Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals (NCT06117020), a randomized phase 1 study. The primary outcome is Incidence, Severity and Relatedness of Treatment-emergent Adverse Events (TEAEs). TEAE Severity Will be Measured as Mild, Moderate or Severe, and Relatedness Will be Either Related or Not Related..

2. Clinical Question

This is an early-stage safety and pharmacology study, not a trial of clinical benefit in people with the conditions listed in the registry. Its summary also identifies plasma and urine pharmacokinetics, pharmacodynamic effects on muscle strength, muscle biopsy, and other potential mechanistic or predictive markers as objectives. Those objectives describe what the investigators intended to evaluate; numerical results for them are not reported in the registry record.

3. Trial Design

The registry describes a randomized, double-blind, placebo-controlled, first-in-human, phase 1 study with quadruple masking and a parallel-group model. It is titled as a single and multiple ascending dose study of MTR-601 in healthy individuals. The record lists the study as completed, with 89 participants enrolled. The start date is 2023-09-11 and the primary completion date is 2024-10-16.

The active-treatment groups are organized by dose level and dosing schedule. The single ascending dose portion includes MTR-601 Dose Levels 1 through 5, with Dose Level 2 separated into fasted and fed groups. The multiple ascending dose portion includes active MTR-601 Dose Levels 1 through 6. Placebo is recorded separately for the single and multiple ascending dose portions. The listed group sizes range from 6 to 12 participants; these are group-specific counts, not a basis for combining results across cohorts.

The stated purpose was to assess safety and tolerability under fed and fasted conditions and to evaluate pharmacokinetics and pharmacodynamics. The registry names plasma and urine pharmacokinetics, effects on muscle strength, muscle accumulation assessed by biopsy, and other potential mechanistic, predictive, and pharmacodynamic markers. Secondary outcomes listed include maximum plasma concentration and time to maximum plasma concentration for single and multiple ascending doses, including steady-state measures.

The registry does not report the dose amounts, the detailed allocation procedure, the identity of masking roles, or the protocol’s rationale for choosing the cohort sizes. It also does not report a detailed statistical analysis plan. Those omissions limit what can be concluded about the design beyond the features explicitly recorded.

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.

Only the first 6 of 13 groups are shown.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The number of participants and events analyzed does not differ from the number of participants or events assigned to the arm or placebo group.

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.

Only the first 6 of 14 groups are shown.

6. Primary Endpoint

The registry lists the primary outcome as Incidence, Severity and Relatedness of Treatment-emergent Adverse Events (TEAEs). TEAE Severity Will be Measured as Mild, Moderate or Severe, and Relatedness Will be Either Related or Not Related., measured over From baseline to last follow-up visit (Day 7 for SAD Dose Levels 1, 3, and 4, Day 12 for SAD Dose Level 2, Day 20 MAD Dose Levels 1-6).

The primary endpoint is described as the incidence, severity, and relatedness of treatment-emergent adverse events (TEAEs). Severity is categorized as mild, moderate, or severe, and relatedness as related or not related. The recorded unit for the primary outcome is the number of events. The stated purpose is to assess the safety and tolerability of single and multiple doses of MTR-601 in healthy volunteers under fed and fasted conditions.

TEAEs are used in early-phase trials to describe medical events that occur after treatment begins and to support structured monitoring of safety. A count of events is not the same as a count of participants: one participant may experience more than one event. The registry’s primary outcome figures should therefore be read as event counts, not as the number of people affected. Separate adverse-event information in the record reports participant counts for non-serious events and serious events.

The specified follow-up varies by cohort: through Day 7 for SAD Dose Levels 1, 3, and 4; through Day 12 for SAD Dose Level 2; and through Day 20 for MAD Dose Levels 1-6. These differing observation periods matter when reading raw counts, because the opportunity to record events is not identical across all groups. The registry record does not provide severity- or relatedness-specific result breakdowns, event descriptions, or an inferential comparison for the primary outcome. The posted total counts alone cannot establish the clinical importance or causality of individual events.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry posts descriptive outcome counts, but no analysis results are listed for inferential comparisons. The interpretation is therefore limited to what the recorded counts represent and to general statistical principles that help explain their limitations.

What does an event count represent?

In general, a count outcome records how many events were observed within a group during a defined period. It differs from a binary participant outcome, which records whether each participant experienced an event. A participant can experience more than one TEAE, so the event total can be greater than the number of affected participants. For that reason, event totals should not be described as the number of participants with events unless the outcome specifically reports participants.

The registry labels the primary outcome unit as number of events. Its summary results are therefore interpreted as event counts. A separate adverse-event listing reports the number of participants with other adverse events, which answers a different question. Keeping these measures separate avoids confusing how often events occurred with how many people experienced at least one event.

Why are raw counts difficult to compare?

In general, event counts depend on the number of people observed, the length of observation, the frequency of events, and the possibility that a participant experiences repeated events. A larger group or longer follow-up can yield more events even if the underlying event rate is similar. Comparisons of raw counts are most informative when observation time and the population at risk are comparable, or when an appropriate rate-based analysis accounts for differences.

