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 Tesamorelin in Chronic Obstructive Pulmonary Disease (COPD) Subjects With Muscle Wasting (NCT01388920), a randomized phase 2 study. The primary outcome is Change From Baseline in Lean Body Mass at 6 Months.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01388920 |
| Phase | Phase 2 |
| Condition | Chronic Obstructive Pulmonary Disease (COPD) |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Theratechnologies |
| Results posted | Yes |
2. Clinical Question
The study asked whether tesamorelin, used in conjunction with exercise training, affected lean body mass in stable ambulatory people with chronic obstructive pulmonary disease (COPD) and muscle wasting. The planned primary assessment was the change from baseline in lean body mass at 6 months, measured by dual-energy X-ray absorptiometry (DXA). The record describes two tesamorelin doses and a placebo group.
Population
Stable ambulatory COPD subjects with muscle wasting. The registry record reports enrollment of 3 participants.
Intervention
Tesamorelin at 2 mg/day or 3 mg/day. The study objective describes tesamorelin in conjunction with exercise training; details of the exercise training are not reported in the registry record.
Comparator
Placebo. The record lists placebo as a study intervention but does not describe further comparator procedures.
Primary question
What was the change from baseline in DXA-measured lean body mass at 6 months? The registry record does not report a numerical result for this endpoint.
This is therefore a question about the planned comparison, not a completed estimate of treatment effect. The study was halted approximately 1 month after patient enrollment, well before the stated 6-month primary assessment. The posted record does not provide an endpoint estimate that would answer the question.
3. Trial Design
This was a Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel study. It planned to assess two tesamorelin doses—2 mg/day and 3 mg/day—alongside a placebo group. The registry record identifies the masking as quadruple. It does not state which roles or groups were masked, or describe the randomization sequence, allocation concealment, or specific procedures used to maintain masking.
In general, random allocation is intended to reduce systematic differences between treatment groups, while a parallel design assigns participants to separate groups rather than having each participant receive multiple study treatments. Blinding can reduce the influence of expectations on care, reporting, and assessment. These are general design aims; the record does not provide enough procedural detail to assess how they were implemented in this study.
The registry reports enrollment of 3 participants and a status of terminated. The primary endpoint was planned for 6 months, but the record says the study was halted approximately 1 month after patient enrollment. It lists a study start of 2011-09 and primary completion of 2011-12. The reason for termination is not reported in the registry record.
The objective describes evaluating tesamorelin in conjunction with exercise training, but the record does not specify the training regimen, how adherence was assessed, or whether training was delivered consistently across groups. The protocol’s intended allocation among the three listed groups and the number enrolled in each group are not reported in the registry record. These omissions matter when interpreting both the planned comparisons and the small amount of posted safety information.
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 | Tesamorelin 2 mg | Tesamorelin 3 mg | Placebo |
|---|---|---|---|
| STARTED | 1 | 1 | 0 |
| COMPLETED | 1 | 1 | 0 |
| NOT COMPLETED | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The primary objective of the study was the change in lean body mass between baseline and Month 6. However, the study was halted approximately 1 month after patient enrollment.
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 | Tesamorelin 2 mg | Tesamorelin 3 mg | Placebo | Total |
|---|---|---|---|---|---|
| Participants analysed | 1 | 1 | 0 | 2 | |
| Age, Continuous | years | 59 (SD 0) | 51 (SD 0) | — | 55 (SD 0) |
| Sex: Female, Male: Female | Participants | 0 | 1 | — | 1 |
| Sex: Female, Male: Male | Participants | 1 | 0 | — | 1 |
| Region of Enrollment: United States | participants | 1 | 1 | — | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Lean Body Mass at 6 Months, measured over 6 months.
| 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 was change from baseline in lean body mass at 6 months. The record describes assessment by dual-energy X-ray absorptiometry (DXA). DXA is used to characterize body composition, and the study selected lean body mass as a measure relevant to its focus on muscle wasting. The registry record does not give a unit for the endpoint or further details about how the measurement was summarized.
In general, a change-from-baseline endpoint compares a participant’s measurement at follow-up with that participant’s starting measurement. This can make the question directly about change over the study period, although the interpretation depends on the measurement method, the analysis population, and the statistical model. Lean body mass measured by DXA is a body-composition measure; it is not a direct measurement of muscle strength or exercise capacity. The registry lists exercise capacity and peripheral muscle strength among the secondary outcomes, alongside patient-reported outcomes, adverse events, COPD exacerbations, and plasma glucose.
