Outcome-targeted Therapy: Principle and Outcome Evaluation: Clinical Study and Phenotype-genotype Correlation

NCT06632834 PHASE2 RECRUITING

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.

Details

Lead sponsor	National Taiwan University Hospital
Phase	PHASE2
Status	RECRUITING
Enrolment	230
Start date	Sat Aug 15 2020 00:00:00 GMT+0000 (Coordinated Universal Time)
Completion	Sun Jan 19 2025 00:00:00 GMT+0000 (Coordinated Universal Time)

Conditions

• Dilated Cardiomyopathy

Interventions

• simvastatin therapy

Countries

Taiwan