This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for 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), a non-randomized phase 2 study. The primary outcome is Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01830972 |
| Phase | Phase 2 |
| Condition | GNE Myopathy, Hereditary Inclusion Body Myopathy (HIBM) |
| Allocation | Non-Randomized |
| Design model | Crossover |
| Masking | None (open label) |
| Enrollment | 59 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Ultragenyx Pharmaceutical Inc |
| Results posted | Yes |
2. Clinical Question
Population
Participants with GNE myopathy or hereditary inclusion body myopathy were enrolled in this Phase 2 extension study. Crossover participants had completed the 48-week study UX001-CL201; treatment-naïve participants with GNE myopathy were also enrolled. The primary safety outcome population was participants who received at least one dose of study drug.
Intervention
The study evaluated sialic acid extended-release tablets (SA-ER) and immediate-release capsules (SA-IR). The planned treatment descriptions include 6 g/day and 12 g/day regimens. For the 12 g/day regimen, the record describes 1.5 g of SA-ER and 1.5 g of SA-IR taken 4 times per day in relevant parts of the study.
Comparator
There was no randomized control group. The record describes different treatment periods and dose groups, including participants who continued from an earlier study and treatment-naïve participants. These groups are not a like-for-like control comparison, and the crossover design means some participants contributed experience at more than one dose.
Primary question
The primary safety question was how many participants experienced treatment-emergent adverse events, serious treatment-emergent adverse events, or treatment-emergent adverse events leading to discontinuation during treatment and follow-up. Treatment-emergent events were captured from the first dose through up to 30 days after the last dose. The record reports event counts for the overall any-dose group and several dose groups, but does not report a count for events leading to discontinuation.
3. Trial Design
This was a completed, open-label, non-randomized Phase 2 extension study with a crossover model. The record gives enrollment as 59. Participants and study personnel were not masked to treatment, and allocation was not randomized. The study was sponsored by Ultragenyx Pharmaceutical Inc. Its start date was 2013-06-04 and its primary completion date was 2017-02-14.
The record distinguishes crossover participants from treatment-naïve participants. Crossover participants had completed the 48-week UX001-CL201 study and entered Part I, in which they continued 6 g/day SA-ER for 12 weeks. The description then specifies 12 g/day SA, combining SA-ER and SA-IR, for 36 months in Part II, followed by Part III at 6 g/day. The record does not give further detail about the crossover participants’ Part III regimen in that description.
Treatment-naïve participants with GNE myopathy entered Part II at 12 g/day SA for 36 months. Their later parts included 6 g/day or 12 g/day SA using both SA-ER and SA-IR in Part III, and 6 g/day or 12 g/day SA-ER only in Part IV. These descriptions identify treatment sequences, not randomized parallel groups. The numbers of participants in every treatment sequence are not reported in the registry record apart from the outcome group counts.
The primary safety outcome was assessed from first dose through up to 30 days after last dose. Mean (SD) treatment duration was 1170.0 (170.2) days for crossover participants and 897 (380) days for treatment-naïve participants. The study’s open-label, non-randomized structure and differing treatment histories are important when interpreting dose-group counts: they do not establish a controlled comparison of regimens.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: A total of 46 crossover participants started the study in Part 1, with an additional 13 naive participants starting the study in Part 2.
