Using simvastatin to improve heart function in children with dilated cardiomyopathy

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

This study is testing if simvastatin can help improve heart function in children with dilated cardiomyopathy who haven't responded well to other treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 230 (estimated)
• Ages: 0 Years to 99 Years
• Sex: All
• Sponsor: National Taiwan University Hospital (Academic / other)
• Locations: 1 site (Taipei County, Zhongzheng District)
• Trial ID: NCT06632834 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effects of simvastatin, a statin medication, on improving cardiac function in children diagnosed with dilated cardiomyopathy (DCM). The study aims to enroll patients who have not responded adequately to standard anti-congestive medications and have low left ventricular ejection fraction. By utilizing a precision-medicine approach, the trial seeks to determine if simvastatin can enhance heart contractility based on previous promising results from laboratory studies. Participants will be monitored for improvements in heart function over the course of the trial.

Who should consider this trial

Good fit: Ideal candidates include children diagnosed with dilated cardiomyopathy who have low left ventricular ejection fraction and have not responded to standard treatments.

Not a fit: Patients who have severe comorbidities or those who do not meet the specific inclusion criteria for heart function may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve heart function and quality of life for children suffering from dilated cardiomyopathy.

How similar studies have performed: While the approach of using simvastatin in this context is novel, preliminary laboratory studies have shown promising results, indicating potential for success.

Eligibility criteria

Inclusion criteria

Patients diagnosed as dilated cardiomyopathy, classified as NYHA functional class II or III and still have a low left ventricular ejection fraction (LVEF) (LVEF \< 45% and the Z score of the LV end-diastolic diameter \> 2.0) will be enrolled, if they have normal or high level of cholesterol and triglyceride and fulfill any of the following criteria:

1. Patients who have already received anti-congestive medications for at least three months and still have poor LV function (LVEF \< 45% and the Z score of the LV end-diastolic diameter \> 2.0).
2. Patients who have persistent or even worsening heart failure after one month of anti-congestive medications.
3. Patients who have positive family history of dilated cardiomyopathy and have received anti-congestive medications for one month.
4. Patients or their parents must sign an informed consent form.

Exclusion criteria

Patients who fulfill any of the following criteria will be excluded from the trial:

1. Patients who underwent prior cardiac surgery. Those who received DCM related surgery, such as mitral valve plasy, for longer than a year are not subject to this restriction.
2. Patients who had active liver / renal dysfunction.
3. Concomitant use with gemfibrozil, cyclosporine, danazol, strong CYP3A4 inhibitors (eg, boceprevir, clarithromycin, erythromycin, HIV protease inhibitors, itraconazole, ketoconazole, nefazodone, posaconazole, telaprevir, telithromycin, voriconazole), or cobicistat-containing products
4. Patients who are pregnant or plan to pregnancy in the period of study.
5. Patients who are intolerance to simvastatin therapy.

Where this trial is running

Taipei County, Zhongzheng District

• National Taiwan University Hospital — Taipei County, Zhongzheng District, Taiwan (Recruiting)

Study contacts

• Principal investigator: Wei-Chieh Tseng, MD — Principal Investigator
• Study coordinator: Wei-Chieh Tseng

Conditions Dilated Cardiomyopathy; cardiomyopathy