Actively Recruiting
Outcome-targeted Therapy: Principle and Outcome Evaluation: Clinical Study and Phenotype-genotype Correlation
Led by National Taiwan University Hospital · Updated on 2024-10-16
230
Participants Needed
1
Research Sites
231 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Outcome-targeted Therapy: Principle and Outcome Evaluation: Clinical Study and Phenotype-genotype Correlation
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with dilated cardiomyopathy and classified as NYHA functional class II or III
- Left ventricular ejection fraction (LVEF) less than 45% and LV end-diastolic diameter Z score greater than 2.0
- Normal or high levels of cholesterol and triglyceride
- Either have received anti-congestive medications for at least three months with poor LV function, or have worsening heart failure after one month of such medications, or have a positive family history of DCM and have been on anti-congestive medications for one month
- Patients or their parents must sign informed consent
You will not qualify if you...
- Prior cardiac surgery (except DCM-related surgery done over a year ago)
- Active liver or kidney dysfunction
- Use of gemfibrozil, cyclosporine, danazol, strong CYP3A4 inhibitors, or cobicistat-containing products
- Pregnancy or planning pregnancy during the study period
- Intolerance to simvastatin therapy
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Taiwan University Hospital
Taipei County, Zhongzheng District, Taiwan, 100
Actively Recruiting
Research Team
W
Wei-Chieh Tseng
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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