Part I
| Stage | Crossover Participants | Naïve Participants |
|---|---|---|
| STARTED | 46 | 0 |
| COMPLETED | 46 | 0 |
| NOT COMPLETED | 0 | 0 |
Part II
| Stage | Crossover Participants | Naïve Participants |
|---|---|---|
| STARTED | 46 | 13 |
| COMPLETED | 44 | 11 |
| NOT COMPLETED | 2 | 2 |
Reasons for not completing this period:
| Reason | Crossover Participants | Naïve Participants |
|---|---|---|
| Withdrawal by Subject | 2 | 2 |
Part III
| Stage | Crossover Participants | Naïve Participants |
|---|---|---|
| STARTED | 44 | 11 |
| COMPLETED | 42 | 11 |
| NOT COMPLETED | 2 | 0 |
Reasons for not completing this period:
| Reason | Crossover Participants | Naïve Participants |
|---|---|---|
| Withdrawal by Subject | 1 | 0 |
| Noncompliance | 1 | 0 |
Part IV
| Stage | Crossover Participants | Naïve Participants |
|---|---|---|
| STARTED | 42 | 11 |
| COMPLETED | 39 | 10 |
| NOT COMPLETED | 3 | 1 |
Reasons for not completing this period:
| Reason | Crossover Participants | Naïve Participants |
|---|---|---|
| Withdrawal by Subject | 2 | 1 |
| Non-Compliance | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: All participants who received at least one dose of study drug.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: Baseline at the start of Part II (upon study entry for Naïve Participants) is presented.
| Characteristic | Unit | Crossover Participants | Naïve Participants | Total |
|---|---|---|---|---|
| Participants analysed | 46 | 13 | 59 | |
| Age, Continuous | years | 41.1 (SD 10.55) | 35.6 (SD 10.97) | 39.9 (SD 10.79) |
| Sex: Female, Male: Female | Participants | 29 | 8 | 37 |
| Sex: Female, Male: Male | Participants | 17 | 5 | 22 |
| Race/Ethnicity, Customized: White | Participants | 38 | 13 | 51 |
| Race/Ethnicity, Customized: Asian | Participants | 8 | 0 | 8 |
| Race/Ethnicity, Customized: Not Hispanic/Latino | Participants | 44 | 13 | 57 |
| Race/Ethnicity, Customized: Hispanic/Latino | Participants | 2 | 0 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation, measured over 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.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome counts participants with treatment-emergent adverse events (TEAEs), serious TEAEs, and TEAEs leading to discontinuation. An adverse event is defined in the record as an untoward medical occurrence, whether or not considered related to treatment. A TEAE is an event that begins on or after the first dose, or a pre-existing event that worsens in severity or relationship after the first dose. The observation window extends through up to 30 days after the final dose.
The serious-event definition includes events resulting in death, a life-threatening event, inpatient hospitalization or its prolongation, persistent or significant incapacity or substantial disruption of normal life functions, or a congenital anomaly or birth defect. TEAEs were graded from mild through death, and their relationship to study medication was classified as not related, possibly related, or probably related.
Counting participants with events is useful for describing how many people experienced safety events during treatment. It is not the same as counting every event, because one participant can have more than one event. Nor does a participant count alone describe the length of exposure or the timing and recurrence of events. The registry record also names discontinuation due to TEAEs as part of the primary outcome, but a numerical discontinuation result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
For the overall any-dose group, the registry reports 59 participants at risk for the TEAE outcome and 59 with a TEAE. It reports 3 participants with a serious TEAE and 0 deaths. These are descriptive participant counts over the study’s stated safety observation period, not estimates from a randomized treatment comparison. The count of participants with TEAEs leading to discontinuation is not reported in the registry record.
The dose-group counts provide further description but should not be read as evidence that one dose is safer than another. Among crossover participants, the record lists 46 at risk in each of the 6 g/day, 12 g/day and any-dose groups. Serious TEAEs were recorded for 2 participants in the 6 g/day group, 0 in the 12 g/day group and 2 in the any-dose group; TEAEs were recorded for 42, 44 and 46 participants in those groups, respectively. The groups reflect treatment periods in a crossover study, not independent randomized arms. A participant may contribute experience across more than one dose period.
Among treatment-naïve participants, the record lists 1 participant at risk in the 6 g/day group and 13 at risk in the 12 g/day group. There were 0 serious TEAEs and 1 TEAE in the 6 g/day group, and 1 serious TEAE and 13 TEAEs in the 12 g/day group. The very different group sizes, especially the 6 g/day group, make direct numerical contrasts particularly difficult to interpret. The registry does not report a randomized comparison or an adjusted estimate for these groups.
