Trial Outcomes & Findings for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies (NCT NCT02214160)

Last Updated: 2023-08-01

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

94 participants

Primary outcome timeframe

Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days)

Results posted on

2023-08-01

Participant Flow

Participant milestones

Participant milestones
Measure	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.	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.	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.
Overall Study STARTED	24	37	33
Overall Study COMPLETED	20	29	23
Overall Study NOT COMPLETED	4	8	10

Reasons for withdrawal

Reasons for withdrawal
Measure	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.	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.	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.
Overall Study Withdrawal by Subject	1	0	6
Overall Study Death	2	2	1
Overall Study Subject Non-Compliance	0	3	1
Overall Study Adverse Event	0	1	1
Overall Study Sponsor Decision	0	2	0
Overall Study Protocol Violation	1	0	0
Overall Study Physician Decision	0	0	1

Baseline Characteristics

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	IST/Other Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.	Total n=94 Participants Total of all reporting groups
Age, Continuous	13.16 years STANDARD_DEVIATION 14.310 • n=99 Participants	17.69 years STANDARD_DEVIATION 14.490 • n=107 Participants	9.33 years STANDARD_DEVIATION 9.745 • n=206 Participants	13.60 years STANDARD_DEVIATION 13.333 • n=7 Participants
Sex: Female, Male Female	10 Participants n=99 Participants	21 Participants n=107 Participants	14 Participants n=206 Participants	45 Participants n=7 Participants
Sex: Female, Male Male	14 Participants n=99 Participants	16 Participants n=107 Participants	19 Participants n=206 Participants	49 Participants n=7 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=99 Participants	4 Participants n=107 Participants	5 Participants n=206 Participants	12 Participants n=7 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	21 Participants n=99 Participants	30 Participants n=107 Participants	28 Participants n=206 Participants	79 Participants n=7 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	3 Participants n=107 Participants	0 Participants n=206 Participants	3 Participants n=7 Participants
Race/Ethnicity, Customized Asian	1 Participants n=99 Participants	1 Participants n=107 Participants	1 Participants n=206 Participants	3 Participants n=7 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=99 Participants	1 Participants n=107 Participants	2 Participants n=206 Participants	4 Participants n=7 Participants
Race/Ethnicity, Customized White	21 Participants n=99 Participants	33 Participants n=107 Participants	28 Participants n=206 Participants	82 Participants n=7 Participants
Race/Ethnicity, Customized Other, Not Specified	1 Participants n=99 Participants	2 Participants n=107 Participants	2 Participants n=206 Participants	5 Participants n=7 Participants

PRIMARY outcome

Timeframe: Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	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.	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.
Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort Pre-UX007 Period	1.76 events/year Standard Deviation 1.64	—	—
Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort UX007 Treatment Period	1.00 events/year Standard Deviation 1.00	—	—

PRIMARY outcome

Timeframe: Pre-UX007 treatment period (up to 18 months) and post-UX007 treatment period (up to 2072 days)

Population: 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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.	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.
Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort Pre-UX007 Period	—	2.00 events/year Interval 0.67 to 3.33	—
Annualized LC-FAOD MCEs Rate: 18 Months Pre- and Entire UX007 Period Comparison for IST/Other Cohort and Triheptanoin-Naïve Cohort UX007 Treatment Period	0.57 events/year Interval 0.0 to 1.67	0.28 events/year Interval 0.0 to 1.43	—

PRIMARY outcome

Timeframe: Post-UX007 treatment through the end of treatment (up to 2072 days) plus 30-35 days

Population: Safety Analysis Set: participants who received at least 1 dose of study drug.

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).

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs >= 1 TEAE	24 participants	35 participants	32 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs Treatment-Related TEAEs	14 participants	22 participants	28 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs Treatment-Related Gastrointestinal (GI) TEAEs	10 participants	19 participants	27 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs Grade 3 TEAEs	16 participants	26 participants	18 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs Grade 4 TEAEs	3 participants	2 participants	2 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs Serious TEAEs	20 participants	28 participants	22 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs Treatment-Related Serious TEAEs	1 participants	1 participants	3 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs TEAEs Leading to Treatment Discontinuation	1 participants	1 participants	1 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs TEAEs Leading to Study Discontinuation	0 participants	1 participants	1 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious TEAEs TEAEs Leading to Death	2 participants	2 participants	1 participants

SECONDARY outcome

Timeframe: Baseline, Month 12, Month 24, Month 36, Month 48, Month 60

Population: 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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) Change at Month 12	-5.27 g/m Standard Deviation 21.433	4.55 g/m Standard Deviation 21.903	-9.06 g/m Standard Deviation 29.965
Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) Change at Month 24	-2.73 g/m Standard Deviation 23.711	-0.37 g/m Standard Deviation 18.132	1.00 g/m Standard Deviation 39.459
Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) Change at Month 36	-6.75 g/m Standard Deviation 28.933	1.23 g/m Standard Deviation 19.488	12.33 g/m Standard Deviation 38.831
Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) Change at Month 48	-12.33 g/m Standard Deviation 29.263	-1.04 g/m Standard Deviation 21.900	-9.67 g/m Standard Deviation 36.529
Change From Baseline in Echocardiogram (ECHO) Parameters Over Time: Left Ventricular Mass Index (LVMI) Change at Month 60	—	8.83 g/m Standard Deviation 25.639	—

