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

Primary Purpose

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

Status
Terminated
Phase
Not Applicable
Locations
International
Study Type
Interventional
Intervention
CNT-02
Sponsored by
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) focused on measuring neutral lipid storage disease, CNT-02, primary triglyceride deposit cardiomyovasculopathy (TGCV), neutral lipid storage disease with myopathy (NLSD-M)

Eligibility Criteria

20 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • IRCCS San Raffaele
  • San Filippo Neri Hospital
  • Catholic University
  • Fondazione Ospedale San Camillo IRCCS
  • Aomori Prefectural Chuo Hospital
  • Tohoku University
  • Graduate School of Osaka University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Medium-chain Fatty Acid (CNT-02)

Arm Description

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

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.
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.
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.
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.
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.
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.
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.
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.
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.
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.

Full Information

First Posted
July 6, 2016
Last Updated
July 4, 2019
Sponsor
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
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1. Study Identification

Unique Protocol Identification Number
NCT02830763
Brief Title
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

2. Study Status

Record Verification Date
July 2019
Overall Recruitment Status
Terminated
Study Start Date
September 5, 2016 (Actual)
Primary Completion Date
January 2019 (Actual)
Study Completion Date
January 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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)

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Medium-chain Fatty Acid (CNT-02)
Arm Type
Experimental
Intervention Type
Dietary Supplement
Intervention Name(s)
CNT-02
Intervention Description
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 Measure Information:
Title
The maximum walking distance in a 6-minute walk test at 3 months
Description
Measuring maximum walking distance of 6-minute walk test. Calculating variation of those measured values from baseline to 3 months.
Time Frame
3 months
Title
The maximum walking distance in a 6-minute walk test at 6 months
Description
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 Outcome Measure Information:
Title
MRC sum score in manual muscle testing (MMT) at 3 months
Description
Evaluating muscle strength by manual muscle test used MRC score. Assessing change from baseline to 3 months.
Time Frame
3 months
Title
MRC sum score in manual muscle testing (MMT) at 6 months
Description
Evaluating muscle strength by manual muscle test used MRC score. Assessing change from baseline to 6 months.
Time Frame
6 months
Title
Fractions of lung volume such as % vital capacity, measured by spirometer at 3 months
Description
Evaluating the respiratory functions by % Vital capacity measured by spirometer. Assessing change from baseline to 3 months.
Time Frame
3 months
Title
Fractions of lung volume such as % vital capacity, measured by spirometer at 6 months
Description
Evaluating the respiratory functions by % Vital capacity measured by spirometer. Assessing change from baseline to 6 months.
Time Frame
6 months
Title
The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 3 months
Description
Evaluating fat deposition in the skeletal muscle by CT. Assessing change from baseline to 3 months.
Time Frame
3 months
Title
The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 6 months
Description
Evaluating fat deposition in the skeletal muscle by CT. Assessing change from baseline to 6 months.
Time Frame
6 months
Title
Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 3 months
Description
Evaluating cardiac function using Left ventricular ejection fraction etc. by echocardiography. Assessing change from baseline to 3 months.
Time Frame
3 months
Title
Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 6 months
Description
Evaluating cardiac function using Left ventricular ejection fraction etc. by echocardiography. Assessing change from baseline to 6 months.
Time Frame
6 months
Title
Serum free fatty acid levels at 3 months
Description
Analysing concentration of fatty acids in serum using HPLC method. Assessing change from baseline to 3 months.
Time Frame
3 months
Title
Serum free fatty acid levels at 6 months
Description
Analysing concentration of fatty acids in serum using HPLC method. Assessing change from baseline to 6 months.
Time Frame
6 months
Other Pre-specified Outcome Measures:
Title
Incidence, severity, seriousness, causality, and outcomes of adverse events
Description
A frequency counting of the adverse events. LLT code will be assigned to the reported adverse event using the MedDRA dictionary.
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients for whom ATGL gene deletion has been previously confirmed. Patients who can take the investigational product orally. Male and female who are at least 20 years old at the time of consent. Patients who gave written informed consent. Exclusion Criteria: Patients with diabetic ketoacidosis. Patients with the possibility of diabetic ketoacidosis (patients with poorly controlled diabetes mellitus [HbA1c>8.4%, NGSP]) Patients with terminal malignancy. Pregnant or lactating women. Patients who do not consent to using contraception while participating in this study. Patients allergic to MCT oil. Patients participating in other clinical trial. Otherwise, patients determined to be ineligible for this study by the investigator or sub-investigator.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kenichi Hirano, MD, Ph.D
Organizational Affiliation
Department of Cardiovascular Medicine, Faculty of Internal Medicine, Graduate School of Osaka University, Japan
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Elena M Pennisi, MD, Ph.D
Organizational Affiliation
Department of Neurology, San Filippo Neri Hospital, Rome, Italy
Official's Role
Principal Investigator
Facility Information:
Facility Name
IRCCS San Raffaele
City
Rome
State/Province
Lazio
ZIP/Postal Code
00163
Country
Italy
Facility Name
San Filippo Neri Hospital
City
Rome
State/Province
Lazio
ZIP/Postal Code
00193
Country
Italy
Facility Name
Catholic University
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20123
Country
Italy
Facility Name
Fondazione Ospedale San Camillo IRCCS
City
Lido
State/Province
Venice
ZIP/Postal Code
30126
Country
Italy
Facility Name
Aomori Prefectural Chuo Hospital
City
Aomori-city
State/Province
Aomori-prefecture
ZIP/Postal Code
030-8553
Country
Japan
Facility Name
Tohoku University
City
Sendai-city
State/Province
Miyagi-prefecture
ZIP/Postal Code
980-8575
Country
Japan
Facility Name
Graduate School of Osaka University
City
Suita-city
State/Province
Osaka-prefecture
ZIP/Postal Code
565-0874
Country
Japan

12. IPD Sharing Statement

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

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