Trial Outcomes & Findings for Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D (NCT NCT03301532)
NCT ID: NCT03301532
Last Updated: 2024-01-17
Results Overview
Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.
COMPLETED
PHASE2
10 participants
Day 1, Day 4
2024-01-17
Participant Flow
Participant milestones
| Measure |
Experimental: Triheptanoin (C7)
Triheptanoin (C7 oil, liquid). Subjects consumed triheptanoin 4 times over the course of one day. The approach included substituting a fraction of ketogenic diet fat with C7, weight by weight, at the maximum tolerable dose (45% of their their daily calories) in individuals receiving a ketogenic diet prior to enrolment prescribed independently of this study.
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|---|---|
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Overall Study
STARTED
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10
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Overall Study
COMPLETED
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10
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
Baseline characteristics by cohort
| Measure |
Triheptanoin (C7 Oil, Liquid)
n=10 Participants
C7 oil was given at maximum tolerated dose (45% of total daily calories) to 10 Glut1 subjects receiving a ketogenic diet prior to enrolment as medically prescribed independently of this study.
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|---|---|
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Age, Categorical
<=18 years
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9 Participants
n=5 Participants
|
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Age, Categorical
Between 18 and 65 years
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1 Participants
n=5 Participants
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Age, Categorical
>=65 years
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0 Participants
n=5 Participants
|
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Sex: Female, Male
Female
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6 Participants
n=5 Participants
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Sex: Female, Male
Male
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4 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Hispanic or Latino
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1 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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9 Participants
n=5 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
|
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
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Race (NIH/OMB)
Asian
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1 Participants
n=5 Participants
|
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=5 Participants
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Race (NIH/OMB)
White
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9 Participants
n=5 Participants
|
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
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Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
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Region of Enrollment
United States
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9 participants
n=5 Participants
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Region of Enrollment
Argentina
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1 participants
n=5 Participants
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PRIMARY outcome
Timeframe: Day 1, Day 4Population: Subjects with Glut1 deficency
Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.
Outcome measures
| Measure |
Ketosis
n=10 Participants
Participants BHB levels were checked daily on days 1 - 4. Treatment: Triheptanoin (C7), orally, dose of 45% of the total daily calories divided in four equally spaced administrations (i.e., one dose every 4 hours to a total of four doses in one day).
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|---|---|
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Change in Ketosis (Beta-hydroxybutyrate Levels)
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3.82 mM
Standard Deviation 1.9
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SECONDARY outcome
Timeframe: Baseline (Day 1) - Day 4Population: All subjects with Glut1 deficiency studied. Triheptanoin (C7 oil, liquid). Subjects consumed triheptanoin 4 times over the course of one day. The approach included substituting a fraction of ketogenic diet fat with C7, weight by weight, at the maximum tolerable dose (45% of their their daily calories) in individuals receiving a ketogenic diet prior to enrolment prescribed independently of this study.
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.
Outcome measures
| Measure |
Ketosis
n=10 Participants
Participants BHB levels were checked daily on days 1 - 4. Treatment: Triheptanoin (C7), orally, dose of 45% of the total daily calories divided in four equally spaced administrations (i.e., one dose every 4 hours to a total of four doses in one day).
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Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate
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3 Participants
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SECONDARY outcome
Timeframe: Day 1 - Day 4Change in Glycemia is measured as changes in blood glucose levels.
Outcome measures
| Measure |
Ketosis
n=10 Participants
Participants BHB levels were checked daily on days 1 - 4. Treatment: Triheptanoin (C7), orally, dose of 45% of the total daily calories divided in four equally spaced administrations (i.e., one dose every 4 hours to a total of four doses in one day).
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|---|---|
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Change in Glycemia
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81.2 mg/dl
Standard Deviation 13.6
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Adverse Events
Ketosis
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Ketosis
n=10 participants at risk
Participants BHB levels were checked daily on days 1 - 4.
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|---|---|
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Nervous system disorders
Seizures
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20.0%
2/10 • Days 1 - 4
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Additional Information
Dr. Juan Pascual
University of Texas Southwestern Medical Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place