GLUT1DS1Phase 2NCT03301532
Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
A Phase 2 study testing Triheptanoin in people with GLUT1DS1, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet. Read more about this study
- Who is it for?
- Ages 30 months to 35 years
- Conditions the study is for
- GLUT1DS1
- What is being tested?
- Triheptanoin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jun 2018to 31 Jul 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 30 months to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical genotyping at a CLIA-certified laboratory.
- Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have taken place for 2 months). The initiation of a ketogenic diet is previous to - and thus is not part of this study.
- Males and females 30 months to 35 years and 11 months old inclusive.
Who cannot join
- Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
- Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome, crohn's disease, or colitis that could increase the subject's risk of developing diarrhea or stomach pain.
- Subjects with a BMI (body mass index) greater than or equal to 30.
- Subjects currently not on ketogenic diet.
- Women who are pregnant or breast feeding may not participate. Women who plan to become pregnant during the course of the study, or who are unwilling to use birth control to prevent pregnancy (including abstinence) may not participate. Females age 10 and over will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects will be asked to agree to abstinence or another form of birth control for the duration of the study.
- Allergy/sensitivity to C7
Show all 12Show fewer
- Previous use of triheptanoin less than 1 month prior to study initiation.
- Treatment with medium chain triglycerides in the last 24 hours.
- Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain disorder (such as Alzheimer's disease) that would confound assessment of cognitive changes, in the opinion of the investigator.
- Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements.
- Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17,
- Addition of a new antiseizure drug in the previous 3 months.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency
To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
Read the detailed description
This is a single site, open label proof of principle exploratory trial to investigate the compatibility of C7 oil with the ketogenic diet in subjects diagnosed with G1D. The ketogenic diet will have been previously described by the patient's treating physician independently of this study and for clinical reasons. The ketogenic diet supplies over 50% of calories from fat, subjects, who are already tolerating over this much fat as part of their previously prescribed ketogenic diet, will replace 45% of their daily caloric intake with the triheptanoin for 24 hours, during a 48 hour inpatient stay. Subjects will have a continuous EEG to monitor for any potential C7 related changes in seizure before, during, and after triheptanoin oil ingestion.
- Conditions
- GLUT1DS1
- Being tested
- Triheptanoin
- Keywords
- G1D, glucose, transporter
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TriheptanoinDrug
Also called C7 oil
Dietary supplementation with triheptanoin
| Study group | Role | What they receive |
|---|---|---|
| Patients on a ketogenic diet | Experimental | This is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil. Receives: Drug: Triheptanoin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Ketosis (Beta-hydroxybutyrate Levels)
Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.
Measured: Day 1, Day 4
Secondary outcomes (2)
Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate
The percent change in observable seizure rate is measured as the change in observable seizure numbers from baseline to Day 4.
Seizure rate is defined as count of seizures per patient per day.
Measured: Baseline (Day 1) - Day 4
Change in Glycemia
Change in Glycemia is measured as changes in blood glucose levels.
Measured: Day 1 - Day 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2017
- Study start5 Jun 2018
- Primary completion18 Jun 2021
- Study completion31 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GLUT1DS1Studies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Juan Pascual, University of Texas Southwestern Medical Center
- Investigators
- Juan Pascual, M.D., Principal investigator, Study Principal Investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03301532
- Sponsor's protocol ID
- STU 102015-091
- Other IDs
- R01NS094257-01A1
- Status last verified
- Dec 2023
- First submitted
- 29 Aug 2017
- Last updated
- 17 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Avila A, Malaga I, Sirsi D, Kayani S, Primeaux S, Kathote GA, Jakkamsetti V, Kallem RR, Putnam WC, Park JY, Shinnar S, Pascual JM. Combination of triheptanoin with the ketogenic diet in Glucose transporter type 1 deficiency (G1D). Sci Rep. 2023 Jun 2;13(1):8951. doi: 10.1038/s41598-023-36001-x.
PubMed 37268656
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency
- ClinicalTrials.gov ID
- NCT03301532
- Acronym / sponsor ID
- STU 102015-091
- Conditions
- GLUT1DS1
- Keywords
- G1D, glucose, transporter
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- 5 Jun 2018 (actual)
- Primary completion
- 18 Jun 2021 (actual)
- Study completion
- 31 Jul 2022 (actual)
- First posted
- 4 Oct 2017 (actual)
- Results
- Posted 17 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
Detailed description
This is a single site, open label proof of principle exploratory trial to investigate the compatibility of C7 oil with the ketogenic diet in subjects diagnosed with G1D. The ketogenic diet will have been previously described by the patient's treating physician independently of this study and for clinical reasons. The ketogenic diet supplies over 50% of calories from fat, subjects, who are already tolerating over this much fat as part of their previously prescribed ketogenic diet, will replace 45% of their daily caloric intake with the triheptanoin for 24 hours, during a 48 hour inpatient stay. Subjects will have a continuous EEG to monitor for any potential C7 related changes in seizure before, during, and after triheptanoin oil ingestion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients on a ketogenic diet | Experimental | Drug: TriheptanoinThis is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil. |
Who may qualify
- Ages
- 30 months to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical genotyping at a CLIA-certified laboratory.
- Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have taken place for 2 months). The initiation of a ketogenic diet is previous to - and thus is not part of this study.
- Males and females 30 months to 35 years and 11 months old inclusive.
Key exclusion criteria (12 listed)
- Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
- Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome, crohn's disease, or colitis that could increase the subject's risk of developing diarrhea or stomach pain.
- Subjects with a BMI (body mass index) greater than or equal to 30.
- Subjects currently not on ketogenic diet.
- Women who are pregnant or breast feeding may not participate. Women who plan to become pregnant during the course of the study, or who are unwilling to use birth control to prevent pregnancy (including abstinence) may not participate. Females age 10 and over will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects will be asked to agree to abstinence or another form of birth control for the duration of the 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 |
|---|---|---|
| TriheptanoinAlso: C7 oilDietary supplementation with triheptanoin | Drug | Patients on a ketogenic diet |
Primary outcome
Change in Ketosis (Beta-hydroxybutyrate Levels)
Time frame: Day 1, Day 4
Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.
2 secondary outcomes
Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate
Time frame: Baseline (Day 1) - Day 4
Change in Glycemia
Time frame: Day 1 - Day 4
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 37268656Avila A, Malaga I, Sirsi D, Kayani S, Primeaux S, Kathote GA, Jakkamsetti V, Kallem RR, Putnam WC, Park JY, Shinnar S, Pascual JM. Combination of triheptanoin with the ketogenic diet in Glucose transporter type 1 deficiency (G1D). Sci Rep. 2023 Jun 2;13(1):8951. doi: 10.1038/s41598-023-36001-x.
Related trials for GLUT1DS1
RecruitingObservational study
Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease
Glut1 Deficiency Syndrome 1, Glucose Metabolism Disorders
RecruitingPhase 2
A Phase II Study With Exploratory Outcomes of Fucose Supplementation in GLUT1 Deficiency Syndrome
Glut1 Deficiency, GLUT1DS1
Active, not recruitingObservational study
Harmonic Ratio in Patients With GLUT1 Deficiency Syndrome
GLUT1DS1, Dystonia
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.
Juan Pascual, M.D. principal investigator
Study Principal Investigator
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Triheptanoin in people with GLUT1DS1, run by University of Texas Southwestern Medical Center at 1 site in United States.
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 3 inclusion and 12 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: finished, as of the sponsor's last update on 17 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change in Ketosis (Beta-hydroxybutyrate Levels) (time frame: Day 1, Day 4). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03301532, which also lists 1 linked publication.
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 17 Jan 2024.