Adults 24 Months to 35, any sex, with GLUT1DS1 or Epilepsy. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Neuropsychological Score of Sustained AttentionPrimary· Change post 6 months of treatment
Sustained attention was evaluated using a subtest of Conners' Kiddie Continuous Performance Test Second Edition (K-CPT 2): CPT-Hit Reaction Time Block Change. CPT HRT BC indicates the change in mean response speed as the administration of the test progresses in blocks. A decrease in CPT HRT BC indicates a decrease in reaction time, which means the participant's information processing efficiency increases, and an improvement in sustained attention is noted. The number of participants that showed a decrease in the CPT HRT BC score after 6 months of treatment as compared to baseline is noted here
Group
Value
95% CI
Triheptanoin
11
Neuropsychological Score of Sustained AttentionPrimary· Change after 3 months off treatment
Sustained attention was evaluated using a subtest of Conners' Kiddie Continuous Performance Test Second Edition (K-CPT 2): CPT-Hit Reaction Time Block Change. CPT HRT BC indicates the change in mean response speed as the administration of the test progresses in blocks. A decrease in CPT HRT BC indicates a decrease in reaction time, which means the participant's information processing efficiency increases, and an improvement in sustained attention is noted. The off-treatment period of 3 months was implemented to study whether the impact of treatment persists or goes back to baseline. The number
Group
Value
95% CI
Triheptanoin
14
Neuropsychological Score of Working Memory Index Scale (WMI)Primary· Change post 6 months of treatment
Subjects were administered the Working Memory Index Scale (WMI) from either the Wechsler Primary and Preschool Scale of Intelligence, 4th Edition (WPPSI-IV), Wechsler Intelligence Scale for Children, 5th Edition (WISC-V), or the Wechsler Adult Intelligence Scale, 4th Edition (WAIS-IV) according to the age of subject. An increase WMI score would indicate an improvement in the cognitive ability of identifying, reorganizing and retaining information for a brief period of time. The number of participants that showed an increase in the WMI score after 6 months of treatment is noted here.
Group
Value
95% CI
Triheptanoin
7
Neuropsychological Score of Working Memory Index Scale (WMI)Primary· Change after 3 months off-treatment
Subjects were administered the Working Memory Index Scale (WMI) from either the Wechsler Primary and Preschool Scale of Intelligence, 4th Edition (WPPSI-IV), Wechsler Intelligence Scale for Children, 5th Edition (WISC-V), or the Wechsler Adult Intelligence Scale, 4th Edition (WAIS-IV) according to the age of subject. An increase WMI score would indicate an improvement in the cognitive ability of identifying, reorganizing and retaining information for a brief period of time. The 3 months off-treatment period was designed to study whether the impact of treatment persists or goes back to baseline
Group
Value
95% CI
Triheptanoin
5
Neuropsychological Score of Processing Speed Index (PSI)Primary· Change post 6 months of treatment
Subjects were administered the Processing Speed Index Scale (PSI) from either the Wechsler Primary and Preschool Scale of Intelligence, 4th Edition (WPPSI-IV), Wechsler Intelligence Scale for Children, 5th Edition (WISC-V) or the Wechsler Adult Intelligence Scale, 4th Edition (WAIS-IV) according to the age of subject. An increase PSI score would indicate an improvement in the motor-based estimate of the subject's cognitive processing speed. The number of participants that showed an increase in the PSI score after 6 months of treatment as compared to baseline is noted here.
Group
Value
95% CI
Triheptanoin
6
Neuropsychological Score of Processing Speed Index (PSI)Primary· Change after 3 months off-treatment
Subjects were administered the Processing Speed Index Scale (PSI) from either the Wechsler Primary and Preschool Scale of Intelligence, 4th Edition (WPPSI-IV), Wechsler Intelligence Scale for Children, 5th Edition (WISC-V) or the Wechsler Adult Intelligence Scale, 4th Edition (WAIS-IV) according to the age of subject. An increase PSI score would indicate an improvement in the motor-based estimate of the subject's cognitive processing speed. The 3 months off-treatment period was designed to study whether the impact of treatment persists or goes back to baseline. The number of participants that
Group
Value
95% CI
Triheptanoin
7
EEG Changes: Generalized Spike Wave Activity and BurstSecondary· Change post 6 months of treatment
The number of generalized spike wave (GSW) activity and bursts were extracted from the patient EEGs. GSW and bursts per hour was calculated. A decrease in the spike wave and burst indicated an improvement. The number of patients that displayed a decrease in GSW and burst per hour after 6 months of treatment in noted here.
