Safety Study of CNT-01 in Patients With Idiopathic TGCV

Study Overview
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Brief Summary
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The purpose of this study is to assess the safety and to examine the index of the efficacy after repeated dosing of CNT-01 in patients with idiopathic triglyceride deposit cardiomyovasculopathy.

Official Title
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Phase I/IIa Study of CNT-01 in Patients With Idiopathic Triglyceride Deposit

Conditions
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Idiopathic Triglyceride Deposit Cardiomyovasculopathy

Intervention / Treatment
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* Drug: CNT-01 500 mg capsule


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have a definite diagnosis of idiopathic triglyceride deposit cardiomyovasculopathy After receiving a sufficient explanation upon participation in this study, on a full understanding, patients who obtained the written informed consent from each patient More than 20 years old at the time of informed consent Is able to oral intake Must be willing to comply with protocol-required examination such as BMIPP myocardial scintigraphy Exclusion Criteria: Have diabetic ketoacidosis Have a malignancy that is being treated by anticancer drug or anticipated survival is less than three years Female with pregnant or lactating Is not able to agree to using intrauterine device, oral contraceptive, condom or having sexual intercourse from the start of taking investigational product to 30 days after the end of the study Have a New York Heart Association functional classification IV Have a known history of drug dependence Is allergic to any component of the investigational product Is allergic to BMIPP or iodine Have a known history of clinically significant drug allergy Have a severe liver dysfunction (Child classification B and C) Participated in other clinical study within the past 3 months and received an investigational agent including placebo Being treated with diet containing medium chain fatty acid Is considered unfit for the study by the Investigator's medical decision

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CNT-01<br>Patients will receive CNT-01 500 mg orally three times daily for 14 days. On Day 15, patients will take CNT-01 500 mg only once after blood drawing. | Drug: CNT-01 500 mg capsule<br>* After being informed about the study and potential risks, all patients giving written informed consent undergo screening tests to determine eligibility for study enrollment. On Day 1, patients who meet the eligibility criteria will start taking CNT-01 500mg orally three times daily for 14 days. On Day 15, patients will take CNT-01 500 mg only once after blood drawing.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of adverse event |  | Baseline to Day 29 | 
| Change in 12-lead ECG recordings from Baseline |  | Baseline to Day 29 | 
| Change in Blood Pressure from Baseline |  | Baseline to Day 29 | 
| Change in clinical laboratory testings as measured by serum chemistry, hematology and urinalysis from Baseline (composite) |  | Baseline to Day 29 | 
| Change in Body temperature from Baseline |  | Baseline to Day 29 | 
| Change in Pulse rate from Baseline |  | Baseline to Day 29 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in uptake and washout rate in BMIPP myocardial scintigraphy |  | Baseline to Day 15 | 
| Change in blood concentration of fatty acid fraction from Baseline (Octanoic acid, Capric acid, Myristic acid, Palmitic acid and Stearic acid) |  | Baseline to Day 16 | 
| Change in lipase activity in peripheral polynuclear leucocyte from Baseline |  | Baseline to Day 29 | 
| Change in blood lipoprotein fraction from Baseline |  | Baseline to Day 29 | 
| Change in vacuolation rate in polynuclear leucocyte from Baseline |  | Baseline to Day 29 | 
| Change in frequency in use of nitroglycerin from Baseline |  | Baseline to Day 29 | 
| Change in New York Heart Association (NYHA) functional classification from Baseline |  | Baseline to Day 29 | 
| Change in reactive hyperemic index from Baseline |  | Baseline to Day 29 | 
| Change in score of SF-36 from Baseline |  | Baseline to Day 29 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Triglyceride Deposit Cardiomyovasculopathy