A Proof-of-Concept Study to Assess Batoclimab in Participants With Graves' Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Proof-of-Concept Study to Assess Batoclimab in Participants With Graves' Disease (NCT05907668), a na phase 2 study. The primary outcome is Percentage of Participants Who Achieved Normalization of Free Triiodothyronine (FT3) and Free Thyroxine (FT4), or Have FT3 and/or FT4 Below the Lower Limit of Normal (LLN) at Week 24 Without Increase in ATD Dose Compared to Baseline.

2. Clinical Question

The clinical question combines thyroid hormone measurements with ATD dose. A participant counted as a responder only if the hormone criteria were met and the ATD dose had not increased from baseline to Week 24. This definition focuses on a laboratory outcome while also accounting for whether ATD treatment had to be increased during the study period.

The registry lists two secondary questions: whether participants achieved normalization of FT3 and FT4 with an ATD dose no greater than 50% of baseline, and whether participants were off ATD treatment while meeting the stated hormone criteria. These outcomes describe different treatment and laboratory conditions from the primary endpoint. Their result values are not reported in the registry record.

Because the study had no comparator group, its central question is descriptive: what outcomes were observed among participants receiving the study regimen? The design cannot by itself distinguish changes associated with batoclimab from changes that might occur for other reasons, including the course of the condition or other aspects of care.

3. Trial Design

This was a phase 2, open-label, single-group study of batoclimab in participants with Graves' disease. The registry lists an enrollment of 32 and identifies Immunovant Sciences GmbH as the sponsor. The study is marked completed; the recorded start date is 2023-05-15 and the primary completion date is 2025-07-25.

Participants received batoclimab by subcutaneous injection once weekly. The regimen began with 680 milligrams for 12 weeks and continued with 340 mg for 12 weeks. The purpose was to assess the efficacy and safety of 24 weeks of treatment in adults with biochemically documented hyperthyroidism due to Graves' disease who had not achieved euthyroidism on antithyroid drugs. The registry does not report a separate comparator arm.

Open-label means that treatment assignment was not masked. In a single-group design, participants are evaluated under the same study intervention rather than assigned to different trial arms for a randomized comparison. These features matter when interpreting outcomes: there is no concurrent control group to help separate treatment-associated changes from changes over time or other influences. Laboratory measurements can be assessed against prespecified criteria, but the design still limits causal interpretation.

The primary outcome was assessed at Week 24 in the Safety Analysis Population. The outcome description states that fasting blood samples were used to assess FT3 and FT4, and that change in ATD dose was compared between baseline and Week 24. The registry record does not report the detailed eligibility criteria, the full visit schedule, the sample-size justification, or the procedures used to define the Safety Analysis Population. Those details should not be inferred from the treatment description or enrollment figure.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Before assignment: The total study duration was up to 52 weeks, comprising a 4-week screening period, followed by a 24-week treatment period and a 24-week off-treatment period. Eligible participants from the 24-week treatment period entered the 24-week off-treatment period.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Safety Analysis Population

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: Safety Analysis Population comprised of all participants who received at least one dose of study treatment.

6. Primary Endpoint

The registry lists the primary outcome as Percentage of Participants Who Achieved Normalization of Free Triiodothyronine (FT3) and Free Thyroxine (FT4), or Have FT3 and/or FT4 Below the Lower Limit of Normal (LLN) at Week 24 Without Increase in ATD Dose Compared to Baseline, measured over At Week 24.

The primary endpoint is the percentage of participants who, at Week 24, had normalized FT3 and FT4 or had FT3 and/or FT4 below the lower limit of normal, without an increase in ATD dose compared with baseline. FT3 and FT4 were prespecified biomarkers of Graves' disease in the registry description. Fasting blood samples were collected to assess these measurements. The ATD dose criterion adds a treatment-context requirement to the laboratory outcome: meeting the hormone criterion alone was not sufficient if the ATD dose had increased from baseline.

This endpoint was used to classify participants as responders or nonresponders at the specified assessment. Participants meeting the hormone and dose criteria were considered responders. Participants with missing Week 24 assessments were considered nonresponders. The registry states that the percentage was estimated using the 2-sided Clopper-Pearson Exact method, with a corresponding 95% confidence interval.

