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| Name | Class |
|---|---|
| Ultragenyx Pharmaceutical Inc | INDUSTRY |
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This study is being done to assess the safety and long-term efficacy of triheptanoin in pediatric patients with Glut1 DS over a 5-year treatment period. Glut 1 is a protein that helps transport glucose to the brain. Glucose is the brain's primary source of energy. Glut 1 DS prevents this protein from being effectively produced, causing deprivation of energy to the neurons of the of the brain.
Glut1 DS is a severely debilitating disease characterized by seizures, developmental delay and movement disorder. There are currently no approved treatments specific to Glut1 DS. Treatment generally includes medications for control of seizures. The use of a ketogenic diet can be effective in controlling seizures when medications are ineffective or provide insufficient control. However, the ketogenic diet may be very difficult for patients to maintain for long periods of time, and there may be negative secondary long-term effects of ketogenic diet.. Triheptanoin is metabolized to molecules that can provide an alternative energy source to the brain, and appears to help in controlling seizures without many of the difficulties of the ketogenic diet.
Eligible patients may be those who have been diagnosed with GLUT1 DS, and have discontinued or are not currently on ketogenic diet, or are able to tolerate triheptanoin if they have been treated or are currently being treated with triheptanoin and do not qualify for any other clinical trial.
Triheptanoin is proposed for the treatment of seizures in glucose transporter type-1 deficiency syndrome (Glut1 DS). Glut1 DS is a rare disease with an estimated US prevalence of ~3,300.
The proposed study is an open-label study to assess the safety and long-term efficacy of triheptanoin in patients with Glut1 DS over a 5-year treatment period. Eligible patients may be those who are able to tolerate triheptanoin if they have been treated or are currently being treated with triheptanoin and do not qualify for any other clinical trial. Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (~1-4g/kg/day, depending on age). Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories.
The primary objective of the study is to evaluate the safety of triheptanoin via adverse event rates and laboratory values. The secondary objective is to evaluate the long-term efficacy of triheptanoin as measured by the change in seizure frequency from historical baseline.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Schedule A | Experimental | Subjects previously treated with triheptanoin |
|
| Schedule B | Experimental | Naïve to triheptanoin |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Triheptanoin | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Reported Change in Seizures Frequency From Baseline at 13 Weeks | 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. | Baseline and 13 weeks |
| Reported Change in Seizures Frequency From Baseline at 26 Weeks | 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. | Baseline and 26 weeks |
| Reported Change in Seizures Frequency From Baseline at 1 Year | 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. | Baseline and one yr |
| Reported Change in Seizures Frequency From Baseline at 18 Months | 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. |
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Inclusion Criteria:
Individuals eligible to participate in this study must meet all of the following criteria:
Exclusion Criteria:
Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
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| Name | Affiliation | Role |
|---|---|---|
| Adrian Lacy, MD | Cook Children's Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cook Childrens Medical Center | Fort Worth | Texas | 76104 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 11800053 | Background | Johnson W Jr; Cosmetic Ingredient Review Expert Panel. Final report on the safety assessment of trilaurin, triarachidin, tribehenin, tricaprin, tricaprylin, trierucin, triheptanoin, triheptylundecanoin, triisononanoin, triisopalmitin, triisostearin, trilinolein, trimyristin, trioctanoin, triolein, tripalmitin, tripalmitolein, triricinolein, tristearin, triundecanoin, glyceryl triacetyl hydroxystearate, glyceryl triacetyl ricinoleate, and glyceryl stearate diacetate. Int J Toxicol. 2001;20 Suppl 4:61-94. | |
| 23443458 |
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Between February 2014 and February 2019, 20 subjects enrolled on this study. Enrollment occured in the outpatient specialty clinic offices.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Reported Change in Seizures Frequency From Baseline at 13 Weeks | 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 number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and 13 weeks |
