Clinical trial NCT02830763: Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M
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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV)
Neutral Lipid Storage Disease With Myopathy (NLSD-M)
Keywords: neutral lipid storage disease
CNT-02
primary triglyceride deposit cardiomyovasculopathy (TGCV)
neutral lipid storage disease with myopathy (NLSD-M)
Lead sponsor: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
Sponsor class: OTHER
First posted: 2016-07-13
Enrollment: 2
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: The maximum walking distance in a 6-minute walk test at 3 months
timeFrame: 3 months
measure: The maximum walking distance in a 6-minute walk test at 6 months
timeFrame: 6 months
Secondary outcomes:
measure: MRC sum score in manual muscle testing (MMT) at 3 months
timeFrame: 3 months
measure: MRC sum score in manual muscle testing (MMT) at 6 months
timeFrame: 6 months
measure: Fractions of lung volume such as % vital capacity, measured by spirometer at 3 months
timeFrame: 3 months
measure: Fractions of lung volume such as % vital capacity, measured by spirometer at 6 months
timeFrame: 6 months
measure: The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 3 months
timeFrame: 3 months
measure: The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 6 months
timeFrame: 6 months
measure: Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 3 months
timeFrame: 3 months
measure: Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 6 months
timeFrame: 6 months
measure: Serum free fatty acid levels at 3 months
timeFrame: 3 months
measure: Serum free fatty acid levels at 6 months
timeFrame: 6 months
Other outcomes:
measure: Incidence, severity, seriousness, causality, and outcomes of adverse events
timeFrame: 6 months
Locations:
city: Rome
country: Italy
facility: IRCCS San Raffaele
state: Lazio
city: Rome
country: Italy
facility: San Filippo Neri Hospital
state: Lazio
city: Milano
country: Italy
facility: Catholic University
state: Lombardia
city: Lido
country: Italy
facility: Fondazione Ospedale San Camillo IRCCS
state: Venice
city: Aomori-city
country: Japan
facility: Aomori Prefectural Chuo Hospital
state: Aomori-prefecture
city: Sendai-city
country: Japan
facility: Tohoku University
state: Miyagi-prefecture
city: Suita-city
country: Japan
facility: Graduate School of Osaka University
state: Osaka-prefecture
Study officials:
affiliation: Department of Cardiovascular Medicine, Faculty of Internal Medicine, Graduate School of Osaka University, Japan
name: Kenichi Hirano, MD, Ph.D
role: PRINCIPAL_INVESTIGATOR
affiliation: Department of Neurology, San Filippo Neri Hospital, Rome, Italy
name: Elena M Pennisi, MD, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: