1 and older, any sex, with Glucose Transporter Type 1 Deficiency Syndrome. 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.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and DeathsPrimary· From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the firs
TEAEs
Group
Value
95% CI
UX007 (Triheptanoin)
13
Serious TEAEs
Group
Value
95% CI
UX007 (Triheptanoin)
2
Related TEAEs
Group
Value
95% CI
UX007 (Triheptanoin)
10
Serious and Related TEAEs
Group
Value
95% CI
UX007 (Triheptanoin)
0
Grade 3 or 4 TEAEs
Group
Value
95% CI
UX007 (Triheptanoin)
1
Gastrointestinal TEAEs
Group
Value
95% CI
UX007 (Triheptanoin)
9
TEAEs Leading to Treatment Discontinuation
Group
Value
95% CI
UX007 (Triheptanoin)
0
TEAEs Leading to Study Discontinuation
Group
Value
95% CI
UX007 (Triheptanoin)
0
Change From Baseline Over Time in Overall Seizure Frequency Per 4 WeeksSecondary· Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36
The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence.
Change at Month 0-3
Group
Value
95% CI
UX007 (Triheptanoin)
-64.22
± 185.564
Change at Month 4-6
Group
Value
95% CI
UX007 (Triheptanoin)
-64.19
± 142.460
Change at Month 7-9
Group
Value
95% CI
UX007 (Triheptanoin)
-61.81
± 164.770
Change at Month 10-12
Group
Value
95% CI
UX007 (Triheptanoin)
-91.93
± 216.834
Change at Month 13-18
Group
Value
95% CI
UX007 (Triheptanoin)
-75.56
± 206.406
Change at Month 19-24
Group
Value
95% CI
UX007 (Triheptanoin)
-110.12
± 233.492
Change at Month 25-30
Group
Value
95% CI
UX007 (Triheptanoin)
-135.60
± 249.871
Change at Month 31-36
Group
Value
95% CI
UX007 (Triheptanoin)
-51.88
± 101.378
Change From Baseline Over Time in CNS Total ScoreSecondary· Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36
The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance \& Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone \& Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function.
Change at Month 0
Group
Value
95% CI
UX007 (Triheptanoin)
11.85
± 20.331
Change at Month 6
Group
Value
95% CI
UX007 (Triheptanoin)
9.36
± 18.495
Change at Month 12
Group
Value
95% CI
UX007 (Triheptanoin)
13.64
± 21.371
Change at Month 24
Group
Value
95% CI
UX007 (Triheptanoin)
3.38
± 3.092
Change at Month 36
Group
Value
95% CI
UX007 (Triheptanoin)
0.00
± NA
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary ScoreSecondary· Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30
The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.
Change at Month 0
Group
Value
95% CI
UX007 (Triheptanoin)
0.25
± 16.917
Change at Month 6
Group
Value
95% CI
UX007 (Triheptanoin)
9.56
± 21.522
Change at Month 12
Group
Value
95% CI
UX007 (Triheptanoin)
-2.67
± 9.475
Change at Month 18
Group
Value
95% CI
UX007 (Triheptanoin)
-9.35
± 12.702
Change at Month 24
Group
Value
95% CI
UX007 (Triheptanoin)
-8.96
± 20.075
Change at Month 30
Group
Value
95% CI
UX007 (Triheptanoin)
7.74
± 2.273
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary ScoreSecondary· Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30
The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.
Change at Month 0
Group
Value
95% CI
UX007 (Triheptanoin)
0.59
± 11.049
Change at Month 6
Group
Value
95% CI
UX007 (Triheptanoin)
-2.29
± 12.366
Change at Month 12
Group
Value
95% CI
UX007 (Triheptanoin)
-7.43
± 15.514
Change at Month 18
Group
Value
95% CI
UX007 (Triheptanoin)
-6.60
± 14.987
Change at Month 24
Group
Value
95% CI
UX007 (Triheptanoin)
-8.25
± 15.316
Change at Month 30
Group
Value
95% CI
UX007 (Triheptanoin)
8.91
± 12.599
Change From Baseline Over Time in SF-12v2 Health Survey PCS ScoreSecondary· Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18
SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.
Change at Month 0
Group
Value
95% CI
UX007 (Triheptanoin)
10.25
± 5.077
Change at Month 6
Group
Value
95% CI
UX007 (Triheptanoin)
12.37
± 5.367
Change at Month 12
Group
Value
95% CI
UX007 (Triheptanoin)
5.09
± 1.619
Change at Month 18
Group
Value
95% CI
UX007 (Triheptanoin)
-0.35
± NA
Change From Baseline Over Time in SF-12v2 Health Survey MCS ScoreSecondary· Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18
SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.
Change at Month 0
Group
Value
95% CI
UX007 (Triheptanoin)
8.63
± 2.249
Change at Month 6
Group
Value
95% CI
UX007 (Triheptanoin)
5.84
± 3.316
Change at Month 12
Group
Value
95% CI
UX007 (Triheptanoin)
6.90
± 5.706
Change at Month 18
Group
Value
95% CI
UX007 (Triheptanoin)
16.96
± NA
Adverse events — posted to ClinicalTrials.gov
Time frame: From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
NCT02960217 — Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucos
· Phase 3
· terminated
NCT02214160 — Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Stu
· Phase 2
· completed
NCT01993186 — Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
· Phase 2
· completed
NCT01886378 — A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
· Phase 2
· completed
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· Phase 2
· terminated
NCT04783428 — Tumor-induced Osteomalacia Disease Monitoring Program
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· completed
Publications: Europe PMC API search by NCT ID, retrieved 9 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 Ultragenyx Pharmaceutical Inc
Last refreshed: 11 June 2020
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/NCT02599961.