|
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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Prolonged generalized tonic-clonic seizure | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Otalgia | Ear and labyrinth disorders | MedDRA (19.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director of Research Operations | Cook Children's Health Care System | 682-885-2103 | CookChildrensResearch@cookchildrens.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 4, 2019 | Jan 25, 2021 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| C536830 | Glut1 Deficiency Syndrome |
| D004827 | Epilepsy |
| D012640 | Seizures |
| ID | Term |
|---|---|
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009461 | Neurologic Manifestations |
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| ID | Term |
|---|---|
| C531010 | triheptanoin |
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|
| Baseline and 18 months |
| Reported Change in Seizures Frequency From Baseline at 2 Years | 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. | Baseline and two yrs |
| Reported Change in Seizures Frequency From Baseline at 3 Years | 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. | Baseline and three yrs |
| Reported Change in Seizure Frequency From Baseline at 4 Years | 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. | Baseline and four yrs |
| Reported Change in Seizure Frequency From Baseline at 5 Years | 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. | Baseline and five yrs |
| Background |
| Pearson TS, Akman C, Hinton VJ, Engelstad K, De Vivo DC. Phenotypic spectrum of glucose transporter type 1 deficiency syndrome (Glut1 DS). Curr Neurol Neurosci Rep. 2013 Apr;13(4):342. doi: 10.1007/s11910-013-0342-7. |
| 22812641 | Background | Pong AW, Geary BR, Engelstad KM, Natarajan A, Yang H, De Vivo DC. Glucose transporter type I deficiency syndrome: epilepsy phenotypes and outcomes. Epilepsia. 2012 Sep;53(9):1503-10. doi: 10.1111/j.1528-1167.2012.03592.x. Epub 2012 Jul 19. |
| 12069537 | Background | Roe CR. Inherited disorders of mitochondrial fatty acid oxidation: a new responsibility for the neonatologist. Semin Neonatol. 2002 Feb;7(1):37-47. doi: 10.1053/siny.2002.0097. |
| 16705058 | Background | Kinman RP, Kasumov T, Jobbins KA, Thomas KR, Adams JE, Brunengraber LN, Kutz G, Brewer WU, Roe CR, Brunengraber H. Parenteral and enteral metabolism of anaplerotic triheptanoin in normal rats. Am J Physiol Endocrinol Metab. 2006 Oct;291(4):E860-6. doi: 10.1152/ajpendo.00366.2005. Epub 2006 May 16. |
| 19666922 | Background | Deng S, Zhang GF, Kasumov T, Roe CR, Brunengraber H. Interrelations between C4 ketogenesis, C5 ketogenesis, and anaplerosis in the perfused rat liver. J Biol Chem. 2009 Oct 9;284(41):27799-27807. doi: 10.1074/jbc.M109.048744. Epub 2009 Aug 8. |
| 19903863 | Background | Gu L, Zhang GF, Kombu RS, Allen F, Kutz G, Brewer WU, Roe CR, Brunengraber H. Parenteral and enteral metabolism of anaplerotic triheptanoin in normal rats. II. Effects on lipolysis, glucose production, and liver acyl-CoA profile. Am J Physiol Endocrinol Metab. 2010 Feb;298(2):E362-71. doi: 10.1152/ajpendo.00384.2009. Epub 2009 Nov 10. |
| 23072752 | Background | Marin-Valencia I, Good LB, Ma Q, Malloy CR, Pascual JM. Heptanoate as a neural fuel: energetic and neurotransmitter precursors in normal and glucose transporter I-deficient (G1D) brain. J Cereb Blood Flow Metab. 2013 Feb;33(2):175-82. doi: 10.1038/jcbfm.2012.151. Epub 2012 Oct 17. |
| 20691264 | Background | Willis S, Stoll J, Sweetman L, Borges K. Anticonvulsant effects of a triheptanoin diet in two mouse chronic seizure models. Neurobiol Dis. 2010 Dec;40(3):565-72. doi: 10.1016/j.nbd.2010.07.017. Epub 2010 Aug 4. |
| 18565731 | Background | Samala R, Willis S, Borges K. Anticonvulsant profile of a balanced ketogenic diet in acute mouse seizure models. Epilepsy Res. 2008 Oct;81(2-3):119-27. doi: 10.1016/j.eplepsyres.2008.05.001. Epub 2008 Jun 18. |
| 23196212 | Background | Kim TH, Borges K, Petrou S, Reid CA. Triheptanoin reduces seizure susceptibility in a syndrome-specific mouse model of generalized epilepsy. Epilepsy Res. 2013 Jan;103(1):101-5. doi: 10.1016/j.eplepsyres.2012.09.016. Epub 2012 Nov 26. |
| Background | AtaÃde 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 |
| 12122118 | Background | Roe CR, Sweetman L, Roe DS, David F, Brunengraber H. Treatment of cardiomyopathy and rhabdomyolysis in long-chain fat oxidation disorders using an anaplerotic odd-chain triglyceride. J Clin Invest. 2002 Jul;110(2):259-69. doi: 10.1172/JCI15311. |
| 16763896 | Background | Roe CR, Mochel F. Anaplerotic diet therapy in inherited metabolic disease: therapeutic potential. J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):332-40. doi: 10.1007/s10545-006-0290-3. |
