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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-000322-19 | EudraCT Number |
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The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in LC-FAOD and evaluate the impact of UX007 on clinical events associated with LC-FAOD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| UX007 | Experimental | Participants previously treated with UX007 or treatment-naive participants will begin or continue treatment with daily open-label UX007 while maintaining their other dietary restrictions. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| UX007 | Drug | Administered orally (PO) with food or by gastrostomy tube, at the target dose range of 25-35% of total calories. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort | 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 | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) |
| Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | 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 | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
| Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California San Francisco | San Francisco | California | 94143 | United States | ||
| Children's National Health System |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32885845 | Background | Vockley J, Burton B, Berry G, Longo N, Phillips J, Sanchez-Valle A, Chapman K, Tanpaiboon P, Grunewald S, Murphy E, Lu X, Cataldo J. Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study. J Inherit Metab Dis. 2021 Jan;44(1):253-263. doi: 10.1002/jimd.12313. Epub 2020 Sep 14. | |
| 37276053 |
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| ID | Title | Description |
|---|---|---|
| FG000 | UX007-CL201-Rollover Cohort | Participants who participated in the UX007-CL201 study (NCT01886378) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 1, 2019 | Sep 15, 2021 |
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| Post-UX007 treatment through the end of treatment (up to 2072 days) plus 30-35 days |
| Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |
| 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 |
| Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) | 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. | Baseline, Month 12, Month 24, Month 36 |
| Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) | 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. | Baseline, Month 12, Month 24, Month 30, Month 36, Month 48, Month 60 |
| Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) | 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. | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |
| Annualized Duration Rate of All MCEs | The annualized duration rate of LC-FAOD MCEs, inclusive of skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia) and cardiomyopathy events, and defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. | Post-UX007 treatment through the end of the study (up to 2072 days) |
| Annualized Event Rate of Rhabdomyolysis MCEs | The annualized event rate of LC-FAOD major events of skeletal myopathy (rhabdomyolysis), defined as any visit to the 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. | Post-UX007 treatment through the end of the study (up to 2072 days) |
| Annualized Duration Rate of Rhabdomyolysis MCEs | The annualized duration rate of LC-FAOD skeletal myopathy (rhabdomyolysis) MCEs, defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. | Post-UX007 treatment through the end of the study (up to 2072 days) |
| Annualized Event Rate of Cardiomyopathy MCEs | The annualized event rate of LC-FAOD major events inclusive of cardiomyopathy events, defined as any visit to the 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. | Post-UX007 treatment through the end of the study (up to 2072 days) |
| Annualized Duration Rate of Cardiomyopathy MCEs | The annualized duration rate of LC-FAOD cardiomyopathy MCEs, defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25 | Post-UX007 treatment through the end of the study (up to 2072 days) |
| Annualized Event Rate of Hypoglycemic MCEs | The annualized event rate of LC-FAOD major events of hepatic (hypoglycemia) events, defined as any visit to the 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. | Post-UX007 treatment through the end of the study (up to 2072 days) |
| Annualized Duration Rate of Hypoglycemic MCEs | The annualized duration rate of LC-FAOD hepatic (hypoglycemia) MCEs, defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. | Post-UX007 treatment through the end of the study (up to 2072 days) |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| University of South Florida | Tampa | Florida | 33606 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Vanderbilt Medical Center | Nashville | Tennessee | 37232 | United States |
| University of Utah | Salt Lake City | Utah | 84108 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| National Hospital for Neurology and Neurosurgery | London | WC1N 3BP | United Kingdom |
| Great Ormond Street Hospital | London | WC1N 3JH | United Kingdom |
| Vockley J, Burton BK, Berry G, Longo N, Phillips J, Sanchez-Valle A, Chapman KA, Tanpaiboon P, Grunewald S, Murphy E, Lu X, Rahman S, Ray K, Reineking B, Pisani L, Ramirez AN. Triheptanoin for the treatment of long-chain fatty acid oxidation disorders: Final results of an open-label, long-term extension study. J Inherit Metab Dis. 2023 Sep;46(5):943-955. doi: 10.1002/jimd.12640. Epub 2023 Jun 19. |
| IST/Other Cohort |
Participants who were previously treated with UX007/triheptanoin (including food-grade triheptanoin) in an investigator sponsored trial (IST) or another UX007/triheptanoin study receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
| FG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | UX007-CL201-Rollover Cohort | Participants who participated in the UX007-CL201 study (NCT01886378) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
| BG001 | IST/Other Cohort | Participants who were previously treated with UX007/triheptanoin (including food-grade triheptanoin) in an investigator sponsored trial (IST) or another UX007/triheptanoin study receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
| BG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort | 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 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Mean | Standard Deviation | events/year | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) |
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| Primary | Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort | 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 | 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. | Posted | Median | Inter-Quartile Range | events/year | Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days) |
