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Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (GNEM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02377921
Recruitment Status : Completed
First Posted : March 4, 2015
Results First Posted : July 9, 2018
Last Update Posted : June 27, 2019
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Hereditary Inclusion Body Myopathy
Distal Myopathy With Rimmed Vacuoles
Distal Myopathy, Nonaka Type
GNE Myopathy
Interventions Drug: aceneuramic acid extended-release (Ace-ER)
Drug: Placebo
Enrollment 89
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description Aceneuramic acid extended-release (Ace-ER) 6 g/day, divided 3 times per day (TID) for 48 weeks. Matching placebo TID for 48 weeks.
Period Title: Overall Study
Started 45 44
Completed 44 43
Not Completed 1 1
Reason Not Completed
Subject non-compliance             1             1
Arm/Group Title Ace-ER 6 g/Day Placebo Total
Hide Arm/Group Description Ace-ER 6 g/day, divided TID for 48 weeks. Matching placebo TID for 48 weeks. Total of all reporting groups
Overall Number of Baseline Participants 45 43 88
Hide Baseline Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 45 participants 43 participants 88 participants
33.8  (7.91) 36.2  (8.59) 35.0  (8.29)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 43 participants 88 participants
Female
20
  44.4%
20
  46.5%
40
  45.5%
Male
25
  55.6%
23
  53.5%
48
  54.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 43 participants 88 participants
Hispanic or Latino
5
  11.1%
7
  16.3%
12
  13.6%
Not Hispanic or Latino
39
  86.7%
33
  76.7%
72
  81.8%
Unknown or Not Reported
1
   2.2%
3
   7.0%
4
   4.5%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 43 participants 88 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
6
  13.3%
7
  16.3%
13
  14.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
36
  80.0%
32
  74.4%
68
  77.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
3
   6.7%
4
   9.3%
7
   8.0%
Upper Extremity Composite (UEC) Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 45 participants 43 participants 88 participants
55.99  (26.950) 56.31  (29.287) 56.14  (27.954)
[1]
Measure Description: Muscle strength based on the maximum voluntary isometric contraction (MVIC) against a dynamometer was measured bilaterally in the following upper extremity muscle groups: gross grip, shoulder abductors, elbow flexors, and elbow extensors. The UEC is derived from the sum of the average of the right and left total force values (measured in kg).
Muscle Strength in the Knee Extensors   [1] 
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 45 participants 43 participants 88 participants
26.53  (9.035) 26.65  (8.969) 26.59  (8.951)
[1]
Measure Description: Lower extremity muscle strength in the knee extensors was measured by dynamometry. Bilateral total force was defined as the average of the right and left force values (measured in kg).
Lower Extremity Composite (LEC) Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 45 participants 43 participants 88 participants
53.52  (33.751) 55.17  (39.324) 54.33  (36.378)
[1]
Measure Description: Muscle strength based on MVIC against a dynamometer was measured bilaterally in the following lower extremity muscle groups: knee flexors, hip flexors, hip extensors, hip abductors and hip adductors. The LEC is derived from the sum of the average of the right and left total force values (measured in kg).
Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy Functional Activities Scale(GNEM-FAS) Mobility Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 45 participants 43 participants 88 participants
26.38  (7.581) 26.23  (6.403) 26.31  (6.990)
[1]
Measure Description: Lower extremity use and function was assessed using the Mobility domain of the GNEM-FAS instrument a disease specific measure developed to assess the functional impact of changes in muscle strength on mobility (reflective of the lower extremities). This mobility score ranges from 0 to 40 with higher scores representing greater mobility.
Stands in Sit-to-Stand Test   [1] 
Mean (Standard Deviation)
Unit of measure:  Stands
Number Analyzed 45 participants 43 participants 88 participants
12.38  (4.103) 12.58  (4.316) 12.48  (4.185)
[1]
Measure Description: Lower extremity function was assessed using a sit-to-stand test. The number of times the participant can rise from a seated to a standing position in a 30-second period was recorded.
