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Outcome-targeted Therapy: Principle and Outcome Evaluation: Clinical Study and Phenotype-genotype Correlation
Sponsor: National Taiwan University Hospital
Summary
Brief Summary(Use lay language. Include a statement of the study hypothesis.): Dilated cardiomyopathy (DCM) is the most common childhood cardiomyopathy and is associated with significant early morbidity and mortality. Those who fail to improve within the first year of diagnosis usually deteriorated even upon aggressive anti-congestive medications. Investigators had conducted precision-medicine-based approach to provide strategic approach as drug repurposing to identify new treatments. Investigators have identified the beneficial effects from a statin, simvastatin, to restore the cardiac contractility in the human induced pluripotent stem cell lines derived from a DCM proband and the proband's father. The mutant mouse consequently confirmed the beneficial effects. The initial experience in the proband is promising. This clinical trial is to find out if simvastatin will benefit the cardiac function of DCM patients.
Official title: Outcome-targeted Therapy: Principle and Outcome Evaluation: Clinical Study, and Phenotype-genotype Correlation
Key Details
Gender
All
Age Range
0 Years - 99 Years
Study Type
INTERVENTIONAL
Enrollment
230
Start Date
2020-08-15
Completion Date
2025-01-19
Last Updated
2024-10-16
Healthy Volunteers
No
Conditions
Interventions
simvastatin therapy
Simvastatin should be administered initially with the dose of 10 mg once daily for adult and 0.25 mg/Kg once daily for children. Dose of simvastatin will be increased to the dose of 20 mg once daily for adult and 0.5 mg/Kg once daily for children after the check at 3 months without adverse effects.
Locations (1)
National Taiwan University Hospital
Taipei County, Zhongzheng District, Taiwan