Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome (GLUT1DS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02036853
Recruitment Status : Completed
First Posted : January 15, 2014
Results First Posted : January 28, 2021
Last Update Posted : January 28, 2021
Sponsor:
Collaborator:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Adrian Lacy, Cook Children's Health Care System

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Intervention Drug: Triheptanoin
Enrollment 20
Recruitment Details Between February 2014 and February 2019, 20 subjects enrolled on this study. Enrollment occured in the outpatient specialty clinic offices.
Pre-assignment Details  
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description

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.

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.

Period Title: Overall Study
Started 6 14
Completed 6 8
Not Completed 0 6
Reason Not Completed
Adverse Event             0             2
Lack of Efficacy             0             2
Physician Decision             0             1
Withdrawal by Subject             0             1
Arm/Group Title Schedule A Schedule B Total
Hide Arm/Group Description

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.

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 of all reporting groups
Overall Number of Baseline Participants 6 14 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 14 participants 20 participants
14.82  (9.77) 5.49  (3.09) 8.29  (7.13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 14 participants 20 participants
Female
2
  33.3%
6
  42.9%
8
  40.0%
Male
4
  66.7%
8
  57.1%
12
  60.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 14 participants 20 participants
Hispanic or Latino
1
  16.7%
4
  28.6%
5
  25.0%
Not Hispanic or Latino
5
  83.3%
10
  71.4%
15
  75.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 14 participants 20 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
6
 100.0%
14
 100.0%
20
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Seizure Count per Reporting Period   [1] 
Mean (Full Range)
Unit of measure:  Seizures
Number Analyzed 5 participants 12 participants 17 participants
23.8
(0 to 106)
170.7
(0 to 974)
127.5
(0 to 974)
[1]
Measure Analysis Population Description: 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   [1] 
Mean (Full Range)
Unit of measure:  Units on a scale
Number Analyzed 6 participants 14 participants 20 participants
8.5
(0 to 20)
7.8
(0 to 22)
8
(0 to 22)
[1]
Measure Description: The Barry-Albright Dystonia Scale is a 5-point ordinal scale that was designed to assess dystonia in 8 body regions: eyes, mouth, neck, trunk, and the four extremities. The score in each region is rated 0 (no dystonia) to 4 (severe dystonia.) The minimum score is 0 and the maximum scale is 32 and is the sum of all of the regions measured.
PedsQL Physical Health Summary Score   [1] 
Mean (Full Range)
Unit of measure:  Units on a scale
Number Analyzed 6 participants 14 participants 20 participants
48.1
(5 to 100)
70.8
(45 to 100)
55.26
(5 to 100)
[1]
Measure Description: The PedsQL (Pediatric Quality of Life) was designed to measure perceived health-related quality of life in pediatric patients with chronic health conditions. The Generic Core scale includes assessment of physical, emotional, social, and school functioning. The summary scores are: Total Quality of Life Score, Physical Health Summary Score (avg of physical functioning), and Psychosocial Health Summary Score (avg of emotional, social, and school items). Items (5-point scale) are reversed scored and transformed to a 0-100 scale so higher scores are better. https://www.pedsql.org/about_pedsql.html
PedsQL Psychosocial Health Summary Score   [1] 
Mean (Full Range)
Unit of measure:  Units on a scale
Number Analyzed 6 participants 14 participants 20 participants
64.7
(42.5 to 90)
78.6
(65 to 95)
69.1
(42.5 to 95)
[1]
Measure Description: The PedsQL (Pediatric Quality of Life) was designed to measure perceived health-related quality of life in pediatric patients with chronic health conditions. The Generic Core scale includes assessment of physical, emotional, social, and school functioning. The summary scores are: Total Quality of Life Score, Physical Health Summary Score (avg of physical functioning), and Psychosocial Health Summary Score (avg of emotional, social, and school items). Items (5-point scale) are reversed scored and transformed to a 0-100 scale so higher scores are better. https://www.pedsql.org/about_pedsql.html
1.Primary Outcome
Title Reported Change in Seizures Frequency From Baseline at 13 Weeks
Hide Description 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.
Time Frame Baseline and 13 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 5 12
Mean (Full Range)
Unit of Measure: seizures/two weeks
4.4
(-10 to 22)
189.5
(-228 to 2420)
2.Primary Outcome
Title Reported Change in Seizures Frequency From Baseline at 26 Weeks
Hide Description 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.
Time Frame Baseline and 26 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 5 6
Mean (Full Range)
Unit of Measure: seizures/two weeks
-5.6
(-25 to 8)
-78
(-265 to 0)
3.Primary Outcome
Title Reported Change in Seizures Frequency From Baseline at 1 Year
Hide Description 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.
Time Frame Baseline and one yr
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 4 6
Mean (Full Range)
Unit of Measure: seizures/two weeks
-6.5
(-15 to 0)
-110.5
(-331 to 0)
4.Primary Outcome
Title Reported Change in Seizures Frequency From Baseline at 18 Months
Hide Description 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.
Time Frame Baseline and 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 5 6
Mean (Full Range)
Unit of Measure: seizures/two weeks
-5.8
(-20 to 2)
-112.7
(-340 to 3)
5.Primary Outcome
Title Reported Change in Seizures Frequency From Baseline at 2 Years
Hide Description 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.
Time Frame Baseline and two yrs
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 4 4
Mean (Full Range)
Unit of Measure: seizures/two weeks
-6
(-39 to 5)
-61.25
(-245 to 0)
6.Primary Outcome
Title Reported Change in Seizures Frequency From Baseline at 3 Years
Hide Description 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.
Time Frame Baseline and three yrs
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 4 3
Mean (Full Range)
Unit of Measure: seizures/two weeks
-0.8
(-12 to 19)
-77
(-231 to 0)
7.Primary Outcome
Title Reported Change in Seizure Frequency From Baseline at 4 Years
Hide Description 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.
Time Frame Baseline and four yrs
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 3 3
Mean (Full Range)
Unit of Measure: seizures/two weeks
-8.3
(-26 to 1)
-80.3
(-241 to 0)
8.Primary Outcome
Title Reported Change in Seizure Frequency From Baseline at 5 Years
Hide Description 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.
Time Frame Baseline and five yrs
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period.
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description:

