Cardiomyopathy, DilatedAssiutUNCT07536880
Effect of Sildenafil on Left Ventricular Function in Pediatric With Primary Dilated Cardiomyopathy
An observational study of Cardiomyopathy, Dilated, run by Assiut University.
Trial 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)… Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Cardiomyopathy, Dilated
- How many people
- 70 participants
- Where
- No sites listed yet
- Study dates
- 10 May 2026to 30 May 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
None listed.
Who cannot join
- 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.
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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)
Read the 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)
- Conditions
- Cardiomyopathy, Dilated
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| sildenafil & standard therapy | pt receving sildenafil | |
| stadard therapy only | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Left Ventricular Ejection Fraction (EF%)
Measured: Baseline, 6 months, and 12 months
Secondary outcomes (1)
Change in Left Ventricular Fractional Shortening (FS%)
by clinical and echo measurements
Measured: Baseline, 6 months, and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2026
- Study start10 May 2026 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion30 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Cardiomyopathy, DilatedStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, kholoudali, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07536880
- Sponsor's protocol ID
- sildenafil on DCM
- Acronym
- AssiutU
- Status last verified
- Apr 2026
- First submitted
- 12 Apr 2026
- Last updated
- 17 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 40217158Abdelaziz 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.
PubMed 33764636Maron 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.
PubMed 16567565Myers 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.
PubMed 39420975Schultheiss 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.
PubMed 31073128
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Sildenafil on Left Ventricular Function in Pediatric Patients With Primary Dilated Cardiomyopathy Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT07536880
- Acronym / sponsor ID
- AssiutU / sildenafil on DCM
- Conditions
- Cardiomyopathy, Dilated
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 10 May 2026 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 30 May 2027 (estimated)
- First posted
- 17 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| sildenafil & standard therapy | – | –pt receving sildenafil |
| stadard therapy only | – | – |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (0 listed)
Key exclusion criteria (4 listed)
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in Left Ventricular Ejection Fraction (EF%)
Time frame: Baseline, 6 months, and 12 months
1 secondary outcome
Change in Left Ventricular Fractional Shortening (FS%)
Time frame: Baseline, 6 months, and 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2026.
- Background: PubMed 40217158Cox 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.
- Background: PubMed 33764636Abdelaziz 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.
- Background: PubMed 16567565Maron 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.
- Background: PubMed 39420975Myers 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.
- Background: PubMed 31073128Schultheiss 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
An observational study of Cardiomyopathy, Dilated, run by Assiut University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 17 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Nagwa Ali Mohamed, PHD (01096260950, nammaali2015@aun.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Left Ventricular Ejection Fraction (EF%) (time frame: Baseline, 6 months, and 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07536880, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2026.