Trial Outcomes & Findings for An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome (NCT NCT02036853)

NCT ID: NCT02036853

Last Updated: 2021-01-28

Results Overview

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 20 participants
• Primary outcome timeframe: Baseline and 13 weeks
• Results posted on: 2021-01-28

Participant Flow

Between February 2014 and February 2019, 20 subjects enrolled on this study. Enrollment occured in the outpatient specialty clinic offices.

Participant milestones

Measure	Schedule A Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Overall Study STARTED	6	14
Overall Study COMPLETED	6	8
Overall Study NOT COMPLETED	0	6

Reasons for withdrawal

Measure	Schedule A Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Overall Study Adverse Event	0	2
Overall Study Lack of Efficacy	0	2
Overall Study Physician Decision	0	1
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

The screening visit was waived for some subjects for whom a treatment gap would have been clinically unsafe. Thus baseline seizure data were not collected prospectively for those individuals.

Baseline characteristics by cohort

Measure	Schedule A n=6 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=14 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Total n=20 Participants Total of all reporting groups
Age, Continuous	14.82 years STANDARD_DEVIATION 9.77 • n=6 Participants	5.49 years STANDARD_DEVIATION 3.09 • n=14 Participants	8.29 years STANDARD_DEVIATION 7.13 • n=20 Participants
Sex: Female, Male Female	2 Participants n=6 Participants	6 Participants n=14 Participants	8 Participants n=20 Participants
Sex: Female, Male Male	4 Participants n=6 Participants	8 Participants n=14 Participants	12 Participants n=20 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=6 Participants	4 Participants n=14 Participants	5 Participants n=20 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=6 Participants	10 Participants n=14 Participants	15 Participants n=20 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=6 Participants	0 Participants n=14 Participants	0 Participants n=20 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=6 Participants	0 Participants n=14 Participants	0 Participants n=20 Participants
Race (NIH/OMB) Asian	0 Participants n=6 Participants	0 Participants n=14 Participants	0 Participants n=20 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=6 Participants	0 Participants n=14 Participants	0 Participants n=20 Participants
Race (NIH/OMB) Black or African American	0 Participants n=6 Participants	0 Participants n=14 Participants	0 Participants n=20 Participants
Race (NIH/OMB) White	6 Participants n=6 Participants	14 Participants n=14 Participants	20 Participants n=20 Participants
Race (NIH/OMB) More than one race	0 Participants n=6 Participants	0 Participants n=14 Participants	0 Participants n=20 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=6 Participants	0 Participants n=14 Participants	0 Participants n=20 Participants
Seizure Count per Reporting Period	23.8 seizures n=5 Participants • The screening visit was waived for some subjects for whom a treatment gap would have been clinically unsafe. Thus baseline seizure data were not collected prospectively for those individuals.	170.7 seizures n=12 Participants • The screening visit was waived for some subjects for whom a treatment gap would have been clinically unsafe. Thus baseline seizure data were not collected prospectively for those individuals.	127.5 seizures n=17 Participants • The screening visit was waived for some subjects for whom a treatment gap would have been clinically unsafe. Thus baseline seizure data were not collected prospectively for those individuals.
Barry Albright Dystonia Scale	8.5 units on a scale n=6 Participants	7.8 units on a scale n=14 Participants	8 units on a scale n=20 Participants
PedsQL Physical Health Summary Score	48.1 units on a scale n=6 Participants	70.8 units on a scale n=14 Participants	55.26 units on a scale n=20 Participants
PedsQL Psychosocial Health Summary Score	64.7 units on a scale n=6 Participants	78.6 units on a scale n=14 Participants	69.1 units on a scale n=20 Participants

PRIMARY outcome

Timeframe: Baseline and 13 weeks

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit.

Outcome measures

Measure	Schedule A n=5 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=12 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizures Frequency From Baseline at 13 Weeks	4.4 seizures/two weeks Interval -10.0 to 22.0	189.5 seizures/two weeks Interval -228.0 to 2420.0

PRIMARY outcome

Timeframe: Baseline and 26 weeks

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.

Outcome measures

Measure	Schedule A n=5 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=6 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizures Frequency From Baseline at 26 Weeks	-5.6 seizures/two weeks Interval -25.0 to 8.0	-78 seizures/two weeks Interval -265.0 to 0.0

PRIMARY outcome

Timeframe: Baseline and one yr

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.

Outcome measures

Measure	Schedule A n=4 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=6 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizures Frequency From Baseline at 1 Year	-6.5 seizures/two weeks Interval -15.0 to 0.0	-110.5 seizures/two weeks Interval -331.0 to 0.0

PRIMARY outcome

Timeframe: Baseline and 18 months

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.

Outcome measures

Measure	Schedule A n=5 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=6 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizures Frequency From Baseline at 18 Months	-5.8 seizures/two weeks Interval -20.0 to 2.0	-112.7 seizures/two weeks Interval -340.0 to 3.0

PRIMARY outcome

Timeframe: Baseline and two yrs

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.

Outcome measures

Measure	Schedule A n=4 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=4 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizures Frequency From Baseline at 2 Years	-6 seizures/two weeks Interval -39.0 to 5.0	-61.25 seizures/two weeks Interval -245.0 to 0.0

PRIMARY outcome

Timeframe: Baseline and three yrs

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.

Outcome measures

Measure	Schedule A n=4 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=3 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizures Frequency From Baseline at 3 Years	-0.8 seizures/two weeks Interval -12.0 to 19.0	-77 seizures/two weeks Interval -231.0 to 0.0

PRIMARY outcome

Timeframe: Baseline and four yrs

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.

