Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD (NCT02515669), a randomized phase 1, phase 2 study. The primary outcome is Safety Summary for the 24 Week Double-Blind Phase.

2. Clinical Question

3. Trial Design

NCT02515669 is listed as a Phase 1 and Phase 2 study of RO7239361 (BMS-986089) in ambulatory boys with Duchenne muscular dystrophy. The registry record describes a multisite, randomized, placebo-controlled, double-blind study with multiple ascending subcutaneous doses. Its design is recorded as parallel, with quadruple masking. The sponsor is Hoffmann-La Roche.

The study began on 2015-12-02, and its primary completion date is recorded as 2018-02-08. The status is TERMINATED. Enrollment is listed as 43. These registry fields describe the planned and administrative features of the trial; they do not, by themselves, explain why the study was terminated or whether the planned enrollment was reached. The reason for termination is not reported in the registry record.

The double-blind dose groups were organized into panels. Panel 1 received 4 mg RO7239361. Panel 2 received 12.5 mg for participants weighing between 15 and 45 kg or 20 mg for those weighing >45 kg. Panel 3 and the Expansion Panel received 35 mg for participants weighing between 15 and 45 kg or 50 mg for those weighing >45 kg. Placebo was also administered by subcutaneous injection on specified days. The registry record lists open-label groups as well as double-blind groups, but does not report the detailed sequence or rules governing entry into the open-label phase.

The endpoint description sets the primary safety observation period from baseline to Week 24. Secondary outcomes include maximum serum concentration, time to maximum concentration, area under the concentration-time curve, trough concentrations, and the percentage of participants with positive anti-RO7239361 antibody assessments. The record lists 20 outcome measures, but does not report analysis records for the primary endpoint. Details such as the randomization method, allocation concealment, exact visit schedule, and prespecified statistical analysis plan 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.

Before assignment: Starting at Week 5, participants whose weight exceeded or dropped below the dosing weight tier to which they were assigned (by \>1kg) were assigned to the new corresponding body weight-based dose within the participants assigned panel.

24 Week Double-Blind Phase

228 Week Open-Label Phase

Reasons for not completing this period:

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: Doses tested in this study were based upon achieving and maintaining moderate (\> 50% suppression), high (\> 85%), and near complete (\> 95%) suppression of serum free myostatin levels for Panels 1, 2, and 3 and Expansion Panel, respectively. Panel 3 was expanded after at least 2 weeks of dosing had been complete by at least 6 participants.

6. Primary Endpoint

The registry lists the primary outcome as Safety Summary for the 24 Week Double-Blind Phase, measured over Baseline to Week 24.

The primary endpoint is a safety summary for the 24 Week Double-Blind Phase. It measures the percentage of participants with fatalities, adverse events, and serious adverse events from baseline to Week 24. In general, adverse-event endpoints are used to describe harms and tolerability during a defined observation period. They do not directly measure whether a treatment improves the disease being studied.

The registry’s posted group figures give counts at risk and counts affected for serious adverse events and other adverse events, alongside deaths. These counts provide a descriptive account of how many participants in each listed group had an event. They should not be mistaken for event totals: a participant can potentially experience more than one event, and the record does not give enough detail to determine whether every event category is mutually exclusive.

Because the primary endpoint combines several kinds of safety information, it does not reduce to a single treatment-versus-placebo comparison. The endpoint title and description do not specify a single summary measure that combines fatalities, adverse events, and serious adverse events. The registry record also does not report a primary hypothesis test or an analysis plan for these categories. Consequently, the posted figures are best read as descriptive safety data within the listed groups and observation periods.

For interpretation, timing and study phase matter. The primary endpoint refers to the double-blind period through Week 24, while the registry separately lists open-label safety groups. Those open-label group counts should not be treated as if they were the randomized placebo comparison or automatically combined with the double-blind figures. The record does not report the participant-level rules needed to determine overlap between phases.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What do the counts describe?

The primary endpoint is expressed as a percentage of participants, while the posted safety data also provide counts at risk and affected. A count states how many participants in a listed group met the event definition; a percentage expresses that count relative to a denominator. In general, percentages can make groups with different sizes easier to compare, but they do not remove uncertainty or differences in study design. The registry’s group counts should be read within their labels, phase, and observation period.

The endpoint covers fatalities, adverse events, and serious adverse events through Week 24 in the double-blind phase. These categories are clinically and statistically distinct. A serious adverse event is not interchangeable with any adverse event, and a participant with an event may contribute to more than one category depending on the definitions and reporting rules. The registry record does not provide the full event definitions or participant-level data needed to reconstruct category overlap.

Why are the group counts not a direct treatment comparison?

In a randomized comparison, the intervention and comparator groups are intended to be comparable on average at allocation. That principle supports causal interpretation when the analysis follows the trial design and appropriate populations. Here, the posted figures are separated into dose panels and also include open-label groups. The registry record does not report a single analysis that combines or contrasts these groups under a stated statistical model. Therefore, the counts alone cannot substitute for a randomized treatment-effect estimate.