| 20512158 | Background | Mochel F, Duteil S, Marelli C, Jauffret C, Barles A, Holm J, Sweetman L, Benoist JF, Rabier D, Carlier PG, Durr A. Dietary anaplerotic therapy improves peripheral tissue energy metabolism in patients with Huntington's disease. Eur J Hum Genet. 2010 Sep;18(9):1057-60. doi: 10.1038/ejhg.2010.72. Epub 2010 May 26. |
| 23053472 | Background | Baruteau J, Sachs P, Broue P, Brivet M, Abdoul H, Vianey-Saban C, Ogier de Baulny H. Clinical and biological features at diagnosis in mitochondrial fatty acid beta-oxidation defects: a French pediatric study of 187 patients. J Inherit Metab Dis. 2013 Sep;36(5):795-803. doi: 10.1007/s10545-012-9542-6. Epub 2012 Oct 3. |
| Background | Goldstein A, Barone AR, DeWard SJ, Payne N, Vockley J. Triheptanoin therapy for inherited disorders of fatty acid oxidation. Mitochondrion. 2012;12(5):566 |
| Background | Sparrow, SS, Cicchetti D, & Balla DA. Vineland Adaptive Behavior Scales - 2nd Edition manual. Minneapolis, MN: NCS Pearson, Inc; 2005 |
| 10400175 | Background | Barry MJ, VanSwearingen JM, Albright AL. Reliability and responsiveness of the Barry-Albright Dystonia Scale. Dev Med Child Neurol. 1999 Jun;41(6):404-11. doi: 10.1017/s0012162299000870. |
| 19807613 | Background | Varni JW, Burwinkle TM, Seid M. The PedsQL as a pediatric patient-reported outcome: reliability and validity of the PedsQL Measurement Model in 25,000 children. Expert Rev Pharmacoecon Outcomes Res. 2005 Dec;5(6):705-19. doi: 10.1586/14737167.5.6.705. |
| Physician Decision |
|
| Withdrawal by Subject |
|
| BG001 | 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. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Seizure Count per Reporting Period | 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. | Mean | Full Range | seizures |
|
| Barry Albright Dystonia Scale | 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. | Mean | Full Range | units on a scale |
|
| PedsQL Physical Health Summary Score | 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 | Mean | Full Range | units on a scale |
|
| PedsQL Psychosocial Health Summary Score | 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 | Mean | Full Range | units on a scale |
|
| OG001 | 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. |
|
|
| Primary | Reported Change in Seizures Frequency From Baseline at 26 Weeks | 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. | The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and 26 weeks |
|
|
|
| Primary | Reported Change in Seizures Frequency From Baseline at 1 Year | 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. | The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and one yr |
|
|
|
| Primary | Reported Change in Seizures Frequency From Baseline at 18 Months | 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. | The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and 18 months |
|
|
|
| Primary | Reported Change in Seizures Frequency From Baseline at 2 Years | 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. | The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and two yrs |
|
|
|
| Primary | Reported Change in Seizures Frequency From Baseline at 3 Years | 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. | The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and three yrs |
|
|
|
| Primary | Reported Change in Seizure Frequency From Baseline at 4 Years | 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. | The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and four yrs |
|
|
|
| Primary | Reported Change in Seizure Frequency From Baseline at 5 Years | 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. | The number analyzed over time reflects both subject drop out and subjects that failed to record seizure counts during the respective reporting period. | Posted | Mean | Full Range | seizures/two weeks | Baseline and five yrs |
|
|
|
| 0 |
| 6 |
| 0 |
| 6 |
| 6 |
| 6 |
| EG001 | 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. | 0 | 14 | 1 | 14 | 14 | 14 |
| Status Epilepticus | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Hirsutism | Endocrine disorders | MedDRA (19.0) | Systematic Assessment |
|
| Conjuctivitis | Eye disorders | MedDRA (19.0) | Systematic Assessment |
|
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Abdominal Distention | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Emesis | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Gastric Reflux | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Gastroenteritis | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Rectal Leakage | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Teething | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Tongue Pain | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (19.0) | Systematic Assessment |
|
