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 Short-term Effects of Leptin in People With Lipodystrophy (NCT01778556), a non-randomized phase 2 study. The primary outcome is Total Body Insulin Sensitivity.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01778556 |
| Phase | Phase 2 |
| Condition | Lipodystrophy |
| Allocation | Non-Randomized |
| Design model | Crossover |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
| Results posted | Yes |
2. Clinical Question
Population
People with lipodystrophy were eligible; the registry summary gives an age range of 14 to 70 years. The record lists enrollment of 25. It does not report outcome results separately by participant characteristics, so the extent to which the recorded population represents people with lipodystrophy more broadly cannot be judged from these details.
Intervention
The intervention was metreleptin, a biological intervention. One described sequence was a 5-day period without metreleptin followed by 14 days taking it. The study summary describes an inpatient evaluation, regular blood and urine testing, and tests related to insulin, sugar and fat metabolism, and hormones.
Comparator
The other described sequence began with 5 days while taking metreleptin and continued with 14 days of withdrawal. The registry labels the study non-randomized and unmasked, and cautions that the arms are not a like-for-like control comparison. The groups therefore should not be read as randomized treatment and control groups.
Primary question
The primary outcome was total body insulin sensitivity, measured as glucose disposal rate during a hyperinsulinemic, euglycemic clamp. The record lists assessment during Intervention 1 (5 days), Intervention 2 (14 days), and long-term follow-up (6 months). Numerical outcome values and an estimated contrast are not reported in the registry record, so the direction and precision of any change cannot be described.
The clinical question is whether measurements of insulin sensitivity differ across the metreleptin exposure and withdrawal periods described in this study. Because allocation was non-randomized and the sequences differed, a comparison between the arms would not have the protection against baseline differences that random assignment can provide. The registry also does not report a numerical primary outcome result, a confidence interval, or a statistical test. Thus, the record identifies what was measured and when, but does not establish the size, direction, or precision of a change associated with metreleptin exposure or withdrawal.
3. Trial Design
This completed phase 2 study enrolled 25 people and is recorded as non-randomized, unmasked, and crossover. The registry describes two sequences: participants in the Leptin naive arm were studied for 5 days without metreleptin and then for 14 days while taking metreleptin; participants in the On-leptin arm were studied for 5 days while taking metreleptin and then for 14 days during withdrawal. The brief summary describes an inpatient stay of 19 days at the National Institutes of Health Clinical Center. It also describes regular blood and urine tests and metabolic and hormone assessments.
The crossover label signals that measurements were planned across periods in which treatment status changed for at least one sequence. It does not make the study randomized: the registry explicitly records non-random allocation and warns that the arms are not a like-for-like control comparison. The record does not report how participants were assigned to the sequences, whether sequence order was determined by a specific rule, or whether period effects were handled in the analysis.
The primary outcome was assessed during Intervention 1 (5 days), Intervention 2 (14 days), and long-term follow-up (6 months). The registry lists the study as completed, with a start date of 2013-01-26 and primary completion on 2018-02-23. Those dates describe the registry timeline; they do not supply outcome results. Numerical results for the primary outcome and statistical analysis details are not reported in the registry record.
The arm descriptions and outcome timeframes define the intended comparison structure, but they do not reveal how many participants contributed data at each time point. The registry notes one withdrawal in the Leptin naive arm at Period 2 (14 days), due to a protocol violation. Other details about follow-up completeness and analysis populations 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.