Lifts in Weighted Arm Lift Test   [1] 
Mean (Standard Deviation)
Unit of measure:  Arm lifts
Number Analyzed 45 participants 43 participants 88 participants
30.50  (10.452) 28.18  (9.824) 29.33  (10.137)
[1]
Measure Description: Upper extremity function was assessed using a weighted arm lift test performed bilaterally. The number of times the subject can raise a 1 kg weight above the head in a 30-second period was recorded.
Distance Walked in 6-Minute Walk Test (6MWT)   [1] 
Mean (Standard Deviation)
Unit of measure:  Meters
Number Analyzed 45 participants 43 participants 88 participants
367.0  (115.07) 361.2  (109.87) 364.2  (111.95)
[1]
Measure Description: The total distance walked (meters) in a 6-minute period was measured.
Percent of Predicted Distance Walked in 6MWT   [1] 
Mean (Standard Deviation)
Unit of measure:  Percentage of predicted meters
Number Analyzed 45 participants 43 participants 88 participants
49.97  (15.521) 49.87  (14.823) 49.92  (15.097)
[1]
Measure Description: The total distance walked (meters) in a 6-minute period was measured, and the percent predicted distance based on normative data for age and gender was estimated. Predicted Six-Minute Walk Test Distance (meters) = 868.8 - (2.99 x Age) - (74.7 x Sex), where Age is baseline age in years, and Sex = 0 for males, and 1 for females.
1.Primary Outcome
Title Change From Baseline in UEC Score (Total Force in kg) at Week 48
Hide Description Muscle strength based on the maximum voluntary isometric contraction (MVIC) against a dynamometer was measured bilaterally in the following upper extremity muscle groups: gross grip, shoulder abductors, elbow flexors, and elbow extensors. The UEC is derived from the sum of the average of the right and left total force values (measured in kg).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kg
-2.25
(-3.77 to -0.74)
-2.99
(-4.69 to -1.28)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5387
Comments Generalized estimating equation (GEE) model includes change from Baseline (BL) as dependent variable, visit, treatment and visit by treatment as fixed factors, and BL values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 0.74
Confidence Interval (2-Sided) 95%
-1.61 to 3.09
Estimation Comments Difference = Ace-ER - placebo
2.Secondary Outcome
Title Change From Baseline in Muscle Strength in the Knee Extensors at Week 48
Hide Description Lower extremity muscle strength in the knee extensors was measured by dynamometry. Bilateral total force was defined as the average of the right and left force values (measured in kg).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kg
0.05
(-1.19 to 1.29)
0.45
(-1.20 to 2.10)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6938
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -0.40
Confidence Interval (2-Sided) 95%
-2.38 to 1.58
Estimation Comments Difference = Ace-ER - placebo
3.Secondary Outcome
Title Change From Baseline in LEC Score (Total Force in kg) at Week 48
Hide Description Muscle strength based on MVIC against a dynamometer was measured bilaterally in the following lower extremity muscle groups: knee flexors, hip flexors, hip extensors, hip abductors and hip adductors. The LEC is derived from the sum of the average of the right and left total force values (measured in kg).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kg
-1.92
(-4.49 to 0.65)
-0.44
(-3.96 to 3.09)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5023
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -1.49
Confidence Interval (2-Sided) 95%
-5.83 to 2.86
Estimation Comments Difference = Ace-ER - placebo
4.Secondary Outcome
Title Change From Baseline in GNEM FAS Mobility Domain Score at Week 48
Hide Description Lower extremity use and function was assessed using the Mobility domain of the GNEM-FAS instrument a disease-specific measure developed to assess the functional impact of changes in muscle strength on mobility (reflective of the lower extremities). This mobility score ranges from 0 to 40 with higher scores representing greater mobility.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
-2.49
(-3.56 to -1.42)
-1.77
(-2.59 to -0.95)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2739
