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 Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy (NCT02858908), a non-randomized phase 2 study. The primary outcome is Safety (Adverse Events).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02858908 |
| Phase | Phase 2 |
| Condition | Myotonic Dystrophy 1 |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | Single |
| Enrollment | 16 |
| Arms in the primary outcome | n/a |
| Lead sponsor | AMO Pharma Limited |
| Results posted | Yes |
2. Clinical Question
Population
Adolescents and adults with congenital and juvenile-onset myotonic dystrophy were the intended population. The condition is listed as Myotonic Dystrophy 1. The registry record reports enrollment of 16 participants. Age limits, diagnostic criteria, and other eligibility details are not reported in the registry record.
Intervention
Participants received tideglusib orally once daily. Cohort 1 received 1000 mg, and Cohort 2 received 400 mg. The study also set out to investigate tideglusib and its primary metabolite pharmacokinetics.
Comparator
The two dose cohorts are not randomized control groups. Allocation is listed as non-randomized and the study model as sequential. The registry record does not identify a placebo group or an untreated control group, and the cohorts should not be read as a like-for-like control comparison.
Primary question
The primary endpoint concerns safety: the incidence of adverse events, including serious adverse events, from baseline to the end of the study, with a stated timeframe of 12 weeks. The primary outcome is the count of participants with events in the Safety Analysis Set. The record also lists functional, respiratory, imaging, clinical-impression, and pharmacokinetic secondary outcomes.
The central registry question is therefore descriptive: what adverse events were recorded among participants in each dose cohort during the stated observation period? The posted results report serious adverse events and other adverse events by cohort. They do not establish a randomized comparison of doses, and the registry record does not report an inferential test that would quantify evidence for a difference between cohorts. Because dose groups were sequentially assigned, observed contrasts may reflect differences between the cohorts as well as dose. The record does not report enough detail to assess whether the groups were comparable at baseline or whether differences in observation or follow-up affected event reporting.
3. Trial Design
This was a phase 2, single-blind, non-randomized study sponsored by AMO Pharma Limited. It enrolled 16 participants with Myotonic Dystrophy 1. The record gives a study start date of 2016-07-20, a primary completion date of 2018-01, and a completed status. The study title describes adolescent and adult participants with congenital and juvenile-onset disease.
Two sequential cohorts received oral tideglusib once daily: Cohort 1 received 1000 mg and Cohort 2 received 400 mg. Each cohort had 8 participants at risk for serious adverse events and 8 at risk for other adverse events in the posted safety results. Allocation is explicitly identified as non-randomized. The record does not report the sequence or timing of cohort enrollment in more detail, whether dose assignment followed a prespecified decision rule, or which parties were blinded. It also does not describe any placebo or active-control arm.
The primary outcome was safety, measured as the count of participants with adverse events, including serious adverse events, between baseline and the end of study. Its timeframe is listed as 12 weeks, and its analysis population is the Safety Analysis Set. The registry lists 20 outcome measures and 26 analyses. Secondary outcomes include plasma concentration and blood pharmacokinetics of tideglusib, area under the plasma concentration versus time curve, a 10 Metre Walk/Run Test, grip strength and muscle relaxation time assessed with a computerised handgrip myometer, respiratory forced vital capacity, dual-energy X-ray absorptiometry, and Clinical Global Impressions-Severity.
The record does not report a protocol-level sample-size justification or the rationale for the cohort sizes. It describes planning illustrations based on an observed effect, but those are not the trial's own design justification. The registry record also does not report the detailed statistical analysis plan, inclusion and exclusion criteria, or a more detailed schedule of assessments.
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: Subjects were to be male or female aged 12 to 45 years with a diagnosis of genetically confirmed congenital or juvenile-onset type 1 myotonic dystrophy. Subjects were to have a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2) and were to be ambulatory and able to complete the 10-metre walk/run test.