Intervention 1 (5 Days)
| Stage | Leptin Naive | On-leptin |
|---|---|---|
| STARTED | 15 | 10 |
| COMPLETED | 15 | 8 |
| NOT COMPLETED | 0 | 2 |
Reasons for not completing this period:
| Reason | Leptin Naive | On-leptin |
|---|---|---|
| Protocol Violation | 0 | 1 |
| Withdrawal by Subject | 0 | 1 |
Intervention 2 (14 Days)
| Stage | Leptin Naive | On-leptin |
|---|---|---|
| STARTED | 15 | 8 |
| COMPLETED | 14 | 8 |
| NOT COMPLETED | 1 | 0 |
Reasons for not completing this period:
| Reason | Leptin Naive | On-leptin |
|---|---|---|
| Protocol Violation | 1 | 0 |
Long-term Follow-up
| Stage | Leptin Naive | On-leptin |
|---|---|---|
| STARTED | 15 | 0 |
| COMPLETED | 14 | 0 |
| NOT COMPLETED | 1 | 0 |
Reasons for not completing this period:
| Reason | Leptin Naive | On-leptin |
|---|---|---|
| noncompliance with metreleptin | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: One patient at Period 2 (14 days) in Leptin naive arm was withdrew due to protocol violation.
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 | Leptin Naive | On-leptin | Total |
|---|---|---|---|---|
| Participants analysed | 15 | 8 | 23 | |
| Age, Continuous | years | 32 (SD 17) | 25 (SD 6) | 28 (SD 14) |
| Sex: Female, Male: Female | Participants | 12 | 5 | 17 |
| Sex: Female, Male: Male | Participants | 3 | 3 | 6 |
| Race/Ethnicity, Customized: White | Participants | 9 | 4 | 13 |
| Race/Ethnicity, Customized: Hispanic | Participants | 4 | 2 | 6 |
| Race/Ethnicity, Customized: African American | Participants | 0 | 2 | 2 |
| Race/Ethnicity, Customized: Asian | Participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Other | Participants | 1 | 0 | 1 |
| Type of lipodystrophy: Generalized | Participants | 3 | 8 | 11 |
| Type of lipodystrophy: Partial | Participants | 12 | 0 | 12 |
| Subtype of lipodystrophy: Congenital generalized lipodystrophy | Participants | 3 | 7 | 10 |
| Subtype of lipodystrophy: Familial partial lipodystrophy | Participants | 12 | 0 | 12 |
| Subtype of lipodystrophy: Acquired generalized lipodystrophy | Participants | 0 | 1 | 1 |
| Endogenous leptin level | ng/dl | 9.5 (SD 10.2) | 1.2 (SD 0.5) | 7.4 (SD 10.1) |
6. Primary Endpoint
The registry lists the primary outcome as Total Body Insulin Sensitivity, measured over Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months).
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | mg/kg fat-free mass/min |
| Endpoint classification | Continuous Endpoint |
Total body insulin sensitivity was the primary endpoint. The registry defines its measurement as glucose disposal rate during a hyperinsulinemic, euglycemic clamp, with the unit mg/kg fat-free mass/min. In general, a clamp is a controlled metabolic measurement: insulin is infused while glucose is maintained at a target level, and glucose disposal is used to assess the body’s response to insulin. This explains why the measure is relevant to a study focused on metabolic effects in lipodystrophy; it is a physiological measure rather than a report of a clinical event or symptom.
The listed outcome type is continuous, with a mean as the parameter and standard deviation as the dispersion measure. In general, a mean describes the average measurement in a group, while a standard deviation describes how spread out individual measurements are around that average. These quantities can help characterize both the typical measured level and participant-to-participant variability.
The registry names three assessment windows: Intervention 1 (5 days), Intervention 2 (14 days), and long-term follow-up (6 months). The measure could therefore describe short-term values across the treatment-status periods and a later follow-up value. However, numerical means, standard deviations, changes, and comparisons are not reported in the registry record. The endpoint definition alone does not establish whether insulin sensitivity changed or how precisely any change was estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the study question, intervention sequences, and primary outcome, but it does not report numerical primary outcome results. No group means, standard deviations, estimated differences, confidence intervals, or p-values are reported in the registry record. As a result, the posted information does not show the direction or magnitude of the measured insulin-sensitivity values across the intervention periods, nor does it show how uncertain any comparison would be.
That absence matters for interpretation. An outcome definition explains what researchers intended to measure; it is not itself a finding. Likewise, study completion and an enrollment entry do not indicate whether the primary outcome changed. Without values and an analysis, there is no basis in this record for saying whether measured glucose disposal was higher, lower, or similar between periods or sequences.