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -0.72
Confidence Interval (2-Sided) 95%
-2.01 to 0.57
Estimation Comments Difference = Ace-ER - placebo
5.Secondary Outcome
Title Change From Baseline in Number of Lifts in the 30 Second Weighted Arm Lift Test at Week 48
Hide Description Upper extremity function was assessed using a weighted arm lift test performed bilaterally. The number of times the participant can raise a 1 kg weight above the head in a 30-second period was recorded.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: arm lifts
0.03
(-2.19 to 2.26)
2.79
(0.21 to 5.38)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1235
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -2.76
Confidence Interval (2-Sided) 95%
-6.27 to 0.75
Estimation Comments Difference: Ace-ER 6 g/day - placebo
6.Secondary Outcome
Title Change From Baseline in Number of Stands in the Sit to Stand Test at Week 48
Hide Description Lower extremity function was assessed using a sit-to-stand test. The number of times the participant can rise from a seated to a standing position in a 30-second period was recorded.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: stands
0.11
(-0.55 to 0.77)
0.53
(-0.21 to 1.28)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3907
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -0.43
Confidence Interval (2-Sided) 95%
-1.40 to 0.55
Estimation Comments Difference: Ace-ER 6 g/day - placebo
7.Secondary Outcome
Title Change From Baseline in Meters Walked in the 6MWT at Week 48
Hide Description The total distance walked (meters) in a 6-minute period was measured.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: meters
-17.79
(-32.09 to -3.50)
-6.81
(-16.83 to 3.21)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1964
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -10.98
Confidence Interval (2-Sided) 95%
-27.64 to 5.68
Estimation Comments Difference: Ace-ER 6 g/day - placebo
8.Secondary Outcome
Title Change From Baseline in Percent Predicted Meters Walked in the 6MWT at Week 48
Hide Description The total distance walked (meters) in a 6-minute period was measured, and the percent predicted distance based on normative data for age and gender was estimated.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percentage of predicted meters
-2.37
(-4.30 to -0.44)
-0.97
(-2.32 to 0.38)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2241
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -1.40
Confidence Interval (2-Sided) 95%
-3.66 to 0.86
Estimation Comments Difference: Ace-ER - placebo
9.Secondary Outcome
Title Change From Baseline in GNEM FAS Upper Extremity Domain Score at Week 48
Hide Description Upper extremity use and function was assessed using the Mobility domain of the GNEM-FAS instrument a disease-specific measure developed to assess the functional impact of changes in muscle strength on mobility (reflective of the upper extremities). This mobility score ranges from 0 to 40 with higher scores representing greater mobility.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description:
Ace-ER 6 g/day, divided TID for 48 weeks.
Matching placebo TID for 48 weeks.
Overall Number of Participants Analyzed 45 43
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
-1.40
(-2.21 to -0.58)
-1.08
(-1.86 to -0.29)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ace-ER 6 g/Day, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5608
Comments GEE model includes change from Baseline as dependent variable, visit, treatment and visit by treatment as fixed factors, and Baseline values, sex, and region as covariates, with compound symmetry covariance structure.
Method GEE model
Comments [Not Specified]
Method of Estimation Estimation Parameter LS Mean Difference
Estimated Value -0.32
Confidence Interval (2-Sided) 95%
-1.39 to 0.75
Estimation Comments Difference: Ace-ER 6 g/day - placebo
Time Frame Screening through Week 48 plus 28 days (+5 days). The mean (SD) duration of treatment was 340.2 (12.02) days and 332.9 (40.86) days for the Ace-ER and placebo groups, respectively.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ace-ER 6 g/Day Placebo
Hide Arm/Group Description Ace-ER 6 g/day, divided TID for 48 weeks. Matching placebo TID for 48 weeks.