Panel membership is linked to dose, and the dose assignment described for some panels also depends on weight. A group-specific count can therefore reflect the group’s size, dose, follow-up, and the participants who contributed data. The record does not report participant-level covariates, exposure time, or enough detail on phase transitions to adjust for those factors. This limits any interpretation of apparent differences between group counts.

What would an uncertainty interval mean?

In general, a confidence interval describes a range of values that are compatible with the observed data under a specified statistical model and repeated-sampling procedure. A narrow interval indicates greater precision under that model; a wide interval indicates less precision. For event proportions, the interval’s width depends in part on the number of observations and the number of events. With limited event counts, uncertainty can remain substantial even when the observed proportion looks high or low.

The registry record does not report confidence intervals or a comparative effect estimate for the primary safety endpoint. No interval can be inferred from the reported counts without making additional analytical choices, and this interpretation does not recalculate one. Similarly, no reported p-value establishes whether a difference between groups is compatible with chance under a prespecified test.

What do the term-specific percentages add?

The registry lists selected serious adverse event terms with percentages. Such a list can identify the terms recorded in the safety data, but its meaning depends on the denominator, coding rules, whether participants or events form the basis of the percentage, and how terms were selected for display. Those details are not reported in the registry record. The listed percentages should therefore be quoted as registry entries rather than treated as comparable incidence estimates across terms or groups.

In general, adverse-event coding organizes reports into standardized terms. A term identifies the reported event category; it does not by itself establish causation. A listed event occurring during a trial is not necessarily caused by the study drug. The registry data presented do not supply a causal assessment for individual events.

How should planning illustrations be understood?

Any sample-size illustration based on an observed effect is a planning exercise, not a description of the trial’s observed power. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. For time-to-event outcomes the stated convention is a total number of events, while other outcomes use participants per arm. Such illustrations depend on assumptions and should not be mistaken for the trial’s own design justification. The sample-size justification and its assumptions are not reported in the registry record.

In general, power describes the chance that a planned statistical procedure will reject a null hypothesis under specified assumptions. It is not a measure of whether the findings are true, and it should not be calculated retrospectively from an observed result and presented as an observed property of the trial. The registry record does not report a formal power calculation or the statistical assumptions underlying the trial’s enrollment target.

What remains unavailable?

The registry lists no analysis records for the primary endpoint. Details of the analysis population, handling of missing safety observations, exposure-adjusted methods, multiplicity control, and prespecified comparisons are not reported in the registry record. Without these details, the posted counts support descriptive interpretation, but not reconstruction of a complete inferential analysis.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 20 outcome measures, including the primary safety summary and secondary pharmacokinetic and antibody outcomes. When a study assesses multiple outcomes, time points, dose groups, or event categories, the chance of obtaining at least one apparently notable result can increase if many statistical tests are conducted. In general, multiplicity procedures are used to control or describe that issue. The registry record does not report a multiplicity strategy or a hierarchy of confirmatory tests.

There are no analysis records reported for the primary endpoint. The registry record does not report the analysis population, whether all randomized participants were included in each safety summary, how participants with incomplete follow-up were handled, or how missing event information was treated. For safety outcomes, missingness can matter if participants differ in their opportunity for observation. Exposure time and reasons for ending follow-up are not reported in the registry record, so exposure-adjusted comparisons cannot be reconstructed.

The endpoint description specifies a baseline-to-Week-24 period for the double-blind phase, while the results also identify open-label groups. The registry record does not report whether the open-label summaries were prespecified, how participants entered those groups, or whether any participants contributed to more than one phase’s summaries. These omissions prevent a clear account of analysis populations and phase-specific follow-up.

The record describes the trial as randomized and quadruple masked, but does not report the randomization sequence method, allocation concealment, or a detailed statistical analysis plan. It also does not report interim analyses. These items should not be inferred from the design labels. As a result, the posted data can be described by group, but a full account of confirmatory inference, missing-data handling, and multiplicity is not available.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: influenza (12.5%), rhabdomyolysis (12.5%), conversion disorder (12.5%), appendicitis perforated (11.1%), skull fracture (9.1%).

For the double-blind phase, the serious adverse event figures are 1 affected participant among 11 at risk in the placebo group, 0 among 7 in Panel 1 RO7239361, 0 among 6 in Panel 2 RO7239361, and 1 among 19 in Panel 3 and the Expansion Panel RO7239361. The registry lists 0 deaths in each of these groups. These are group-specific counts; they do not establish a difference in risk or a causal relationship with treatment.

The open-label figures are separate: Panel 1 has 1 serious adverse event among 9 at risk, Panel 2 has 2 among 8, and Panel 3 and the Expansion Panel has 1 among 26. The registry lists 0 deaths in each open-label group. They should not be combined with the double-blind counts as though they arose from the same randomized comparison. The registry record does not report participant overlap between phases or exposure-adjusted rates.