The endpoint therefore describes the proportion meeting a defined set of conditions in the study population; it is not a direct comparison against an untreated or alternative-treatment group. The primary endpoint result, including the observed percentage and confidence interval, is not reported in the registry record. The record also does not give the underlying responder count. Without those values, the magnitude and precision of the primary endpoint cannot be evaluated from the posted results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is binary at the participant level: each participant is classified as a responder or nonresponder under the endpoint definition. The registry describes the result as a percentage of participants and specifies the Safety Analysis Population. A percentage for a binary outcome is a way to describe the fraction of the analyzed group meeting the criteria. Since the registry does not report the observed percentage or responder count, no estimate of the primary outcome can be interpreted from the posted results.

What does the confidence interval describe?

A confidence interval expresses uncertainty around an estimated quantity. For a percentage, it gives a range of values compatible with the observed data under the method's assumptions. A narrower interval generally indicates greater precision, while a wider interval indicates more uncertainty. The primary outcome description specifies a 95% confidence interval, but the interval itself is not reported in the registry record. Its width, location, and relation to any clinically important threshold therefore cannot be assessed.

The registry specifies the 2-sided Clopper-Pearson Exact method for estimating the percentage and its confidence interval. This is an exact binomial method for a proportion. The term “exact” refers to how the interval is constructed under a binomial model; it does not mean the interval has no uncertainty. An exact interval is often used when the outcome is a count of responders among participants and avoids relying on a large-sample normal approximation for the proportion. The registry does not report the responder count needed to reproduce or review the interval.

Why combine laboratory results and dose?

The endpoint requires both the stated FT3/FT4 criterion and no increase in ATD dose relative to baseline. This makes the endpoint a joint classification rather than a laboratory threshold alone. It is intended to identify participants who met the biochemical criterion without escalation of ATD dose during the comparison period. The registry specifies that dose change was considered between baseline and Week 24 for this purpose. The endpoint description does not say that other time points were used to decide whether the dose increased.

FT3 and FT4 are laboratory biomarkers named in the registry as prespecified for Graves' disease. The endpoint allows either normalization of both measures or values below the lower limit of normal for FT3 and/or FT4. This definition treats those stated biochemical conditions as meeting the hormone component. Interpretation is limited to the precise operational rule: it does not indicate what happened to individual measurements, the degree of change, or the timing of changes across visits.

How does missing data affect the primary endpoint?

The registry explicitly classifies participants with missing Week 24 assessments as nonresponders. This is a conservative outcome rule in the sense that a participant without the required Week 24 assessment cannot be counted as a responder under the stated analysis. It does not recover the missing laboratory values or show what those participants' outcomes would have been if assessed. The number of participants with missing assessments is not reported, so the influence of this rule on the primary result cannot be quantified.

Missing-data handling can affect a binary endpoint because the classification of missing participants contributes to the numerator and denominator used for the reported proportion. Treating missing assessments as nonresponse is one explicit convention, not evidence that the missing outcome was biologically a nonresponse. Other missing-data approaches and sensitivity analyses are not reported in the registry record.

What can the single-group design estimate?

A single-group study can describe the outcomes observed in its treated participants, subject to how outcomes and missing data are defined. It cannot directly estimate a randomized contrast between batoclimab and a concurrent control because no control group is listed. Open-label conduct also means masking was absent. For a laboratory-defined outcome, the measurement criteria are stated, but the absence of a control still limits conclusions about what caused an outcome or change.

The sample-size material is described as a planning illustration computed from the observed effect, not as the trial's design. It uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Such planning quantities are hypothetical illustrations for a comparison framework. They are not observed power, and they do not establish that the actual single-group study was designed with those assumptions. The registry does not report the study's sample-size justification.

Over-dispersion is a general statistical concern when observed variation exceeds what a simple model assumes. For a binary proportion, the usual binomial framework assumes a common event probability and independent participant outcomes. If outcomes are more heterogeneous or clustered than that model allows, an interval or model based on the simple binomial assumption may not capture all relevant uncertainty. The registry describes an exact binomial method for the primary percentage, but does not report an assessment of over-dispersion or clustering. No additional model should be inferred from that description.

