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Name:
UMIN ID:

Recruitment status No longer recruiting
Unique ID issued by UMIN UMIN000023043
Receipt No. R000026523
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)
Date of disclosure of the study information 2016/08/13
Last modified on 2019/01/08

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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
Middle name
Last name Ken-ichi Hirano / Elena M. Pennisi
Organization Osaka University / San Filippo Neri Hospital, Rome, Italy
Division name Department of Cardiovascular Medicine Graduate School of Medicine, Faculty of Medicine / Department of Neurology
Zip code
Address 6-2-3 Furuedai, Suita, Osaka, Japan 565-0874
TEL 06-6872-8215
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, 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
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)、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
Anticipated trial start date
2016 Year 08 Month 01 Day
Last follow-up date
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
2019 Year 01 Month 08 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000026523

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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