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

Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan

Status: Terminated

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

Treatments: Dietary Supplement: CNT-02

Study type: Interventional

Funder types: Other

Identifiers

NCT02830763

TRICVD1527

UMIN000023043 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 2 patients

Sex: All

Ages: 20+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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2 participants in 1 patient group

Medium-chain Fatty Acid (CNT-02)

Experimental group

Treatment:

Dietary Supplement: CNT-02