GSW/hr
Group
Value
95% CI
Triheptanoin
10
Bursts/hr
Group
Value
95% CI
Triheptanoin
7
EEG Changes: Generalized Spike Wave Activity and BurstSecondary· Change after 3 months off-treatment
The number of generalized spike wave (GSW) activity and bursts were extracted from the patient EEGs. GSW and bursts per hour was calculated. A decrease in the spike wave and burst indicated an improvement. The off-treatment period of 3 months was implemented to study whether the impact of treatment persists or goes back to baseline. The number of patients that displayed an increase in GSW and burst per hour (towards baseline) after 3 months off treatment as compared to 6 months on treatment is noted here.
GSW/hr
Group
Value
95% CI
Triheptanoin
13
Bursts/hr
Group
Value
95% CI
Triheptanoin
11
Brief Ataxia Rating ScaleSecondary· Change post 6 months of treatment
Ataxia is scored as per the Brief ataxia rating scale (BARS) - a modified form of the International Cooperative Ataxia Rating Scale (ICARS). The possible range for the scores is from 0 (normal, no ataxia) to 30 (severe ataxia). A decrease in the BARS score indicates an improvement in the ataxia symptoms. The number of participants that showed a decrease in BARS score after 6 months of intervention is reported here.
Group
Value
95% CI
Triheptanoin
13
Brief Ataxia Rating ScaleSecondary· Change after 3 months off treatment
Ataxia is scored as per the Brief ataxia rating scale (BARS) - a modified form of the International Cooperative Ataxia Rating Scale (ICARS). The possible range for the scores is from 0 (normal, no ataxia) to 30 (severe ataxia). A decrease in the BARS score indicates an improvement in the ataxia symptoms. The off-treatment period of 3 months was implemented to study whether the impact of treatment persists or goes back to baseline. The number of participants that showed a subsequent increase in BARS score (towards baseline) after the of 3 months of no treatment as compared to 6 months on treatm
Group
Value
95% CI
Triheptanoin
11
Clinical Global Impression Severity ScaleSecondary· Change post 6 months of treatment
The Clinical global impression - Severity (CGI-S) scale is used to evaluate the illness severity where the scores range from 1 (very much improved) through to 7 (very much worse). A decrease in the CGI-S score indicates a decrease in illness severity. The number of participants that showed a decrease in CGI-S score after 6 months of intervention as compared to baseline is reported here.
Group
Value
95% CI
Triheptanoin
10
Clinical Global Impression Severity ScaleSecondary· Change after 3 months off-treatment
The Clinical global impression - Severity (CGI-S) scale is used to evaluate the illness severity where the scores range from 1 (very much improved) through to 7 (very much worse). A decrease in the CGI-S score indicates a decrease in illness severity. The off-treatment period of 3 months was designed to study whether the impact of treatment persists or goes back to baseline. The number of participants that showed an increase in CGI-S score (towards baseline) after 3 months off treatment as compared to 6 months on treatment is reported
Group
Value
95% CI
Triheptanoin
9
Adverse events — posted to ClinicalTrials.gov
Time frame: 9 months.
Reporting threshold: 3%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Forty-five subjects receiving no dietary therapy with a proven G1D diagnosis will be enrolled. To evaluate the effect of C7 supplementation of a regular diet on a EEG activity in addition to IQ, language, working memory, processing speed, emotional and behavioral functioning, ataxia, and other neuropsychological and neurological performance indices in children and adults genetically diagnosed with G1D receiving a regular diet at enrollment.
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by University of Texas Southwestern Medical Center
Last refreshed: 7 August 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03181399.