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| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs | 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). | Safety Analysis Set: participants who received at least 1 dose of study drug. | Posted | Number | participants | Post-UX007 treatment through the end of treatment (up to 2072 days) plus 30-35 days |
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| Secondary | Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) | 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. | Posted | Mean | Standard Deviation | g/m | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |
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| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) | 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. | Posted | Mean | Standard Deviation | g | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |
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| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) | 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. | Posted | Mean | Standard Deviation | mm | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |
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| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) | 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. | Posted | Mean | Standard Deviation | percent of blood ejected during systole | Baseline, Month 12, Month 18, Month 24, Month 30, Month 36, Month 48, Month 60 |
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| Secondary | Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) | 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. | 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. | Posted | Mean | Standard Deviation | Z-score | Baseline, Month 12, Month 24, Month 36 |
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| Secondary | Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) | 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. | 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. | Posted | Mean | Standard Deviation | % change in left ventricular diameter | Baseline, Month 12, Month 24, Month 30, Month 36, Month 48, Month 60 |
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| Secondary | Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) | 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. | 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. | Posted | Mean | Standard Deviation | Z-score | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |
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| Secondary | Annualized Duration Rate of All MCEs | The annualized duration rate of LC-FAOD MCEs, inclusive of skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia) and cardiomyopathy events, and defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Median | Inter-Quartile Range | days/year | Post-UX007 treatment through the end of the study (up to 2072 days) |
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| Secondary | Annualized Event Rate of Rhabdomyolysis MCEs | The annualized event rate of LC-FAOD major events of skeletal myopathy (rhabdomyolysis), defined as any visit to the 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. | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Median | Inter-Quartile Range | events/year | Post-UX007 treatment through the end of the study (up to 2072 days) |
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| Secondary | Annualized Duration Rate of Rhabdomyolysis MCEs | The annualized duration rate of LC-FAOD skeletal myopathy (rhabdomyolysis) MCEs, defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Median | Inter-Quartile Range | days/year | Post-UX007 treatment through the end of the study (up to 2072 days) |
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| Secondary | Annualized Event Rate of Cardiomyopathy MCEs | The annualized event rate of LC-FAOD major events inclusive of cardiomyopathy events, defined as any visit to the 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. | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Median | Inter-Quartile Range | event/year | Post-UX007 treatment through the end of the study (up to 2072 days) |
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| Secondary | Annualized Duration Rate of Cardiomyopathy MCEs | The annualized duration rate of LC-FAOD cardiomyopathy MCEs, defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25 | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Median | Inter-Quartile Range | days/year | Post-UX007 treatment through the end of the study (up to 2072 days) |
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| Secondary | Annualized Event Rate of Hypoglycemic MCEs | The annualized event rate of LC-FAOD major events of hepatic (hypoglycemia) events, defined as any visit to the 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. | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Median | Inter-Quartile Range | events/year | Post-UX007 treatment through the end of the study (up to 2072 days) |
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| Secondary | Annualized Duration Rate of Hypoglycemic MCEs | The annualized duration rate of LC-FAOD hepatic (hypoglycemia) MCEs, defined as any visit to the 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 duration rate is calculated as the total duration (days) of events divided by the duration of data collection period in days/365.25. | Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment. | Posted | Median | Inter-Quartile Range | days/year | Post-UX007 treatment through the end of the study (up to 2072 days) |
|
Post-UX007 treatment through the end of treatment (up to 2072 days) plus 30-35 days
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | UX007-CL201-Rollover Cohort | Participants who participated in the UX007-CL201 study (NCT01886378) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. | 2 | 24 | 20 | 24 | 24 | 24 |
| EG001 | IST/Other Cohort | Participants who were previously treated with UX007/triheptanoin (including food-grade triheptanoin) in an investigator sponsored trial (IST) or another UX007/triheptanoin study receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. | 2 | 37 | 28 | 37 | 34 | 37 |
| EG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. | 1 | 33 | 22 | 33 | 32 | 33 |