Mean treatment duration differed between the crossover and treatment-naïve populations: 1170.0 (170.2) days and 897 (380) days, respectively. Participant counts do not account for time exposed, so the overall counts cannot be treated as rates per unit of treatment time. The record does not report exposure-adjusted event rates or confidence intervals for these safety outcomes. General statistical interpretation: uncertainty is important when comparing event counts, especially when groups are small, exposure differs, and treatment assignment is not randomized.
The serious-event terms listed in the record include Femur fracture at 7.7%, Chest pain at 2.2%, Quadriparesis at 2.2%, and Deep vein thrombosis at 2.2%. The record does not identify the denominator or provide dose-specific attribution for these listed percentages, so they should be read as registry-listed terms and percentages rather than as a basis for comparing treatment groups. Overall, the posted results describe reported safety experience; they do not establish a causal difference between doses or provide a treatment efficacy conclusion.
9. Statistical Methods Explained
The primary outcome is a binary participant-level measure: whether a participant experienced an event in a specified category during the safety observation period. The registry reports counts of participants with events and counts at risk. General statistical explanation: for a binary outcome, a participant is counted as having the outcome if the event criterion is met; the count is distinct from the number of event episodes. One person with multiple events may still count once in a participant-level result.
What does a participant count capture?
A participant count gives a direct description of how many people had at least one event of interest. It is useful for communicating the frequency of participants affected in the study population. However, it does not describe how many total event episodes occurred, how long events lasted, how severe every event was, or when events occurred. Those details would require additional event-level results. Although the registry defines TEAE severity grades and treatment relationship categories, detailed grade-specific or relationship-specific counts are not reported in the registry record.
Counts also depend on the population and observation period. The primary outcome population is all participants who received at least one dose of study drug. The outcome window began at first dose and continued through up to 30 days after the last dose. Participants with longer exposure have more time in which an event may be observed. The reported mean (SD) duration was 1170.0 (170.2) days for crossover participants and 897 (380) days for treatment-naïve participants. General statistical explanation: where exposure time varies, raw participant counts do not by themselves adjust for differing opportunity to experience an event. An exposure-adjusted rate would answer a different question, but such a rate is not reported in the registry record.
Why is the design relevant to comparisons?
The study was non-randomized and open label, with a crossover model. General statistical explanation: random assignment can help balance known and unknown factors between groups, while a non-randomized comparison may reflect differences in participant characteristics, treatment history, or clinical decisions as well as treatment. In a crossover study, the same participant may receive more than one regimen at different times. Outcomes across dose periods therefore are not necessarily independent observations, and the groups cannot automatically be interpreted as separate sets of participants.
The record explicitly cautions that this is not a randomized comparison and that the arms are not a like-for-like control comparison. It does not report an adjusted statistical comparison that accounts for repeated observations or differences in treatment history. The dose-group counts are best understood as descriptive registry results, not estimates of a causal treatment effect.
How should uncertainty be understood?
General statistical explanation: a confidence interval describes a range of values compatible with the data under a specified statistical method. It helps show how precisely an effect or event frequency has been estimated. Confidence intervals for the primary safety outcomes are not reported in the registry record. Without them, the numerical counts still describe what was recorded, but they do not communicate the precision of an estimated difference or rate.
Small groups can yield especially imprecise comparisons. The treatment-naïve 6 g/day group has 1 participant at risk, while the treatment-naïve 12 g/day group has 13. These counts do not support a stable comparison of group-level event frequencies, and no inferential comparison for those groups is reported. Similarly, seeing 0 serious TEAEs in a group does not demonstrate that the underlying risk is zero; it means no participant in that group was recorded with the outcome in the posted results.
What do the planning illustrations represent?
The sample-size illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They describe planning for a stated fraction of the observed effect. The record identifies these as planning illustrations computed from the observed effect, not as the trial’s own design. They are not observed power, and they should not be interpreted as a feature of the original study’s enrollment plan. Numerical planning values are not reported in the registry record. The trial’s sample-size justification and statistical planning rationale are also not reported in the registry record.
