Clinical Trial: Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official 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 Dis

Brief Summary: This study is planning to evaluate the safety and clinical efficacy of medium-chain fatty acid capsules (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.

Detailed Summary:
Sponsor: Translational Research Informatics Center, Kobe, Hyogo, Japan

Current Primary Outcome:

• The maximum walking distance in a 6-minute walk test at 3 months [ Time Frame: 3 months ]
  Measuring maximum walking distance of 6-minute walk test. Calculating variation of those measured values from baseline to 3 months.
• The maximum walking distance in a 6-minute walk test at 6 months [ Time Frame: 6 months ]
  Measuring maximum walking distance of 6-minute walk test . Calculating variation of those measured values from baseline to 6 months.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• MRC sum score in manual muscle testing (MMT) at 3 months [ Time Frame: 3 months ]
  Evaluating muscle strength by manual muscle test used MRC score. Assessing change from baseline to 3 months.
• MRC sum score in manual muscle testing (MMT) at 6 months [ Time Frame: 6 months ]
  Evaluating muscle strength by manual muscle test used MRC score. Assessing change from baseline to 6 months.
• Fractions of lung volume such as % vital capacity, measured by spirometer at 3 months [ Time Frame: 3 months ]
  Evaluating the respiratory functions by % Vital capacity measured by spirometer. Assessing change from baseline to 3 months.
• Fractions of lung volume such as % vital capacity, measured by spirometer at 6 months [ Time Frame: 6 months ]
  Evaluating the respiratory functions by % Vital capacity measured by spirometer. Assessing change from baseline to 6 months.
• The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 3 months [ Time Frame: 3 months ]
  Evaluating fat deposition in the skeletal muscle by CT. Assessing change from baseline to 3 months.
• The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 6 months [ Time Frame: 6 months ]
  Evaluating fat deposition in the skeletal muscle by CT. Assessing change from baseline to 6 months.
• Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 3 months [ Time Frame: 3 months ]
  Evaluating cardiac function using Left ventricular ejection fraction etc. by echocardiography. Assessing change from baseline to 3 months.
• Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 6 months [ Time Frame: 6 months ]
  Evaluating cardiac function using Left ventricular ejection fraction etc. by echocardiography. Assessing change from baseline to 6 months.
• Serum free fatty acid levels at 3 months [ Time Frame: 3 months ]
  Analysing concentration of fatty acids in serum using HPLC method. Assessing change from baseline to 3 months.
• Serum free fatty acid levels at 6 months [ Time Frame: 6 months ]
  Analysing concentration of fatty acids in serum using HPLC method. Assessing change from baseline to 6 months.

Original Secondary Outcome: Same as current

Information By: Translational Research Informatics Center, Kobe, Hyogo, Japan

Dates:
Date Received: July 6, 2016
Date Started: July 2016
Date Completion: May 2021
Last Updated: July 18, 2016
Last Verified: July 2016