Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV)NCT02830763
Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M
A study testing CNT-02 in people with Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Neutral Lipid Storage Disease With Myopathy (NLSD-M), run by Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan at 7 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV), Neutral Lipid Storage Disease With Myopathy (NLSD-M)
- What is being tested?
- CNT-02
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 5 Sep 2016to Jan 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
Italy4 sites
Fondazione Ospedale San Camillo IRCCS
Lido, Venice, 30126
Catholic University
Milan, Lombardy, 20123
IRCCS San Raffaele
Rome, Lazio, 00163
San Filippo Neri Hospital
Rome, Lazio, 00193
Japan3 sites
Aomori Prefectural Chuo Hospital
Aomori, Aomori, 030-8553
Tohoku University
Sendai, Miyagi, 980-8575
Graduate School of Osaka University
Suita, Osaka, 565-0874
About this study
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)
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.
- Conditions
- Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV), Neutral Lipid Storage Disease With Myopathy (NLSD-M)
- Being tested
- CNT-02
- Keywords
- neutral lipid storage disease, CNT-02, primary triglyceride deposit cardiomyovasculopathy (TGCV), neutral lipid storage disease with myopathy (NLSD-M)
- Medical subject headings
- Neutral Lipid Storage Disease with Myopathy, Chanarin-Dorfman Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| Medium-chain Fatty Acid (CNT-02) | Experimental | Not described Receives: Dietary Supplement: CNT-02 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 6 months
Secondary outcomes (10)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2016 (estimated)
- Study start5 Sep 2016
- Primary completionJan 2019
- Study completionJan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kenichi Hirano, MD, Ph.D, Principal investigator, Department of Cardiovascular Medicine, Faculty of Internal Medicine, Graduate School of Osaka University, JapanElena M Pennisi, MD, Ph.D, Principal investigator, Department of Neurology, San Filippo Neri Hospital, Rome, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02830763
- Sponsor's protocol ID
- TRICVD1527
- Other IDs
- UMIN000023043
- Status last verified
- Jul 2019
- First submitted
- 6 Jul 2016
- Last updated
- 8 Jul 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02830763
- Acronym / sponsor ID
- TRICVD1527
- 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)
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Translational Research Center for Medical Innovation, Kobe, Hyogo, JapanUniversity, hospital or other
- Study start
- 5 Sep 2016 (actual)
- Primary completion
- Jan 2019 (actual)
- Study completion
- Jan 2019 (actual)
- First posted
- 13 Jul 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Medium-chain Fatty Acid (CNT-02) | Experimental | Dietary Supplement: CNT-02 |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CNT-02Each 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. | Dietary supplement | Medium-chain Fatty Acid (CNT-02) |
Primary outcome
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.
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.
10 secondary outcomes
MRC sum score in manual muscle testing (MMT) at 3 months
Time frame: 3 months
MRC sum score in manual muscle testing (MMT) at 6 months
Time frame: 6 months
Fractions of lung volume such as % vital capacity, measured by spirometer at 3 months
Time frame: 3 months
Fractions of lung volume such as % vital capacity, measured by spirometer at 6 months
Time frame: 6 months
The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 3 months
Time frame: 3 months
The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 6 months
Time frame: 6 months
Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 3 months
Time frame: 3 months
Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 6 months
Time frame: 6 months
Serum free fatty acid levels at 3 months
Time frame: 3 months
Serum free fatty acid levels at 6 months
Time frame: 6 months
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2019.
Related trials for Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV)
RecruitingObservational study
International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases
Neutral Lipid Storage Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenichi Hirano, MD, Ph.D principal investigator
Department of Cardiovascular Medicine, Faculty of Internal Medicine, Graduate School of Osaka University, Japan
Elena M Pennisi, MD, Ph.D principal investigator
Department of Neurology, San Filippo Neri Hospital, Rome, Italy
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CNT-02 in people with Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Neutral Lipid Storage Disease With Myopathy (NLSD-M), run by Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Italy, Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The maximum walking distance in a 6-minute walk test at 3 months (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2019.