The record gives no analyses for the primary outcome beyond the posted counts. It does not report a formal hypothesis test, adjusted effect estimate, confidence interval, or exposure-adjusted analysis. In this setting, descriptive interpretation is more appropriate than treating the dose groups as if they were a randomized efficacy or safety comparison.
10. Multiplicity, Missing Data and Analysis Populations
The registry record does not report a statistical plan for handling multiple outcomes or multiple comparisons. The primary outcome names three safety categories: TEAEs, serious TEAEs, and TEAEs leading to discontinuation. The record does not specify whether these were tested as separate hypotheses, whether any adjustment for multiplicity was planned, or how other safety summaries were prioritized. No inferential analyses are reported for these outcomes in the registry record.
Missing-data methods are not reported in the registry record. It does not describe whether participants who stopped treatment early were followed for safety, how incomplete follow-up was handled, or whether any imputation was used. The posted primary outcome population is participants who received at least one dose, but details about exclusions, withdrawals, and outcome-specific missingness are not reported in the registry record. A count at risk should therefore be interpreted as the registry’s stated denominator for that group, not as evidence about the completeness of every participant’s follow-up.
The record does not report interim analyses, a stopping rule, or a sample-size justification. These omissions prevent assessment of whether the study used planned monitoring or prespecified decision thresholds. The sample-size illustrations associated with the posted material are planning illustrations based on observed effects; they do not replace a prospectively specified design rationale and do not constitute observed power.
There is also no reported adjustment for the non-randomized allocation, crossover treatment history, or differing exposure durations. The group counts should consequently remain descriptive. They do not support a multiplicity-adjusted or covariate-adjusted claim about differences between treatment regimens.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Crossover Participants; 6 g/Day | 2 | 46 | 4.3% |
| Crossover Participants: 12 g/Day | 0 | 46 | 0.0% |
| Crossover Participants: Any Dose | 2 | 46 | 4.3% |
| Naïve Participants: 6 g/Day | 0 | 1 | 0.0% |
| Naïve Participants: 12 g/Day | 1 | 13 | 7.7% |
| Overall: Any Dose | 3 | 59 | 5.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Crossover Participants; 6 g/Day | 0 of 46 |
| Crossover Participants: 12 g/Day | 0 of 46 |
| Crossover Participants: Any Dose | 0 of 46 |
| Naïve Participants: 6 g/Day | 0 of 1 |
| Naïve Participants: 12 g/Day | 0 of 13 |
| Overall: Any Dose | 0 of 59 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Crossover Participants; 6 g/Day | 42 of 46 |
| Crossover Participants: 12 g/Day | 44 of 46 |
| Crossover Participants: Any Dose | 46 of 46 |
| Naïve Participants: 6 g/Day | 1 of 1 |
| Naïve Participants: 12 g/Day | 13 of 13 |
| Overall: Any Dose | 59 of 59 |
Most frequent serious adverse event terms
| Event term | Organ system | Crossover Participants; 6 g/Day | Crossover Participants: 12 g/Day | Crossover Participants: Any Dose | Naïve Participants: 6 g/Day | Naïve Participants: 12 g/Day | Overall: Any Dose |
|---|---|---|---|---|---|---|---|
| Chest pain | General disorders | 1 of 46 | 0 of 46 | 1 of 46 | 0 of 1 | 0 of 13 | 1 of 59 |
| Quadriparesis | Nervous system disorders | 1 of 46 | 0 of 46 | 1 of 46 | 0 of 1 | 0 of 13 | 1 of 59 |
| Deep vein thrombosis | Vascular disorders | 1 of 46 | 0 of 46 | 1 of 46 | 0 of 1 | 0 of 13 | 1 of 59 |
