Trial · NCT02214160
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
NCT02214160, Phase 2, Completed, Ultragenyx Pharmaceutical Inc; results first posted 2021-12-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies. Trials, record NCT02214160. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02214160
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
- NCT number
- NCT02214160
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 94 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency, Carnitine-acylcarnitine Translocase (CACT) Deficiency
- Interventions
- UX007
- First posted
- 2014-08-12
- Last updated
- 2023-08-01
- Other registries' IDs
- eudract:2016-000322-19
- Results first posted
- 2021-12-08
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) | The annualized LC-FAOD MCE rate, inclusive of skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia) and cardiomyopathy events, defined as any visit to the emergency room (ER)/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized event rate was calculated at the number of events divided by the duration of data collection period in days/365.25 |
| Primary | Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) | The annualized LC-FAOD MCE rate, inclusive of skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia) and cardiomyopathy events, defined as any visit to the emergency room (ER)/acute care, hospitalization, emergency intervention (i.e. any unscheduled administration of therapeutics at home or in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD. The annualized event rate was calculated at the number of events divided by the duration of data collection period in days/365.25 |
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | Post-UX007 treatment through the end of treatment (up to 2072 days) plus 30-35 days | An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. A serious adverse event (SAE) 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; an important medical event. AEs were graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 (mild=1, moderate=2, severe=3, life-threatening=4, death=5). |
| Secondary | Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) | Baseline, Month 12, Month 18, Month 24, Month 30, Month 36, Month 48, Month 60 | |
| Secondary | Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) | Baseline, Month 12, Month 24, Month 36 | The Z-scores express the deviation (or how far away) the measure is from the mean LVEF based on the size or age of the pediatric participants: Z-score=0 indicates the participant is exactly the same as the mean of the healthy general population. Z-score=-1 indicates it's 1 standard deviation below the mean of the healthy population. Z-score=+1 indicates it's 1 standard deviation above the mean. |
| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) | Baseline, Month 12, Month 24, Month 30, Month 36, Month 48, Month 60 | Fractional shortening is calculated by measuring the percentage change in left ventricular diameter during systole. A negative value indicates less ventricular/muscular contractility, and a positive value indicates more ventricular/muscular contractility. |
| Secondary | Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | The Z-scores express the deviation (or how far away) the measure is from the mean LVSF based on the size or age of the pediatric participants: Z-score=0 indicates the participant is exactly the same as the mean of the healthy general population. Z-score=-1 indicates it's 1 standard deviation below the mean of the healthy population. Z-score=+1 indicates it's 1 standard deviation above the mean. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | events/year | MEAN |
| PRIMARY | Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Per protocol, pre-UX007 MCE data was not collected in the IST/Other Cohort. | events/year | MEDIAN |
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | Post-UX007 treatment through the end of treatment (up to 2072 days) plus 30-35 days | Safety Analysis Set: participants who received at least 1 dose of study drug. | participants | NUMBER |
| SECONDARY | Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Participants with a value at baseline and given time point. | g/m | MEAN |
| SECONDARY | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Participants with a value at baseline and given time point. | g | MEAN |
| SECONDARY | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Participants with a value at baseline and given time point. | mm | MEAN |
| SECONDARY | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) | Baseline, Month 12, Month 18, Month 24, Month 30, Month 36, Month 48, Month 60 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Participants with a value at baseline and given time point. | percent of blood ejected during systole | MEAN |