Overall Study
| Stage | Cohort 1 | Cohort 2 |
|---|---|---|
| STARTED | 8 | 8 |
| COMPLETED | 8 | 8 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety Analysis Set
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.
| Characteristic | Unit | Cohort 1 | Cohort 2 | Total |
|---|---|---|---|---|
| Participants analysed | 8 | 8 | 16 | |
| Age, Customized: Mean | years | 21.8 (SD 5.5) | 20.3 (SD 6.36) | 21 (SD 5.8) |
| Sex: Female, Male: Female | Participants | 5 | 1 | 6 |
| Sex: Female, Male: Male | Participants | 3 | 7 | 10 |
| Race/Ethnicity, Customized: Not Hispanic or latino | Participants | 8 | 8 | 16 |
| Race/Ethnicity, Customized: White | Participants | 8 | 7 | 15 |
| Race/Ethnicity, Customized: Asian | Participants | 0 | 1 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Safety (Adverse Events), measured over 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is safety, expressed as the count of participants who experienced adverse events, including serious adverse events. The stated assessment runs from baseline to the end of study, with a timeframe of 12 weeks. The analysis population is the Safety Analysis Set. In the posted results, the counts are separated into serious adverse events and other adverse events for each dose cohort.
Counting participants with an event is a way to describe how many people experienced at least one event in the relevant category. It does not, by itself, describe how many separate events each participant had, how long events lasted, their severity, or whether they were judged related to treatment. Those details are not reported in the registry record. The event count also does not establish a cause: an adverse event occurring during a study is not automatically an event caused by the study intervention.
Safety is used as the primary outcome because the stated study purpose includes determining whether tideglusib is safe in the target population. In general, safety outcomes can describe the types and frequency of events observed during a specified follow-up period. Their interpretation depends on how events were defined, solicited, classified, and followed. Those operational details are not reported in the registry record. As the study was non-randomized and had no reported control group, these counts describe the experience of the enrolled cohorts; they do not isolate what would have occurred without tideglusib.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety results report no serious adverse events among 8 participants in Cohort 1 and none among 8 participants in Cohort 2. Deaths are recorded as 0 in each cohort. Other adverse events were recorded for 8 of 8 participants in Cohort 1 and 6 of 8 participants in Cohort 2. These are descriptive counts within the reported safety population.
The registry record does not report confidence intervals, hypothesis tests, adjusted estimates, or a statistical comparison between the cohorts for this endpoint. Accordingly, the observed counts should not be treated as evidence that the doses differ, or that their safety profiles are equivalent. A difference in observed counts may occur by chance, may reflect differences between the sequential cohorts, or may be affected by how events were collected and reported. The non-randomized allocation is especially relevant: the cohorts are not a randomized dose comparison and are not like-for-like control groups.
Zero recorded serious adverse events in a cohort means that no participant in that cohort was recorded as having a serious event during the defined observation period. It does not demonstrate that such events cannot occur or that the underlying risk is zero. The amount of information about uncommon events is limited when the recorded cohort size is small. This general statistical point applies regardless of whether the observed count is zero. The record does not provide event-level details, severity grades beyond the serious/non-serious distinction, timing, duration, treatment relatedness, or the method used to solicit and adjudicate events. The absence of these details limits interpretation of the event counts.
Other adverse events were recorded for all participants in Cohort 1 and for 6 participants in Cohort 2. These counts do not reveal the number of events per participant or whether participants experienced similar kinds of events. They also do not establish a dose-response pattern. Cohort order, participant characteristics, exposure, and ascertainment could all be relevant, but the registry record does not provide enough information to assess them.
The primary endpoint is a safety measure, while the registry also lists pharmacokinetic and functional, respiratory, imaging, and clinical-impression outcomes. No numerical results for those secondary outcomes are included in the registry record for this interpretation. Their direction, precision, and relationship to the safety counts therefore cannot be assessed. Overall, the results support a limited description of recorded adverse events in these two cohorts over the stated timeframe, not a causal or comparative conclusion about dose or treatment effect.
9. Statistical Methods Explained
What does a participant count represent?
The primary outcome is a count of participants with adverse events, not a count of all event episodes. If a participant experiences more than one event, a participant-level count may still include that person only once in the relevant category. The posted results give the number at risk and the number affected for serious events and other events in each cohort. They do not give individual event histories or the number of episodes. This distinction matters because participant counts describe how widespread an event category was across people, whereas episode counts also reflect repeated events in the same person.