| Femur fracture | Injury, poisoning and procedural complications | 0 of 46 | 0 of 46 | 0 of 46 | 0 of 1 | 1 of 13 | 1 of 59 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Crossover Participants; 6 g/Day | Crossover Participants: 12 g/Day | Crossover Participants: Any Dose | Naïve Participants: 6 g/Day | Naïve Participants: 12 g/Day | Overall: Any Dose |
|---|---|---|---|---|---|---|---|
| Flatulence | Gastrointestinal disorders | 8 of 46 | 24 of 46 | 27 of 46 | 0 of 1 | 11 of 13 | 38 of 59 |
| Headache | Nervous system disorders | 11 of 46 | 16 of 46 | 21 of 46 | 0 of 1 | 6 of 13 | 27 of 59 |
| Procedural pain | Injury, poisoning and procedural complications | 25 of 46 | 0 of 46 | 25 of 46 | 0 of 1 | 2 of 13 | 27 of 59 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 9 of 46 | 10 of 46 | 17 of 46 | 0 of 1 | 8 of 13 | 25 of 59 |
| Influenza like illness | General disorders | 9 of 46 | 13 of 46 | 17 of 46 | 0 of 1 | 6 of 13 | 23 of 59 |
| Back pain | Musculoskeletal and connective tissue disorders | 9 of 46 | 12 of 46 | 17 of 46 | 0 of 1 | 3 of 13 | 20 of 59 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 7 of 46 | 8 of 46 | 13 of 46 | 1 of 1 | 7 of 13 | 20 of 59 |
| Diarrhoea | Gastrointestinal disorders | 6 of 46 | 11 of 46 | 13 of 46 | 1 of 1 | 5 of 13 | 18 of 59 |
| Dyspepsia | Gastrointestinal disorders | 3 of 46 | 13 of 46 | 14 of 46 | 0 of 1 | 3 of 13 | 17 of 59 |
| Cough | Respiratory, thoracic and mediastinal disorders | 7 of 46 | 10 of 46 | 15 of 46 | 0 of 1 | 1 of 13 | 16 of 59 |
Serious events with the highest arm-level rate: femur fracture (7.7%), chest pain (2.2%), quadriparesis (2.2%), deep vein thrombosis (2.2%).
The overall any-dose results report 3 participants with serious TEAEs among 59 at risk, 59 participants with a TEAE among 59 at risk, and 0 deaths. In the crossover groups, serious TEAEs were reported for 2 participants at 6 g/day, 0 at 12 g/day and 2 at any dose, with 46 at risk in each group. In the treatment-naïve groups, serious TEAEs were reported for 0 of 1 at 6 g/day and 1 of 13 at 12 g/day. These are participant counts; they do not indicate the total number of serious event episodes.
The listed serious-event terms are Femur fracture (7.7%), Chest pain (2.2%), Quadriparesis (2.2%), and Deep vein thrombosis (2.2%). Their denominator and dose attribution are not reported in the registry record. The percentages should not be used to reconstruct unreported counts or compare regimens. The open-label, non-randomized crossover design and differing treatment durations also limit dose-group comparisons. No count for TEAEs leading to discontinuation is reported in the registry record.
12. What the Result Does, and Does Not, Mean
The posted results describe safety events recorded during a long-term extension study of SA-ER and SA-IR regimens in participants with GNE myopathy or hereditary inclusion body myopathy. They report that TEAEs were recorded for 59 participants in the overall any-dose group, serious TEAEs for 3 participants, and deaths for 0 participants. They also provide counts for the listed dose groups and define how treatment-emergent and serious events were classified.
These results do not establish that a regimen caused an event, nor do they establish that one dose has a different safety profile from another. Participants were not randomized, the study was open label, and crossover participants had treatment experience across periods. The groups therefore are not independent randomized comparison arms. Differing treatment duration further complicates interpretation of raw counts, and the record does not report exposure-adjusted rates or inferential comparisons.
The findings also do not quantify treatment efficacy. Although the study description refers to safety and efficacy, the primary outcome reported in the record is a safety endpoint. Efficacy results are not reported in the registry record. The numerical count for TEAEs leading to discontinuation is also not reported in the registry record. Lack of a reported result should not be interpreted as a result of zero events.