| EG003 | All Participants | All participants received UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. | 5 | 94 | 70 | 94 | 90 | 94 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ARRHYTHMIA | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CARDIAC ARREST | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CARDIAC FAILURE | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CARDIAC FAILURE ACUTE | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CARDIO-RESPIRATORY ARREST | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CARDIOMYOPATHY | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CARDIOPULMONARY FAILURE | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CONGESTIVE CARDIOMYOPATHY | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| LEFT VENTRICULAR HYPERTROPHY | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| LONG QT SYNDROME | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MYOCARDITIS | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CHRONIC GASTRITIS | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| GASTRIC ULCER HAEMORRHAGE | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| GASTRITIS | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROINTESTINAL DISORDER | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROOESOPHAGEAL REFLUX DISEASE | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ILEUS | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PANCREATITIS ACUTE | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RECTAL POLYP | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| STEATORRHOEA | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| VOLVULUS | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CELLULITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| CLOSTRIDIUM DIFFICILE INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| CROUP INFECTIOUS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| DEVICE RELATED INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| DIVERTICULITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| ESCHERICHIA URINARY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| GASTRITIS VIRAL | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROENTERITIS VIRAL | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROINTESTINAL VIRAL INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| HERPES SIMPLEX | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| INFLUENZA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| KLEBSIELLA BACTERAEMIA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| KLEBSIELLA INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| ORAL CANDIDIASIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PARAINFLUENZAE VIRUS INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PHARYNGITIS STREPTOCOCCAL | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PNEUMONIA STAPHYLOCOCCAL | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PYELONEPHRITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| RESPIRATORY TRACT INFECTION VIRAL | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| RHINOVIRUS INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| SEPSIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| STAPHYLOCOCCAL BACTERAEMIA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| STAPHYLOCOCCAL INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| TONSILLITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| TRACHEITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| VIRAL CARDIOMYOPATHY | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| VIRAL UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| OVERDOSE | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| PROCEDURAL PAIN | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| AMMONIA INCREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| BLOOD CREATINE PHOSPHOKINASE INCREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| BRAIN NATRIURETIC PEPTIDE INCREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| INFLUENZA A VIRUS TEST POSITIVE | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| TRANSAMINASES INCREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| ACIDOSIS | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DEHYDRATION | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPERAMMONAEMIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOKALAEMIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOPHAGIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| METABOLIC DISORDER | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| JOINT INSTABILITY | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MYALGIA | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| OSTEOPOROSIS | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RHABDOMYOLYSIS | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| SCOLIOSIS | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PLEOMORPHIC ADENOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 20.0 | Systematic Assessment |
| |
| HAEMORRHAGE INTRACRANIAL | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ISCHAEMIC STROKE | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| LETHARGY | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MIGRAINE | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| NEUROPATHY PERIPHERAL | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| POLYNEUROPATHY | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| SEIZURE | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ANXIETY | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| LISTLESS | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ACUTE KIDNEY INJURY | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RENAL FAILURE | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ATELECTASIS | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CHRONIC RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PULMONARY OEDEMA | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RESPIRATORY DISTRESS | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| TONSILLAR HYPERTROPHY | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ELECTIVE PROCEDURE | Surgical and medical procedures | MedDRA 20.0 | Systematic Assessment |
| |
| HEART TRANSPLANT | Surgical and medical procedures | MedDRA 20.0 | Systematic Assessment |
| |
| HAEMODYNAMIC INSTABILITY | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| POOR VENOUS ACCESS | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CARDIOMYOPATHY | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PALPITATIONS | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| EAR PAIN | Ear and labyrinth disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MOTION SICKNESS | Ear and labyrinth disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ASTIGMATISM | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPERMETROPIA | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MYOPIA | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ABDOMINAL DISCOMFORT | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ABDOMINAL DISTENSION | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ABDOMINAL PAIN UPPER | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DENTAL CARIES | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DYSPEPSIA | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROINTESTINAL DISORDER | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROINTESTINAL PAIN | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROOESOPHAGEAL REFLUX DISEASE | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| TOOTH IMPACTED | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| TOOTHACHE | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| CHEST