For safety outcomes, the event definition and collection process shape what the count means. General statistical interpretation depends on whether events were actively solicited or spontaneously reported, how serious events were defined, how duplicate reports were handled, and whether events were assessed for relatedness. These details are not reported in the registry record. The listed endpoint covers the period between baseline and end of study, with a stated timeframe of 12 weeks; event counts should be understood within that specified observation window rather than as lifetime experience.
Why does the non-randomized design matter?
In a randomized comparison, random assignment is intended to make treatment groups comparable on average, including on characteristics that may affect outcomes. This is a general principle, not a claim about any unreported feature of this study. Here, allocation is listed as non-randomized and the model as sequential. The dose cohorts may therefore differ for reasons other than dose. Without random allocation or a reported control group, a numerical contrast between cohorts cannot by itself identify a causal dose effect.
Single-blind describes the masking status recorded for the study, but the registry record does not say who was blinded or how masking was maintained. That information is important for interpreting outcomes affected by participant or investigator expectations, as well as the reporting and assessment of adverse events. The record does not describe concealment, blinding procedures, or whether masking could have been broken.
How should uncertainty be understood?
A confidence interval is a statistical range that expresses the uncertainty around an estimated quantity under specified assumptions. In general, a narrow interval indicates greater precision, while a wide interval indicates less precision. No confidence intervals are reported for the primary safety counts in the registry information. Therefore, the precision of any estimated event frequency cannot be read from a reported interval, and no additional interval should be inferred from the counts alone.
When counts are small, a change in the experience of a few participants can materially alter the descriptive picture. This is a general property of small samples. It is particularly relevant to serious adverse events: zero observed events does not rule out an underlying possibility of such events. The record gives the cohort counts but does not report a model for rare events, a formal uncertainty analysis, or a prespecified test of differences between cohorts.
What do the planning illustrations mean?
The registry record describes sample-size planning illustrations computed from an observed effect, rather than the trial's original design. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is specified as a fraction of the observed effect. Such illustrations are hypothetical planning calculations. They are not observed power, do not demonstrate that the completed study had a particular probability of detecting an effect, and do not replace a protocol's design rationale. The protocol's sample-size justification is not reported in the registry record.
General power calculations depend on assumptions about the outcome, effect size, variability, significance criterion, and allocation. When an effect is taken from observed results, the planning calculation is conditional on that estimate and does not make the estimate more reliable. In this study, the registry entries do not include numeric planning results, so no sample-size values or power conclusions can be stated. Any planning display should be read as an illustration under its stated assumptions, not as evidence about the outcome or as a report of the trial's original planning.
What analysis details are absent?
The registry identifies the primary population as the Safety Analysis Set, but it does not define membership in that set or report the numbers analyzed for every outcome. It does not describe the statistical tests or models used, any covariate adjustment, handling of missing outcome data, or whether analyses followed a prespecified hierarchy. It also does not report whether the sponsor conducted interim analyses. These omissions mean that the posted safety counts can be described, but the detailed analytical route from collected data to results cannot be reconstructed.
Over-dispersion is a general issue for count outcomes when variability exceeds what a chosen model expects; a model that ignores it may understate uncertainty. It is not possible to determine whether over-dispersion is relevant to these participant-level binary counts without a reported model and analysis plan. More broadly, a statistical method can only support conclusions that its design and assumptions permit. The registry record's non-randomized structure and limited reporting call for a descriptive reading rather than a causal comparison.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 20 outcome measures and 26 analyses. It does not report a multiplicity strategy, such as a prespecified hierarchy, family-wise error control, or another adjustment for testing multiple outcomes. When many outcomes or analyses are considered, general statistical practice recognizes that the chance of at least one apparently notable result can increase if each is interpreted separately. An adjustment or prespecified prioritization can address some of that issue, but no such approach is reported in the registry record. The primary endpoint is identified as safety, but the relationship between the listed analyses and any formal testing plan is not described.
