Clinical trial NCT02502578: Safety Study of CNT-01 in Patients With Idiopathic TGCV
Official title: Phase I/IIa Study of CNT-01 in Patients With Idiopathic Triglyceride Deposit
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Idiopathic Triglyceride Deposit Cardiomyovasculopathy
Keywords: Triglyceride Deposit Cardiomyovasculopathy
Lead sponsor: Osaka University
Sponsor class: OTHER
First posted: 2015-07-20
Enrollment: 5
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of adverse event
timeFrame: Baseline to Day 29
measure: Change in 12-lead ECG recordings from Baseline
timeFrame: Baseline to Day 29
measure: Change in Blood Pressure from Baseline
timeFrame: Baseline to Day 29
measure: Change in clinical laboratory testings as measured by serum chemistry, hematology and urinalysis from Baseline (composite)
timeFrame: Baseline to Day 29
measure: Change in Body temperature from Baseline
timeFrame: Baseline to Day 29
measure: Change in Pulse rate from Baseline
timeFrame: Baseline to Day 29
Secondary outcomes:
measure: Change in uptake and washout rate in BMIPP myocardial scintigraphy
timeFrame: Baseline to Day 15
measure: Change in blood concentration of fatty acid fraction from Baseline (Octanoic acid, Capric acid, Myristic acid, Palmitic acid and Stearic acid)
timeFrame: Baseline to Day 16
measure: Change in lipase activity in peripheral polynuclear leucocyte from Baseline
timeFrame: Baseline to Day 29
measure: Change in blood lipoprotein fraction from Baseline
timeFrame: Baseline to Day 29
measure: Change in vacuolation rate in polynuclear leucocyte from Baseline
timeFrame: Baseline to Day 29
measure: Change in frequency in use of nitroglycerin from Baseline
timeFrame: Baseline to Day 29
measure: Change in New York Heart Association (NYHA) functional classification from Baseline
timeFrame: Baseline to Day 29
measure: Change in reactive hyperemic index from Baseline
timeFrame: Baseline to Day 29
measure: Change in score of SF-36 from Baseline
timeFrame: Baseline to Day 29
Other outcomes:
Locations:
city: Suita
country: Japan
facility: Cardiovascular Medicine, Osaka University Hospital
state: Osaka
Study officials:
affiliation: Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine
name: Ken-ichi Hirano, M.D., Ph.D.
role: STUDY_CHAIR
References: