Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

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ClinicalTrials.gov Identifier: NCT02599961

Recruitment Status : Terminated (Study was halted prematurely due to lack of efficacy)

First Posted : November 9, 2015

Results First Posted : April 30, 2020

Last Update Posted : June 11, 2020

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Ultragenyx Pharmaceutical Inc

Study Type	Interventional
Study Design	Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition	Glucose Transporter Type 1 Deficiency Syndrome
Intervention	Drug: UX007
Enrollment	15

Participant Flow

Recruitment Details	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.
Pre-assignment Details	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.


Arm/Group Title	UX007 (Triheptanoin)
Arm/Group Description	UX007 dosing was targeted and/or ma...
Arm/Group Description	UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Period Title: Overall Study
Started	15
Completed	0
Not Completed	15
Reason Not Completed
Withdrawal by Subject	1
Subject Non-Compliance	2
Discontinuation of Study by Sponsor	9
Other, Not Specified	3

Baseline Characteristics

Arm/Group Title		UX007 (Triheptanoin)
Arm/Group Description		UX007 dosing was targeted and/or ma...
Arm/Group Description		UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Baseline Participants		15
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Continuous Mean (Standard Deviation) Unit of measure: Years
	Number Analyzed	15 participants
		15.24 (6.106)
Age, Customized Measure Type: Count of Participants Unit of measure: Participants
2 to < 12 years	Number Analyzed	15 participants
2 to < 12 years		5 33.3%
12 to < 18 years	Number Analyzed	15 participants
12 to < 18 years		5 33.3%
18 to < 65 years	Number Analyzed	15 participants
18 to < 65 years		5 33.3%
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	15 participants
	Female	11 73.3%
	Male	4 26.7%
Ethnicity (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	15 participants
	Hispanic or Latino	1 6.7%
	Not Hispanic or Latino	13 86.7%
	Unknown or Not Reported	1 6.7%
Race (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	15 participants
	American Indian or Alaska Native	1 6.7%
	Asian	1 6.7%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	1 6.7%
	White	11 73.3%
	More than one race	0 0.0%
	Unknown or Not Reported	1 6.7%
Race/Ethnicity, Customized Measure Type: Count of Participants Unit of measure: Participants
American Indian or Alaska Native	Number Analyzed	15 participants
American Indian or Alaska Native		1 6.7%
Asian	Number Analyzed	15 participants
Asian		1 6.7%
Black or African American	Number Analyzed	15 participants
Black or African American		1 6.7%
White	Number Analyzed	15 participants
White		11 73.3%
Other, Not Specified	Number Analyzed	15 participants
Other, Not Specified		1 6.7%
Overall Seizure Frequency Per 4 Weeks ^[1] Mean (Standard Deviation) Unit of measure: Seizures per 4 weeks
	Number Analyzed	15 participants
		312.17 (528.057)
		[1] Measure Description: 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.
Baseline (NCT01993186) Columbia Neurological Score (CNS) Total Score ^[1] [2] Mean (Standard Deviation) Unit of measure: Score on a scale
	Number Analyzed	12 participants
		38.63 (25.428)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: participants with a baseline assessment
Baseline (NCT01993186) Short Form (SF) 10 (SF-10) Health Survey for Children Physical Summary Score ^[1] [2] Mean (Standard Deviation) Unit of measure: T-score
	Number Analyzed	10 participants
		32.63 (17.027)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: participants with a baseline assessment
Baseline (NCT01993186) SF-10 Health Survey for Children Psychosocial Summary Score ^[1] [2] Mean (Standard Deviation) Unit of measure: T-score
	Number Analyzed	10 participants
		48.29 (10.196)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: participants with a baseline assessment
Baseline (NCT01993186) SF12 Version 2 (SF-12v2) Health Survey Physical Component Summary (PCS) Score ^[1] [2] Mean (Standard Deviation) Unit of measure: T-score
	Number Analyzed	2 participants
		39.37 (0.523)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: participants with a baseline assessment
Baseline (NCT01993186) SF-12v2 Health Survey MCS Score ^[1] [2] Mean (Standard Deviation) Unit of measure: T-score
	Number Analyzed	2 participants
		43.49 (4.356)
		[1] Measure Description: 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. [2] Measure Analysis Population Description: participants with a baseline assessment

Outcome Measures

1.Primary Outcome


Title	Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths
Description	An adverse event (AE) was defined as any untoward medical occurrence, ...
Description	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.
Time Frame	From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	UX007 (Triheptanoin)
Arm/Group Description:	UX007 dosing was targeted and/or ma...
Arm/Group Description:	UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Participants Analyzed	15
Measure Type: Count of Participants Unit of Measure: Participants
TEAEs	13 86.7%
Serious TEAEs	2 13.3%
Related TEAEs	10 66.7%
Serious and Related TEAEs	0 0.0%
Grade 3 or 4 TEAEs	1 6.7%
Gastrointestinal TEAEs	9 60.0%
TEAEs Leading to Treatment Discontinuation	0 0.0%
TEAEs Leading to Study Discontinuation	0 0.0%
TEAEs Leading to Death	0 0.0%

2.Secondary Outcome


Title	Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks
Description	The number of observable seizures were recorded by the subject or care...
Description	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.
Time Frame	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

Outcome Measure Data

Analysis Population Description

Participants with a baseline and postbaseline assessment at given time point.


Arm/Group Title		UX007 (Triheptanoin)
Arm/Group Description:		UX007 dosing was targeted and/or ma...
Arm/Group Description:		UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Participants Analyzed		15
Mean (Standard Deviation) Unit of Measure: seizures per 4 weeks
Change at Month 0-3	Number Analyzed	10 participants
Change at Month 0-3		-64.22 (185.564)
Change at Month 4-6	Number Analyzed	7 participants
Change at Month 4-6		-64.19 (142.460)
Change at Month 7-9	Number Analyzed	7 participants
Change at Month 7-9		-61.81 (164.770)
Change at Month 10-12	Number Analyzed	7 participants
Change at Month 10-12		-91.93 (216.834)
Change at Month 13-18	Number Analyzed	8 participants
Change at Month 13-18		-75.56 (206.406)
Change at Month 19-24	Number Analyzed	6 participants
Change at Month 19-24		-110.12 (233.492)
Change at Month 25-30	Number Analyzed	5 participants
Change at Month 25-30		-135.60 (249.871)
Change at Month 31-36	Number Analyzed	2 participants
Change at Month 31-36		-51.88 (101.378)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	UX007 (Triheptanoin)
	Comments	Month 0-3
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	< 0.0001
	Comments	From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.
	Method	generalized estimating equation (GEE)
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-82.73
	Confidence Interval	(2-Sided) 95% -105.00 to -60.46
	Parameter Dispersion	Type: Standard Error of the Mean Value: 11.363
	Estimation Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	UX007 (Triheptanoin)
	Comments	Month 4-6
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0006
	Comments	From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.
	Method	GEE model
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS mean
	Estimated Value	-54.91
	Confidence Interval	(2-Sided) 95% -86.46 to -23.36
	Parameter Dispersion	Type: Standard Error of the Mean Value: 16.097
	Estimation Comments	[Not Specified]

Statistical Analysis 3

Statistical Analysis Overview	Comparison Group Selection	UX007 (Triheptanoin)
	Comments	Month 7-9
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	0.0019
	Comments	From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.
	Method	GEE model
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-52.53
	Confidence Interval	(2-Sided) 95% -85.62 to -19.45
	Parameter Dispersion	Type: Standard Error of the Mean Value: 16.882
	Estimation Comments	[Not Specified]

Statistical Analysis 4

Statistical Analysis Overview	Comparison Group Selection	UX007 (Triheptanoin)
	Comments	Month 10-12
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	< 0.0001
	Comments	From the GEE model which includes the reduction from baseline for each visit period as the response variable, baseline as covariates, visit period as a factor, identity link function and exchangeable within subject working correlation matrix.
	Method	GEE model
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean
	Estimated Value	-82.65
	Confidence Interval	(2-Sided) 95% -113.13 to -52.17
	Parameter Dispersion	Type: Standard Error of the Mean Value: 15.550
	Estimation Comments	[Not Specified]

3.Secondary Outcome


Title	Change From Baseline Over Time in CNS Total Score
Description	The CNS evaluates measures of neurological function and development de...
Description	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.
Time Frame	Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36

Outcome Measure Data

Analysis Population Description

Participants with a baseline and postbaseline assessment at given time point.


Arm/Group Title		UX007 (Triheptanoin)
Arm/Group Description:		UX007 dosing was targeted and/or ma...
Arm/Group Description:		UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Participants Analyzed		12
Mean (Standard Deviation) Unit of Measure: score on a scale
Change at Month 0	Number Analyzed	10 participants
Change at Month 0		11.85 (20.331)
Change at Month 6	Number Analyzed	7 participants
Change at Month 6		9.36 (18.495)
Change at Month 12	Number Analyzed	7 participants
Change at Month 12		13.64 (21.371)
Change at Month 24	Number Analyzed	4 participants
Change at Month 24		3.38 (3.092)
Change at Month 36	Number Analyzed	1 participants
Change at Month 36		0.00 ^[1] (NA)

[1]

1 participant assessed

4.Secondary Outcome


Title	Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score
Description	The SF-10 Health Survey for Children was administered to caregivers of...
Description	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.
Time Frame	Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30

Outcome Measure Data

Analysis Population Description

Pediatric participants with a baseline and postbaseline assessment at given time point.


Arm/Group Title		UX007 (Triheptanoin)
Arm/Group Description:		UX007 dosing was targeted and/or ma...
Arm/Group Description:		UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Participants Analyzed		10
Mean (Standard Deviation) Unit of Measure: T-score
Change at Month 0	Number Analyzed	9 participants
Change at Month 0		0.25 (16.917)
Change at Month 6	Number Analyzed	7 participants
Change at Month 6		9.56 (21.522)
Change at Month 12	Number Analyzed	6 participants
Change at Month 12		-2.67 (9.475)
Change at Month 18	Number Analyzed	5 participants
Change at Month 18		-9.35 (12.702)
Change at Month 24	Number Analyzed	4 participants
Change at Month 24		-8.96 (20.075)
Change at Month 30	Number Analyzed	2 participants
Change at Month 30		7.74 (2.273)

5.Secondary Outcome


Title	Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score
Description	The SF-10 Health Survey for Children was administered to caregivers of...
Description	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.
Time Frame	Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30

Outcome Measure Data

Analysis Population Description

Pediatric participants with a baseline and postbaseline assessment at given time point.


Arm/Group Title		UX007 (Triheptanoin)
Arm/Group Description:		UX007 dosing was targeted and/or ma...
Arm/Group Description:		UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Participants Analyzed		10
Mean (Standard Deviation) Unit of Measure: T-score
Change at Month 0	Number Analyzed	9 participants
Change at Month 0		0.59 (11.049)
Change at Month 6	Number Analyzed	7 participants
Change at Month 6		-2.29 (12.366)
Change at Month 12	Number Analyzed	6 participants
Change at Month 12		-7.43 (15.514)
Change at Month 18	Number Analyzed	5 participants
Change at Month 18		-6.60 (14.987)
Change at Month 24	Number Analyzed	4 participants
Change at Month 24		-8.25 (15.316)
Change at Month 30	Number Analyzed	2 participants
Change at Month 30		8.91 (12.599)

6.Secondary Outcome


Title	Change From Baseline Over Time in SF-12v2 Health Survey PCS Score
Description	SF-12v2 was assessed for adults 18 years of age and older. Eight domai...
Description	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.
Time Frame	Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18

Outcome Measure Data

Analysis Population Description

Adult participants with a baseline and postbaseline assessment at given time point.


Arm/Group Title		UX007 (Triheptanoin)
Arm/Group Description:		UX007 dosing was targeted and/or ma...
Arm/Group Description:		UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Participants Analyzed		2
Mean (Standard Deviation) Unit of Measure: T-score
Change at Month 0	Number Analyzed	2 participants
Change at Month 0		10.25 (5.077)
Change at Month 6	Number Analyzed	2 participants
Change at Month 6		12.37 (5.367)
Change at Month 12	Number Analyzed	2 participants
Change at Month 12		5.09 (1.619)
Change at Month 18	Number Analyzed	1 participants
Change at Month 18		-0.35 ^[1] (NA)

[1]

1 participant assessed.

7.Secondary Outcome


Title	Change From Baseline Over Time in SF-12v2 Health Survey MCS Score
Description	SF-12v2 was assessed for adults 18 years of age and older. Eight domai...
Description	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.
Time Frame	Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18

Outcome Measure Data

Analysis Population Description

Adult participants with a baseline and postbaseline assessment at given time point.


Arm/Group Title		UX007 (Triheptanoin)
Arm/Group Description:		UX007 dosing was targeted and/or ma...
Arm/Group Description:		UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Participants Analyzed		2
Mean (Standard Deviation) Unit of Measure: T-score
Change at Month 0	Number Analyzed	2 participants
Change at Month 0		8.63 (2.249)
Change at Month 6	Number Analyzed	2 participants
Change at Month 6		5.84 (3.316)
Change at Month 12	Number Analyzed	2 participants
Change at Month 12		6.90 (5.706)
Change at Month 18	Number Analyzed	1 participants
Change at Month 18		16.96 ^[1] (NA)

[1]

1 participant assessed.

Adverse Events

Time Frame	From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	UX007 (Triheptanoin)
Arm/Group Description	UX007 dosing was targeted and/or ma...
Arm/Group Description	UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
All-Cause Mortality
	UX007 (Triheptanoin)
	Affected / at Risk (%)
Total	0/15 (0.00%)
Serious Adverse Events Serious Adverse Events
	UX007 (Triheptanoin)
	Affected / at Risk (%)
Total	2/15 (13.33%)
Gastrointestinal disorders
Intestinal Obstruction ^† 1	1/15 (6.67%)
Infections and infestations
Croup Infectious ^† 1	1/15 (6.67%)
Ear Infection ^† 1	1/15 (6.67%)
Otitis Media ^† 1	1/15 (6.67%)
Otitis Media Acute ^† 1	1/15 (6.67%)
1 Term from vocabulary, MedDRA 20.0 † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	UX007 (Triheptanoin)
	Affected / at Risk (%)
Total	13/15 (86.67%)
Cardiac disorders
Tachycardia ^† 1	1/15 (6.67%)
Gastrointestinal disorders
Abdominal Pain ^† 1	3/15 (20.00%)
Abdominal Pain Upper ^† 1	1/15 (6.67%)
Breath Odour ^† 1	1/15 (6.67%)
Constipation ^† 1	1/15 (6.67%)
Diarrhoea ^† 1	7/15 (46.67%)
Dysphagia ^† 1	1/15 (6.67%)
Flatulence ^† 1	1/15 (6.67%)
Haematochezia ^† 1	1/15 (6.67%)
Nausea ^† 1	3/15 (20.00%)
Vomiting ^† 1	5/15 (33.33%)
General disorders
Pyrexia ^† 1	1/15 (6.67%)
Thirst ^† 1	1/15 (6.67%)
Immune system disorders
Seasonal Allergy ^† 1	1/15 (6.67%)
Infections and infestations
Ear Infection ^† 1	1/15 (6.67%)
Influenza ^† 1	1/15 (6.67%)
Otitis Media ^† 1	1/15 (6.67%)
Otitis Media Acute ^† 1	1/15 (6.67%)
Pharyngitis Streptococcal ^† 1	1/15 (6.67%)
Upper Respiratory Tract Infection ^† 1	3/15 (20.00%)
Viral Upper Respiratory Tract Infection ^† 1	3/15 (20.00%)
Injury, poisoning and procedural complications
Contusion ^† 1	1/15 (6.67%)
Head Injury ^† 1	1/15 (6.67%)
Ligament Sprain ^† 1	1/15 (6.67%)
Procedural Pain ^† 1	1/15 (6.67%)
Investigations
Alanine Aminotransferase Increased ^† 1	1/15 (6.67%)
Blood Glucose Increased ^† 1	1/15 (6.67%)
Weight Increased ^† 1	1/15 (6.67%)
Metabolism and nutrition disorders
Hypoglycaemia ^† 1	1/15 (6.67%)
Hyponatraemia ^† 1	1/15 (6.67%)
Musculoskeletal and connective tissue disorders
Back Pain ^† 1	2/15 (13.33%)
Muscle Spasms ^† 1	1/15 (6.67%)
Muscular Weakness ^† 1	1/15 (6.67%)
Musculoskeletal Pain ^† 1	1/15 (6.67%)
Pain In Extremity ^† 1	1/15 (6.67%)
Nervous system disorders
Disturbance In Attention ^† 1	1/15 (6.67%)
Dysarthria ^† 1	1/15 (6.67%)
Head Titubation ^† 1	1/15 (6.67%)
Headache ^† 1	4/15 (26.67%)
Lethargy ^† 1	3/15 (20.00%)
Petit Mal Epilepsy ^† 1	2/15 (13.33%)
Seizure ^† 1	1/15 (6.67%)
Tremor ^† 1	1/15 (6.67%)
Psychiatric disorders
Insomnia ^† 1	1/15 (6.67%)
Reproductive system and breast disorders
Dysmenorrhoea ^† 1	1/15 (6.67%)
Vulvovaginal Pruritus ^† 1	1/15 (6.67%)
Respiratory, thoracic and mediastinal disorders
Cough ^† 1	1/15 (6.67%)
Nasal Congestion ^† 1	1/15 (6.67%)
Oropharyngeal Pain ^† 1	1/15 (6.67%)
Skin and subcutaneous tissue disorders
Hair Growth Abnormal ^† 1	1/15 (6.67%)
Vascular disorders
Hot Flush ^† 1	1/15 (6.67%)
Hypertension ^† 1	1/15 (6.67%)
1 Term from vocabulary, MedDRA 20.0 † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

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.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Medical Information
Organization:	Ultragenyx Pharmaceutical Inc
Phone:	1-888-756-8657
EMail:	medinfo@ultragenyx.com

Layout table for additonal information

Responsible Party:	Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier:	NCT02599961
Other Study ID Numbers:	UX007G-CL202 2015-000389-69 ( EudraCT Number )
First Submitted:	November 3, 2015
First Posted:	November 9, 2015
Results First Submitted:	April 17, 2020
Results First Posted:	April 30, 2020
Last Update Posted:	June 11, 2020

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