Missing-data handling is not reported in the registry record. The record does not state whether all enrolled participants completed the 12-week assessment, how incomplete follow-up was treated, or whether participants with missing safety information remained in the Safety Analysis Set. The listed denominators for the safety results are 8 participants in each cohort, but these counts alone do not describe the completeness of follow-up or missingness at individual visits.
The primary analysis population is named as the Safety Analysis Set; its definition and any additional analysis populations are not reported in the registry record. It is not possible to determine whether participants were analyzed according to assigned cohort, actual exposure, or another rule. The registry also does not report an analysis plan for secondary outcomes, sensitivity analyses, or interim analyses. These gaps limit assessment of the robustness and multiplicity-adjusted interpretation of results, particularly for outcomes beyond the descriptive safety counts.
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 |
|---|---|---|---|
| Cohort 1 | 0 | 8 | 0.0% |
| Cohort 2 | 0 | 8 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Cohort 1 | 0 of 8 |
| Cohort 2 | 0 of 8 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Cohort 1 | 8 of 8 |
| Cohort 2 | 6 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cohort 1 | Cohort 2 |
|---|---|---|---|
| Nasopharyngitis | Infections and infestations | 2 of 8 | 3 of 8 |
| Vomiting | Gastrointestinal disorders | 1 of 8 | 2 of 8 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 8 | 1 of 8 |
| Diarrhea | Gastrointestinal disorders | 2 of 8 | 0 of 8 |
| Lower respiratory tract infection | Infections and infestations | 1 of 8 | 1 of 8 |
| Back injury | Injury, poisoning and procedural complications | 1 of 8 | 0 of 8 |
| Fall | Injury, poisoning and procedural complications | 1 of 8 | 0 of 8 |
| Joint dislocation | Injury, poisoning and procedural complications | 1 of 8 | 0 of 8 |
| Laceration | Injury, poisoning and procedural complications | 1 of 8 | 0 of 8 |
| Limb injury | Injury, poisoning and procedural complications | 0 of 8 | 1 of 8 |
For serious adverse events, the posted results record 0 affected participants among 8 at risk in Cohort 1 and 0 among 8 at risk in Cohort 2. Deaths are recorded as 0 in both cohorts. These are observed counts over the study's stated safety timeframe; they are not proof that serious events or deaths are impossible, and they do not establish a zero underlying risk.
For other adverse events, the record reports 8 affected participants among 8 at risk in Cohort 1 and 6 among 8 at risk in Cohort 2. The word “other” distinguishes these events from the serious adverse-event category in the results. The record does not provide event-level descriptions, number of events per participant, severity details for these events, or judgments about whether they were caused by tideglusib. The counts therefore indicate how many participants were recorded as affected, not the full clinical course of their events.
The cohorts were sequential and non-randomized. Their counts should not be interpreted as a controlled estimate of the difference between 1000 mg and 400 mg. Cohort composition, exposure, follow-up, and event ascertainment may matter, but relevant details are not reported in the registry record. The results are best read as descriptive safety observations in the enrolled cohorts.
12. What the Result Does, and Does Not, Mean
The results describe recorded safety outcomes in a completed, single-blind phase 2 study of tideglusib involving 16 participants with Myotonic Dystrophy 1. The posted counts show no serious adverse events or deaths recorded in either dose cohort, alongside other adverse events recorded for 8 participants in Cohort 1 and 6 participants in Cohort 2. They address the primary safety endpoint as defined in the registry: participants with events from baseline to end of study, within a stated timeframe of 12 weeks.
These counts do not establish that tideglusib caused any reported event, nor do they establish that it prevented or reduced events. They do not show that one dose is safer than the other. The study was not randomized, and the registry does not report a control group or an inferential comparison. Observed cohort differences are consequently descriptive and cannot separate dose effects from other potential cohort differences.
The absence of recorded serious events in these cohorts should not be generalized to all people with the condition or to longer periods of exposure. A small set of observations has limited ability to characterize uncommon outcomes. The record does not report confidence intervals or event-level information that would provide more detail about precision, timing, severity, or relatedness. The safety counts also cannot answer questions about efficacy. Although the study's stated purpose included efficacy and the record lists secondary functional, respiratory, imaging, clinical-impression, and pharmacokinetic measures, numerical results for those outcomes are not reported in the registry record for this analysis.