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.

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 Number of Participants Analyzed 1 0
Mean (Full Range)
Unit of Measure: seizures/two weeks
23
(23 to 23)
Time Frame 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.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Schedule A Schedule B
Hide Arm/Group Description

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.

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.

All-Cause Mortality
Schedule A Schedule B
Affected / at Risk (%) Affected / at Risk (%)
Total   0/6 (0.00%)      0/14 (0.00%)    
Hide Serious Adverse Events
Schedule A Schedule B
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/6 (0.00%)      1/14 (7.14%)    
Nervous system disorders     
Prolonged generalized tonic-clonic seizure  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Status Epilepticus  1  0/6 (0.00%)  0 1/14 (7.14%)  2
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Schedule A Schedule B
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/6 (100.00%)      14/14 (100.00%)    
Ear and labyrinth disorders     
Otalgia  1  1/6 (16.67%)  1 1/14 (7.14%)  1
Endocrine disorders     
Hirsutism  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Eye disorders     
Conjuctivitis  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Gastrointestinal disorders     
Abdominal Discomfort  1  1/6 (16.67%)  1 8/14 (57.14%)  8
Abdominal Distention  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Constipation  1  1/6 (16.67%)  1 2/14 (14.29%)  3
Diarrhea  1  5/6 (83.33%)  7 12/14 (85.71%)  14
Emesis  1  1/6 (16.67%)  1 11/14 (78.57%)  19
Flatulence  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Gastric Reflux  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Gastroenteritis  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Nausea  1  1/6 (16.67%)  1 3/14 (21.43%)  3
Rectal Leakage  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Teething  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Tongue Pain  1  1/6 (16.67%)  1 0/14 (0.00%)  0
General disorders     
Fatigue  1  0/6 (0.00%)  0 3/14 (21.43%)  3
Fever  1  2/6 (33.33%)  3 5/14 (35.71%)  6
Lethargy  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Infections and infestations     
Abcess  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Acute Pharyngitis  1  0/6 (0.00%)  0 2/14 (14.29%)  3
Acute Tonsillitis  1  0/6 (0.00%)  0 1/14 (7.14%)  2
Acute Viral Syndrome  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Croup  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Impetigo  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Influenza  1  0/6 (0.00%)  0 3/14 (21.43%)  5
Onychomycosis  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Otitis Media  1  0/6 (0.00%)  0 5/14 (35.71%)  5
Rhinovirus  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Sinusitis  1  0/6 (0.00%)  0 2/14 (14.29%)  2
Stomach Virus  1  2/6 (33.33%)  2 1/14 (7.14%)  1
Strep Throat  1  0/6 (0.00%)  0 3/14 (21.43%)  3
Virus  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Injury, poisoning and procedural complications     
Fall-Witnessed  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Fracture Of Left Wrist  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Head Injury  1  0/6 (0.00%)  0 2/14 (14.29%)  2
Nasal Fracture  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Right Arm Fracture  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Right Hand Index And Middle Finger Buckle Fracture  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Right Wrist Fracture  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Investigations     
Decreased Hematocrit  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Elevated ALT  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Increased Ast  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Weight Gain  1  1/6 (16.67%)  1 5/14 (35.71%)  5