The non-randomized allocation also limits what a simple comparison between the Leptin naive and On-leptin arms could establish. The registry itself cautions that the arms are not a like-for-like control comparison. In general, randomization helps balance measured and unmeasured participant characteristics across treatment groups. Without it, differences between groups may reflect baseline or sequence differences as well as treatment status. The record does not report baseline outcome values or an adjusted analysis that would clarify the influence of such differences.
The crossover structure may permit comparisons within participants across treatment-status periods, but that interpretation depends on the actual analysis and on how time and order are handled. In general, measurements taken at different times can be influenced by period effects, changes unrelated to the intervention, or residual effects from an earlier period. The registry record does not report whether these issues were assessed or how they were addressed. It would therefore be inappropriate to presume that the posted design label alone resolves them.
The registry lists one participant in the Leptin naive arm as withdrawn at Period 2 (14 days) due to a protocol violation. The record does not explain how that observation was handled in the primary analysis, or whether other measurements were missing. Missing observations can affect both the estimated result and its precision, especially when missingness is related to participants’ health or study experience. That impact cannot be assessed without analysis-population and missing-data details.
The safety record lists serious and other adverse-event figures for several time periods and sequences, but these are not efficacy results. The adverse-event information is useful as a record of reported safety events; it does not supply an estimate of the primary metabolic outcome. Overall, the registry supports a description of the study’s intent and design, but does not permit a statistical conclusion about the primary endpoint.
9. Statistical Methods Explained
The primary outcome is continuous, and the registry specifies a mean and standard deviation. In general, the mean is the arithmetic average of the recorded measurements, while the standard deviation describes how dispersed those measurements are around the mean. A mean can provide an interpretable summary of a group’s measurements, but it does not show the full distribution: participants may have values spread broadly or tightly around the average. The standard deviation helps describe that variability, but it is not a confidence interval for the mean and does not by itself quantify uncertainty in a comparison.
A comparison of means would ordinarily need to identify the contrast being estimated. For this study, possible contrasts might concern different intervention periods or sequences, but the registry record does not report the actual primary analysis or a numerical contrast. It also does not report a statistical model, an adjustment set, or a test statistic. Consequently, no particular method should be attributed to the investigators based only on the outcome type and design labels.
What does a confidence interval tell a reader?
In general, a confidence interval gives a range of estimates compatible with the observed data under the method’s assumptions. Its width reflects precision: a narrower interval indicates greater precision than a wider interval, all else equal. The interval also helps readers assess which effect sizes remain plausible, rather than reducing interpretation to whether a p-value crosses a threshold. No confidence interval for the primary endpoint is reported in the registry record, so its precision cannot be assessed.
Why does the study structure matter?
A crossover study observes participants across more than one treatment-status period. In general, within-participant comparisons can account for stable characteristics of each participant, because those characteristics do not change between that participant’s measurements. But period and sequence can still matter. Measurements may change over time for reasons unrelated to treatment status, and an earlier treatment period may influence a later measurement. These are general considerations, not evidence that such effects occurred in this study. The registry does not report whether the primary analysis modeled period, sequence, or possible carryover effects.
The non-randomized allocation further affects interpretation. In general, random assignment supports comparable treatment groups by distributing baseline factors without systematic selection. With non-random allocation, participant characteristics or clinical circumstances may differ between sequences. The registry explicitly cautions against treating the arms as a like-for-like control comparison. No baseline comparison or adjustment method is reported in the registry record.
How should the planning illustrations be read?
The registry describes sample-size illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. The illustrations are framed around detecting a stated fraction of the observed effect. In general, a planning calculation asks how many participants per arm would be needed under specified assumptions to attain a chosen power for a target effect. It is a hypothetical design exercise, not a count of observed participants and not proof that the completed study had that power.
These illustrations should not be read as the trial’s original design justification or as an observed-power analysis. The registry does not report the trial’s own sample-size justification. Because numerical primary outcome results are not reported in the registry record, the effect underlying any such illustration cannot be independently interpreted from this record. Any planning figure also depends on assumptions about variability, allocation, and the statistical model; departures from those assumptions can change the required sample size.
