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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
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
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)
Hide 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
Arm/Group Title UX007 (Triheptanoin)
Hide Arm/Group Description UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake.
Overall Number of Baseline Participants 15
Hide 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
5
  33.3%
12 to < 18 years Number Analyzed 15 participants
5
  33.3%
18 to < 65 years Number Analyzed 15 participants
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
1
   6.7%
Asian Number Analyzed 15 participants
1
   6.7%
Black or African American Number Analyzed 15 participants
1
   6.7%
White Number Analyzed 15 participants
11
  73.3%
Other, Not Specified Number Analyzed 15 participants
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
1.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths
Hide 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.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title UX007 (Triheptanoin)
Hide 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
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with a baseline and postbaseline assessment at given time point.
Arm/Group Title UX007 (Triheptanoin)
Hide 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
-64.22  (185.564)
Change at Month 4-6 Number Analyzed 7 participants
-64.19  (142.460)
Change at Month 7-9 Number Analyzed 7 participants
-61.81  (164.770)
Change at Month 10-12 Number Analyzed 7 participants
-91.93  (216.834)
Change at Month 13-18 Number Analyzed 8 participants
-75.56  (206.406)
Change at Month 19-24 Number Analyzed 6 participants
-110.12  (233.492)
Change at Month 25-30 Number Analyzed 5 participants
-135.60  (249.871)
Change at Month 31-36 Number Analyzed 2 participants
-51.88  (101.378)
Hide 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]
Hide 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]
Hide 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]
Hide 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
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with a baseline and postbaseline assessment at given time point.
Arm/Group Title UX007 (Triheptanoin)
Hide 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
11.85  (20.331)
Change at Month 6 Number Analyzed 7 participants
9.36  (18.495)
Change at Month 12 Number Analyzed 7 participants
13.64  (21.371)
Change at Month 24 Number Analyzed 4 participants
3.38  (3.092)
Change at Month 36 Number Analyzed 1 participants
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
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Pediatric participants with a baseline and postbaseline assessment at given time point.
Arm/Group Title UX007 (Triheptanoin)
Hide 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
0.25  (16.917)
Change at Month 6 Number Analyzed 7 participants
9.56  (21.522)
Change at Month 12 Number Analyzed 6 participants
-2.67  (9.475)
Change at Month 18 Number Analyzed 5 participants
-9.35  (12.702)
Change at Month 24 Number Analyzed 4 participants
-8.96  (20.075)
Change at Month 30 Number Analyzed 2 participants
7.74  (2.273)
5.Secondary Outcome
Title Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Pediatric participants with a baseline and postbaseline assessment at given time point.
Arm/Group Title UX007 (Triheptanoin)
Hide 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
0.59  (11.049)
Change at Month 6 Number Analyzed 7 participants
-2.29  (12.366)
Change at Month 12 Number Analyzed 6 participants
-7.43  (15.514)
Change at Month 18 Number Analyzed 5 participants
-6.60  (14.987)
Change at Month 24 Number Analyzed 4 participants
-8.25  (15.316)
Change at Month 30 Number Analyzed 2 participants
8.91  (12.599)
6.Secondary Outcome
Title Change From Baseline Over Time in SF-12v2 Health Survey PCS Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Adult participants with a baseline and postbaseline assessment at given time point.
Arm/Group Title UX007 (Triheptanoin)
Hide 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
10.25  (5.077)
Change at Month 6 Number Analyzed 2 participants
12.37  (5.367)
Change at Month 12 Number Analyzed 2 participants
5.09  (1.619)
Change at Month 18 Number Analyzed 1 participants
-0.35 [1]   (NA)
[1]
1 participant assessed.
7.Secondary Outcome
Title Change From Baseline Over Time in SF-12v2 Health Survey MCS Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Adult participants with a baseline and postbaseline assessment at given time point.
Arm/Group Title UX007 (Triheptanoin)
Hide 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
8.63  (2.249)
Change at Month 6 Number Analyzed 2 participants
5.84  (3.316)
Change at Month 12 Number Analyzed 2 participants
6.90  (5.706)
Change at Month 18 Number Analyzed 1 participants
16.96 [1]   (NA)
[1]
1 participant assessed.
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)
Hide 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%) 
Hide 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
Hide 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
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