Weight Loss  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Decrease Carbondioxide  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Decreased Serum Carbon Dioxide  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Metabolism and nutrition disorders     
Decreased Appetite  1  1/6 (16.67%)  1 9/14 (64.29%)  11
Electrolyte Imbalance  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Musculoskeletal and connective tissue disorders     
Hand Pain  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Muscle Cramps  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Nervous system disorders     
Balance Issues  1  0/6 (0.00%)  0 1/14 (7.14%)  3
Blurred Vision  1  0/6 (0.00%)  0 1/14 (7.14%)  2
Convulsive Seizures  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Headache  1  2/6 (33.33%)  2 4/14 (28.57%)  4
Impulsive Behavior  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Increased Seizures  1  0/6 (0.00%)  0 2/14 (14.29%)  2
Lost Ability To Independently Walk  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Poor Balance  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Seizure  1  1/6 (16.67%)  1 1/14 (7.14%)  1
Somnolence  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Worsening Paroxysmal Exercise Dyskinesia  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Psychiatric disorders     
Aggression  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Agitation  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Excessive Daytime Sleepiness  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Hyperactivity  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Hyperactivity (Adhd)  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Inattention  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Insomnia  1  1/6 (16.67%)  1 2/14 (14.29%)  2
Moodiness  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Sleep Difficulties  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Renal and urinary disorders     
Incontinence  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Urinary Incontinence  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Reproductive system and breast disorders     
Amenorrhea  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Acute Bronchitis  1  0/6 (0.00%)  0 1/14 (7.14%)  2
Rhinorrhea  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Seasonal Allergies  1  0/6 (0.00%)  0 2/14 (14.29%)  2
Upper Respiratory Infection  1  1/6 (16.67%)  1 2/14 (14.29%)  2
Viral Upper Respiratory Infection  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Skin and subcutaneous tissue disorders     
Cellulitis  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Chin Laceration  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Rash-Right Leg  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Scalp Laceration  1  0/6 (0.00%)  0 1/14 (7.14%)  1
Skin Scale Hands  1  1/6 (16.67%)  1 0/14 (0.00%)  0
Vascular disorders     
Contusion of Left Foot  1  0/6 (0.00%)  0 1/14 (7.14%)  1
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Director of Research Operations
Organization: Cook Children's Health Care System
Phone: 682-885-2103
EMail: CookChildrensResearch@cookchildrens.org
Publications:
Ataíde TdaR, de Olivera SK, da Silva FM, Vitorino Filha LGC, do N Tavares MC, Sant'Ana AEG. Toxicological analysis of the chronic consumption of diheptanoin and triheptanoin in rats. Intl J Food Sci Tech. 2009;44:484-492
Goldstein A, Barone AR, DeWard SJ, Payne N, Vockley J. Triheptanoin therapy for inherited disorders of fatty acid oxidation. Mitochondrion. 2012;12(5):566
Sparrow, SS, Cicchetti D, & Balla DA. Vineland Adaptive Behavior Scales - 2nd Edition manual. Minneapolis, MN: NCS Pearson, Inc; 2005
Layout table for additonal information
Responsible Party: Adrian Lacy, Cook Children's Health Care System
ClinicalTrials.gov Identifier: NCT02036853    
Other Study ID Numbers: 2013-NEUR-001
First Submitted: January 13, 2014
First Posted: January 15, 2014
Results First Submitted: January 2, 2020
Results First Posted: January 28, 2021
Last Update Posted: January 28, 2021