All-Cause Mortality
Ace-ER 6 g/Day Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/45 (0.00%)   0/44 (0.00%) 
Hide Serious Adverse Events
Ace-ER 6 g/Day Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   2/45 (4.44%)   1/44 (2.27%) 
Cardiac disorders     
Acute myocardial infarction † 1  1/45 (2.22%)  0/44 (0.00%) 
Gastrointestinal disorders     
Gastritis † 1  1/45 (2.22%)  0/44 (0.00%) 
Pregnancy, puerperium and perinatal conditions     
Abortion † 1  0/45 (0.00%)  1/44 (2.27%) 
1
Term from vocabulary, MedDRA 18.1
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ace-ER 6 g/Day Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   38/45 (84.44%)   30/44 (68.18%) 
Gastrointestinal disorders     
Abdominal distension † 1  4/45 (8.89%)  4/44 (9.09%) 
Abdominal pain upper † 1  10/45 (22.22%)  3/44 (6.82%) 
Diarrhoea † 1  8/45 (17.78%)  6/44 (13.64%) 
Flatulence † 1  6/45 (13.33%)  5/44 (11.36%) 
Frequent bowel movements † 1  3/45 (6.67%)  0/44 (0.00%) 
Nausea † 1  5/45 (11.11%)  2/44 (4.55%) 
General disorders     
Asthenia † 1  2/45 (4.44%)  3/44 (6.82%) 
Fatigue † 1  4/45 (8.89%)  4/44 (9.09%) 
Influenza like illness † 1  4/45 (8.89%)  11/44 (25.00%) 
Pain † 1  1/45 (2.22%)  3/44 (6.82%) 
Infections and infestations     
Influenza † 1  1/45 (2.22%)  4/44 (9.09%) 
Upper respiratory tract infection † 1  5/45 (11.11%)  1/44 (2.27%) 
Injury, poisoning and procedural complications     
Contusion † 1  4/45 (8.89%)  0/44 (0.00%) 
Fall † 1  7/45 (15.56%)  7/44 (15.91%) 
Skin abrasion † 1  0/45 (0.00%)  3/44 (6.82%) 
Investigations     
Alanine aminotransferase increased † 1  4/45 (8.89%)  2/44 (4.55%) 
Musculoskeletal and connective tissue disorders     
Arthralgia † 1  9/45 (20.00%)  5/44 (11.36%) 
Back pain † 1  5/45 (11.11%)  4/44 (9.09%) 
Muscular weakness † 1  6/45 (13.33%)  3/44 (6.82%) 
Musculoskeletal pain † 1  4/45 (8.89%)  2/44 (4.55%) 
Myalgia † 1  6/45 (13.33%)  1/44 (2.27%) 
Pain in extremity † 1  4/45 (8.89%)  3/44 (6.82%) 
Nervous system disorders     
Dizziness † 1  3/45 (6.67%)  1/44 (2.27%) 
Dysgeusia † 1  3/45 (6.67%)  0/44 (0.00%) 
Headache † 1  7/45 (15.56%)  7/44 (15.91%) 
Sciatica † 1  0/45 (0.00%)  3/44 (6.82%) 
Psychiatric disorders     
Sleep disorder † 1  0/45 (0.00%)  4/44 (9.09%) 
Respiratory, thoracic and mediastinal disorders     
Cough † 1  5/45 (11.11%)  4/44 (9.09%) 
Nasal congestion † 1  4/45 (8.89%)  1/44 (2.27%) 
Oropharyngeal pain † 1  4/45 (8.89%)  2/44 (4.55%) 
1
Term from vocabulary, MedDRA 18.1
†
Indicates events were collected by systematic assessment
[Not Specified]
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: 1-888-756-8567
EMail: medinfo@ultragenyx.com
Publications of Results:
Lochmuller H, Behin A, Caraco Y, Lau H, Mirabella M, Tournev I, Tarnopolsky M, Pogoryelova O, Woods C, Lai A, Shah J, Koutsoukos T, Skrinar A, Mansbach H, Kakkis E, Mozaffar T. A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy. Neurology. 2019 Apr 30;92(18):e2109-e2117. doi: 10.1212/WNL.0000000000006932. Epub 2019 Jan 25.
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT02377921    
Other Study ID Numbers: UX001-CL301
First Submitted: February 27, 2015
First Posted: March 4, 2015
Results First Submitted: June 7, 2018
Results First Posted: July 9, 2018
Last Update Posted: June 27, 2019