What does the safety count represent statistically?
Adverse-event summaries are descriptive counts over specified risk groups and study periods. They do not automatically provide a comparative estimate of treatment-related risk. In general, the interpretation of such counts depends on the definition of an event, the observation period, the number at risk, and whether the same participant can contribute events across periods. The registry reports counts at risk and affected for its listed groups, but does not provide a comparative statistical analysis of those figures. Small counts, in particular, give limited information about the range of plausible event rates; no rate or risk difference should be inferred by recalculating the listed numbers.
The record reports no analyses in its analysis listing and no numerical primary outcome results. Statistical concepts can explain how a complete analysis might be interpreted, but they cannot fill in results absent from the registry. In this case, the correct statistical reading is descriptive: the endpoint and design are specified, while the primary estimate, its uncertainty, and the analysis method are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary outcome and two secondary outcome names: Insulin-mediated Suppression of Hepatic Glucose Production and Endogenous Rate of Appearance of Palmitate. It also lists three outcome measures overall, but does not provide numerical outcome results or analysis entries. The analysis listing reports 0 analyses. The statistical plan, including any prespecified testing sequence or adjustment for multiple comparisons, is not reported in the registry record.
In general, multiplicity arises when investigators conduct several statistical tests or examine several outcomes, time points, or contrasts. Repeated testing can increase the chance of at least one apparently unusual result, so interpretation may depend on whether outcomes and comparisons were selected in advance and whether adjustments were planned. These are general considerations; the registry does not establish what multiplicity procedures were used for this study. The listed outcomes and timeframes alone are not enough to infer a testing strategy.
The registry notes that one patient at Period 2 (14 days) in the Leptin naive arm was withdrawn due to a protocol violation. It does not report how that participant’s data were handled, what analysis population was used, or whether additional observations were missing. It also does not describe an imputation method, a sensitivity analysis, or a missing-data assumption. These details are not reported in the registry record.
In general, an analysis population specifies which participants or measurements contribute to an estimate, while a missing-data approach describes how absent measurements are handled. Those choices can affect both an estimate and its precision. With no outcome results or analysis-population description, readers cannot determine the extent or statistical impact of missing data.
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 |
|---|---|---|---|
| Intervention 1 (5 Days) : Leptin Naive | 0 | 15 | 0.0% |
| Intervention 2 (14 Days): Leptin Naive | 1 | 15 | 6.7% |
| Long-term Follow-up (6 Months): Leptin Naive | 2 | 15 | 13.3% |
| Intervention 1 ( 5 Days) : On-Leptin | 0 | 8 | 0.0% |
| Intervention 1 ( 14 Days): On-Leptin | 0 | 8 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Intervention 1 (5 Days) : Leptin Naive | 0 of 15 |
| Intervention 2 (14 Days): Leptin Naive | 0 of 15 |
| Long-term Follow-up (6 Months): Leptin Naive | 0 of 15 |
| Intervention 1 ( 5 Days) : On-Leptin | 0 of 8 |
| Intervention 1 ( 14 Days): On-Leptin | 0 of 8 |
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 |
|---|---|
| Intervention 1 (5 Days) : Leptin Naive | 0 of 15 |
| Intervention 2 (14 Days): Leptin Naive | 2 of 15 |
| Long-term Follow-up (6 Months): Leptin Naive | 4 of 15 |
| Intervention 1 ( 5 Days) : On-Leptin | 0 of 8 |
| Intervention 1 ( 14 Days): On-Leptin | 0 of 8 |
Most frequent serious adverse event terms
| Event term | Organ system | Intervention 1 (5 Days) : Leptin Naive | Intervention 2 (14 Days): Leptin Naive | Long-term Follow-up (6 Months): Leptin Naive | Intervention 1 ( 5 Days) : On-Leptin | Intervention 1 ( 14 Days): On-Leptin |