Here, the registry lists varying group sizes and follow-up windows. The single ascending dose follow-up is through Day 7 for Dose Levels 1, 3, and 4, through Day 12 for Dose Level 2, and the multiple ascending dose follow-up is through Day 20 for Dose Levels 1-6. The record does not report a statistical model that adjusts event counts for these differences, nor does it post person-time or exposure-adjusted rates. Consequently, the raw counts are best read within their respective cohorts rather than as a dose-response comparison across the entire study.

What would an inferential analysis add?

In general, an inferential analysis can quantify uncertainty around an estimated difference or rate and may account for design features or differing observation time. A confidence interval expresses a range of values compatible with the data and the analysis assumptions; it helps show precision, not certainty that a particular value is true. A hypothesis test evaluates how compatible the observed data are with a specified null model. Neither a confidence interval nor a p-value establishes clinical importance on its own.

No confidence intervals, p-values, or adjusted comparisons for the TEAE count endpoint are reported in the registry record. The analysis count is recorded as 0. This means the posted record does not supply inferential results for interpreting the apparent differences among the group counts. It would be inappropriate to calculate significance from the summaries without a prespecified analysis and the needed event-level information.

Why can event data require specialized models?

In general, count outcomes may be analyzed with models for event rates. A simple Poisson model assumes that, conditional on modeled factors, variability is tied to the mean. If the observed counts are more variable than that assumption allows, over-dispersion can make conventional uncertainty estimates too optimistic. Negative binomial models or other approaches can accommodate extra variability, depending on the data and design. Repeated events within the same participant can also create dependence, so analyses may need to account for clustering or recurrent events.

These are general considerations, not methods reported for this trial. The registry record does not identify a count model, a recurrent-event method, an over-dispersion assessment, or a plan for handling participant-level clustering. Without those details and the event-level records, no such analysis can be attributed to the study.

What do the dose cohorts imply for analysis?

In general, a dose-escalation study has a different analytic purpose from a large comparative efficacy trial. Dose cohorts can be designed to describe tolerability and pharmacology at successive dose levels, while placebo groups provide context for observed events. Cohort size and the number of events may be insufficient to estimate rare risks precisely. The registry does not report a sample-size justification, an interim analysis plan, or a prespecified method for combining cohorts. It is therefore not possible to infer whether the study was designed to detect a particular difference in adverse-event counts.

The record includes planned pharmacokinetic and pharmacodynamic outcomes, but it does not provide their numerical results or any analysis linking those measures to safety events. Such relationships should not be inferred from the adverse-event counts alone.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 36 outcome measures and 35 secondary outcomes posted. The secondary outcomes include pharmacokinetic measures such as maximum plasma concentration and time to maximum plasma concentration for single and multiple ascending doses, including steady-state measures. The record does not report results for those outcomes in the information available for this analysis.

When a study examines multiple outcomes, dose cohorts, or time points, the chance of at least one apparently notable result can increase if many tests are conducted. General statistical approaches include prespecifying a primary comparison, adjusting for multiple testing, or treating secondary analyses as exploratory. The registry record does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or a detailed analysis plan. There are also no inferential analysis results posted for the primary event-count outcome. Accordingly, no claim about statistical significance across outcomes or dose groups can be made from the record.

The registry provides the number assigned to each listed arm and states that the number analyzed does not differ from the number assigned for the primary endpoint. However, it does not report a detailed missing-data analysis, participant-level disposition, or how incomplete follow-up was handled. The record does not describe an intention-to-treat or per-protocol analysis population, nor does it state how participants with missing outcome information would be treated. These matters are not reported in the registry record and should not be inferred from the posted group counts.

In general, missing-data methods depend on why observations are absent and on the estimand being targeted. With safety outcomes, incomplete observation can reduce the opportunity to record events. Without participant-level follow-up information or a stated method, the effect of any missingness on the reported counts cannot be assessed.

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 non-serious adverse event terms

The separate adverse-event listing reports zero participants with serious adverse events in every listed active MTR-601 group and in both placebo groups. It also reports zero deaths in every listed group. The number at risk is recorded for each group, including 6 for most active single and multiple ascending dose cohorts, 7 for Multiple Ascending Dose Level 4, 10 for single ascending dose placebo, and 12 for multiple ascending dose placebo.

These entries describe what was recorded during the specified study follow-up. A zero count means no serious adverse event or death was reported in that group in this registry listing; it does not establish that the underlying risk is zero. The cohorts are limited in size, and the record does not provide confidence intervals for the serious-event counts. Rare events may not be observed in a study of this scale.

The listing also reports participants with other, non-serious adverse events. Those counts are not interchangeable with the primary endpoint’s event totals: the primary endpoint counts events, while this listing counts participants affected. The registry does not provide event descriptions, severity breakdowns, or relatedness results in the posted outcome figures. The absence of reported serious events should therefore be read narrowly, rather than as a complete characterization of safety or as evidence about risks outside the study’s observation period.

