Hypothyroidism Treated With Calcitonin: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Hypothyroidism Treated With Calcitonin (NCT03342001), a na phase 4 study. The primary outcome is Modified City of Hope Quality of Life Questionnaire for Patients With Thyroid Disease.

2. Clinical Question

The trial’s stated rationale concerns people with hypothyroidism who continue to experience symptoms despite adequate thyroid-hormone treatment. Its primary assessment was a quality-of-life score change during calcitonin use, rather than a comparison of outcomes between randomized groups. Consequently, the central statistical question is descriptive: what change was observed in the people who completed the trial, and how precisely was that change estimated? The registry record identifies the intended endpoint and timeframe but does not report an estimate, dispersion value, confidence interval, or analysis result. Without those results, the size and precision of any observed change cannot be assessed from the record.

3. Trial Design

The registry classifies NCT03342001 as a phase 4, completed, single-group trial with open-label masking and allocation listed as not applicable. Enrollment was 11. The record identifies hypothyroidism as the condition and calcitonin as the intervention. The treatment group is described as receiving calcitonin nasal spray, 200 mcg daily. No separate control or comparison group is described, so the design does not support a like-for-like treatment contrast between groups.

The trial began on 2018-06-18, and the primary completion date is 2019-06-30. The primary assessment timeframe was 6 weeks. The outcome population is described as participants completing the trial, but the count completing is not reported in the registry record. The record also does not report the number who started treatment, adherence, withdrawals, or reasons for any missing endpoint observations.

The primary outcome was a continuous questionnaire score, with the mean and standard deviation specified as the measure and dispersion. Although the registry describes the intended summary, it lists no outcome analyses. The primary score change is not reported in the registry record, and no statistical analysis plan, model, or inferential procedure is reported in the registry record. The sample-size assumptions associated with the page are a planning illustration based on an observed effect; they are not the trial’s own design assumptions and do not establish that the trial was planned as a two-arm study. The actual trial design remained single group. The registry record does not report a protocol-based sample-size justification or an interim-analysis plan.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: participants completing trial

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: people with hypothyroidism

6. Primary Endpoint

The registry lists the primary outcome as Modified City of Hope Quality of Life Questionnaire for Patients With Thyroid Disease, measured over 6 weeks.

The primary endpoint is the change after 6 weeks in score on the Modified City of Hope Quality of Life Questionnaire for Patients With Thyroid Disease. The instrument contains 40 questions, each using a 1-10 scale; higher scores indicate more severe symptoms. The total score can reach 400 points. This endpoint is intended to represent participants’ reported quality of life and thyroid-related symptom burden, rather than a laboratory measure of thyroid function.

Using change in score focuses the assessment on how participants’ reported status differed over the trial period. In a single-group study, however, a change over time is not itself a comparison with what would have happened without calcitonin. Changes could also reflect the passage of time, expectations in an open-label study, other care, or variation in questionnaire responses. These are general interpretive considerations, not findings about this trial.

The registry specifies the mean and standard deviation for the continuous endpoint and identifies participants completing the trial as the outcome population. It does not report baseline or follow-up scores, the mean change, the standard deviation of change, or the number of participants with endpoint data. The endpoint’s intended measurement is therefore clear, but the registry record does not report the result needed to judge the magnitude or precision of change.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a continuous primary outcome summarized by a mean and standard deviation. A mean represents the central value of a set of measurements, while a standard deviation describes how spread out individual measurements are around that mean. For a change endpoint, the value of interest is the difference between each participant’s follow-up and baseline score, summarized across participants. The registry identifies change after 6 weeks as the primary assessment but does not report the values needed to reproduce or inspect that summary.

What does a change score describe?

A change score describes how measurements differ over time within the assessed participants. In a single-group study, the average change can describe the course observed during the treatment period. It cannot by itself establish how the same participants would have changed over the same period without the intervention. A comparator group can help estimate that missing counterfactual contrast, but no separate comparator is described for this trial. This is a general design distinction, not an additional finding about the participants.

Interpretation also depends on the variability of individual changes. A mean change alone would not show whether most participants changed in a similar direction or whether individual changes varied widely. A standard deviation of change can describe that spread. A confidence interval, if reported, would express the range of values compatible with the estimate under the statistical model and its assumptions. The registry record does not report a confidence interval or the underlying change-score dispersion, so precision cannot be assessed from the posted outcome information.

What does the open-label design imply?

Masking is listed as none. As a general consideration, awareness of treatment assignment can affect participant expectations and responses, particularly for self-reported outcomes. An open-label design does not prove that responses were biased; it means that such influences cannot be ruled out by masking. The questionnaire captures reported quality of life and symptom severity, so the interpretation concerns those reports rather than an independently masked assessment.

Because this is a single-group design, statistical methods can describe the observed within-participant change, but no method can turn the record into a randomized comparison. Adjustment for measured variables or a more elaborate model would not, on its own, create an untreated comparison group. The registry record does not report a specific model, test, adjustment strategy, or confidence-interval method.

How should the planning illustration be understood?

The planning illustration is described as using a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation. It considers effects defined as stated fractions of an observed effect. The illustration is not the trial’s own design: the registry classifies the actual study as single group and allocation as not applicable. It must therefore be read as a hypothetical planning exercise, not as a reconstruction of the trial protocol or an analysis of its participants. No planning result should be called observed power. The observed endpoint effect on which the illustration is based is not reported in the registry record.

In general, power calculations estimate how often a planned analysis would meet a chosen statistical criterion under specified assumptions. They depend on assumptions about the effect, variability, allocation, and analysis. A planning calculation does not show that a result was observed, and it does not establish the probability that a particular study finding is true. Here, the planning assumptions describe a hypothetical two-arm setting, whereas the clinical trial record describes a single group. The registry record does not report its own sample-size justification.

What can be concluded from the posted analysis information?

The registry lists one outcome measure and no outcome analyses. It specifies a mean and standard deviation but gives no estimate or uncertainty measure. The methods information therefore supports an account of the intended endpoint and its general statistical interpretation, not an assessment of the observed primary outcome. The analysis population is described as participants completing the trial, but the number in that population is not reported in the registry record. The handling of missing data and any sensitivity analyses are also not reported in the registry record.

These points are general statistical principles applied to the design described in the registry. They do not supply an unreported test, model, result, or assumption. A reader can identify the endpoint and see why a within-group change has a limited causal interpretation, but cannot evaluate the numerical magnitude or precision of the primary result without the posted outcome analysis.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Treatment Group, Open Label")
n   <- c(9)
m   <- c(-36.33)
sp  <- c(39.41)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Treatment Group, Open Label  9  -36.330  39.410  -66.623  -6.037

11. Multiplicity, Missing Data and Analysis Populations

The registry lists one outcome measure, the Modified City of Hope Quality of Life Questionnaire for Patients With Thyroid Disease, and no outcome analyses. With no reported outcome analysis, there is no reported collection of primary-outcome tests or estimates to assess. The registry record does not report whether multiple time points, subscales, or other outcomes were analyzed beyond the stated primary assessment.

Multiplicity is a general concern when several outcomes or repeated tests are considered: the chance of obtaining a nominally notable result can rise when many tests are conducted. Whether an adjustment was planned or used is not reported in the registry record. No adjustment should be inferred from the description of a single primary measure.

The stated outcome population is participants completing the trial. The registry enrollment was 11, but the number who completed the trial and the number with available primary endpoint data are not reported in the registry record. The record does not report reasons for missing data, how missing questionnaire items or missing visits were handled, or whether any sensitivity analysis was performed. It also does not report whether the primary analysis included all enrolled participants or only participants with complete endpoint measurements beyond the stated population description.

These omissions matter because a complete-case result may describe only those with observed data, and the interpretation can depend on how missingness arose. That is a general methodological issue, not evidence that missing data affected this trial. Without participant-flow and analysis details, the degree of missingness and its implications cannot be evaluated. The numerical primary result is also not reported in the registry record, so neither its completeness nor its robustness can be assessed.

12. 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 registry reports that 11 participants were at risk for serious adverse events and that 0 were affected. It reports 11 participants at risk for other adverse events and that 2 were affected. Deaths are reported as 0. No serious adverse-event terms are listed.

These are counts in the treatment group, not comparative risk estimates. Because there is no separate comparator group, the figures do not show how event occurrence compares with an alternative treatment or with no treatment. The record does not describe the specific other adverse events, their timing, severity, relationship to calcitonin, or whether participants experienced more than one event. The absence of listed serious-event terms does not add detail about the events beyond the reported counts.

As a general statistical consideration, a small group can provide limited information about uncommon events. The counts should therefore be read as the adverse events recorded among this group, not as a complete characterization of possible risks. The registry record does not report additional safety analyses or longer-term follow-up beyond the trial information summarized in the record.

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

The record establishes that a completed phase 4 study enrolled 11 participants in an open-label, single-group trial of calcitonin nasal spray, 200 mcg daily. It identifies a 6-week primary assessment of change in a thyroid-disease quality-of-life questionnaire. It also reports 0 participants affected by serious adverse events, 2 affected by other adverse events, and 0 deaths among the 11 at risk in the treatment group.