Outcome measures

Measure	Schedule A n=3 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=3 Participants Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizure Frequency From Baseline at 4 Years	-8.3 seizures/two weeks Interval -26.0 to 1.0	-80.3 seizures/two weeks Interval -241.0 to 0.0

PRIMARY outcome

Timeframe: Baseline and five yrs

Population: The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.

A seizure diary was used to track date, type, number, and unusual presentation of seizures. Subjects were given a seizure diary at screening to record daily seizure activity for incremental periods of time. Unless otherwise waived, subjects complete this form daily during the screening period and for two weeks prior to each subsequent study visit. The table below represents the change in seizure frequency from baseline for each time point.

Outcome measures

Measure	Schedule A n=1 Participants Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Reported Change in Seizure Frequency From Baseline at 5 Years	23 seizures/two weeks Interval 23.0 to 23.0	—

Adverse Events

Schedule A

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Schedule B

Serious events: 1 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Measure	Schedule A n=6 participants at risk Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=14 participants at risk Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Nervous system disorders Prolonged generalized tonic-clonic seizure	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Status Epilepticus	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.

Other adverse events

Measure	Schedule A n=6 participants at risk Subjects previously treated with triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.	Schedule B n=14 participants at risk Naïve to triheptanoin Triheptanoin: Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age). Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established.
Ear and labyrinth disorders Otalgia	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Endocrine disorders Hirsutism	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Eye disorders Conjuctivitis	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Abdominal Discomfort	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	57.1% 8/14 • Number of events 8 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Abdominal Distention	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Constipation	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 3 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Diarrhea	83.3% 5/6 • Number of events 7 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	85.7% 12/14 • Number of events 14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Emesis	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	78.6% 11/14 • Number of events 19 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Flatulence	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Gastric Reflux	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Gastroenteritis	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Nausea	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	21.4% 3/14 • Number of events 3 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Rectal Leakage	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Teething	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Gastrointestinal disorders Tongue Pain	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
General disorders Fatigue	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	21.4% 3/14 • Number of events 3 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
General disorders Fever	33.3% 2/6 • Number of events 3 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	35.7% 5/14 • Number of events 6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
General disorders Lethargy	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Abcess	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Acute Pharyngitis	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 3 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Acute Tonsillitis	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Acute Viral Syndrome	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Croup	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Impetigo	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Influenza	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	21.4% 3/14 • Number of events 5 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Onychomycosis	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Otitis Media	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	35.7% 5/14 • Number of events 5 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Rhinovirus	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Sinusitis	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Stomach Virus	33.3% 2/6 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Strep Throat	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	21.4% 3/14 • Number of events 3 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Infections and infestations Virus	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Injury, poisoning and procedural complications Fall-Witnessed	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Injury, poisoning and procedural complications Fracture Of Left Wrist	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Injury, poisoning and procedural complications Head Injury	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Injury, poisoning and procedural complications Nasal Fracture	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Injury, poisoning and procedural complications Right Arm Fracture	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Injury, poisoning and procedural complications Right Hand Index And Middle Finger Buckle Fracture	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Injury, poisoning and procedural complications Right Wrist Fracture	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Investigations Decreased Hematocrit	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Investigations Elevated ALT	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Investigations Increased Ast	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Investigations Weight Gain	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	35.7% 5/14 • Number of events 5 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Investigations Weight Loss	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Investigations Decrease Carbondioxide	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Investigations Decreased Serum Carbon Dioxide	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Metabolism and nutrition disorders Decreased Appetite	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	64.3% 9/14 • Number of events 11 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Metabolism and nutrition disorders Electrolyte Imbalance	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Musculoskeletal and connective tissue disorders Hand Pain	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Musculoskeletal and connective tissue disorders Muscle Cramps	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Balance Issues	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 3 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Blurred Vision	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Convulsive Seizures	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Headache	33.3% 2/6 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	28.6% 4/14 • Number of events 4 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Impulsive Behavior	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Increased Seizures	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Lost Ability To Independently Walk	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Poor Balance	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Seizure	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Somnolence	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Nervous system disorders Worsening Paroxysmal Exercise Dyskinesia	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Aggression	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Agitation	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Excessive Daytime Sleepiness	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Hyperactivity	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Hyperactivity (Adhd)	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Inattention	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Insomnia	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Moodiness	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Psychiatric disorders Sleep Difficulties	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Renal and urinary disorders Incontinence	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Renal and urinary disorders Urinary Incontinence	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Reproductive system and breast disorders Amenorrhea	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Respiratory, thoracic and mediastinal disorders Acute Bronchitis	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Respiratory, thoracic and mediastinal disorders Rhinorrhea	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Respiratory, thoracic and mediastinal disorders Seasonal Allergies	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	14.3% 2/14 • Number of events 2 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Respiratory, thoracic and mediastinal disorders Viral Upper Respiratory Infection	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Skin and subcutaneous tissue disorders Cellulitis	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Skin and subcutaneous tissue disorders Chin Laceration	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Skin and subcutaneous tissue disorders Rash-Right Leg	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Skin and subcutaneous tissue disorders Scalp Laceration	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Skin and subcutaneous tissue disorders Skin Scale Hands	16.7% 1/6 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	0.00% 0/14 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.
Vascular disorders Contusion of Left Foot	0.00% 0/6 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.	7.1% 1/14 • Number of events 1 • Adverse Event data were collected for the duration of study participation for each subject from baseline through study completion or discontinuation. The longest subject participation was 5 years, 3 months.

Additional Information

Director of Research Operations

Cook Children's Health Care System

Phone: 682-885-2103

Email: CookChildrensResearch@cookchildrens.org

Results disclosure agreements

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