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Dilated cardiomyopathy (DCM) is the most common childhood cardiomyopathy and is associated with significant early morbidity and mortality. About half of patients die or require heart transplantation within 5 years of diagnosis. The medical therapy for DCM with heart failure includes anti-congestive medications and antiplatelet therapy. Those who fail to improve within the first year of diagnosis usually deteriorated even upon aggressive anti-congestive medications. The investigators had conducted precision-medicine-based approach to provide strategic approach as drug repurposing to identify new treatments. The investigators have identified the beneficial effects from a statin, simvastatin, to restore the cardiac contractility. The investigators would further assess the efficacy of simvastatin to improve the cardiac function in patients with DCM.
Dilated cardiomyopathy (DCM) is the most common childhood cardiomyopathy and is associated with significant early morbidity and mortality. About half of patients die or require heart transplantation within 5 years of diagnosis. The survival advantage from transplantation is limited, particularly in DCM infants.
The medical therapy for DCM with heart failure includes anti-congestive medications and antiplatelet therapy. Those who fail to improve within the first year of diagnosis usually deteriorated even upon aggressive anti-congestive medications. The investigators had conducted precision-medicine-based approach to provide strategic approach as drug repurposing to identify new treatments. The investigators have identified the beneficial effects from a statin, simvastatin, to restore the cardiac contractility in a DCM proband.The initial experience in the proband is promising.
Simvastatin is effective in lowing LDL and cholesterol, thereby to improve the outcome of patients with coronary arterial disease, familiar hypercholesterolemia, etc. For children, though the dosage range and the indication remain unclear, it had been used in children with various diseases. Simvastatin had been given in a small cohort of adult DCM. Patients treated with simvastatin had a lower New York Heart Association functional class compared with those receiving placebo. The LVEF also improved in the simvastatin group. The investigators would further assess the efficacy of simvastatin to improve the cardiac function in patients with DCM.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Simvastatin | Experimental | Simvastatin, 0.5mg/kg/d(maximum 20mg), once daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Simvastatin | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 1st month(The 1st month follow-up time tolerates a 0.5-month window ) | |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 3rd month(The 3rd month follow-up time tolerates a 1-month window ) | |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 6th month(The 6th month follow-up time tolerates a 1-month window ) | |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 9th month(The 9th month follow-up time tolerates a 1-month window ) | |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 12th month(The 12th month follow-up time tolerates a 1-month window ) | |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 15th month(The 15th month follow-up time tolerates a 1-month window ) | |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 18th month(The 18th month follow-up time tolerates a 1-month window ) | |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE v3.0 | We will check patient's biochemistry profile including, lipid profile, liver function and renal function. Treatment-related adverse events would be assessed by CTCAE v3.0 | Up to 24 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Taiwan University Hospital | Taipei | 100 | Taiwan |
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| ID | Term |
|---|---|
| D002311 | Cardiomyopathy, Dilated |
| ID | Term |
|---|---|
| D006332 | Cardiomegaly |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D009202 | Cardiomyopathies |
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| ID | Term |
|---|---|
| D019821 | Simvastatin |
| ID | Term |
|---|---|
| D008148 | Lovastatin |
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
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|
| baseline, 21st month(The 21th month follow-up time tolerates a 1-month window ) |
| Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. | baseline, 24th month(The 24th month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 3rd month(The 3rd month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 6th month(The 6th month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 9th month(The 9th month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 12th month(The 12th month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 15th month(The 15th month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 18th month(The 18th month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 21st month(The 21th month follow-up time tolerates a 1-month window ) |
| Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level. | baseline, 24th month(The 24th month follow-up time tolerates a 1-month window ) |
| D000083083 |
| Laminopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006844 |
| Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D011083 | Polycyclic Compounds |