Trial · NCT02599961
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
NCT02599961, Phase 2, Terminated, Ultragenyx Pharmaceutical Inc; results first posted 2020-04-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS). Trials, record NCT02599961. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02599961
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
- NCT number
- NCT02599961
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 15 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Glucose Transporter Type 1 Deficiency Syndrome
- Interventions
- UX007
- First posted
- 2015-11-09
- Last updated
- 2020-06-11
- Other registries' IDs
- eudract:2015-000389-69
- Results first posted
- 2020-04-30
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | 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 first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE. |
| Secondary | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | 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. |
| Secondary | Change From Baseline Over Time in CNS Total Score | 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. |
| Secondary | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | 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. |
| Secondary | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | 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. |
| Secondary | Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | 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. |
| Secondary | Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | 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. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days. | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | 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 | Participants with a baseline and postbaseline assessment at given time point. | seizures per 4 weeks | MEAN |
| SECONDARY | Change From Baseline Over Time in CNS Total Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36 | Participants with a baseline and postbaseline assessment at given time point. | score on a scale | MEAN |
| SECONDARY | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 | Pediatric participants with a baseline and postbaseline assessment at given time point. | T-score | MEAN |
| SECONDARY | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 | Pediatric participants with a baseline and postbaseline assessment at given time point. | T-score | MEAN |
| SECONDARY | Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 | Adult participants with a baseline and postbaseline assessment at given time point. | T-score | MEAN |
| SECONDARY | Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 | Adult participants with a baseline and postbaseline assessment at given time point. | T-score | MEAN |
Source ClinicalTrials.gov
Results by group42
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | TEAEs | 13 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | Serious TEAEs | 2 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | Related TEAEs | 10 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | Serious and Related TEAEs | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | Grade 3 or 4 TEAEs | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | Gastrointestinal TEAEs | 9 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | TEAEs Leading to Treatment Discontinuation | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | TEAEs Leading to Study Discontinuation | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | UX007 (Triheptanoin) | TEAEs Leading to Death | 0 | Participants | |
| Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | UX007 (Triheptanoin) | Change at Month 0-3 | -64.22 | Standard Deviation 185.564 | seizures per 4 weeks |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | UX007 (Triheptanoin) | 15 | |
| Overall Study | COMPLETED | UX007 (Triheptanoin) | 0 | |
| Overall Study | NOT COMPLETED | UX007 (Triheptanoin) | 15 |
Source ClinicalTrials.gov
Adverse events57
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Tachycardia | Cardiac disorders | UX007 (Triheptanoin) | 1 | 15 | |
| other | Abdominal Pain | Gastrointestinal disorders | UX007 (Triheptanoin) | 3 | 15 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | UX007 (Triheptanoin) | 1 | 15 | |
| other | Breath Odour | Gastrointestinal disorders | UX007 (Triheptanoin) | 1 | 15 | |
| other | Constipation | Gastrointestinal disorders | UX007 (Triheptanoin) | 1 | 15 | |
| other | Diarrhoea | Gastrointestinal disorders | UX007 (Triheptanoin) | 7 | 15 | |
| other | Dysphagia | Gastrointestinal disorders | UX007 (Triheptanoin) | 1 | 15 | |
| other | Flatulence | Gastrointestinal disorders | UX007 (Triheptanoin) | 1 | 15 | |
| other | Haematochezia | Gastrointestinal disorders | UX007 (Triheptanoin) | 1 | 15 | |
| other | Nausea | Gastrointestinal disorders | UX007 (Triheptanoin) | 3 | 15 |
1 to 10 of 57Source ClinicalTrials.gov
See all 57 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | UX007 (Triheptanoin) | 0 | 15 |
| Total, other adverse events | UX007 (Triheptanoin) | 13 | 15 |
| Total, serious adverse events | UX007 (Triheptanoin) | 2 | 15 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02599961
GET https://conductscience.org/api/trials/v1/NCT02599961/results?page=2