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 Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (NCT03907072), a randomized phase 2, phase 3 study. The primary outcome is Change From Baseline in Dystrophin Level (% Normal Dystrophin).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03907072 |
| Phase | Phase 2, Phase 3 |
| Condition | Duchenne Muscular Dystrophy |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Wave Life Sciences USA, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Ambulatory male pediatric patients with Duchenne muscular dystrophy amenable to exon 51 skipping intervention. The registry record lists an enrollment of 6.
Intervention
WVE-210201 (suvodirsen), assigned in the trial arms as weekly intravenous administrations at 3 mg/kg or 4.5 mg/kg.
Comparator
Weekly intravenous administrations of placebo, described in the registry record as phosphate buffered saline visually identical in appearance to the study drug.
Primary question
How did dystrophin level, expressed as % normal dystrophin, change from baseline at the listed follow-up points? The registry record says no statistical analysis was performed because the study terminated early.
The trial was designed to compare suvodirsen with placebo in a population selected for the stated exon 51 skipping eligibility. Its primary endpoint was change from baseline in dystrophin level, with the timeframe listed as Day 1 to Week 12, Week 22, or Week 46. The record does not provide an analyzed comparison of change between treatment groups, nor does it report a numerical treatment effect or its uncertainty.
This distinction matters: the clinical question describes what the trial was set up to assess, not a conclusion about whether the intervention changed dystrophin. The posted record states that early termination prevented statistical analysis of the primary outcome. It therefore does not answer the primary comparison in a way that permits an estimate of the size or precision of any difference.
Secondary outcomes listed in the record include change in the North Star Ambulatory Assessment, upper limb proximal strength, the 4-stair climb, the 10-meter walk/run test, forced vital capacity, the 95th percentile of stride velocity, and dystrophin level. These are listed as planned outcome measures; the registry record does not provide analyzed results for them. The clinical question should consequently be read as the trial's intended question, not as a finding established by the posted results.
3. Trial Design
DYSTANCE 51 is described as a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension. The registry record identifies a parallel-group model and lists WVE-210201 (suvodirsen) and placebo as the interventions. The treatment arms were WVE-210201 at 3 mg/kg, WVE-210201 at 4.5 mg/kg, and placebo. Each arm description specifies weekly intravenous administration.
Randomization is a design method for assigning participants to study groups, intended to reduce systematic differences at the start of a trial. Double masking means that the record describes the study as double-blind; the registry record does not specify which roles were masked. The placebo is described as visually identical in appearance to the study drug, a feature relevant to maintaining masking. These design features describe the intended study structure. They do not establish that the groups were balanced, especially in a trial with an enrollment of 6.
The study is listed as terminated. Its start date is 2019-09-04, and its primary completion date is 2019-12-16. The registry record's primary outcome description explicitly states that no statistical analysis was performed due to early study termination. The planned open-label extension is part of the study description, but the record does not establish whether participants entered or completed that extension.
The registry arm descriptions contain inconsistent product names: the arm labels use WVE-210201, while the dosing text refers to WVE-210210; the placebo text ends with WVE-21021. These inconsistencies make the wording of the descriptions less clear, although the intervention name and arm labels identify WVE-210201 (suvodirsen). The record also does not describe the randomization procedure, allocation ratio, treatment duration before termination, or the reasons for stopping. Those details are not reported in the registry record.
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.
Overall Study
| Stage | WVE-210201 (3 mg/kg) | WVE-210201 (4.5 mg/kg) | Placebo |
|---|---|---|---|
| STARTED | 2 | 2 | 2 |
| COMPLETED | 0 | 0 | 0 |
| NOT COMPLETED | 2 | 2 | 2 |
Reasons for not completing this period:
| Reason | WVE-210201 (3 mg/kg) | WVE-210201 (4.5 mg/kg) | Placebo |
|---|---|---|---|
| Study Terminated by Sponsor | 2 | 2 | 2 |
Analysis population for the primary outcome, as defined in the registry: No statistical analysis has been performed due to early study termination
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 | WVE-210201 (3 mg/kg) | WVE-210201 (4.5 mg/kg) | Placebo | Total |
|---|---|---|---|---|---|
| Participants analysed | 2 | 2 | 2 | 6 | |
| Age, Categorical: <=18 years | Participants | 2 | 2 | 2 | 6 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 |
| Age, Continuous | Years | 6.5 (5 to 8) | 9 (8 to 10) | 7 (5 to 9) | 7.5 (5 to 10) |
| Sex: Female, Male: Female | Participants | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Male | Participants | 2 | 2 | 2 | 6 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 1 | 2 | 2 | 5 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 0 | 1 |
| Region of Enrollment: Sweden | participants | 0 | 1 | 0 | 1 |
| Region of Enrollment: Belgium | participants | 1 | 0 | 0 | 1 |
| Region of Enrollment: Italy | participants | 0 | 0 | 2 | 2 |
| Region of Enrollment: France | participants | 1 | 1 | 0 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Dystrophin Level (% Normal Dystrophin), measured over Day 1 to Week 12, Week 22, or Week 46.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is change from baseline in dystrophin level, expressed as % normal dystrophin. It is listed for Day 1 to Week 12, Week 22, or Week 46. The endpoint is a measure of dystrophin level relative to a normal reference, rather than a direct measure of a participant's walking ability or daily function. The registry record's endpoint description adds only “US/other regions (as applicable)” and does not provide further detail about how the measure was collected or analyzed.
