UMIN-CTR Clinical Trial

Basic information
Public title	Clinical study on the safety and efficacy of medium-chain fatty acid capsules (CNT-02) for primary triglyceride deposit cardiomyovasculopathy (TGCV) and neutral lipid storage disease with myopathy (NLSD-M)
Acronym	Clinical study on the safety of CNT-02 for TGCV and NLSD-M
Scientific Title	Clinical study on the safety and efficacy of medium-chain fatty acid capsules (CNT-02) for primary triglyceride deposit cardiomyovasculopathy (TGCV) and neutral lipid storage disease with myopathy (NLSD-M)
Scientific Title:Acronym	Clinical study on the safety of CNT-02 for TGCV and NLSD-M
Region	Japan Europe

Condition
Condition	Primary triglyceride deposit cardiomyovasculopathy (TGCV) Neutral lipid storage disease with myopathy (NLSD-M)
Classification by specialty	Cardiology Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the safety and clinical efficacy of capsules containing medium-chain fatty acid (food-grade CNT-02) in subjects with primary triglyceride deposit cardiomyovasculopathy (TGCV) and neutral lipid storage disease with myopathy (NLSD-M) associated with adipose triglyceride lipase (ATGL) genetic defects.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase	Not applicable

Assessment
Primary outcomes	The maximum walking distance in a 6-minute walk test.
Key secondary outcomes	1) MRC sum score in manual muscle testing (MMT) 2) Fractions of lung volume such as % vital capacity, measured by spirometer 3) The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) 4) Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography 5) Serum free fatty acid levels

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification	NO
Dynamic allocation	NO
Institution consideration	Institution is not considered as adjustment factor.
Blocking	NO
Concealment	No need to know

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Food
Interventions/Control_1	Introduction period: Take the CNT-02, 1.5 g/day (0.5 g/day after each meal) for 4 days and take 3.0g/day (1.0 g/day after each meal) for 4 days. Treatment period: Take the CNT-02, 6.0g/day (2.0g/day after each meal) for24 months.
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) Patients for whom ATGL gene deletion has been previously confirmed. 2) Patients who can take the investigational product orally. 3) Male and female who are at least 20 years old at the time of consent. 4) Patients who gave written informed consent.
Key exclusion criteria	1) Patients with diabetic ketoacidosis. 2) Patients with the possibility of diabetic ketoacidosis (patients with poorly controlled diabetes mellitus [HbA1c>8.4%, NGSP]) 3) Patients with terminal malignancy. 4) Pregnant or lactating women. 5) Patients who do not consent to using contraception while participating in this study. 6) Patients allergic to MCT oil. 7) Patients participating in other clinical trial. 8) Otherwise, patients determined to be ineligible for this study by the investigator or sub-investigator.
Target sample size	10

Research contact person
Name of lead principal investigator	1st name Ken-ichi Middle name Last name Hirano
Organization	Osaka University
Division name	Department of Cardiovascular Medicine Graduate School of Medicine, Faculty of Medicine / Department of Neurology
Zip code	565-0874
Address	6-2-4 Furuedai, Suita, Osaka, Japan 565-0874
TEL	06-6872-8215
Email	khirano@cnt-osaka.com

Public contact
Name of contact person	1st name Daisaku Middle name Last name Nakatani
Organization	Osaka University Hospital
Division name	Department of Medical Innovation
Zip code	565-0871
Address	2-2, Yamadaoka, Suita, Osaka, Japan 565-0871
TEL	06-6210-8289
Homepage URL
Email	nakatani@cardiology.med.osaka-u.ac.jp

Sponsor
Institute	Translational Research Informatics Center, Foundation for Biomedical Research and Innovation
Institute
Department

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

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

IRB Contact (For public release)
Organization	Osaka University Clinical Research Review Committee
Address	2-2, Yamadaoka, Suita, Osaka, Japan 565-0871
Tel	06-6879-8290
Email	rinri@hp-crc.med.osaka-u.ac.jp

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）、San Filippo Neri Hospital (Rome, Italy)

Other administrative information
Date of disclosure of the study information	2016 Year 08 Month 13 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	No longer recruiting
Date of protocol fixation	2016 Year 05 Month 09 Day
Date of IRB	2016 Year 07 Month 15 Day
Anticipated trial start date	2016 Year 08 Month 01 Day
Last follow-up date	2019 Year 10 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2016 Year 07 Month 06 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=R000026523