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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02830763
Recruitment Status : Terminated
First Posted : July 13, 2016
Last Update Posted : July 8, 2019
Sponsor:
Information provided by (Responsible Party):
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan

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.

Condition or disease Intervention/treatment Phase
Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) Neutral Lipid Storage Disease With Myopathy (NLSD-M) Dietary Supplement: CNT-02 Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : September 5, 2016
Actual Primary Completion Date : January 2019
Actual Study Completion Date : January 2019


Arm Intervention/treatment
Experimental: Medium-chain Fatty Acid (CNT-02) Dietary Supplement: CNT-02
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.




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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.


Other Outcome Measures:
  1. Incidence, severity, seriousness, causality, and outcomes of adverse events [ Time Frame: 6 months ]
    A frequency counting of the adverse events. LLT code will be assigned to the reported adverse event using the MedDRA dictionary.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02830763


Locations
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Italy
IRCCS San Raffaele
Rome, Lazio, Italy, 00163
San Filippo Neri Hospital
Rome, Lazio, Italy, 00193
Catholic University
Milano, Lombardia, Italy, 20123
Fondazione Ospedale San Camillo IRCCS
Lido, Venice, Italy, 30126
Japan
Aomori Prefectural Chuo Hospital
Aomori-city, Aomori-prefecture, Japan, 030-8553
Tohoku University
Sendai-city, Miyagi-prefecture, Japan, 980-8575
Graduate School of Osaka University
Suita-city, Osaka-prefecture, Japan, 565-0874
Sponsors and Collaborators
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
Investigators
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Principal Investigator: 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

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Responsible Party: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
ClinicalTrials.gov Identifier: NCT02830763    
Other Study ID Numbers: TRICVD1527
UMIN000023043 ( Other Identifier: UMIN-CTR )
First Posted: July 13, 2016    Key Record Dates
Last Update Posted: July 8, 2019
Last Verified: July 2019
Keywords provided by Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan:
neutral lipid storage disease
CNT-02
primary triglyceride deposit cardiomyovasculopathy (TGCV)
neutral lipid storage disease with myopathy (NLSD-M)
Additional relevant MeSH terms:
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Muscular Diseases
Lipidoses
Lipid Metabolism, Inborn Errors
Ichthyosiform Erythroderma, Congenital
Metabolic Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Ichthyosis
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Infant, Newborn, Diseases
Keratosis
Skin Diseases