Safety Study of CNT-01 in Patients With Idiopathic TGCV

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

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.

Study Overview

• Status: Completed
• Conditions: Idiopathic Triglyceride Deposit Cardiomyovasculopathy
• Intervention / Treatment: Drug: CNT-01 500 mg capsule

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Japan
  • Osaka
    • Suita, Osaka, Japan, 565-0871
      • Cardiovascular Medicine, Osaka University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CNT-01; 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; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Osaka University
• Investigators: Study Chair: Ken-ichi Hirano, M.D., Ph.D., Department of Cardiovascular Medicine, Osaka University Graduate School of Medicine

Study record dates

Study Major Dates

• Study Start: September 1, 2015
• Primary Completion (Actual): June 1, 2016

Study Registration Dates

• First Submitted: July 16, 2015
• First Submitted That Met QC Criteria: July 17, 2015
• First Posted (Estimate): July 20, 2015

Study Record Updates

• Last Update Posted (Estimate): September 28, 2016
• Last Update Submitted That Met QC Criteria: September 27, 2016
• Last Verified: September 1, 2016

More Information

Terms related to this study

• Keywords: Triglyceride Deposit Cardiomyovasculopathy
• Other Study ID Numbers: CNT-01-001