UMIN-CTR Clinical Trial

Basic information
Public title	Phase I/IIa Study of CNT-01 in Patients with Idiopathic Triglyceride Deposit Cardiomyovasculopathy
Acronym	Safety Study of CNT-01 in Patients with Idiopathic TGCV
Scientific Title	Phase I/IIa Study of CNT-01 in Patients with Idiopathic Triglyceride Deposit Cardiomyovasculopathy
Scientific Title:Acronym	Safety Study of CNT-01 in Patients with Idiopathic TGCV
Region	Japan

Condition
Condition	Idiopathic Triglyceride Deposit Cardiomyovasculopathy
Classification by specialty	Cardiology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	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.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Others
Trial characteristics_2
Developmental phase	Phase I,II

Assessment
Primary outcomes	-Adverse event -Change in ECG parameters from Baseline (QTc, QT, QRS) -Change in vital signs from Baseline (Body temperature, Blood pressure, Pulse rate) -Change in clinical laboratory data from Baseline (Hematology, Serum chemistry, Urinalysis)
Key secondary outcomes	-Change in uptake and washout rate in BMIPP myocardial scintigraphy -Change in blood concentration of fatty acid fraction from Baseline -Change in lipase activity in peripheral polynuclear leucocyte from Baseline -Change in blood lipoprotein fraction from Baseline (Total cholesterol in Chylomicron, VLDL, LDL, HDL, Triglyceride in Chylomicron, VLDL, LDL, HDL) -Change in vacuolation rate in polynuclear leucocyte from Baseline -Change in frequency in use of nitroglycerin from Baseline -Change in New York Heart Association (NYHA) functional classification from Baseline -Change in vascular endothelial function from Baseline (Reactive hyperemic index) -Change in score of SF-36 from Baseline

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	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.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1) Have a definite diagnosis of idiopathic triglyceride deposit cardiomyovasculopathy 2) After receiving a sufficient explanation upon participation in this study, on a full understanding, patients who obtained the written informed consent from each patient 3) More than 20 years old at the time of informed consent 4) Is able to oral intake 5) Must be willing to comply with protocol-required examination such as BMIPP myocardial scintigraphy
Key exclusion criteria	1) Have diabetic ketoacidosis 2) Have a malignancy that is being treated by anticancer drug or anticipated survival is less than three years 3) Female with pregnant or lactating 4) 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 5) Have a New York Heart Association functional classification IV 6) Have a known history of drug dependence 7) Is allergic to any component of the investigational product 8) Is allergic to BMIPP or iodine 9) Have a known history of clinically significant drug allergy 10) Have a severe liver dysfunction (Child classification B and C) 11) Participated in other clinical study within the past 3 months and received an investigational agent including placebo 12) Being treated with diet containing medium chain fatty acid 13) Is considered unfit for the study by the Investigator's medical decision
Target sample size	6

Research contact person
Name of lead principal investigator	1st name Middle name Last name Ken-ichi Hirano
Organization	Osaka University Grduate School of Medicine
Division name	Department of Cardiovascular Medicine
Zip code
Address	6-2-3, Furuedai, Suita, Osaka 565-0874, JAPAN
TEL	+81-6-6872-8215
Email	khirano@cnt-osaka.com

Public contact
Name of contact person	1st name Middle name Last name Daisaku Nakatani
Organization	Osaka University Hospital
Division name	Department of Medical Innovation
Zip code
Address	2-2, Yamadaoka, Suita, Osaka 565-0871, JAPAN
TEL	+81-6-6210-8289
Homepage URL
Email	nakatani@cardiology.med.osaka-u.ac.jp

Sponsor
Institute	Ken-ichi Hirano, Department of Cardiovascular Medicine, Osaka University Grduate School of Medicine
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions	大阪大学医学部附属病院（大阪府）　　　　　　　Osaka University Hospital(Osaka)

Other administrative information
Date of disclosure of the study information	2015 Year 07 Month 14 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 04 Month 15 Day
Date of IRB
Anticipated trial start date	2015 Year 09 Month 01 Day
Last follow-up date	2016 Year 06 Month 30 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2015 Year 07 Month 13 Day
Last modified on	2020 Year 03 Month 09 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000021168