UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000023043 |
|---|---|
| Receipt number | 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 | 2020/03/09 13:14:31 |
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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 | 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
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
nakatani@cardiology.med.osaka-u.ac.jp
Sponsor or person
Institute
Translational Research Informatics Center, Foundation for Biomedical Research and Innovation
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000026523
