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Safety Study of CNT-01 in Patients With Idiopathic TGCV

Phase 1
Completed
Conditions
Idiopathic Triglyceride Deposit Cardiomyovasculopathy
Interventions
Drug: CNT-01 500 mg capsule
Registration Number
NCT02502578
Lead Sponsor
Osaka University
Brief Summary

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
5
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
CNT-01CNT-01 500 mg capsulePatients 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.
Primary Outcome Measures
NameTimeMethod
Change in Blood Pressure from BaselineBaseline to Day 29
Number of adverse eventBaseline to Day 29
Change in 12-lead ECG recordings from BaselineBaseline 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 BaselineBaseline to Day 29
Change in Pulse rate from BaselineBaseline to Day 29
Secondary Outcome Measures
NameTimeMethod
Change in lipase activity in peripheral polynuclear leucocyte from BaselineBaseline to Day 29
Change in New York Heart Association (NYHA) functional classification from BaselineBaseline to Day 29
Change in reactive hyperemic index from BaselineBaseline to Day 29
Change in blood lipoprotein fraction from BaselineBaseline to Day 29
Change in vacuolation rate in polynuclear leucocyte from BaselineBaseline to Day 29
Change in frequency in use of nitroglycerin from BaselineBaseline to Day 29
Change in uptake and washout rate in BMIPP myocardial scintigraphyBaseline 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 score of SF-36 from BaselineBaseline to Day 29

Trial Locations

Locations (1)

Cardiovascular Medicine, Osaka University Hospital

🇯🇵

Suita, Osaka, Japan

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