Dilated CardiomyopathyPhase 2SavDCMNCT03775070
Simvastatin Therapy in Patients With Dilated Cardiomyopathy.
A Phase 2 study testing Simvastatin in people with Dilated Cardiomyopathy, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Dilated Cardiomyopathy
- What is being tested?
- Simvastatin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteTaiwan
- Study dates
- 17 Jan 2019to 31 Jul 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open Label, Single-armed, Exploratory Study of Simvastatin Therapy on the Cardiac Function in Patients With Dilated Cardiomyopathy.
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.
Read the 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 infants.
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 in a DCM proband.The initial experience in the proband is promising.
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. The investigators would further assess the efficacy of simvastatin to improve the cardiac function in patients with DCM.
- Conditions
- Dilated Cardiomyopathy
- Being tested
- Simvastatin
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SimvastatinDrug
- 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.
| Study group | Role | What they receive |
|---|---|---|
| Simvastatin | Experimental | Simvastatin, 0.5mg/kg/d(maximum 20mg), once daily Receives: Drug: Simvastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
We will check patient's biochemistry profile including, lipid profile, liver function and renal function. Treatment-related adverse events would be assessed by CTCAE v3.0
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2018
- Study start17 Jan 2019
- Primary completion31 Jul 2022
- Study completion31 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dilated CardiomyopathyStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03775070
- Sponsor's protocol ID
- 201806014MINB
- Acronym
- SavDCM
- Status last verified
- Aug 2022
- First submitted
- 3 Dec 2018
- Last updated
- 3 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Single-armed, Exploratory Study of Simvastatin Therapy on the Cardiac Function in Patients With Dilated Cardiomyopathy.
- ClinicalTrials.gov ID
- NCT03775070
- Acronym / sponsor ID
- SavDCM / 201806014MINB
- Conditions
- Dilated Cardiomyopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- 17 Jan 2019 (actual)
- Primary completion
- 31 Jul 2022 (actual)
- Study completion
- 31 Jul 2022 (actual)
- First posted
- 13 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 infants.
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 in a DCM proband.The initial experience in the proband is promising.
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. The investigators would further assess the efficacy of simvastatin to improve the cardiac function in patients with DCM.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Simvastatin | Experimental | Drug: SimvastatinSimvastatin, 0.5mg/kg/d(maximum 20mg), once daily |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Simvastatin* 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. | Drug | Simvastatin |
Primary outcome
Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: baseline, 24th month(The 24th month follow-up time tolerates a 1-month window )
1 secondary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v3.0
Time frame: Up to 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Simvastatin in people with Dilated Cardiomyopathy, run by National Taiwan University Hospital at 1 site in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from base line in left ventricular ejection fraction and end-diastolic dimention by cardiac ultrasound. (time frame: baseline, 1st month(The 1st month follow-up time tolerates a 0.5-month window )). The study lists 17 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2022.