Simvastatin Therapy in Patients With Dilated Cardiomyopathy.

NCT03775070 | Completed Phase 2

• 1 other identifier: 201806014MINB
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

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 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. The investigators had conducted precision-medicine-based approach to provide strategic approach as drug repurposing to identify new treatments. The investigators have identified the beneficial effects from a statin, simvastatin, to restore the cardiac contractility. The investigators would further assess the efficacy of simvastatin to improve the cardiac function in patients with DCM.

Conditions

Dilated Cardiomyopathy

Outcome Measures

Primary Outcomes (17)

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 1st month(The 1st month follow-up time tolerates a 0.5-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 3rd month(The 3rd month follow-up time tolerates a 1-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 6th month(The 6th month follow-up time tolerates a 1-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 9th month(The 9th month follow-up time tolerates a 1-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 12th month(The 12th month follow-up time tolerates a 1-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 15th month(The 15th month follow-up time tolerates a 1-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 18th month(The 18th month follow-up time tolerates a 1-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 21st month(The 21th month follow-up time tolerates a 1-month window )

• Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.

  baseline, 24th month(The 24th month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 3rd month(The 3rd month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 6th month(The 6th month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 9th month(The 9th month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 12th month(The 12th month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 15th month(The 15th month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 18th month(The 18th month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 21st month(The 21th month follow-up time tolerates a 1-month window )

• Change from baseline in N-terminal pro-brain natriuretic peptide level.natriuretic peptide level.

  baseline, 24th month(The 24th month follow-up time tolerates a 1-month window )

Secondary Outcomes (1)

• Number of participants with treatment-related adverse events as assessed by CTCAE v3.0

  Up to 24 months

Study Arms (1)

Simvastatin

EXPERIMENTAL

Simvastatin, 0.5mg/kg/d(maximum 20mg), once daily

Drug: Simvastatin

Interventions

Simvastatin DRUG

* Starting dosage: in adult, the dose of simvastatin is 10 mg once daily. In children, the dose is 0.25mg/Kg/day (maximum dose: 10 mg/d). * Target dosage: in adult, the dose of simvastatin is 20 mg once daily. In children, the dose is 0.5mg/Kg/day (maximum dose: 20 mg/d). * The basic anti-congestive medication will be kept as the same. * The dosage may be titrated to a lesser dose by investigators according to the patient's condition.

Simvastatin

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients who have already received anti-congestive medications for at least three months and still have compromised LV function (LVEF \< 45% and the Z score of the LV end-diastolic diameter \> 2.0).
• Patients who have persistent or even worsening heart failure after one month of anti-congestive medications.
• Patients who have positive family history of dilated cardiomyopathy and have received anti-congestive medications for one week.

You may not qualify if:

• Patients who underwent prior cardiac surgery. Those who received DCM related cardiac surgery, such as mitral valve plasty, for longer than a year are not subject to this restriction.
• Patients who had liver / renal dysfunction.
• Patients who are pregnant or plan to pregnancy in the period of study.
• Patients who are intolerance to simvastatin therapy.

Sponsors & Collaborators

• National Taiwan University Hospital: lead

Study Sites (1)

National Taiwan University Hospital

Taipei, 100, Taiwan

Location

MeSH Terms

Conditions

Cardiomyopathy, Dilated

Interventions

Simvastatin

Condition Hierarchy (Ancestors)

Cardiomegaly; Heart Diseases; Cardiovascular Diseases; Cardiomyopathies; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Lovastatin; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2018
• First Posted: December 13, 2018
• Study Start: January 17, 2019
• Primary Completion: July 31, 2022
• Study Completion: July 31, 2022
• Last Updated: August 3, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

TW (1)