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

The registry results describe observed adverse-event counts in healthy volunteers receiving single or multiple ascending doses of MTR-601 or placebo. They also report no serious adverse events and no deaths in the listed groups. These findings are descriptive safety observations from a completed phase 1 study. They do not establish the frequency of events in a broader population or during longer-term use.

The posted primary outcome is a count of events. Because participants may experience repeated events, an event count is not equivalent to a count of people affected. The separate listing of participants with other adverse events provides a different measure. Neither measure, as posted, includes confidence intervals or a formal statistical comparison. Differences in group size, dose schedule, and follow-up further limit direct comparisons among the counts.

The study was conducted in healthy volunteers, according to its summary. Although the registry lists muscle spasticity, hypertonia, and several related disorders in its conditions field, the record does not establish that the trial enrolled people with those conditions. The results therefore should not be interpreted as evidence about clinical effects in those patient populations. The record’s stated interests in pharmacokinetics, muscle strength, muscle biopsy, and other markers are objectives; numerical results for those measures are not reported in the registry record.

These data do not show whether MTR-601 is effective for any condition, whether a particular TEAE was caused by treatment, or whether the event burden differs reliably between active treatment and placebo. They also do not establish that no unobserved or uncommon safety concerns exist. The appropriate reading is limited to the recorded events and the study’s defined follow-up, with uncertainty remaining because inferential results and detailed event information are not reported.

13. Why This Trial Matters Statistically

Early-phase safety studies often present outcomes as counts across dose cohorts rather than as a single pooled treatment contrast. That structure makes it important to identify what each number counts, which participants contribute to it, and over what observation period events were collected. In this study, the primary endpoint is an event count, while the separate adverse-event listing reports participants affected. Confusing those quantities would change the meaning of the results.

The trial also illustrates why cohort-level counts require context. The study includes single and multiple ascending dose groups, fed and fasted conditions for a single-dose cohort, separate placebo groups, and different stated follow-up windows. Group sizes and observation periods are not uniform across all cohorts. Raw counts alone therefore do not provide a clean comparison of rates or a test of a dose-response relationship.

Reporting zero serious adverse events and zero deaths is useful descriptive information, but small cohort counts cannot rule out uncommon outcomes. Precision matters: an observed zero is not equivalent to proof of no risk. The registry does not post uncertainty intervals or an inferential analysis for these safety counts, which limits quantitative interpretation.

Finally, the record lists pharmacokinetic and pharmacodynamic objectives alongside safety outcomes, but does not provide corresponding numerical results. This distinction between stated aims and posted findings is central to evidence appraisal. Statistical interpretation should follow the outcomes and analyses actually reported, rather than treating the presence of an objective as evidence that it was achieved or that a clinical benefit was demonstrated.

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 provides aggregate outcome counts rather than event-level data. It does not identify the types of TEAEs, their severity distribution, or the results of the relatedness assessments described in the endpoint definition. Without those details, the total count cannot distinguish mild from severe events or events judged related from those judged not related.

Counts are reported across multiple dose cohorts and schedules, with different group sizes and follow-up windows. The record does not provide an exposure-adjusted rate or an analysis accounting for these differences. Nor does it report a method for handling repeated events within participants. As a result, direct comparisons of raw counts across cohorts may be misleading.

The registry does not report confidence intervals, p-values, a statistical analysis plan, a sample-size justification, or interim-analysis details. The posted analysis count is 0, so the listed results do not support inferential conclusions about differences between active treatment and placebo or across dose levels. The record also does not report detailed missing-data handling or define analysis populations beyond stating that the analyzed count does not differ from the assigned count for the primary endpoint.

The study summary describes healthy volunteers, limiting the direct relevance of these safety observations to people with the disorders listed in the conditions field. Numerical results for the pharmacokinetic and pharmacodynamic objectives are not reported in the registry record. Finally, the registry’s reporting does not establish how completely adverse events were captured beyond the stated follow-up periods, or what may have occurred outside them.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals — NCT06117020.
• Registry record last updated 2026-06-05; status last verified 2026-05.
• Results first posted 2026-06-05.
• 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

NCT06117020 is a completed phase 1 randomized, quadruple-masked, placebo-controlled, parallel-group study of single and multiple ascending doses of MTR-601 in healthy volunteers. The registry lists 89 enrolled participants and describes safety and tolerability as the main purpose, alongside pharmacokinetic and pharmacodynamic objectives.

The primary outcome is the number of treatment-emergent adverse events, with severity and relatedness specified in the outcome definition. The posted counts vary by dose cohort; the separate adverse-event listing reports zero serious adverse events and zero deaths across the listed groups. The primary counts are events, while the separate non-serious adverse-event figures are participants affected.

The registry record does not report inferential comparisons, uncertainty intervals, event-level details, or numerical results for the listed pharmacokinetic and pharmacodynamic objectives. The results are therefore descriptive and should be interpreted in light of the cohort structure and differing follow-up periods.