In general, a biological measure can be used to assess whether an intervention is associated with a change in a target-related quantity. Its interpretation depends on the measurement procedure, the comparison being made, and the relationship between the measured quantity and outcomes that matter to participants. The registry record does not provide enough information to assess those relationships or to interpret a numerical change in this trial.
The endpoint is presented as change from baseline, which generally means that follow-up values are considered relative to an initial measurement. For a randomized comparison, the key treatment question would ordinarily concern how that change differs between groups, with an estimate and an indication of its precision. Here, no numerical endpoint results or statistical analysis are reported. The record specifically attributes the lack of analysis to early termination, so the endpoint's intended role should not be mistaken for an observed finding.
The record lists several follow-up time points rather than one single endpoint time. The registry data do not clarify which time point would have been primary for a completed analysis, how multiple time points would have been handled, or whether the endpoint was assessed in all participants. Those details are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the registry record reports no statistical analysis of the primary endpoint because the study terminated early. Although the trial was designed to assess change from baseline in dystrophin level, the posted record gives no analyzed treatment-group estimates, no between-group effect, and no measure of statistical uncertainty. It is therefore not possible to use this record to quantify the primary comparison.
The listed enrollment is 6, and the posted serious and other adverse event counts show 2 participants at risk in each of the three groups. Such small group counts leave little information for estimating comparative effects. Even if numerical outcome summaries were available, estimates from groups of this size would generally be imprecise and sensitive to individual observations. This is a general statistical limitation, not a calculated property of an unreported endpoint estimate.
A confidence interval, in general, describes a range of values compatible with the data and the method used to calculate it. A narrow interval indicates greater precision than a wide interval. No confidence interval for the primary endpoint is reported, so the precision of any treatment contrast cannot be assessed. Likewise, no p-value for the primary endpoint is reported. The absence of a statistical result is not evidence that the groups were the same; it means that this registry record does not present an analyzed comparison.
The listed secondary outcomes include functional assessments, strength, pulmonary function, and dystrophin-related measures. Their presence in the outcome list does not mean results were posted. The record reports 0 analyses, and does not provide numerical results for these outcomes in the registry record. The outcome list should therefore be treated as a description of planned assessment areas, not as evidence of effects or lack of effects.
The safety figures are descriptive counts. Serious adverse events affected 0 participants in the 3 mg/kg group, 1 in the 4.5 mg/kg group, and 0 in the placebo group; each group had 2 participants at risk. Other adverse events affected 2 participants in each group, and deaths were 0 in each group. With so few participants, these counts cannot by themselves establish a comparative safety pattern or determine whether an event was caused by treatment. The record's top serious term is Cardiac Disorder, listed as 50.0%, but the term-level denominator and group attribution are not reported in the registry record.
The most defensible reading is consequently limited: the registry describes a randomized, double-blind comparison that ended early, records a very small enrollment, and provides adverse event counts, but it does not provide an analyzed primary efficacy result. This is a limitation of what can be concluded from the posted record, not a finding for or against suvodirsen.
9. Statistical Methods Explained
Statistical methods are the procedures used to turn observations into estimates and to describe how uncertain those estimates are. In a completed randomized trial, an analysis might compare the outcome between assigned groups, account for baseline values, and report an estimate with an uncertainty interval. The registry record does not report the primary analysis method, a treatment-effect estimate, or an uncertainty interval. It states that no statistical analysis was performed because of early termination. Any discussion of methods below is therefore general explanation, not a description of analyses actually conducted in this trial.