PAIN | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| INFUSION SITE EXTRAVASATION | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PAIN | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DRUG HYPERSENSITIVITY | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPERSENSITIVITY | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| SEASONAL ALLERGY | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| BRONCHITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| CELLULITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| CROUP INFECTIOUS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| EAR INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROENTERITIS VIRAL | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| GASTROINTESTINAL VIRAL INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| INFECTIOUS MONONUCLEOSIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| INFLUENZA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| LOWER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| OTITIS EXTERNA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| OTITIS MEDIA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PARAINFLUENZAE VIRUS INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PHARYNGITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PHARYNGITIS STREPTOCOCCAL | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| POST PROCEDURAL INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| RHINITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| SINUSITIS | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| VIRAL UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| CONTUSION | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| HAND FRACTURE | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| LIGAMENT SPRAIN | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| PROCEDURAL PAIN | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| STOMA SITE HYPERGRANULATION | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| BLOOD CHOLESTEROL INCREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| BLOOD CREATINE PHOSPHOKINASE INCREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| CARNITINE DECREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| ELECTROENCEPHALOGRAM ABNORMAL | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| INFLUENZA A VIRUS TEST POSITIVE | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| WEIGHT INCREASED | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| ABNORMAL WEIGHT GAIN | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DECREASED APPETITE | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DEHYDRATION | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| FLUID OVERLOAD | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPERGLYCAEMIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOCALCAEMIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOKALAEMIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOMAGNESAEMIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOPHAGIA | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| OBESITY | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MUSCLE SPASMS | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MUSCULAR WEAKNESS | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MYALGIA | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RHABDOMYOLYSIS | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOTONIA | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| LETHARGY | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| MIGRAINE | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| NEUROPATHY PERIPHERAL | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ANXIETY | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ATTENTION DEFICIT/HYPERACTIVITY DISORDER | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DEPRESSION | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ENURESIS | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| INSOMNIA | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ACUTE KIDNEY INJURY | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ATELECTASIS | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPOXIA | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| NASAL CONGESTION | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| OROPHARYNGEAL PAIN | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RESPIRATORY DISORDER | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RHINORRHOEA | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| ACNE | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DERMATITIS | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DERMATITIS CONTACT | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| DERMATITIS DIAPER | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| RASH | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| HYPERTENSION | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| POOR VENOUS ACCESS | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
|
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Ultragenyx Pharmaceutical Inc | 1-888-756-8657 | medinfo@ultragenyx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 21, 2020 | Sep 22, 2021 | SAP_001.pdf |
| ID | Term |
|---|---|
| C536353 | VLCAD deficiency |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C531010 | triheptanoin |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black or African American |
|
| White |
|
| Other, Not Specified |
|
|
|
|
| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
|
|
|
|
|
|
|
|
|
|
| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
|
|
| OG002 |
| Triheptanoin-Naïve Cohort |
Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
|
|
| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
|
|
| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
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| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
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| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
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| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
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| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
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| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
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| OG002 | Triheptanoin-Naïve Cohort | Participants who are UX007 treatment-naïve (i.e., naïve to both UX007 and food-grade triheptanoin), or who had failed conventional therapy (including those who participated in UX007-CL201 study previously but were off UX007 for more than 2 years preceding enrollment into CL202) receive UX007, administered orally with food or by gastronomy tube (usually 4 times per day: breakfast, lunch, dinner, and before bed), at the target dose range of 25-35% of total calories. |
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