Ignite Creation Date:
2024-10-26 @ 3:42 PM
Last Modification Date:
2024-10-26 @ 3:42 PM
Study NCT ID:
NCT06632834
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-10-07
Brief Title:
Outcome-targeted Therapy Principle and Outcome Evaluation Clinical Study and Phenotype-genotype Correlation
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Outcome-targeted Therapy Principle and Outcome Evaluation Clinical Study and Phenotype-genotype Correlation
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Brief SummaryUse 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 probands 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
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 probands 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
Detailed Description:
Dilated cardiomyopathy DCM is the most common childhood cardiomyopathy and is associated with significant early morbidity and mortality About half of patients die or require heart transplantation within 5 years of diagnosis The survival advantage from transplantation is limited particularly in DCM infantsThe medical therapy for DCM with heart failure includes anti-congestive medications and antiplatelet therapy Those who fail to improve within the first year of diagnosis usually deteriorated even upon aggressive anti-congestive medications Investigators have 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 probands father The mutant mouse consequently confirmed the beneficial effects The initial experience in the proband is promising The proband was diagnosed as DCM with severe heart failure during infancy Family screening identified that the probands father had dilated left ventricle LV and low LV ejection fraction LVEF Genetic examination of this DCM family revealed that the proband and the probands father had heterogeneous missense mutation About 2 years after the disease onset participants growth was slow the LVEF remained poor and the NT-pro BNP level was high under aggressive anti-congestive medications Because of the beneficial effects of simvastatin from in vitro study and in vivo animal study off-label use of simvastatin was initiated after obtaining the parents agreement Participants general condition and cardiac condition 4 years after the disease onset and 15 years after the simvastatin therapy improved without adverse effects The LVEF increased from 175 to 392 and the NT-pro BNP level dropped from 1200 to 363 pgml Simvastatin is effective in lowing LDL and cholesterol thereby to improve the outcome of patients with coronary arterial disease familiar hypercholesterolemia etc For children though the dosage range and the indication remain unclear it had been used in children with various diseases Simvastatin had been given in a small cohort of adult DCM Patients treated with simvastatin had a lower New York Heart Association functional class compared with those receiving placebo The LVEF also improved in the simvastatin group This clinical trial is to find out if simvastatin will benefit the cardiac function of DCM patients
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None