What randomization contributes
In general, random assignment aims to make treatment groups comparable at the point of assignment, including with respect to factors that are measured and factors that are not. It supports an interpretation of group differences as related to assignment, subject to the quality of trial conduct and analysis. Randomization does not guarantee balance in a small study. With the listed enrollment of 6, chance differences between groups can remain substantial, and an observed difference could be strongly affected by one participant. The registry record identifies the design as randomized but does not report the randomization sequence, allocation concealment method, or procedure used to implement assignment.
What a between-group estimate would represent
For a change-from-baseline endpoint, a treatment comparison generally focuses on the difference in change between groups, rather than interpreting a change in one group alone. This helps separate change occurring over time in both groups from a difference associated with assignment. Such an estimate would normally be accompanied by an interval describing its precision. The registry record does not provide group-level change values or a between-group estimate for dystrophin level. It is not possible to reconstruct the primary comparison in the registry record in this record.
The endpoint is listed across Day 1 to Week 12, Week 22, or Week 46. In general, repeated time points raise choices about which follow-up is the main comparison and how results across time are summarized. The registry record does not state how those choices were to be made. Nor does it report whether the different time points correspond to separate analysis populations, a single model, or another approach.
Precision and uncertainty
In general, a confidence interval communicates uncertainty around an estimate. It reflects both the observed data and the assumptions of the analysis method. A trial with few participants usually has limited ability to estimate effects precisely, although the exact interval depends on the outcome data and analysis. There is no primary endpoint interval in the posted record. The sample-size assumptions mentioned for planning illustrations are not a substitute for an observed confidence interval and do not provide evidence about the trial's actual treatment effect.
A p-value, in general, measures how unusual the data would be under a specified null hypothesis and analysis model. It does not measure the probability that a treatment works, and it does not express the size or clinical importance of an effect. No primary-endpoint p-value is reported. In the absence of an analysis, there is no basis to characterize the endpoint comparison as statistically significant or not statistically significant.
Planning illustrations are not trial findings
The planning assumptions described for the sample-size illustrations are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect set as a stated fraction of the observed effect. These are assumptions for hypothetical planning calculations, not a report of the original trial's design justification. The registry record does not report the trial's sample-size justification. The illustrations should not be interpreted as observed power, as evidence about the primary result, or as a record of the sponsor's planned sample size.
In general, a planning calculation asks how much information might be needed under specified assumptions. Its result depends on those assumptions and is not a property measured from the participants. Here, the primary outcome was not analyzed, so the record does not report an observed effect that can be interpreted as a treatment result. The illustrations do not remedy the absence of that analysis.
Over-dispersion and count outcomes
In general, over-dispersion occurs when count data vary more than a chosen statistical model expects. If a model assumes less variability than is present, its uncertainty estimates may be too small. This concept may matter in analyses of event counts, but the registry record does not report a model for adverse events or any over-dispersion assessment. The posted adverse event information is presented as participant counts, so no model-based conclusion should be inferred from it.
What can be learned from the posted methods information
The record identifies the broad design features—randomized, double-blind, placebo-controlled, parallel-group, and Phase 2/3—and gives the primary endpoint and its listed timeframe. It does not report a statistical analysis plan, model specification, covariate adjustment, handling of repeated measures, interim analyses, missing-data method, or analysis population for the efficacy outcome. These items are not reported in the registry record. They cannot be inferred from the trial title, its design labels, or the planned endpoint list.
As a result, the statistical reading rests on a clear distinction: design describes how the study was intended to operate, while analysis reports what was estimated from collected data. The first is present in the registry record; the primary efficacy analysis is explicitly absent. The tables, plots, and planning section should be read in that context, with descriptive posted counts kept separate from unreported efficacy estimates.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study evaluates several outcomes, time points, doses, or comparisons. In general, making many statistical tests can increase the chance of finding an apparently notable result by chance if no adjustment or hierarchy is specified. A statistical plan may address this by defining a primary comparison, ordering secondary tests, or applying another procedure. The registry record lists one primary endpoint, multiple secondary outcomes, several follow-up time points, and three arms, but it does not report how multiplicity would have been handled.
The record's primary outcome timeframe includes Day 1 to Week 12, Week 22, or Week 46. It does not clarify how the listed times relate to the primary analysis or whether any one time point would control the main inference. The record also does not report an analysis plan describing how comparisons between the two suvodirsen dose groups and placebo would be prioritized. These details are not reported in the registry record, so the outcome list cannot be used to infer an adjustment strategy.
Missing-data methods are also not reported in the registry record. There is no description of how incomplete endpoint measurements, withdrawals, or measurements after early termination would have been handled. The record does not report an analysis population, such as whether an efficacy analysis would include all randomized participants or another defined group. Because no primary statistical analysis was performed, it is not possible to assess the impact of missing observations or determine whether the observed participants represent the intended analysis population.