|---|---|---|---|---|---|---|
| abdominal pain of unknown etiology | Gastrointestinal disorders | 0 of 15 | 0 of 15 | 1 of 15 | n/a of 8 | n/a of 8 |
| angioedema secondary to angiotensin- converting enzyme inhibitor use | Cardiac disorders | 0 of 15 | 0 of 15 | 1 of 15 | n/a of 8 | n/a of 8 |
| anemia secondary to menorrhagia | Endocrine disorders | 0 of 15 | 1 of 15 | 0 of 15 | n/a of 8 | n/a of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Intervention 1 (5 Days) : Leptin Naive | Intervention 2 (14 Days): Leptin Naive | Long-term Follow-up (6 Months): Leptin Naive | Intervention 1 ( 5 Days) : On-Leptin | Intervention 1 ( 14 Days): On-Leptin |
|---|---|---|---|---|---|---|
| decreased appetite | Metabolism and nutrition disorders | 0 of 15 | 0 of 15 | 1 of 15 | n/a of 8 | n/a of 8 |
| weight loss | Metabolism and nutrition disorders | 0 of 15 | 0 of 15 | 1 of 15 | n/a of 8 | n/a of 8 |
| hair loss | Skin and subcutaneous tissue disorders | 0 of 15 | 0 of 15 | 1 of 15 | n/a of 8 | n/a of 8 |
| hypoglycemia (in a subject treated with insulin) | Metabolism and nutrition disorders | 0 of 15 | 0 of 15 | 1 of 15 | n/a of 8 | n/a of 8 |
| injection site reaction | Injury, poisoning and procedural complications | 0 of 15 | 1 of 15 | 0 of 15 | n/a of 8 | n/a of 8 |
| menorrhagia | Endocrine disorders | 0 of 15 | 1 of 15 | 0 of 15 | n/a of 8 | n/a of 8 |
Serious events with the highest arm-level rate: abdominal pain of unknown etiology (6.7%), angioedema secondary to angiotensin- converting enzyme inhibitor use (6.7%), anemia secondary to menorrhagia (6.7%).
The registry reports serious and other adverse-event counts by intervention period and sequence. For the Leptin naive sequence, the serious-event figures are 0 during Intervention 1 (5 Days), 1 during Intervention 2 (14 Days), and 2 during Long-term Follow-up (6 Months); the corresponding other-event figures are 0, 2, and 4. Each of those groups lists 15 at risk. For the On-Leptin sequence, both listed periods have 0 serious and 0 other events; each lists 8 at risk. These are the registry’s period-specific figures, not a calculated comparison between arms.
The registry also lists abdominal pain of unknown etiology, angioedema secondary to angiotensin-converting enzyme inhibitor use, and anemia secondary to menorrhagia among the top serious terms, each at 6.7%. The denominator and the relation of these terms to the period-specific counts are not reported in the registry record. The counts and terms describe what the registry records; they do not establish cause or a comparative risk.
In general, adverse-event counts should be read in the context of the group at risk, observation period, event definitions, and whether repeated events or affected participants are counted. The registry does not provide a formal safety comparison or further detail on event attribution. There were 0 deaths listed in each of the reported groups.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase 2 study of metreleptin in people with lipodystrophy was completed and identifies total body insulin sensitivity as its primary endpoint. It also describes treatment initiation and withdrawal sequences and a later follow-up assessment. These details explain the study’s question and measurement schedule, but they are not results demonstrating a change in insulin sensitivity.
Numerical primary outcome findings are not reported in the registry record. There is no reported mean, standard deviation, comparison estimate, confidence interval, or p-value from which to describe the size, direction, or precision of an outcome difference. The record therefore does not support a conclusion that the outcome increased, decreased, or remained unchanged across the periods.
Even if numerical results were available, the non-randomized allocation and the registry’s warning that the arms are not a like-for-like control comparison would need to guide interpretation. General limitations of non-randomized comparisons include possible differences between groups before treatment. The crossover structure also makes period and sequence important considerations, but the analysis approach for these factors is not reported in the registry record.