The endpoint was intended to be assessed at 6 months. Because the study was halted approximately 1 month after patient enrollment, the registry record does not report a numerical primary endpoint result. It also does not report participant-level measurements, a group-level change, an estimate of the contrast between groups, or its uncertainty. The endpoint’s planned role can be described, but its observed effect cannot be evaluated from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted record establishes the intended clinical question and the study’s early termination, but it does not provide an efficacy estimate for the primary endpoint. The primary outcome is change from baseline in lean body mass at 6 months, measured by DXA. The record says the study was halted approximately 1 month after patient enrollment. It lists enrollment of 3 participants, but gives no numerical result for the planned 6-month assessment.
There is consequently no posted between-group estimate to interpret, and no reported confidence interval or p-value for the primary endpoint. A confidence interval, in general, describes the statistical uncertainty around an estimated effect; without an estimate and its interval, the precision and range of effects compatible with the data cannot be assessed. The record also contains no analysis entries. It does not show a completed statistical comparison for either tesamorelin dose versus placebo.
The registry’s status is terminated, but the reason for termination is not reported in the registry record. Nor does the record say whether any measurements were collected before the halt, whether participants completed any interim assessments, or how any such measurements were handled. The stated 6-month endpoint should therefore be read as the planned assessment, not as evidence that the assessment was completed.
Enrollment of 3 participants is the total listed for the study, not a basis for inferring what happened in each group. The record does not state group-specific enrollment for the efficacy endpoint, and it does not provide a reported analysis population. The posted adverse-event denominators are not a substitute for efficacy results: they describe the safety information displayed in the registry, not an estimate of change in lean body mass.
The calculated tables and plots on the page should be read in light of this absence of outcome estimates. A visual display cannot supply a missing comparison. Any sample-size illustrations are planning calculations under stated assumptions; they are not observed power and do not establish that this terminated study could detect an effect. The record does not report the original sample-size justification or the study’s statistical analysis plan.
Overall, the record supports a description of the planned Phase 2 design and the fact that the trial stopped early, but not a conclusion about the direction, size, or precision of an effect on lean body mass. It also cannot establish whether the study’s secondary outcomes changed or whether any difference between groups was statistically distinguishable.
9. Statistical Methods Explained
The registry describes a randomized, double-blind, placebo-controlled, parallel Phase 2 study with two tesamorelin dose groups and a placebo group. Those design labels describe how a trial is intended to compare interventions; they do not, by themselves, show what analysis was performed or what results were obtained. The registry record does not report a statistical analysis plan, a model for the primary endpoint, or analysis entries.
What randomization contributes
In general, randomization assigns participants to groups by chance. Its purpose is to make treatment assignment less dependent on participant characteristics or investigator judgment. When enrollment is very limited, however, randomization cannot ensure that groups are similar in all relevant characteristics. Here, the registry lists enrollment of 3 participants but does not report the number assigned to each group or provide baseline characteristics by group. It is not possible to assess balance or the practical implementation of randomization from the posted record.
The parallel design means that different participants were assigned to the tesamorelin 2 mg/day, tesamorelin 3 mg/day, or placebo group, rather than each participant necessarily receiving each option. This is the general meaning of a parallel-group design. The registry does not provide further details about treatment duration, adherence, or the exercise-training procedures described in the study objective.
What masking means
The record describes the study as double-blind and lists masking as quadruple. In general, masking is intended to limit the influence of knowledge about assigned treatment on participants, investigators, outcome assessment, or other study processes. The record does not identify which parties were masked or describe how masking was maintained. It also does not say whether masking was assessed or broken during the study. The labels alone cannot establish whether masking was successful.
How the primary endpoint would be interpreted
The primary endpoint was change from baseline in lean body mass at 6 months by DXA. In general, a treatment comparison for a change endpoint might summarize change within each group and compare those changes between groups, or use another prespecified model that accounts for baseline measurements. Which approach was planned is not reported in the registry record. No group-level changes, between-group estimates, confidence intervals, or p-values for this endpoint are posted.
A confidence interval is a way to communicate uncertainty around an estimate. It helps distinguish a result that is estimated precisely from one that remains compatible with a broad range of effects. A p-value, in general, measures how unusual data would be under a specified statistical null model; it does not measure the size or clinical importance of an effect. Neither kind of result is reported for the primary outcome here, so there is no numerical inferential result to interpret.