SECONDARY outcome

Timeframe: Baseline, Month 12, Month 24, Month 36, Month 48, Month 60

Population: 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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) Change at Month 12	-1.50 g Standard Deviation 29.374	12.58 g Standard Deviation 22.648	-0.75 g Standard Deviation 25.935
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) Change at Month 24	9.71 g Standard Deviation 27.034	5.61 g Standard Deviation 34.465	3.25 g Standard Deviation 38.833
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) Change at Month 36	6.21 g Standard Deviation 30.355	16.36 g Standard Deviation 36.898	15.75 g Standard Deviation 48.922
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) Change at Month 48	33.25 g Standard Deviation 12.842	19.72 g Standard Deviation 42.645	10.25 g Standard Deviation 52.753
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Mass (LVM) Change at Month 60	—	46.29 g Standard Deviation 19.102	—

SECONDARY outcome

Timeframe: Baseline, Month 12, Month 24, Month 36, Month 48, Month 60

Population: 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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) Change at Month 12	2.19 mm Standard Deviation 9.138	2.38 mm Standard Deviation 8.719	1.57 mm Standard Deviation 4.308
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) Change at Month 24	3.26 mm Standard Deviation 11.158	3.00 mm Standard Deviation 8.535	0.67 mm Standard Deviation 4.755
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) Change at Month 36	-0.11 mm Standard Deviation 13.174	3.23 mm Standard Deviation 8.883	4.78 mm Standard Deviation 5.094
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) Change at Month 48	10.88 mm Standard Deviation 17.264	3.82 mm Standard Deviation 9.115	2.60 mm Standard Deviation 5.177
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Diameter (LVD) Change at Month 60	—	40.29 mm Standard Deviation 5.407	50.00 mm Standard Deviation NA 1 participant at this time point

SECONDARY outcome

Timeframe: Baseline, Month 12, Month 18, Month 24, Month 30, Month 36, Month 48, Month 60

Population: 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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) Change at Month 12	0.76 percent of blood ejected during systole Standard Deviation 7.602	1.32 percent of blood ejected during systole Standard Deviation 7.254	0.71 percent of blood ejected during systole Standard Deviation 6.474
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) Change at Month 18	—	—	0.00 percent of blood ejected during systole Standard Deviation NA 1 participant at this time point
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) Change at Month 24	-1.86 percent of blood ejected during systole Standard Deviation 9.062	0.24 percent of blood ejected during systole Standard Deviation 8.875	2.31 percent of blood ejected during systole Standard Deviation 8.400
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) Change at Month 30	0.00 percent of blood ejected during systole Standard Deviation NA 1 participant at this time point	—	—
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) Change at Month 36	-1.10 percent of blood ejected during systole Standard Deviation 7.259	-1.92 percent of blood ejected during systole Standard Deviation 5.986	3.44 percent of blood ejected during systole Standard Deviation 7.601
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) Change at Month 48	-1.33 percent of blood ejected during systole Standard Deviation 3.445	-1.57 percent of blood ejected during systole Standard Deviation 7.464	1.60 percent of blood ejected during systole Standard Deviation 5.459
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Ejection Fraction (LVEF) Change at Month 60	—	-5.00 percent of blood ejected during systole Standard Deviation 7.394	1.00 percent of blood ejected during systole Standard Deviation NA 1 participant at this time point

SECONDARY outcome

Timeframe: Baseline, Month 12, Month 24, Month 36

Population: 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.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=5 Participants 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.	Triheptanoin-Naïve Cohort n=1 Participants 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.	Triheptanoin-Naïve Cohort n=8 Participants 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.
Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) Change at Month 12	0.34 Z-score Standard Deviation 1.229	1.84 Z-score Standard Deviation NA 1 participant at this time point	0.21 Z-score Standard Deviation 1.492
Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) Change at Month 24	-0.05 Z-score Standard Deviation 1.469	2.28 Z-score Standard Deviation NA 1 participant at this time point	0.72 Z-score Standard Deviation 1.103
Change From Baseline in ECHO Parameters Over Time: LVEF Z-Score (Pediatric Participants) Change at Month 36	—	-0.52 Z-score Standard Deviation NA 1 participant at this time point	-0.66 Z-score Standard Deviation NA 1 participant at this time point

