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

NCT07536880 | Not Yet Recruiting

• 1 other identifier: sildenafil on DCM
• Study Type: observational
• Target: 70
• Locations: 0 countries
• Sites: N/A

Brief Summary

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)

Conditions

Cardiomyopathy, Dilated

Outcome Measures

Primary Outcomes (1)

• Change in Left Ventricular Ejection Fraction (EF%)

  Baseline, 6 months, and 12 months

Secondary Outcomes (1)

• Change in Left Ventricular Fractional Shortening (FS%)

  Baseline, 6 months, and 12 months

Study Arms (2)

sildenafil & standard therapy

pt receving sildenafil

stadard therapy only

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

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

You may not qualify if:

• \- 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.

Sponsors & Collaborators

• Assiut University: lead

Related Publications (5)

• Cox D, Byrne B, Hammers DW, Landry J, Sweeney HL. Effect of Tadalafil on cardiac function and left ventricular dimensions in Duchenne muscular dystrophy: safety and cardiac MRI substudy results from a randomized, placebo-controlled trial. BMC Cardiovasc Disord. 2025 Apr 11;25(1):276. doi: 10.1186/s12872-025-04727-3.

  PMID: 40217158 BACKGROUND

• Abdelaziz SM, Hussein RRS, El Mokadem M, Mahmoud HB. Clinical and hemodynamic effects of oral sildenafil on biventricular function on patients with left ventricular systolic dysfunction. Int J Clin Pract. 2021 Jul;75(7):e14171. doi: 10.1111/ijcp.14171. Epub 2021 Mar 30.

  PMID: 33764636 BACKGROUND

• Maron BJ, Towbin JA, Thiene G, Antzelevitch C, Corrado D, Arnett D, Moss AJ, Seidman CE, Young JB; American Heart Association; Council on Clinical Cardiology, Heart Failure and Transplantation Committee; Quality of Care and Outcomes Research and Functional Genomics and Translational Biology Interdisciplinary Working Groups; Council on Epidemiology and Prevention. Contemporary definitions and classification of the cardiomyopathies: an American Heart Association Scientific Statement from the Council on Clinical Cardiology, Heart Failure and Transplantation Committee; Quality of Care and Outcomes Research and Functional Genomics and Translational Biology Interdisciplinary Working Groups; and Council on Epidemiology and Prevention. Circulation. 2006 Apr 11;113(14):1807-16. doi: 10.1161/CIRCULATIONAHA.106.174287. Epub 2006 Mar 27.

  PMID: 16567565 BACKGROUND

• Myers MC, Breznen B, Zhong Y, Maruyama S, Bueno C, Bastien A, Fazeli MS, Golchin N. Diverse Concepts in Definitions of Dilated Cardiomyopathy: Theory and Practice. Cardiol Res. 2024 Oct;15(5):319-329. doi: 10.14740/cr1679. Epub 2024 Sep 16.

  PMID: 39420975 BACKGROUND

• Schultheiss HP, Fairweather D, Caforio ALP, Escher F, Hershberger RE, Lipshultz SE, Liu PP, Matsumori A, Mazzanti A, McMurray J, Priori SG. Dilated cardiomyopathy. Nat Rev Dis Primers. 2019 May 9;5(1):32. doi: 10.1038/s41572-019-0084-1.

  PMID: 31073128 BACKGROUND

MeSH Terms

Conditions

Cardiomyopathy, Dilated

Condition Hierarchy (Ancestors)

Cardiomegaly; Heart Diseases; Cardiovascular Diseases; Cardiomyopathies; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Nagwa Ali Mohamed, PHD

CONTACT

01096260950; nammaali2015@aun.edu.eg

Amr mohamed Kotb, PHD

CONTACT

01001936031; amrkotb336@yahoo.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: principle investigator

Study Record Dates

• First Submitted: April 12, 2026
• First Posted: April 17, 2026
• Study Start (Estimated): May 10, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): May 30, 2027
• Last Updated: April 17, 2026

Record last verified: 2026-04