The posted adverse-event figures are descriptive safety information. They should not be treated as proof that metreleptin caused or prevented an event, and they do not substitute for a comparative safety analysis. The listed event counts apply to specified study periods and groups; their interpretation depends on definitions and follow-up details that are not fully reported.
In general, a clinical trial result is best interpreted by considering the estimated effect, its uncertainty, the design, and the analysis population together. Here, the design and outcome definition are available, but the numerical efficacy result and key statistical details are not. The registry record therefore permits a description of what was studied, not a conclusion about the magnitude or clinical meaning of an effect.
13. Why This Trial Matters Statistically
This trial is statistically notable for the way treatment status changes across sequences. A crossover structure can offer within-participant information, which in general may reduce the influence of stable differences between people. At the same time, the sequence and timing of measurements become central: changes over time and residual effects from an earlier period can complicate interpretation. The registry identifies the design as crossover but does not report how these issues were analyzed.
The non-randomized allocation is equally important. The record explicitly warns that the arms are not a like-for-like control comparison. That caution prevents a casual interpretation of the two sequences as equivalent groups differing only in treatment. Understanding the rationale for allocation and any adjustment strategy would help assess comparability, but those details are not reported in the registry record.
The study’s primary endpoint is a physiological measure defined through a clamp procedure, with an explicit unit and assessment windows. This specificity helps readers understand what was intended to be measured. Yet precision in endpoint definition cannot replace the missing numerical result: without an estimate and uncertainty measure, the statistical evidence about the primary question cannot be evaluated.
The record also illustrates why registry entries should be read for both design and reporting completeness. It identifies a withdrawal related to a protocol violation and records adverse events by period, while leaving the primary outcome analysis and missing-data handling unreported. Those gaps limit the ability to assess robustness, multiplicity, and the relationship between the design and the reported findings. The principal statistical contribution of the record is therefore clarity about the intended measurement and structure, alongside a clear boundary on what can be concluded from the posted information.
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 primary limitation for statistical interpretation is that numerical efficacy results are not reported in the registry record. Without values for the primary endpoint, readers cannot assess the observed pattern, the size of a contrast, or its precision. The record also does not report a statistical analysis plan, a model, a test, or an adjustment strategy.
The non-randomized allocation limits a direct comparison of the two arms, consistent with the registry’s explicit caution that they are not a like-for-like control comparison. The record does not explain how participants entered each sequence or report baseline measurements that could help assess group comparability.
Although the study is labelled crossover, the analysis handling period, sequence, and possible carryover effects is not reported in the registry record. These issues are general considerations for interpreting measurements taken across treatment-status periods; the record does not show whether they were present or how they were addressed.
The registry identifies one withdrawal at Period 2 (14 days) in the Leptin naive arm due to a protocol violation. It does not report the analysis population, the treatment of that observation, or whether other data were missing. Follow-up completeness and missing-data methods are also not reported in the registry record.
Finally, the safety information is presented as period-specific counts and listed terms, without a reported comparative safety analysis or further detail about event attribution. These limitations do not establish that the study was poorly conducted; they describe what cannot be evaluated from the registry record alone.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Short-term Effects of Leptin in People With Lipodystrophy — NCT01778556.
- Registry record last updated 2019-11-13; status last verified 2019-02-25.
- Results first posted 2019-11-13.
- 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
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18. Record Summary
This completed phase 2 study examined metreleptin initiation or withdrawal in people with lipodystrophy. The registry lists 25 enrolled participants and describes non-randomized, unmasked, crossover sequences. The primary outcome was total body insulin sensitivity, measured as glucose disposal rate during a hyperinsulinemic, euglycemic clamp, with assessments during Intervention 1 (5 days), Intervention 2 (14 days), and long-term follow-up (6 months).
The registry reports one withdrawal at Period 2 (14 days) in the Leptin naive arm due to a protocol violation, and it lists adverse-event counts by period and sequence. Numerical results for the primary endpoint, the statistical analysis method, and details of analysis populations and missing-data handling are not reported in the registry record. The record therefore supports a description of the study design and planned measurement, but not an estimate of the primary outcome effect or its precision.