SECONDARY outcome

Timeframe: Baseline, Month 12, Month 24, Month 30, Month 36, Month 48, Month 60

Population: 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.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) Change at Month 12	-1.30 % change in left ventricular diameter Standard Deviation 5.564	-0.91 % change in left ventricular diameter Standard Deviation 6.468	-1.48 % change in left ventricular diameter Standard Deviation 5.904
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) Change at Month 24	-1.90 % change in left ventricular diameter Standard Deviation 6.052	1.03 % change in left ventricular diameter Standard Deviation 6.242	-1.36 % change in left ventricular diameter Standard Deviation 7.967
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) Change at Month 30	-7.00 % change in left ventricular diameter Standard Deviation NA 1 participant at this time point	—	—
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) Change at Month 36	-0.10 % change in left ventricular diameter Standard Deviation 6.086	0.25 % change in left ventricular diameter Standard Deviation 4.502	1.11 % change in left ventricular diameter Standard Deviation 5.395
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) Change at Month 48	3.00 % change in left ventricular diameter Standard Deviation 4.320	0.52 % change in left ventricular diameter Standard Deviation 4.501	-0.60 % change in left ventricular diameter Standard Deviation 6.269
Change From Baseline in ECHO Parameters Over Time: Left Ventricular Shortening Fraction (LVSF) Change at Month 60	—	-0.57 % change in left ventricular diameter Standard Deviation 4.392	0.00 % change in left ventricular diameter Standard Deviation NA 1 participant at this time point

SECONDARY outcome

Timeframe: Baseline, Month 12, Month 24, Month 36, Month 48, Month 60

Population: 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.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) Change at Month 12	0.10 Z-score Standard Deviation 1.659	0.25 Z-score Standard Deviation 1.315	-0.38 Z-score Standard Deviation 1.597
Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) Change at Month 24	0.27 Z-score Standard Deviation 2.255	0.30 Z-score Standard Deviation 1.607	0.13 Z-score Standard Deviation 2.119
Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) Change at Month 36	0.75 Z-score Standard Deviation 1.729	0.13 Z-score Standard Deviation 1.115	0.58 Z-score Standard Deviation 1.943
Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) Change at Month 48	0.53 Z-score Standard Deviation 0.316	0.24 Z-score Standard Deviation 1.464	0.10 Z-score Standard Deviation 1.888
Change From Baseline in ECHO Parameters Over Time: LVSF Z-Score (Pediatric Participants) Change at Month 60	—	-0.45 Z-score Standard Deviation 0.834	—

SECONDARY outcome

Timeframe: Post-UX007 treatment through the end of the study (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Annualized Duration Rate of All MCEs	2.123 days/year Interval 0.0 to 9.766	2.487 days/year Interval 0.0 to 6.968	0.796 days/year Interval 0.0 to 5.484

SECONDARY outcome

Timeframe: Post-UX007 treatment through the end of the study (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Annualized Event Rate of Rhabdomyolysis MCEs	0.352 events/year Interval 0.0 to 1.472	0.574 events/year Interval 0.0 to 1.673	0.281 events/year Interval 0.0 to 1.425

SECONDARY outcome

Timeframe: Post-UX007 treatment through the end of the study (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Annualized Duration Rate of Rhabdomyolysis MCEs	2.123 days/year Interval 0.0 to 6.969	2.487 days/year Interval 0.0 to 6.948	0.448 days/year Interval 0.0 to 5.398

SECONDARY outcome

Timeframe: Post-UX007 treatment through the end of the study (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Annualized Event Rate of Cardiomyopathy MCEs	0.000 event/year Interval 0.0 to 0.12	0.000 event/year Interval 0.0 to 0.0	0.000 event/year Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Post-UX007 treatment through the end of the study (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

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

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Annualized Duration Rate of Cardiomyopathy MCEs	0.000 days/year Interval 0.0 to 0.174	0.000 days/year Interval 0.0 to 0.0	0.000 days/year Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Post-UX007 treatment through the end of the study (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Annualized Event Rate of Hypoglycemic MCEs	0.000 events/year Interval 0.0 to 0.0	0.000 events/year Interval 0.0 to 0.0	0.000 events/year Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Post-UX007 treatment through the end of the study (up to 2072 days)

Population: Full Analysis Set: all participants enrolled who had at least 1 post-baseline efficacy assessment.

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.

Outcome measures

Outcome measures
Measure	UX007-CL201-Rollover Cohort n=24 Participants 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.	Triheptanoin-Naïve Cohort n=37 Participants 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.	Triheptanoin-Naïve Cohort n=33 Participants 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.
Annualized Duration Rate of Hypoglycemic MCEs	0.000 days/year Interval 0.0 to 0.0	0.000 days/year Interval 0.0 to 0.0	0.000 days/year Interval 0.0 to 0.0

Adverse Events

UX007-CL201-Rollover Cohort

Serious events: 20 serious events

Other events: 24 other events

Deaths: 2 deaths

IST/Other Cohort

Serious events: 28 serious events

Other events: 34 other events

Deaths: 2 deaths

Triheptanoin-Naïve Cohort

Serious events: 22 serious events

Other events: 32 other events

Deaths: 1 deaths

All Participants

Serious events: 70 serious events

Other events: 90 other events

Deaths: 5 deaths

Serious adverse events

Other adverse events

Additional Information

Medical Information

Ultragenyx Pharmaceutical Inc

Phone: 1-888-756-8657

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER