Neutral Lipid Storage Disease With Myopathy (NLSD-M) Clinical Trial
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 Disease With Myopathy (NLSD-M)
| Verified date | July 2019 |
| Source | Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Terminated |
| Enrollment | 2 |
| Est. completion date | January 2019 |
| Est. primary completion date | January 2019 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 20 Years and older |
| Eligibility |
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. 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. |
| Country | Name | City | State |
|---|---|---|---|
| Italy | Fondazione Ospedale San Camillo IRCCS | Lido | Venice |
| Italy | Catholic University | Milano | Lombardia |
| Italy | IRCCS San Raffaele | Rome | Lazio |
| Italy | San Filippo Neri Hospital | Rome | Lazio |
| Japan | Aomori Prefectural Chuo Hospital | Aomori-city | Aomori-prefecture |
| Japan | Tohoku University | Sendai-city | Miyagi-prefecture |
| Japan | Graduate School of Osaka University | Suita-city | Osaka-prefecture |
| Lead Sponsor | Collaborator |
|---|---|
| Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan |
Italy, Japan,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Incidence, severity, seriousness, causality, and outcomes of adverse events | A frequency counting of the adverse events. LLT code will be assigned to the reported adverse event using the MedDRA dictionary. | 6 months | |
| Primary | The maximum walking distance in a 6-minute walk test at 3 months | Measuring maximum walking distance of 6-minute walk test. Calculating variation of those measured values from baseline to 3 months. | 3 months | |
| Primary | The maximum walking distance in a 6-minute walk test at 6 months | Measuring maximum walking distance of 6-minute walk test . Calculating variation of those measured values from baseline to 6 months. | 6 months | |
| Secondary | MRC sum score in manual muscle testing (MMT) at 3 months | Evaluating muscle strength by manual muscle test used MRC score. Assessing change from baseline to 3 months. | 3 months | |
| Secondary | MRC sum score in manual muscle testing (MMT) at 6 months | Evaluating muscle strength by manual muscle test used MRC score. Assessing change from baseline to 6 months. | 6 months | |
| Secondary | Fractions of lung volume such as % vital capacity, measured by spirometer at 3 months | Evaluating the respiratory functions by % Vital capacity measured by spirometer. Assessing change from baseline to 3 months. | 3 months | |
| Secondary | Fractions of lung volume such as % vital capacity, measured by spirometer at 6 months | Evaluating the respiratory functions by % Vital capacity measured by spirometer. Assessing change from baseline to 6 months. | 6 months | |
| Secondary | The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 3 months | Evaluating fat deposition in the skeletal muscle by CT. Assessing change from baseline to 3 months. | 3 months | |
| Secondary | The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 6 months | Evaluating fat deposition in the skeletal muscle by CT. Assessing change from baseline to 6 months. | 6 months | |
| Secondary | Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 3 months | Evaluating cardiac function using Left ventricular ejection fraction etc. by echocardiography. Assessing change from baseline to 3 months. | 3 months | |
| Secondary | Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 6 months | Evaluating cardiac function using Left ventricular ejection fraction etc. by echocardiography. Assessing change from baseline to 6 months. | 6 months | |
| Secondary | Serum free fatty acid levels at 3 months | Analysing concentration of fatty acids in serum using HPLC method. Assessing change from baseline to 3 months. | 3 months | |
| Secondary | Serum free fatty acid levels at 6 months | Analysing concentration of fatty acids in serum using HPLC method. Assessing change from baseline to 6 months. | 6 months |