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| ID | Type | Description | Link |
|---|---|---|---|
| UMIN000023043 | Other Identifier | UMIN-CTR |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Medium-chain Fatty Acid (CNT-02) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CNT-02 | Dietary Supplement | Each subject will take 2.0g of the investigational product orally 3 times a day after every meal. Unless an apparent worsening of symptoms or unacceptable adverse event occurs, the subject will continue to take the investigational product for up to 6 months. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| MRC sum score in manual muscle testing (MMT) at 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Kenichi Hirano, MD, Ph.D | Department of Cardiovascular Medicine, Faculty of Internal Medicine, Graduate School of Osaka University, Japan | Principal Investigator |
| Elena M Pennisi, MD, Ph.D | Department of Neurology, San Filippo Neri Hospital, Rome, Italy | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| IRCCS San Raffaele | Rome | Lazio | 00163 | Italy | ||
| San Filippo Neri Hospital |
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| ID | Term |
|---|---|
| C565192 | Neutral Lipid Storage Disease with Myopathy |
| C536560 | Chanarin-Dorfman Syndrome |
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|
Evaluating muscle strength by manual muscle test used MRC score. Assessing change from baseline to 6 months.
| 6 months |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Rome |
| Lazio |
| 00193 |
| Italy |
| Catholic University | Milan | Lombardy | 20123 | Italy |
| Fondazione Ospedale San Camillo IRCCS | Lido | Venice | 30126 | Italy |
| Aomori Prefectural Chuo Hospital | Aomori | Aomori | 030-8553 | Japan |
| Tohoku University | Sendai | Miyagi | 980-8575 | Japan |
| Graduate School of Osaka University | Suita | Osaka | 565-0874 | Japan |