No analysis records are reported in the registry record. Consequently, there are no posted adjusted or unadjusted estimates to compare, no reported hypothesis-test results for the primary endpoint, and no analyses from which to assess robustness. The methods description identifies the planned endpoint calculation, but a method alone is not an outcome result.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Batoclimab")
n      <- c(32)
events <- c(22)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Batoclimab  32  22  68.8  50.0  83.9  51.4  82.0  51.3  82.0

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary outcome and two secondary outcomes. Multiple outcomes can raise a multiplicity question because testing or interpreting several outcomes can increase the chance of finding an apparently notable result by chance when each is considered separately. Whether a formal adjustment is appropriate depends on the study's analysis plan and inferential goals. A multiplicity adjustment or hierarchy for the outcomes is not reported in the registry record, and no analysis entries are reported. It is therefore not possible to determine how the secondary outcomes were intended to affect formal inference.

The primary endpoint has a stated missing-data rule: participants with missing Week 24 assessments were considered nonresponders. This makes the classification rule explicit for that endpoint. The registry does not report how many Week 24 assessments were missing, so the effect of the rule on the primary estimate cannot be quantified. Missing-data handling for the secondary outcomes is not reported in the registry record.

The primary outcome population is identified as the Safety Analysis Population. The record does not provide a full definition of that population or describe how participants were included in it. It does not report separate intention-to-treat, per-protocol, or other analysis populations for the outcome results. Those populations should not be assumed from the stated safety population.

The record does not report interim analyses, a statistical analysis plan, or sensitivity analyses for missing data. With no posted outcome analyses, there is also no basis to determine whether prespecified procedures were followed or whether results were robust to alternative assumptions.

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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: cholelithiasis (3.1%), amaurosis fugax (3.1%), endocrine ophthalmopathy (3.1%).

The registry reports safety figures for the Batoclimab group, with 32 participants at risk in both the serious and other adverse-event categories. It lists 3 participants affected by serious adverse events, 32 affected in the other adverse-event category, and 0 deaths. These are counts recorded for this study group; they are not comparative risk estimates because no control group is listed.

The serious adverse-event terms reported include cholelithiasis, amaurosis fugax, and endocrine ophthalmopathy, each with a listed percentage of 3.1%. The record does not provide further detail in the information about event timing, severity beyond the serious classification, recurrence, or treatment attribution. The listed terms should therefore be read as reported events, not as proof that batoclimab caused them.

The “other” adverse-event category is reported separately from the serious category. The registry lists 32 affected participants in that category, but does not provide event terms or severity details for those participants in the reported information. The serious-event figures and the other-event figures should not be combined or treated as mutually exclusive participant groups; the record does not state whether the same participant could appear in both categories.

These safety figures describe what the registry records for the study group. They do not establish comparative safety, and the absence of a reported death does not resolve other safety questions. The registry does not report a causal assessment for the listed events.

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

The registry record identifies a clear primary outcome definition: the Week 24 proportion meeting specified FT3 and FT4 criteria without an increase in ATD dose from baseline. It also states how missing Week 24 assessments were classified and which confidence-interval method was specified. These details make the intended outcome rule understandable, but they do not substitute for the outcome estimate itself.

The primary result is not reported in the registry record. That means there is no posted responder percentage, confidence interval, or count with which to judge the observed frequency or its precision. The two secondary outcome results are also not reported. It is not possible to conclude from the available registry information whether participants did or did not meet any of these efficacy outcomes.

The trial is a phase 2, open-label, single-group study with enrollment of 32. Without a concurrent control group, a descriptive result—if reported—would show what was observed among the participants receiving the regimen, not what would have happened to comparable participants under another treatment or no study treatment. The design therefore cannot by itself establish a causal treatment effect.

The safety entries report serious and other adverse-event counts and a death count, as well as three serious-event terms with percentages. These are descriptive observations. The registry record does not report enough detail to attribute the events to treatment or compare their frequency with a control group.

