate cardiac dys¬function associated with dystrophin deficiency (8, 9). Prophylactic treatment of dogs with golden retriever muscular dystrophy (GRMD) with tadalafil preserved cardiac systolic function, improved measures of circum¬ferential wall strain, and reduced histopathology of car¬diac muscle compared to untreated GRMD animals (9). Congestive heart failure (CHF) occurs when the heart can no longer maintain the baseline metabolic demand triggered by physiological venous pressure. In the pediatric population, it corresponds to a complex entity with multiple etiologies, depending on the age group assessed, being mainly associated with congenital structural malformations, cardiomyopathies, or secondary to arrhythmogenic, infectious, ischemic, toxic or infiltrative events1. cardiomyopathy is the major factor associated with CHF, with a 40% incidence of CHF reported in those patients1. According to Freitas Jr. et al9, the acute administration of sildenafil and sodium nitroprusside has been associated with reverse cardiac remodeling, a reduction in right cardiac chambers and an improvement in biventricular cardiac function9. The use of sildenafil improves oxygen uptake, cardiac index, depression, and quality of life, and reduces aortic stiffness and systemic vascular resistance in patients with CHF.(10)

Study Type

Observational

Enrollment (Estimated)

70

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

Children aged 1-18 years with a diagnosis of primary dilated cardiomyopathy confirmed by echocardiography, recruited from inpatient and outpatient pediatric cardiology services at Assiut University Children Hospital, Egypt.

Description

Inclusion Criteria:- Children from 1-18 year's age, diagnosed primary dilated cardiomyopathy by echocardiography and are admitted at Pediatric Cardiology unit or attend Pediatric Cardiology outpatient clinic in Assiut University children hospital.

  • Patient on anti-failure therapy consistent with pediatric heart failure guidelines.

Exclusion Criteria:. Patients less than 1 year old

- Patients with impaired left ventricular systolic function due to other causes like: i. Congenital heart diseases (anomalous origin of the left coronary artery from the pulmonary artery (ALCAPA), severe coarctation of aorta, critical aortic stenosis, metabolic disorders as mitochondrial dysfunction or storage diseases.

ii. Acquired heart diseases as myocarditis, Kawasaki, or arrhythmias

  • Post-Operative left ventricular dysfunction
  • Known hypersensitivity or contraindications to sildenafil as hypotension (<90/50 mmHg), or severe hepatic/renal impairment or optic neuropathy.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
sildenafil & standard therapy
pt receving sildenafil
stadard therapy only

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change in Left Ventricular Ejection Fraction (EF%)
Time Frame: Baseline, 6 months, and 12 months
Baseline, 6 months, and 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Left Ventricular Fractional Shortening (FS%)
Time Frame: Baseline, 6 months, and 12 months
by clinical and echo measurements
Baseline, 6 months, and 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assiut University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

May 10, 2026

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

May 30, 2027

Study Registration Dates

First Submitted

April 12, 2026

First Submitted That Met QC Criteria

April 12, 2026

First Posted (Actual)

April 17, 2026

Study Record Updates

Last Update Posted (Actual)

April 17, 2026

Last Update Submitted That Met QC Criteria

April 12, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • sildenafil on DCM

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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