Why the early halt changes the analysis context
The primary time point was 6 months, while the record says the study was halted approximately 1 month after patient enrollment. This limits what can be inferred about the planned endpoint from the registry record. The record does not state whether any participants had follow-up measurements, whether an early assessment was planned, or whether any analysis was conducted before termination. The reason for the halt and any decision process around it are also not reported in the registry record.
In general, early termination can reduce the amount of information available and may leave planned outcomes unmeasured. It does not, on its own, establish why a study stopped or the direction of any treatment effect. The registry does not report an interim analysis plan, stopping boundaries, or any interim efficacy or safety analysis, so none should be assumed.
How to read the planning illustrations
The sample-size section presents planning illustrations under a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The illustration is based on fractions of an observed effect, with the required effect described as a stated fraction of that observed effect. These are assumptions for planning calculations, not evidence that the trial used those assumptions or that it had a particular design target. The registry record does not report the original sample-size justification, target enrollment, or statistical plan. Planning sample sizes are not observed power, and they cannot replace missing outcome data.
The stated illustration describes participants per arm for non-time-to-event outcomes and total events for time-to-event tables. It should be treated as a separate analytical aid, not as an analysis of this study. Because the registry record does not report a numerical primary effect, any planning illustration should not be read as a confirmed study result or as a measured effect of tesamorelin.
What cannot be recovered from the record
The registry lists 7 outcome measures and no analysis entries. It does not report model specifications, covariate adjustment, handling of repeated measurements, an analysis population, missing-data methods, or multiplicity procedures. These details determine how an estimate is constructed and how uncertainty is assessed. Without them—and without posted primary outcome results—the reader can describe the planned design but cannot reproduce or evaluate a treatment-effect analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 7 outcome measures. The primary endpoint is change from baseline in lean body mass at 6 months. Listed secondary outcomes include exercise capacity, peripheral muscle strength, patient-reported outcomes, adverse events, COPD exacerbations, and plasma glucose. The record does not report a plan for controlling the chance of false-positive findings across multiple outcomes, doses, or time points. It also does not report a hierarchy of tests or identify which secondary outcomes, if any, were intended to support confirmatory claims.
In general, when researchers examine several outcomes or comparisons, the opportunity for a chance finding can increase. Multiplicity methods can address this, but the appropriate approach depends on the prespecified questions and analysis plan. No such methods are reported in the registry record, and there are no posted analysis entries from which to assess how multiplicity was handled.
Missing-data procedures and analysis populations are also not reported in the registry record. The record does not state whether the intended primary analysis included all randomized participants, only participants with follow-up measurements, or another population. It does not describe how missing baseline or follow-up measurements would be handled. These omissions are particularly relevant because the trial was halted approximately 1 month after patient enrollment, before the planned 6-month primary assessment.
The record does not report an interim analysis plan or whether any outcome data were reviewed before termination. It does not describe stopping rules or the reason for stopping. These are unknowns, not evidence that a particular procedure was or was not used outside what is documented in the registry.
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 |
|---|---|---|---|
| Tesamorelin 2 mg | 0 | 1 | 0.0% |
| Tesamorelin 3 mg | 0 | 1 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Tesamorelin 2 mg | 0 of 1 |
| Tesamorelin 3 mg | 0 of 1 |
| Placebo | 0 of 0 |
The posted serious adverse-event figures list 1 participant at risk and 0 affected in the Tesamorelin 2 mg group, and 1 at risk and 0 affected in the Tesamorelin 3 mg group. For placebo, the record lists 0 at risk and 0 affected. The posted non-serious adverse-event figures show the same group-specific at-risk and affected counts. These are the figures displayed in the registry record; they should not be interpreted as a complete safety comparison.
The registry reports enrollment of 3 participants overall, but does not explain the relationship between overall enrollment and the group-specific denominators shown in the adverse-event record. The exposure duration and completeness of adverse-event collection are not reported in the registry record. The study was halted approximately 1 month after patient enrollment, while the primary endpoint was planned for 6 months. The available counts therefore do not establish how much observation time was available in each group.
Death counts are not reported in the registry record. A displayed count of 0 affected for the listed adverse-event categories is not proof that an intervention is free of risk, especially with the limited information and follow-up. The registry does not provide event narratives or a broader safety analysis.
12. What the Result Does, and Does Not, Mean
This record tells readers that a Phase 2 trial was planned to compare tesamorelin at 2 mg/day and 3 mg/day with placebo in stable ambulatory COPD subjects with muscle wasting. Its primary question concerned change from baseline in DXA-measured lean body mass at 6 months, with the stated objective involving exercise training. The registry reports that the study was terminated and halted approximately 1 month after patient enrollment.