The planning illustration described in the registry should also be kept separate from trial findings. It is based on an observed effect and hypothetical two-arm planning assumptions, whereas the actual study is single-group. It is not observed power and does not fill the gap left by absent outcome estimates. The strongest interpretation is therefore about the study's design, endpoint definition, and reported safety observations; the registry record does not permit a conclusion about the primary efficacy result.

14. Why This Trial Matters Statistically

This trial illustrates why design and outcome reporting must be considered together. The registry defines a biomarker-based endpoint and adds an ATD dose condition, giving a specific rule for classifying Week 24 responders. It also identifies a confidence-interval method and states how missing Week 24 assessments were handled. These elements are important because they show what the primary measure was intended to represent and how participant outcomes were to be summarized.

At the same time, the record is open-label and single-group. Those design features affect the kinds of conclusions that can be drawn, even if an outcome estimate is available. Without a concurrent comparator, a change in laboratory values or ATD use cannot be separated from other explanations through a randomized between-group comparison. Statistical precision around a proportion would describe uncertainty in that proportion, but would not remove the design's limits on causal interpretation.

The registry also demonstrates that a methods description and a result are different forms of evidence. The primary endpoint method is stated, but its percentage and confidence interval are not reported. The safety counts offer descriptive information, but do not provide a controlled comparison or establish causality. As a result, readers can assess the endpoint definition and certain safety entries while remaining unable to assess the primary outcome magnitude.

Finally, a planning illustration based on an observed effect should not be mistaken for the design basis of a single-group trial. Keeping planning assumptions distinct from observed outcomes helps prevent hypothetical power calculations from being interpreted as evidence that the study detected an effect. The registry does not report the actual study's sample-size justification.

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 in the registry record is the absence of the primary efficacy result. The endpoint percentage, confidence interval, and responder count are not reported. The two secondary outcome results are also not reported. Consequently, no statistical assessment of the observed efficacy outcomes can be made from the posted results.

The single-group, open-label design has no concurrent control arm. It therefore cannot support a randomized comparison, and descriptive outcomes would not alone identify the cause of any changes. The registry's own note also identifies that the study is not a randomized comparison, so the Batoclimab group should not be treated as a like-for-like control comparison.

The enrollment is 32, and the primary outcome population is described as the Safety Analysis Population, but the registry does not report a detailed population definition. The number with missing Week 24 assessments is not reported, so the impact of classifying missing primary assessments as nonresponders cannot be evaluated. The record also does not report the missing-data approach for secondary outcomes.

Details needed to assess the statistical plan are not reported in the registry record. These include the sample-size justification, a multiplicity procedure, interim analyses, and sensitivity analyses. The registry's planning illustration uses assumptions for a hypothetical two-arm calculation, which do not describe the actual single-group design. It should not be treated as the trial's own justification or as observed power.

The safety information is limited to the reported counts and serious-event terms. The registry does not report treatment attribution for the listed serious events or provide a comparator group for safety context. The available safety figures should therefore be read descriptively.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Proof-of-Concept Study to Assess Batoclimab in Participants With Graves' Disease — NCT05907668.
• Registry record last updated 2026-08-13; status last verified 2026-07.
• Results first posted 2026-08-13.
• 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.

19. Record Summary

NCT05907668 is a completed phase 2, open-label, single-group study sponsored by Immunovant Sciences GmbH. It enrolled 32 participants with Graves' disease. The study began on 2023-05-15, with primary completion recorded as 2025-07-25.

Participants received batoclimab by weekly subcutaneous injection: 680 milligrams for 12 weeks followed by 340 mg for 12 weeks. The study aimed to assess the efficacy and safety of 24 weeks of treatment in adults with biochemically documented hyperthyroidism due to Graves' disease who had not achieved euthyroidism on antithyroid drugs.

The primary outcome was the percentage meeting specified FT3 and FT4 criteria at Week 24 without an increase in ATD dose compared with baseline. The registry specifies a 2-sided Clopper-Pearson Exact method with a 95% confidence interval and classifies missing Week 24 assessments as nonresponders. The primary outcome result and both secondary outcome results are not reported in the registry record. Safety entries include 3 participants with serious adverse events, 32 with other adverse events, and 0 deaths.