In general, missing-data assumptions matter because conclusions may depend on why measurements are absent and on the method used to address them. A method cannot be selected from the posted information alone. The early termination and the absence of analysis are central limitations: there is no reported primary endpoint estimate to evaluate, and no reported analysis procedure that would clarify how multiplicity or missingness was addressed.
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 |
|---|---|---|---|
| WVE-210201 (3 mg/kg) | 0 | 2 | 0.0% |
| WVE-210201 (4.5 mg/kg) | 1 | 2 | 50.0% |
| Placebo | 0 | 2 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| WVE-210201 (3 mg/kg) | 0 of 2 |
| WVE-210201 (4.5 mg/kg) | 0 of 2 |
| Placebo | 0 of 2 |
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 |
|---|---|
| WVE-210201 (3 mg/kg) | 2 of 2 |
| WVE-210201 (4.5 mg/kg) | 2 of 2 |
| Placebo | 2 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | WVE-210201 (3 mg/kg) | WVE-210201 (4.5 mg/kg) | Placebo |
|---|---|---|---|---|
| Cardiac Disorder | Cardiac disorders | 0 of 2 | 1 of 2 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | WVE-210201 (3 mg/kg) | WVE-210201 (4.5 mg/kg) | Placebo |
|---|---|---|---|---|
| Pyrexia | General disorders | 1 of 2 | 2 of 2 | 0 of 2 |
| Diarrhoea | Gastrointestinal disorders | 1 of 2 | 1 of 2 | 0 of 2 |
| Nasopharyngitis | Infections and infestations | 1 of 2 | 1 of 2 | 0 of 2 |
| Bone contusion | Injury, poisoning and procedural complications | 0 of 2 | 0 of 2 | 1 of 2 |
| C-reactive protein increased | Investigations | 0 of 2 | 1 of 2 | 0 of 2 |
| Headache | Nervous system disorders | 0 of 2 | 1 of 2 | 0 of 2 |
| Vomiting | Gastrointestinal disorders | 0 of 2 | 1 of 2 | 0 of 2 |
| Gastroenteritis | Infections and infestations | 0 of 2 | 0 of 2 | 1 of 2 |
| Oral herpes | Infections and infestations | 0 of 2 | 1 of 2 | 0 of 2 |
Serious events with the highest arm-level rate: cardiac disorder (50.0%).
The posted safety counts are small descriptive summaries. In the WVE-210201 (3 mg/kg) group, 0 of 2 participants at risk had a serious adverse event; in the WVE-210201 (4.5 mg/kg) group, 1 of 2 did; and in the placebo group, 0 of 2 did. Other adverse events affected 2 of 2 participants in each group. Deaths were 0 in each group. These are participant counts as recorded, not estimates of comparative risk.
The registry record lists Cardiac Disorder at 50.0% among the top serious adverse event terms. The term-level denominator and the group to which this percentage applies are not reported in the registry record. It should therefore not be combined with the group counts to infer an event rate by treatment arm.
In general, an adverse event recorded during a trial is not automatically an event caused by the study treatment. Assessing causality requires information beyond whether an event occurred, including clinical evaluation and the trial's attribution procedures. Those details are not reported. Also, when only a few participants are represented, one participant's experience can materially affect the descriptive pattern. These counts do not establish comparative safety, and they should not be read as proof of either harm or absence of harm.
12. What the Result Does, and Does Not, Mean
The record establishes that DYSTANCE 51 was a randomized, double-blind, placebo-controlled study of suvodirsen in ambulatory male pediatric patients with Duchenne muscular dystrophy amenable to exon 51 skipping. It was designed to assess change from baseline in dystrophin level, with additional functional and physiological outcomes listed. It also establishes that the study was terminated and that no statistical analysis of the primary outcome was performed because of early termination.
What the posted record does not establish is the size or direction of a primary endpoint difference between suvodirsen and placebo. There is no reported estimate, confidence interval, or p-value for that comparison. The absence of those results does not mean that the groups had identical outcomes, nor does it show that one group had a better outcome. It means the registry record does not present an analysis from which those claims could be made.
The safety counts document serious and other adverse events among the small number of participants listed as at risk, as well as no deaths recorded in the groups. They do not, by themselves, identify the cause of an event or provide a reliable basis for comparing event rates between groups. The term-level Cardiac Disorder percentage also lacks a reported denominator and group attribution in the record.