What it does not provide is a numerical estimate of change in lean body mass, a comparison between any tesamorelin group and placebo, or an uncertainty interval for such a comparison. There is no reported primary endpoint analysis in the registry record. As a result, the posted information cannot establish the direction or size of an effect, or the precision with which an effect might have been estimated.
Likewise, the record does not provide numerical results for the listed secondary outcomes. The posted adverse-event counts are limited and do not amount to a comprehensive safety assessment. The reason the study was stopped is not reported in the registry record, so the termination should not be attributed to efficacy, safety, recruitment, or any other cause.
In general, a trial’s design describes how researchers intended to answer a question; it is not itself evidence for an answer. Randomization, placebo control, and masking can help structure a comparison, but interpretation also requires adequate outcome data and transparent analysis methods. Here the registry gives design information, a small overall enrollment, and a report of early halting, but does not provide the outcome results needed to judge the planned comparison.
The appropriate reading is therefore limited: this is a record of a planned clinical comparison that did not reach its stated primary assessment as described in the registry. It does not support a conclusion that tesamorelin did or did not change lean body mass, exercise capacity, muscle strength, or any other outcome.
13. Why This Trial Matters Statistically
This trial matters statistically because its design and its available results are quite different things. The registry describes randomization, masking, a placebo comparator, and a primary endpoint selected before the planned 6-month assessment. Those elements define a structured research question. Yet the record also reports termination after enrollment began and provides no numerical primary endpoint comparison. Readers need to distinguish a trial’s intended ability to answer a question from the evidence that is actually posted.
The record also illustrates why enrollment and follow-up are essential to interpretation. It lists 3 participants overall, while the primary assessment was planned for 6 months and the study was halted approximately 1 month after patient enrollment. No numerical result or analysis is reported for the endpoint. Even a well-described design cannot yield an interpretable treatment-effect estimate when the registry record does not include the relevant outcome data.
A further statistical lesson concerns uncertainty. In general, an effect estimate and its confidence interval help readers understand both the estimated contrast and its precision. Neither is available for the primary outcome. The absence of a reported estimate is not evidence of no effect; it means the record does not provide a basis for quantifying the effect.
Finally, the record cautions against treating planning illustrations as trial results. Sample-size calculations describe what might be needed under stated assumptions. They are not observed power, and they do not recover an unreported outcome. Keeping these distinctions clear helps readers avoid overinterpreting an early-terminated study while still recognizing what its registry record does document.
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 main limitation for interpretation is that the registry record contains no numerical result for the primary endpoint. The planned outcome was change from baseline in lean body mass at 6 months, but the study was halted approximately 1 month after patient enrollment. The record does not say whether any follow-up measurements were collected or whether a separate analysis was performed.
Enrollment was 3 participants overall. The record does not give group-specific enrollment for the primary outcome, baseline characteristics, or a reported analysis population. It therefore does not allow readers to assess group comparability or determine which participants contributed to any outcome analysis. No analysis entries are listed.
The record does not report the reason for termination, the original target enrollment, a sample-size justification, or the statistical analysis plan. It also does not report the randomization procedure, the specific parties masked, the exercise-training regimen, or how treatment adherence was assessed. These details limit assessment of implementation and of the intended estimand.
For outcomes, the record does not provide estimates, measures of uncertainty, or methods for handling missing data. It does not describe multiplicity control, an interim analysis plan, stopping rules, or analysis populations. Death counts are not reported, and the safety denominators displayed do not resolve the broader limitations in duration and completeness of follow-up. These gaps mean that neither efficacy nor safety can be characterized beyond the information explicitly posted.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Efficacy and Safety Study of Tesamorelin in Chronic Obstructive Pulmonary Disease (COPD) Subjects With Muscle Wasting — NCT01388920.
- Registry record last updated 2022-03-18; status last verified 2022-03.
- Results first posted 2014-05-07.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01388920 is a Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel study sponsored by Theratechnologies. It planned to compare tesamorelin 2 mg/day and 3 mg/day with placebo in stable ambulatory COPD subjects with muscle wasting. The stated primary endpoint was change from baseline in DXA-measured lean body mass at 6 months.
The registry lists a study start of 2011-09, primary completion of 2011-12, enrollment of 3, and status of terminated. It says the study was halted approximately 1 month after patient enrollment. The registry record does not report numerical primary outcome results, analysis entries, the reason for termination, or the statistical analysis plan. The posted safety figures list 0 affected in the displayed adverse-event categories, with limited at-risk counts; death counts are not reported.