Trial · NCT01830972
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
NCT01830972, Phase 2, Completed, Ultragenyx Pharmaceutical Inc; results first posted 2018-03-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy. Trials, record NCT01830972. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01830972
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
- NCT number
- NCT01830972
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 59 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- GNE Myopathy, Hereditary Inclusion Body Myopathy (HIBM)
- Interventions
- SA-ER 500 mg, SA-IR 500 mg
- First posted
- 2013-04-12
- Last updated
- 2018-04-11
- Results first posted
- 2018-03-13
Source ClinicalTrials.gov
Registered outcomes6
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 TEAEs, and TEAEs Leading to Discontinuation | From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants | An adverse event (AE) is defined as any untoward medical occurrence, whether or not considered drug related. A serious AE (SAE) is an AE that at any dose results in any of the following: death, a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. TEAEs include all adverse events that start on or after the first dose of study medication, or adverse events that are present prior to the first dose of study medication, but their severity or relationship increases after the first dose of study medication up to and including 30 days after the final study medication dosing date. TEAEs were graded as mild (grade 1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). TEAE relationship to study medication was classified as not related, possibly related, or probably related. |
| Primary | Clinically Significant Changes From Baseline in Vital Signs, Physical and Neurological Examination Findings and Laboratory Evaluations | From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants | |
| Primary | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
| Primary | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
| Primary | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
| Primary | Interval History: Typical Number of Falls Per Year | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 6, 12, 18, 24, 30, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
Source ClinicalTrials.gov
Outcomes6
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 TEAEs, and TEAEs Leading to Discontinuation | From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants | All participants who received at least one dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Clinically Significant Changes From Baseline in Vital Signs, Physical and Neurological Examination Findings and Laboratory Evaluations | From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants | Clinically significant changes from Baseline for vital signs, physical and neurological examination findings and laboratory evaluations were recorded as AEs and are reported as part of the Safety section of this record and Outcome Measure 1. | ||
| PRIMARY | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | All participants who received at least one dose of study drug. Participant n at each time point includes those with a yes or no response. Naïve Participants did not enter the study until Part II. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | All participants who received at least one dose of study drug. Participant n at each time point includes those with a yes or no response. Naïve Participants did not enter the study until Part II. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | All participants who received at least one dose of study drug. Participant n at each time point includes those with a yes or no response. Naïve Participants did not enter the study until Part II. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Interval History: Typical Number of Falls Per Year | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 6, 12, 18, 24, 30, 36, study termination | All participants who received at least one dose of study drug. Naïve Participants did not enter the study until Part II. | falls | MEAN |
Source ClinicalTrials.gov
Results by group254
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 TEAEs, and TEAEs Leading to Discontinuation | Crossover Participants; 6 g/Day | >/= 1 TEAE | 43 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Crossover Participants: 12 g/Day | >/= 1 TEAE | 46 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Crossover Participants: Any Dose | >/= 1 TEAE | 46 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Naïve Participants: 6 g/Day | >/= 1 TEAE | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Naïve Participants: 12 g/Day | >/= 1 TEAE | 13 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Overall: Any Dose | >/= 1 TEAE | 59 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Crossover Participants; 6 g/Day | TEAE Maximum Severity = Grade 1 | 19 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Crossover Participants: 12 g/Day | TEAE Maximum Severity = Grade 1 | 23 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Crossover Participants: Any Dose | TEAE Maximum Severity = Grade 1 | 15 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Naïve Participants: 6 g/Day | TEAE Maximum Severity = Grade 1 | 0 | Participants |
1 to 10 of 254Source ClinicalTrials.gov
See all 254 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part I | STARTED | Crossover Participants | 46 | |
| Part I | STARTED | Naïve Participants | 0 | |
| Part I | COMPLETED | Crossover Participants | 46 | |
| Part I | COMPLETED | Naïve Participants | 0 | |
| Part I | NOT COMPLETED | Crossover Participants | 0 | |
| Part I | NOT COMPLETED | Naïve Participants | 0 | |
| Part II | STARTED | Crossover Participants | 46 | |
| Part II | STARTED | Naïve Participants | 13 | |
| Part II | COMPLETED | Crossover Participants | 44 | |
| Part II | COMPLETED | Naïve Participants | 11 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events768
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 | Anaemia | Blood and lymphatic system disorders | Crossover Participants; 6 g/Day | 1 | 46 | |
| other | Anaemia | Blood and lymphatic system disorders | Crossover Participants: 12 g/Day | 2 | 46 | |
| other | Anaemia | Blood and lymphatic system disorders | Crossover Participants: Any Dose | 3 | 46 | |
| other | Anaemia | Blood and lymphatic system disorders | Naïve Participants: 6 g/Day | 0 | 1 | |
| other | Anaemia | Blood and lymphatic system disorders | Naïve Participants: 12 g/Day | 0 | 13 | |
| other | Anaemia | Blood and lymphatic system disorders | Overall: Any Dose | 3 | 59 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Crossover Participants; 6 g/Day | 0 | 46 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Crossover Participants: 12 g/Day | 1 | 46 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Crossover Participants: Any Dose | 1 | 46 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Naïve Participants: 6 g/Day | 0 | 1 |
1 to 10 of 768Source ClinicalTrials.gov
See all 768 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Crossover Participants; 6 g/Day | 0 | 46 |
| Total, all-cause mortality | Crossover Participants: 12 g/Day | 0 | 46 |
| Total, all-cause mortality | Crossover Participants: Any Dose | 0 | 46 |
| Total, all-cause mortality | Naïve Participants: 6 g/Day | 0 | 1 |
| Total, all-cause mortality | Naïve Participants: 12 g/Day | 0 | 13 |
| Total, all-cause mortality | Overall: Any Dose | 0 | 59 |
| Total, other adverse events | Crossover Participants; 6 g/Day | 42 | 46 |
| Total, other adverse events | Crossover Participants: 12 g/Day | 44 | 46 |
| Total, other adverse events | Crossover Participants: Any Dose | 46 | 46 |
| Total, other adverse events | Naïve Participants: 6 g/Day | 1 | 1 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers0
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/NCT01830972
GET https://conductscience.org/api/trials/v1/NCT01830972/results?page=2