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)
Not Applicable
- Conditions
- Primary triglyceride deposit cardiomyovasculopathy (TGCV) Neutral lipid storage disease with myopathy (NLSD-M)
- Registration Number
- JPRN-UMIN000023043
- Lead Sponsor
- Translational Research Informatics Center, Foundation for Biomedical Research and Innovation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up continuing
- Sex
- All
- Target Recruitment
- 10
Inclusion Criteria
Not provided
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The maximum walking distance in a 6-minute walk test.
- Secondary Outcome Measures
Name Time Method 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