| SECONDARY | Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) | Baseline, Month 12, Month 24, Month 36 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Pediatric participants with a value at baseline and given time point. | Z-score | MEAN |
| SECONDARY | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) | Baseline, Month 12, Month 24, Month 30, Month 36, Month 48, Month 60 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Participants with a value at baseline and given time point. | % change in left ventricular diameter | MEAN |
| SECONDARY | Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. Pediatric participants with a value at baseline and given time point. | Z-score | MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group148
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort | UX007-CL201-Rollover Cohort | Pre-UX007 Period | 1.76 | Standard Deviation 1.64 | events/year |
| Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort | UX007-CL201-Rollover Cohort | UX007 Treatment Period | 1.00 | Standard Deviation 1.00 | events/year |
| Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | Triheptanoin-Naïve Cohort | Pre-UX007 Period | 2.00 | Inter-Quartile Range 0.67 to 3.33 | events/year |
| Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | IST/Other Cohort | UX007 Treatment Period | 0.57 | Inter-Quartile Range 0.0 to 1.67 | events/year |
| Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | Triheptanoin-Naïve Cohort | UX007 Treatment Period | 0.28 | Inter-Quartile Range 0.00 to 1.43 | events/year |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | UX007-CL201-Rollover Cohort | >= 1 TEAE | 24 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | IST/Other Cohort | >= 1 TEAE | 35 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | Triheptanoin-Naïve Cohort | >= 1 TEAE | 32 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | UX007-CL201-Rollover Cohort | Treatment-Related TEAEs | 14 | participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | IST/Other Cohort | Treatment-Related TEAEs | 22 | participants |
1 to 10 of 148Source ClinicalTrials.gov
See all 148 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | UX007-CL201-Rollover Cohort | 24 | |
| Overall Study | STARTED | IST/Other Cohort | 37 | |
| Overall Study | STARTED | Triheptanoin-Naïve Cohort | 33 | |
| Overall Study | COMPLETED | UX007-CL201-Rollover Cohort | 20 | |
| Overall Study | COMPLETED | IST/Other Cohort | 29 | |
| Overall Study | COMPLETED | Triheptanoin-Naïve Cohort | 23 | |
| Overall Study | NOT COMPLETED | UX007-CL201-Rollover Cohort | 4 | |
| Overall Study | NOT COMPLETED | IST/Other Cohort | 8 | |
| Overall Study | NOT COMPLETED | Triheptanoin-Naïve Cohort | 10 |
Source ClinicalTrials.gov
Adverse events824
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 | ANAEMIA | Blood and lymphatic system disorders | UX007-CL201-Rollover Cohort | 0 | 24 | |
| other | ANAEMIA | Blood and lymphatic system disorders | IST/Other Cohort | 1 | 37 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Triheptanoin-Naïve Cohort | 2 | 33 | |
| other | ANAEMIA | Blood and lymphatic system disorders | All Participants | 3 | 94 | |
| other | CARDIOMYOPATHY | Cardiac disorders | UX007-CL201-Rollover Cohort | 3 | 24 | |
| other | CARDIOMYOPATHY | Cardiac disorders | IST/Other Cohort | 3 | 37 | |
| other | CARDIOMYOPATHY | Cardiac disorders | Triheptanoin-Naïve Cohort | 1 | 33 | |
| other | CARDIOMYOPATHY | Cardiac disorders | All Participants | 7 | 94 | |
| other | PALPITATIONS | Cardiac disorders | UX007-CL201-Rollover Cohort | 2 | 24 | |
| other | PALPITATIONS | Cardiac disorders | IST/Other Cohort | 0 | 37 |
1 to 10 of 824Source ClinicalTrials.gov
See all 824 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | UX007-CL201-Rollover Cohort | 2 | 24 |
| Total, all-cause mortality | IST/Other Cohort | 2 | 37 |
| Total, all-cause mortality | Triheptanoin-Naïve Cohort | 1 | 33 |
| Total, all-cause mortality | All Participants | 5 | 94 |
| Total, other adverse events | UX007-CL201-Rollover Cohort | 24 | 24 |
| Total, other adverse events | IST/Other Cohort | 34 | 37 |
| Total, other adverse events | Triheptanoin-Naïve Cohort | 32 | 33 |
| Total, other adverse events | All Participants | 90 | 94 |
| Total, serious adverse events | UX007-CL201-Rollover Cohort | 20 | 24 |
| Total, serious adverse events | IST/Other Cohort | 28 | 37 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
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/NCT02214160
GET https://conductscience.org/api/trials/v1/NCT02214160/results?page=2