| Fever | General disorders | MedDRA (19.0) | Systematic Assessment |
|
| Lethargy | General disorders | MedDRA (19.0) | Systematic Assessment |
|
| Abcess | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Acute Pharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Acute Tonsillitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Acute Viral Syndrome | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Croup | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Impetigo | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Onychomycosis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Otitis Media | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Rhinovirus | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Stomach Virus | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Strep Throat | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Virus | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
|
| Fall-Witnessed | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
|
| Fracture Of Left Wrist | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
|
| Head Injury | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
|
| Nasal Fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
|
| Right Arm Fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
|
| Right Hand Index And Middle Finger Buckle Fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
|
| Right Wrist Fracture | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
|
| Decreased Hematocrit | Investigations | MedDRA (19.0) | Systematic Assessment |
|
| Elevated ALT | Investigations | MedDRA (19.0) | Systematic Assessment |
|
| Increased Ast | Investigations | MedDRA (19.0) | Systematic Assessment |
|
| Weight Gain | Investigations | MedDRA (19.0) | Systematic Assessment |
|
| Weight Loss | Investigations | MedDRA (19.0) | Systematic Assessment |
|
| Decrease Carbondioxide | Investigations | MedDRA (19.0) | Systematic Assessment |
|
| Decreased Serum Carbon Dioxide | Investigations | MedDRA (19.0) | Systematic Assessment |
|
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA (19.0) | Systematic Assessment |
|
| Electrolyte Imbalance | Metabolism and nutrition disorders | MedDRA (19.0) | Systematic Assessment |
|
| Hand Pain | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
|
| Muscle Cramps | Musculoskeletal and connective tissue disorders | MedDRA (19.0) | Systematic Assessment |
|
| Balance Issues | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Blurred Vision | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Convulsive Seizures | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Impulsive Behavior | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Increased Seizures | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Lost Ability To Independently Walk | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Poor Balance | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Seizure | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Somnolence | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Worsening Paroxysmal Exercise Dyskinesia | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
|
| Aggression | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Agitation | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Excessive Daytime Sleepiness | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Hyperactivity | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Hyperactivity (Adhd) | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Inattention | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Moodiness | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Sleep Difficulties | Psychiatric disorders | MedDRA (19.0) | Systematic Assessment |
|
| Incontinence | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
|
| Urinary Incontinence | Renal and urinary disorders | MedDRA (19.0) | Systematic Assessment |
|
| Amenorrhea | Reproductive system and breast disorders | MedDRA (19.0) | Systematic Assessment |
|
| Acute Bronchitis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Seasonal Allergies | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Upper Respiratory Infection | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Viral Upper Respiratory Infection | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
|
| Cellulitis | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
|
| Chin Laceration | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
|
| Rash-Right Leg | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
|
| Scalp Laceration | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
|
| Skin Scale Hands | Skin and subcutaneous tissue disorders | MedDRA (19.0) | Systematic Assessment |
|
| Contusion of Left Foot | Vascular disorders | MedDRA (19.0) | Systematic Assessment |
|
Not provided
Not provided
Not provided
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|