Trial · NCT02377921

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)

NCT02377921, Phase 3, Completed, Ultragenyx Pharmaceutical Inc; results first posted 2018-07-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. 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). Trials, record NCT02377921. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02377921

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: 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)
NCT number: NCT02377921
Status: Completed
Phases: Phase 3
Enrollment: 89 participants, actual
Lead sponsor: Ultragenyx Pharmaceutical Inc
Conditions: Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
Interventions: aceneuramic acid extended-release (Ace-ER), Placebo
First posted: 2015-03-04
Last updated: 2019-06-27
Results first posted: 2018-07-09

Source ClinicalTrials.gov

Registered outcomes9

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in UEC Score (Total Force in kg) at Week 48	Baseline, Week 48	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).
Secondary	Change From Baseline in Muscle Strength in the Knee Extensors at Week 48	Baseline, Week 48	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).
Secondary	Change From Baseline in LEC Score (Total Force in kg) at Week 48	Baseline, Week 48	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).
Secondary	Change From Baseline in GNEM FAS Mobility Domain Score at Week 48	Baseline, Week 48	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.
Secondary	Change From Baseline in Number of Lifts in the 30 Second Weighted Arm Lift Test at Week 48	Baseline, Week 48	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.
Secondary	Change From Baseline in Number of Stands in the Sit to Stand Test at Week 48	Baseline, Week 48	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.
Secondary	Change From Baseline in Meters Walked in the 6MWT at Week 48	Baseline, Week 48	The total distance walked (meters) in a 6-minute period was measured.
Secondary	Change From Baseline in Percent Predicted Meters Walked in the 6MWT at Week 48	Baseline, Week 48	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.
Secondary	Change From Baseline in GNEM FAS Upper Extremity Domain Score at Week 48	Baseline, Week 48	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.

Source ClinicalTrials.gov

Outcomes9

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in UEC Score (Total Force in kg) at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	kg	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Muscle Strength in the Knee Extensors at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	kg	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in LEC Score (Total Force in kg) at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	kg	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in GNEM FAS Mobility Domain Score at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	units on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Number of Lifts in the 30 Second Weighted Arm Lift Test at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	arm lifts	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Number of Stands in the Sit to Stand Test at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	stands	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Meters Walked in the 6MWT at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	meters	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Percent Predicted Meters Walked in the 6MWT at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	percentage of predicted meters	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in GNEM FAS Upper Extremity Domain Score at Week 48	Baseline, Week 48	Primary Analysis Set: participants who had a Baseline and at least 1 postbaseline measurement.	units on a scale	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group18

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change From Baseline in UEC Score (Total Force in kg) at Week 48	Ace-ER 6 g/Day	-2.25	95% Confidence Interval -3.77 to -0.74	kg
Change From Baseline in UEC Score (Total Force in kg) at Week 48	Placebo	-2.99	95% Confidence Interval -4.69 to -1.28	kg
Change From Baseline in Muscle Strength in the Knee Extensors at Week 48	Ace-ER 6 g/Day	0.05	95% Confidence Interval -1.19 to 1.29	kg
Change From Baseline in Muscle Strength in the Knee Extensors at Week 48	Placebo	0.45	95% Confidence Interval -1.20 to 2.10	kg
Change From Baseline in LEC Score (Total Force in kg) at Week 48	Ace-ER 6 g/Day	-1.92	95% Confidence Interval -4.49 to 0.65	kg
Change From Baseline in LEC Score (Total Force in kg) at Week 48	Placebo	-0.44	95% Confidence Interval -3.96 to 3.09	kg
Change From Baseline in GNEM FAS Mobility Domain Score at Week 48	Ace-ER 6 g/Day	-2.49	95% Confidence Interval -3.56 to -1.42	units on a scale
Change From Baseline in GNEM FAS Mobility Domain Score at Week 48	Placebo	-1.77	95% Confidence Interval -2.59 to -0.95	units on a scale
Change From Baseline in Number of Lifts in the 30 Second Weighted Arm Lift Test at Week 48	Ace-ER 6 g/Day	0.03	95% Confidence Interval -2.19 to 2.26	arm lifts
Change From Baseline in Number of Lifts in the 30 Second Weighted Arm Lift Test at Week 48	Placebo	2.79	95% Confidence Interval 0.21 to 5.38	arm lifts

1 to 10 of 18Source ClinicalTrials.gov

See all 18 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Ace-ER 6 g/Day	45
Overall Study	STARTED	Placebo	44
Overall Study	COMPLETED	Ace-ER 6 g/Day	44
Overall Study	COMPLETED	Placebo	43
Overall Study	NOT COMPLETED	Ace-ER 6 g/Day	1
Overall Study	NOT COMPLETED	Placebo	1

Source ClinicalTrials.gov

Adverse events66

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Abdominal distension	Gastrointestinal disorders	Ace-ER 6 g/Day	4	45
other	Abdominal distension	Gastrointestinal disorders	Placebo	4	44
other	Abdominal pain upper	Gastrointestinal disorders	Ace-ER 6 g/Day	10	45
other	Abdominal pain upper	Gastrointestinal disorders	Placebo	3	44
other	Diarrhoea	Gastrointestinal disorders	Ace-ER 6 g/Day	8	45
other	Diarrhoea	Gastrointestinal disorders	Placebo	6	44
other	Flatulence	Gastrointestinal disorders	Ace-ER 6 g/Day	6	45
other	Flatulence	Gastrointestinal disorders	Placebo	5	44
other	Frequent bowel movements	Gastrointestinal disorders	Ace-ER 6 g/Day	3	45
other	Frequent bowel movements	Gastrointestinal disorders	Placebo	0	44

1 to 10 of 66Source ClinicalTrials.gov

See all 66 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Ace-ER 6 g/Day	0	45
Total, all-cause mortality	Placebo	0	44
Total, other adverse events	Ace-ER 6 g/Day	38	45
Total, other adverse events	Placebo	30	44
Total, serious adverse events	Ace-ER 6 g/Day	2	45
Total, serious adverse events	Placebo	1	44

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
31036580	RESULT	PubMed
33874971	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Ace-ER 6 g/Day vs Placebo: deaths from any cause	Trial comparisons
Ace-ER 6 g/Day vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02377921

GET https://conductscience.org/api/trials/v1/NCT02377921/events?page=2

API reference For AI assistants