The central limitation for interpreting the primary question is that its numerical result is not reported in the registry record. There is no reported mean change, standard deviation, confidence interval, or test result. These data therefore do not show the direction, magnitude, or precision of change in questionnaire score. The stated endpoint and design alone cannot establish whether participants’ reported symptoms changed during the assessment period.

Even if a within-group change estimate were available, a single-group, open-label study would not provide a randomized comparison with an untreated or alternative-treatment group. A change over time could reflect multiple influences, including natural fluctuation and expectations; these are general possibilities rather than findings from this trial. No conclusion about a causal treatment effect can be drawn from the design description alone.

The safety counts are also descriptive. They do not provide a comparative estimate, detailed event descriptions, or a basis for evaluating uncommon or later events. In addition, the registry does not report how many participants completed the study or had primary outcome data. Thus, the record conveys the intended question, the treatment approach, the design, and limited event counts, but it does not provide the outcome evidence needed to judge the primary endpoint’s observed change.

14. Why This Trial Matters Statistically

This trial is statistically instructive because it makes a distinction visible: measuring change is not the same as estimating a treatment contrast. The primary question concerns change in reported quality of life over 6 weeks, while the design is single group and has no separate comparator. A within-participant assessment can describe what happened during the study period, but it cannot isolate the intervention’s contribution from other influences over time.

The endpoint also illustrates why the direction and scale of a measure matter. Higher questionnaire scores indicate more severe symptoms, so interpreting any reported change would require attention to that orientation. The registry identifies a 40-question instrument with a 1-10 scale and a total of 400 points. Yet it does not report the observed score change, making the planned measurement understandable while leaving the result unavailable for interpretation.

The registry’s stated analysis population and missing result highlight another statistical issue: a defined target population is not enough to evaluate an estimate if the number analyzed and the completeness of outcome data are unknown. The record does not report the number completing the trial or the number with endpoint observations. That information would be needed to understand how the analyzed participants relate to the enrolled group.

Finally, the planning illustration is separate from the trial’s actual design. It describes a hypothetical two-arm planning setup, while the registered study is single group. Keeping that distinction clear prevents a planning calculation from being mistaken for trial evidence, observed power, or a protocol-based sample-size justification. The trial therefore offers a useful case for reading design, endpoint, missingness, and planning assumptions separately rather than treating them as interchangeable evidence.

15. 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 trial-specific limitation is the absence of the primary outcome result in the registry record. Although the endpoint, summary measure, and assessment timeframe are specified, the observed mean change and its variability are not reported. The record therefore does not allow assessment of either the size or precision of change in questionnaire score.

The design is single group, with no separate comparator described, and masking is listed as none. These features limit causal interpretation of any observed within-group change and leave self-reported scores potentially open to expectation effects as a general methodological concern. The record does not report a statistical method that could address the lack of a comparator.

Enrollment was 11, while the endpoint population is described as participants completing the trial. The number completing and the number contributing primary outcome data are not reported in the registry record. Reasons for discontinuation, adherence, handling of missing questionnaire data, and sensitivity analyses are not reported in the registry record. These omissions prevent evaluation of whether missing observations could affect the endpoint summary.

The registry lists no outcome analyses and does not report a statistical analysis plan, sample-size justification for the actual design, or interim-analysis plan. The planning assumptions described separately concern a hypothetical two-arm calculation and should not be read as evidence about the trial’s design or observed results. Safety reporting is also limited to event counts: specific other adverse events, timing, severity, and attribution are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Hypothyroidism Treated With Calcitonin — NCT03342001.
• Registry record last updated 2022-02-21; status last verified 2022-02.
• Results first posted 2021-06-10.
• 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.

19. Record Summary

NCT03342001 is a completed phase 4, open-label, single-group study of calcitonin in participants with hypothyroidism. The treatment group received calcitonin nasal spray, 200 mcg daily. Enrollment was 11; the trial began on 2018-06-18, and its primary completion date was 2019-06-30.

The primary endpoint was change after 6 weeks in the Modified City of Hope Quality of Life Questionnaire for Patients With Thyroid Disease. The instrument has 40 questions, uses a 1-10 scale, and assigns higher scores to more severe symptoms. The registry record does not report the primary outcome result or an outcome analysis.

For safety, 0 of 11 participants were affected by serious adverse events, 2 of 11 by other adverse events, and 0 deaths were reported. No serious adverse-event terms were listed.