General statistical explanation: safety findings from an extension study can contribute descriptive information about events during observed treatment, but causal conclusions require a design and analysis capable of distinguishing treatment effects from other explanations. The counts are most appropriately read as a record of observed participant-level safety outcomes in this study, with uncertainty and design limitations kept in view.
13. Why This Trial Matters Statistically
This study illustrates why the structure behind a safety count matters as much as the count itself. The overall result is straightforward to describe: the registry reports 59 participants with a TEAE, 3 with a serious TEAE and 0 deaths in the any-dose group. But those counts arose in an open-label, non-randomized extension with crossover treatment periods, not in a randomized comparison with a control group.
The record also shows how exposure and treatment history affect interpretation. Crossover and treatment-naïve participants had different mean treatment durations, and the dose groups include different numbers of participants. In particular, the treatment-naïve 6 g/day group has only 1 participant at risk. A numerical contrast between those groups would conflate dose with group size, treatment history and the opportunity to observe events. This is why a participant count should not be mistaken for an exposure-adjusted risk estimate or a causal effect.
The registry’s event definitions are also statistically important. TEAEs include new events and certain worsening pre-existing events during a defined window, while serious events meet additional criteria. These definitions determine what is counted. They do not, by themselves, show event frequency over time, recurrence, or treatment attribution. The record classifies relationship to study medication but does not report detailed counts by that classification.
Finally, the record makes clear that planning illustrations derived from observed effects are separate from the study’s prospective design. They do not supply missing evidence about sample-size justification, statistical analysis plans, or uncertainty around the posted counts. This trial is a useful example of interpreting descriptive safety results in light of study design, exposure, group size and reporting completeness.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation for interpretation is the non-randomized, open-label crossover design. The registry itself cautions that its arms are not a like-for-like control comparison. Differences in event counts across dose groups therefore cannot be attributed to dose alone. Prior treatment and period-specific exposure may also differ, while an adjusted analysis accounting for those features is not reported in the registry record.
Group sizes are uneven, including a treatment-naïve 6 g/day group with 1 participant at risk. Such a small group offers very limited descriptive information for comparing safety outcomes. The record also reports different mean (SD) treatment durations for crossover and treatment-naïve participants, while the posted counts are not exposure-adjusted. The same participant may contribute to more than one crossover treatment group, so those group counts should not be treated as independent samples.
Reporting is incomplete for several questions readers may have. The numerical result for TEAEs leading to discontinuation, detailed event grades, treatment relationships, missing-data handling, confidence intervals, formal comparisons, multiplicity procedures, and efficacy results are not reported in the registry record. For the listed serious-event percentages, the denominator and dose-specific attribution are not reported in the registry record. The sample-size justification and prespecified statistical analysis plan are not reported in the registry record either.
These limitations do not negate the descriptive counts, but they constrain what can be inferred from them. The results characterize registry-reported event experience; they do not provide a controlled estimate of relative safety or a numerical efficacy conclusion.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: 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.
- Registry record last updated 2018-04-11; status last verified 2018-03.
- Results first posted 2018-03-13.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT01830972 is a completed, open-label, non-randomized Phase 2 extension study with a crossover model and enrollment of 59. It evaluated SA-ER tablets and SA-IR capsules in participants with GNE myopathy or hereditary inclusion body myopathy, including crossover participants from UX001-CL201 and treatment-naïve participants with GNE myopathy. The study began on 2013-06-04 and had primary completion on 2017-02-14.
The primary outcome counts participants with TEAEs, serious TEAEs, and TEAEs leading to discontinuation. In the overall any-dose group, the registry reports 59 participants with a TEAE, 3 with a serious TEAE, and 0 deaths. A numerical result for TEAEs leading to discontinuation is not reported in the registry record. The design and unequal group sizes mean that dose-group counts are descriptive rather than a randomized comparison.