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

NCT06632834 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 230

Last updated 2024-10-16

No results posted yet for this study

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.

Conditions

  • Dilated Cardiomyopathy

Interventions

DRUG

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.

Sponsors & Collaborators

  • National Taiwan University Hospital

    lead OTHER

Principal Investigators

  • Wei-Chieh Tseng, MD · Principal Investigator

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-08-15
Primary Completion
2025-01-19
Completion
2025-01-19

Countries

  • Taiwan

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06632834 on ClinicalTrials.gov