Thus, the evidence supports a bounded description of the recorded events among the enrolled participants. It does not support a treatment recommendation, a conclusion of equivalence between doses, or a general claim about safety or efficacy. Further interpretation depends on details and outcome results not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically informative in part because its design and reporting show why the form of a comparison matters. Two dose cohorts can produce separate descriptive event counts, but non-randomized sequential allocation does not make them equivalent to randomized groups. A difference between their observed counts cannot automatically be attributed to dose. Recognizing that distinction prevents a descriptive safety summary from being mistaken for a causal estimate.
The primary endpoint is participant-based and focuses on adverse events, a relevant safety summary for a study whose stated purpose includes safety assessment. At the same time, participant counts compress important information: they do not indicate event frequency per person, timing, severity, or relatedness. Understanding what a measure captures—and what it leaves out—is essential to interpreting clinical results precisely.
The study also lists multiple outcome measures and analyses, while the registry does not report how multiplicity was handled or provide a detailed analysis plan. This illustrates why a clear prespecified plan matters when a study evaluates several outcomes. Without that context, individual findings can be difficult to place in relation to the primary question, and the scope of formal statistical claims is unclear.
Finally, reporting the size and structure of the safety cohorts helps readers distinguish observed counts from evidence about rare-event risk. No serious adverse events were recorded in either cohort, but that observation is not proof of absence. The statistical lesson is not to overstate what a small, non-randomized study can establish: describe the observed data, identify the limits on precision and comparison, and separate the study's aims from results that the registry record does not report.
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 non-randomized, sequential design is a central limitation for comparing the two dose cohorts. The record does not identify a placebo or untreated control group, and it does not report enough information to establish that the cohorts were comparable. The posted counts cannot therefore isolate dose-related differences from other possible differences between cohorts.
The registry reports 16 participants in total, with 8 participants at risk in each cohort for the listed safety categories. The amount of information available for characterizing uncommon events is limited, and zero observed serious adverse events should not be interpreted as zero underlying risk. No confidence intervals or other precision estimates are reported for these safety results.
Important safety details are absent: event descriptions, event counts per participant, severity beyond the serious-event distinction, timing, duration, relatedness assessments, ascertainment procedures, and adjudication methods are not reported in the registry record. The identity of the party or parties masked in this single-blind study and the masking procedures are also not reported. Follow-up completeness, missing-data handling, and the definition of the Safety Analysis Set are not reported.
The record lists 20 outcome measures and 26 analyses but does not report a multiplicity strategy or detailed statistical analysis plan. It also does not provide numerical results for the listed secondary outcomes in the registry record used for this account. The protocol's sample-size rationale and any interim-analysis plan are not reported in the registry record. These limitations constrain interpretation to the posted descriptive safety counts rather than broader claims about comparative safety or efficacy.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy — NCT02858908.
- Registry record last updated 2025-09-11; status last verified 2025-08.
- Results first posted 2025-09-11.
- 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
The registry record describes a completed, single-blind, non-randomized phase 2 study sponsored by AMO Pharma Limited, with 16 enrolled participants and a study start date of 2016-07-20. Primary completion is listed as 2018-01. The study concerned adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy 1.
Participants in Cohort 1 received tideglusib 1000 mg orally once daily; Cohort 2 received 400 mg orally once daily. The primary outcome was the count of participants with adverse events, including serious adverse events, from baseline to end of study, with a timeframe of 12 weeks and the Safety Analysis Set as the named population.
The safety results record no serious adverse events and no deaths in either cohort. Other adverse events were recorded for 8 participants in Cohort 1 and 6 in Cohort 2, with 8 participants at risk in each cohort. The registry also lists pharmacokinetic, functional, respiratory, imaging, and clinical-impression secondary outcomes. Detailed analysis methods, missing-data procedures, multiplicity handling, and numerical secondary-outcome results are not reported in the registry record.