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

Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan2 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV)Neutral Lipid Storage Disease With Myopathy (NLSD-M)

Interventions

CNT-02DIETARY_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.

Eligibility

Sex: ALLMin age: 20 Years

Medical Language ↔ Plain English

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.

Locations (7)

IRCCS San Raffaele

Rome, Lazio, Italy

San Filippo Neri Hospital

Rome, Lazio, Italy

Catholic University

Milan, Lombardy, Italy

Fondazione Ospedale San Camillo IRCCS

Lido, Venice, Italy

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: 6 months

Secondary Outcomes

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.

Time frame: 3 months

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.

Time frame: 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.

Time frame: 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.

Time frame: 6 months

The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 3 months

Data from ClinicalTrials.gov

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