Trial · NCT02736188
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
NCT02736188, Phase 3, Terminated, Ultragenyx Pharmaceutical Inc; results first posted 2019-02-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM). Trials, record NCT02736188. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02736188
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
- NCT number
- NCT02736188
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 143 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy
- Interventions
- Aceneuramic Acid Extended-Release Tablets
- First posted
- 2016-04-13
- Last updated
- 2023-03-24
- Results first posted
- 2019-02-19
- Limitations and caveats
No efficacy result summaries or analyses were performed for participants rolling over from UX001-CL202 or UX001-CL203 because of the limited data from those participants due to the early study closure.
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | From first dose of study drug through the end of treatment plus 30 days (+5 days). Mean (SD) duration of treatment was 256.3 (101.54) days. | An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. An SAE or serious suspected adverse reaction is an AE or suspected adverse reaction that at any dose, in the view of either the Investigator or Ultragenyx, 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 (substantial disruption of the ability to conduct normal life functions); congenital anomaly/birth defect. TEAEs were defined as any AE that occurred after the first dose of study drug. The severity of all AEs were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.03: grade1=mild, grade 2=moderate, grade 3=severe, grade 4=life-threatening, grade 5=death. |
| Primary | Change From Baseline in HHD UEC Score Over Time | Baseline, Weeks 8, 16, 24, 48 | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. The UEC is derived from the sum of the average of the right and left total force (measured in kgf). Analyzed using a repeated measure generalized estimation equation (GEE) model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline in the GNEM-FAS Expanded Version Mobility Domain Score Over Time | Baseline, Weeks 8, 16, 24, 48 | GNEM-FAS Expanded Version Mobility subscale score has 13 items and ranges from 0 to 52 with higher scores representing greater mobility. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline on the GNEM-FAS Upper Extremity Domain Score Over Time | Baseline, Weeks 8, 16, 24, 48 | GNEM-FAS Expanded Version Upper Extremity subscale score has 9 items and ranges from 0 to 36 with higher scores representing more skilled, independent use of the arms during functional activity performance. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline in HHD Lower Extremity Composite (LEC) Score Over Time | Baseline, Weeks 8, 16, 24, and 48 | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. The LEC is derived from the sum of the average of the right and left total force (measured in kgf). Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline in the Number of Stands in the Sit-to-Stand Test Over Time | Baseline, Weeks 8, 16, 24, and 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. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline in Number of Lifts in the 30-Second Weighted Arm Lift Test Over Time | Baseline, Weeks 8, 16, 24, and 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. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline in Meters Walked in 6MWT Over Time | Baseline, Weeks 8, 16, 24, and 48 | The total distance walked (meters) in a 6-minute period was measured. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline in Percent Predicted Meters Walked in 6MWT Over Time | Baseline, Weeks 8, 16, 24, and 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. Predicted 6MWT 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. Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
| Secondary | Change From Baseline in Total Force in Knee Extensors Over Time | Baseline, Weeks 8, 16, 24, and 48 | Hand held dynamometry testing was used to measure strength. The maximum voluntary isometric contraction against a dynamometer was used to measure bilateral strength in the following muscle groups: shoulder abductors, wrist extensors and knee extensors. Specialized dynamometers for the measurement of grip and key pinch strength were also used. The total force (in kgf) for each was recorded. Bilateral total force was defined as the average of the right and left force (measured in kgf). Analyzed using a repeated measure GEE model, which includes the baseline value as a covariate. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | From first dose of study drug through the end of treatment plus 30 days (+5 days). Mean (SD) duration of treatment was 256.3 (101.54) days. | Safety Analysis Set: all participants who received at least one dose of study drug in UX001-CL302. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in HHD UEC Score Over Time | Baseline, Weeks 8, 16, 24, 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | kgf | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the GNEM-FAS Expanded Version Mobility Domain Score Over Time | Baseline, Weeks 8, 16, 24, 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline on the GNEM-FAS Upper Extremity Domain Score Over Time | Baseline, Weeks 8, 16, 24, 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in HHD Lower Extremity Composite (LEC) Score Over Time | Baseline, Weeks 8, 16, 24, and 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | kgf | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Number of Stands in the Sit-to-Stand Test Over Time | Baseline, Weeks 8, 16, 24, and 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | stands | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Number of Lifts in the 30-Second Weighted Arm Lift Test Over Time | Baseline, Weeks 8, 16, 24, and 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | lifts | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Meters Walked in 6MWT Over Time | Baseline, Weeks 8, 16, 24, and 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | meters | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Percent Predicted Meters Walked in 6MWT Over Time | Baseline, Weeks 8, 16, 24, and 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | percent of predicted distance | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Total Force in Knee Extensors Over Time | Baseline, Weeks 8, 16, 24, and 48 | Full Analysis Set: all participants in parent study UX001-CL301 (NCT02377921) with a UX001-CL302 baseline measurement and at least one post-baseline measurement in UX001-CL302. | kgf | LEAST_SQUARES_MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group86
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | Ace-ER 6 g/Day | TEAEs | 104 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | Ace-ER 6 g/Day | Serious TEAEs (SAEs) | 7 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | Ace-ER 6 g/Day | Deaths | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | Ace-ER 6 g/Day | Grade 3 or 4 TEAEs | 11 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | Ace-ER 6 g/Day | TEAEs Leading to Study Drug Discontinuation | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEs (SAEs), and Discontinuations Due to AEs | Ace-ER 6 g/Day | TEAEs Leading to Study Discontinuation | 1 | Participants | |
| Change From Baseline in HHD UEC Score Over Time | Ace-ER 6 g/Day (Parent Study Treatment: Ace-ER 6 g/Day) | Week 8 | 0.88 | 95% Confidence Interval -0.75 to 2.51 | kgf |
| Change From Baseline in HHD UEC Score Over Time | Ace-ER 6 g/Day (Parent Study Treatment: Placebo) | Week 8 | 0.09 | 95% Confidence Interval -1.05 to 1.23 | kgf |
| Change From Baseline in HHD UEC Score Over Time | Ace-ER 6 g/Day (Parent Study Treatment: Ace-ER 6 g/Day) | Week 16 | 0.10 | 95% Confidence Interval -1.47 to 1.67 | kgf |
| Change From Baseline in HHD UEC Score Over Time | Ace-ER 6 g/Day (Parent Study Treatment: Placebo) | Week 16 | -0.26 | 95% Confidence Interval -1.26 to 0.74 | kgf |
1 to 10 of 86Source ClinicalTrials.gov
See all 86 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ace-ER 6 g/Day | 142 | |
| Overall Study | COMPLETED | Ace-ER 6 g/Day | 0 | |
| Overall Study | NOT COMPLETED | Ace-ER 6 g/Day | 142 |
Source ClinicalTrials.gov
Adverse events17
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 | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Ace-ER 6 g/Day | 10 | 142 | |
| other | Flatulence | Gastrointestinal disorders | Ace-ER 6 g/Day | 9 | 142 | |
| other | Fatigue | General disorders | Ace-ER 6 g/Day | 8 | 142 | |
| other | Nasopharyngitis | Infections and infestations | Ace-ER 6 g/Day | 8 | 142 | |
| other | Fall | Injury, poisoning and procedural complications | Ace-ER 6 g/Day | 30 | 142 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Ace-ER 6 g/Day | 17 | 142 | |
| other | Back pain | Musculoskeletal and connective tissue disorders | Ace-ER 6 g/Day | 13 | 142 | |
| other | Musculoskeletal pain | Musculoskeletal and connective tissue disorders | Ace-ER 6 g/Day | 11 | 142 | |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | Ace-ER 6 g/Day | 10 | 142 | |
| other | Headache | Nervous system disorders | Ace-ER 6 g/Day | 8 | 142 |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ace-ER 6 g/Day | 0 | 142 |
| Total, other adverse events | Ace-ER 6 g/Day | 66 | 142 |
| Total, serious adverse events | Ace-ER 6 g/Day | 7 | 142 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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/NCT02736188
GET https://conductscience.org/api/trials/v1/NCT02736188/results?page=2