The record also lists selected serious adverse event terms and percentages, but does not report the denominators or detailed calculation rules for those percentages. The terms are descriptive safety listings, not evidence that the drug caused the events. Serious event counts can be useful for identifying what was reported, while the limited counts and absent comparative analysis constrain conclusions about relative risk.

12. What the Result Does, and Does Not, Mean

The registry record describes a randomized, placebo-controlled study of RO7239361 in ambulatory boys with Duchenne muscular dystrophy, with safety and tolerability as the primary focus. The posted results give counts of participants affected by serious and other adverse events in the listed double-blind and open-label groups. Those counts provide a descriptive record of events during the study.

They do not, on their own, show that RO7239361 caused a reported event or that a group’s event count reflects a treatment effect. A safety event observed during a trial may have other explanations, and attribution requires clinical and analytical assessment not reported in these data. Nor do the counts establish that one dose or group is safer than another: the groups differ in size and dose panel, and no comparative statistical analysis is reported for the primary endpoint.

The findings also do not answer whether the study drug improves Duchenne muscular dystrophy. The primary endpoint is a safety summary, and the listed secondary outcomes focus substantially on pharmacokinetics and antibody assessment. The registry record does not provide efficacy results in the registry record for this analysis, and no claim about clinical benefit is warranted.

The status TERMINATED is an administrative record, not an explanation of the trial’s outcome. The reason for termination is not reported in the registry record. Likewise, the absence of reported analysis records does not establish that no analyses were performed; it means that a statistical analysis of the primary endpoint is not reported in the registry record. The results are therefore most appropriately interpreted as descriptive safety information with substantial limits on comparative and causal conclusions.

13. Why This Trial Matters Statistically

This trial illustrates a common statistical challenge in early-phase clinical research: the main question is safety and tolerability across multiple dose groups, rather than a single confirmatory comparison of clinical effectiveness. Such studies can generate important descriptive information, but their results need to be interpreted in light of dose panels, differing group sizes, and the distinction between blinded and open-label follow-up.

The registry record makes the endpoint’s scope explicit: participants with fatalities, adverse events, and serious adverse events are summarized through Week 24 for the double-blind phase. At the same time, it lists additional open-label safety groups and pharmacokinetic outcomes. This makes clarity about phase, denominator, and analysis population essential. Counts from different phases should not be treated as directly interchangeable, and a list of event terms is not itself a causal analysis.

The record also demonstrates why statistical interpretation depends on more than the raw counts. Comparative uncertainty, missing-data handling, multiplicity, and the prespecified analysis plan all shape what can be concluded. Those details are not reported in the registry record for the primary endpoint. Descriptive results can still communicate what was recorded, but they cannot replace a reported comparative estimate and its uncertainty.

Finally, the TERMINATED status should be distinguished from the scientific interpretation of the posted safety data. Without a reported reason for termination, readers should not infer whether it was connected to safety, feasibility, or another consideration. Keeping administrative status, observed event counts, and causal conclusions separate is central to responsible analysis of clinical trial registries.

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 registry record gives group-level safety counts but not participant-level data. It does not report the detailed definitions used for adverse events and serious adverse events, how events were coded, or whether event categories overlap. The selected serious adverse event term percentages also lack reported denominators and calculation details, limiting their interpretation.

Several design and analysis details are not reported in the registry record: the randomization method, allocation concealment, statistical analysis plan, analysis populations, handling of missing observations, exposure time, and any interim analyses. There are no analysis records reported for the primary endpoint. Without these details, it is not possible to reconstruct a planned comparative analysis or assess its precision from the registry counts alone.

The safety data include both double-blind and open-label group labels. The record does not explain the rules for entering the open-label phase or whether participants may appear in more than one phase summary. The double-blind dose panels also differ, and some dose assignments depend on weight. These features make direct comparisons across all listed groups inappropriate without further design and participant-level information.

The study is recorded as TERMINATED, but the reason is not reported in the registry record. The record does not establish whether the listed results represent all planned observations or explain the extent of follow-up. These limitations do not negate the descriptive counts; they define what those counts can support and prevent strong comparative or causal conclusions.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD — NCT02515669.
• Registry record last updated 2020-11-04; status last verified 2020-10.
• Results first posted 2019-05-08.
• 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.

18. Record Summary

NCT02515669 is a Phase 1 and Phase 2 randomized, placebo-controlled study of RO7239361 in ambulatory boys with Duchenne muscular dystrophy. The registry describes a parallel design with quadruple masking and subcutaneous administration. Enrollment is listed as 43, and the sponsor is Hoffmann-La Roche.

The primary endpoint is a safety summary from baseline to Week 24, covering fatalities, adverse events, and serious adverse events. The registry lists double-blind and open-label groups, as well as secondary pharmacokinetic and antibody outcomes. The study status is TERMINATED; its reason is not reported in the registry record. No analysis records are reported for the primary endpoint, so the posted safety counts are descriptive and do not provide a reported comparative treatment-effect estimate.