Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2015-000389-69 | EudraCT Number |
Not provided
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Study was halted prematurely due to lack of efficacy
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The primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.
The study will enroll up to 40 pediatric, adolescent and adult Glut 1 DS participants who have completed the UX007G-CL201 (NCT019933186) study and, at the discretion of the Sponsor, additional participants from other clinical studies, investigator sponsored trials (ISTs), or expanded access/compassionate use treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| UX007 | Experimental | UX007 dosing targeted and/or maintained at 35% of total daily caloric intake. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| UX007 | Drug | UX007 is a liquid intended for oral (PO) administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE. | From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Colorado - University of Colorado, Denver, School of Medicine | Aurora | Colorado | 80045 | United States | ||
Not provided
| Label | URL |
|---|---|
| Company Website | View source |
Not provided
For continuing UX007G-CL201 participants, the Week 52 visit of that study may have been conducted in conjunction with the Baseline visit for this study to avoid duplication of assessments.
Study enrolled pediatric, adolescent, and adult glucose transporter type 1 deficiency syndrome (Glut1 DS) participants who completed the UX007G-CL201 study (NCT01993186; rollover participants). No non-rollover participants (those from other clinical studies, investigator sponsored trials, or expanded access/compassionate use treatment) enrolled.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | UX007 (Triheptanoin) | UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 20, 2017 | Apr 1, 2020 |
Not provided
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| Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36 |
| Change From Baseline Over Time in CNS Total Score | The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance & Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone & Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function. | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36 |
| Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 |
| Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 |
| Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life. | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 |
| Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life. | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 |
| Miami Children's Hospital |
| Miami |
| Florida |
| 33155 |
| United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Melbourne Brain Centre | Heidelberg | Victoria | 3084 | Australia |
| Copenhagen University Hospital | Copenhagen | 2100 | Denmark |
| Hospital Sant Joan De Deu | Barcelona | 08950 | Spain |
| The Newcastle upon Tyne Hospitals NHS Foundation Trust | Newcastle upon Tyne | NE7 7DN | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | UX007 (Triheptanoin) | UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Overall Seizure Frequency Per 4 Weeks | The number of observable seizures were recorded by the subject or caregiver via diary. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence. | Mean | Standard Deviation | seizures per 4 weeks |
| ||||||||||||||||
| Baseline (NCT01993186) Columbia Neurological Score (CNS) Total Score | The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance & Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone & Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function. | participants with a baseline assessment | Mean | Standard Deviation | score on a scale |
| |||||||||||||||
| Baseline (NCT01993186) Short Form (SF) 10 (SF-10) Health Survey for Children Physical Summary Score | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. | participants with a baseline assessment | Mean | Standard Deviation | T-score |
| |||||||||||||||
| Baseline (NCT01993186) SF-10 Health Survey for Children Psychosocial Summary Score | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. | participants with a baseline assessment | Mean | Standard Deviation | T-score |
| |||||||||||||||
| Baseline (NCT01993186) SF12 Version 2 (SF-12v2) Health Survey Physical Component Summary (PCS) Score | SF-12v2 was assessed for adults aged >=18 years. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to US general population T-scores. Therefore, all scores obtained that are < 50 can be interpreted as below the US general population T-score and scores > 50 can be interpreted as above the US general population T-score. Higher global scores are associated with better quality of life. | participants with a baseline assessment | Mean | Standard Deviation | T-score |
| |||||||||||||||
| Baseline (NCT01993186) SF-12v2 Health Survey MCS Score | SF-12v2 was assessed for adults aged >=18 years. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to US general population T-scores. Therefore, all scores obtained that are < 50 can be interpreted as below the US general population T-score and scores > 50 can be interpreted as above the US general population T-score. Higher global scores are associated with better quality of life. | participants with a baseline assessment | Mean | Standard Deviation | T-score |
|
| 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 | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE. | Posted | Count of Participants | Participants | From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days. |
|
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence. | Participants with a baseline and postbaseline assessment at given time point. | Posted | Mean | Standard Deviation | seizures per 4 weeks | Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline Over Time in CNS Total Score | The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance & Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone & Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function. | Participants with a baseline and postbaseline assessment at given time point. | Posted | Mean | Standard Deviation | score on a scale | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. | Pediatric participants with a baseline and postbaseline assessment at given time point. | Posted | Mean | Standard Deviation | T-score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. | Pediatric participants with a baseline and postbaseline assessment at given time point. | Posted | Mean | Standard Deviation | T-score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life. | Adult participants with a baseline and postbaseline assessment at given time point. | Posted | Mean | Standard Deviation | T-score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life. | Adult participants with a baseline and postbaseline assessment at given time point. | Posted | Mean | Standard Deviation | T-score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 |
|
|
From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | UX007 (Triheptanoin) | UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake. | 0 | 15 | 2 | 15 | 13 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Intestinal Obstruction | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Croup Infectious | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Ear Infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Otitis Media | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Otitis Media Acute | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Breath Odour | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Thirst | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Seasonal Allergy | Immune system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ear Infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Otitis Media | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Otitis Media Acute | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pharyngitis Streptococcal | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Viral Upper Respiratory Tract Infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Head Injury | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Ligament Sprain | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Procedural Pain | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Alanine Aminotransferase Increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood Glucose Increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Weight Increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Muscular Weakness | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Disturbance In Attention | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Head Titubation | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Petit Mal Epilepsy | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vulvovaginal Pruritus | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hair Growth Abnormal | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hot Flush | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
|
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Ultragenyx Pharmaceutical Inc | 1-888-756-8657 | medinfo@ultragenyx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 25, 2018 | Apr 1, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| C536830 | Glut1 Deficiency Syndrome |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C531010 | triheptanoin |
Not provided
Not provided
Not provided
|
|
| 18 to < 65 years |
|
|
|
| Unknown or Not Reported |
|
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
| Black or African American |
|
|
| White |
|
|
| Other, Not Specified |
|
|
| Title | Measurements |
|---|---|
|
| Serious and Related TEAEs |
|
| Grade 3 or 4 TEAEs |
|
| Gastrointestinal TEAEs |
|
| TEAEs Leading to Treatment Discontinuation |
|
| TEAEs Leading to Study Discontinuation |
|
| TEAEs Leading to Death |
|
|
|
|
|
|
|
|