The distinction between a trial's intended question and its posted findings is particularly important. A trial may be designed around a clear endpoint and still provide no analyzable estimate when it ends early. The study description and outcome list explain what investigators intended to evaluate; they do not stand in for results.
Accordingly, the statistical conclusion is about the evidence available in this registry record: it does not quantify the primary efficacy comparison, and its small safety counts remain descriptive. It cannot support a conclusion about whether suvodirsen changed dystrophin level or about the comparative safety of the study arms. Those questions remain unanswered by the posted results.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a well-defined research question and an answer supported by analyzed data. The record describes randomization, double masking, a placebo comparator, and a primary endpoint. Yet it also states that early termination meant no statistical analysis was performed for that endpoint. Design labels and an endpoint specification cannot substitute for an effect estimate.
The enrollment of 6 also highlights how limited information can be when only a small number of participants contribute to a study. In general, estimates based on few observations tend to be imprecise and can be highly sensitive to individual outcomes. That concern applies especially to comparisons among multiple groups, though the registry record does not provide efficacy data from which to calculate precision.
The record is also a useful reminder to separate outcomes that are listed from outcomes that have results. It names several secondary measures, but the presence of those measures does not indicate that they were analyzed or that any difference was found. The record reports 0 analyses, and the primary outcome specifically has no statistical analysis due to early termination.
Finally, this example shows why safety counts need context. Counts by group are useful descriptions of what was recorded, but they are not causal conclusions and are difficult to compare when the groups contain very few participants. A serious-event term percentage without a reported denominator or group attribution has additional interpretive limits.
For readers, the key statistical lesson is to ask whether a result is an estimate from an analysis, a descriptive count, a planned outcome, or a hypothetical planning illustration. Those categories answer different questions. In this record, the design and planned endpoints are described, safety counts are posted, and the primary efficacy analysis is absent. Keeping those evidence types distinct prevents an intended assessment from being mistaken for a demonstrated result.
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.
Several trial-specific limitations constrain interpretation beyond the absence of a primary analysis. The registry lists an enrollment of 6, with 2 participants at risk in each of the three adverse-event groups. This leaves very little information for stable estimates or comparisons. The record does not report participant-level endpoint data, group-level changes, or the distribution of follow-up measurements, so the influence of individual observations cannot be assessed.
The study is terminated, and the record attributes the lack of primary analysis to early termination. It does not report the reason for stopping, the timing of individual discontinuations, or whether any participants completed the listed follow-up points. The open-label extension appears in the study description, but participation in or completion of that extension is not reported in the registry record.
The outcome information includes multiple time points and several secondary measures, but the record does not explain how these would have been prioritized or analyzed. It does not report a statistical analysis plan, analysis populations, a missing-data approach, or methods for addressing multiple comparisons. These omissions prevent a reader from evaluating how an intended efficacy analysis would have been conducted.
The arm descriptions also have inconsistent product names: the treatment labels identify WVE-210201, while the dosing descriptions use WVE-210210 and the placebo description refers to WVE-21021. The record's intervention names and arm labels identify suvodirsen and placebo, but the description text is not fully consistent.
For safety, the serious-event term Cardiac Disorder is listed at 50.0%, but its denominator and group attribution are not reported. The adverse event counts do not provide a basis for assigning causality. Together, these record-specific gaps mean that the posted information supports a description of the study and limited counts, not a comparative efficacy conclusion or a detailed safety assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy — NCT03907072.
- Registry record last updated 2021-05-20; status last verified 2021-04.
- Results first posted 2021-05-20.
- 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
NCT03907072 is a Phase 2/3 randomized, double-blind, placebo-controlled, parallel-group study of WVE-210201 (suvodirsen), with an open-label extension, in ambulatory male pediatric patients with Duchenne muscular dystrophy amenable to exon 51 skipping. The arms were WVE-210201 at 3 mg/kg, WVE-210201 at 4.5 mg/kg, and placebo, described as weekly intravenous administrations. The registry lists an enrollment of 6 and a status of terminated.
The primary endpoint was change from baseline in dystrophin level (% normal dystrophin), with the timeframe listed as Day 1 to Week 12, Week 22, or Week 46. The record states that no statistical analysis was performed because of early study termination, and reports 0 analyses. It lists secondary functional and physiological outcomes but provides no analyzed efficacy results in the record.
Posted safety counts show serious adverse events in 0, 1, and 0 participants across the 3 mg/kg, 4.5 mg/kg, and placebo groups, respectively; other adverse events affected 2 participants in each group, and deaths were 0 in each group. These counts are descriptive and do not establish causality or comparative safety.