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 An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism (NCT04538989), a non-randomized phase 2 study. The primary outcome is Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04538989 |
| Phase | Phase 2 |
| Condition | Congenital Hyperinsulinism |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 23 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Rezolute |
| Results posted | Yes |
2. Clinical Question
Population
Patients with congenital hyperinsulinism were enrolled. The study record reports an enrollment of 23 and identifies the primary endpoint population as the Per Protocol Population. It does not describe the eligibility criteria or provide further detail about the participants in the record.
Intervention
Participants received RZ358 in four sequential cohorts. The cohort names are RZ358 Cohort 1, RZ358 Cohort 2, RZ358 Cohort 3, and RZ358 Cohort 4. Specific dose levels and details of how treatment changed between cohorts are not reported in the registry record.
Comparator
There was no randomized control group. The study is described as non-randomized, open-label, and sequential. The registry record does not identify a concurrent comparator arm, so comparisons between cohorts should not be interpreted as like-for-like treatment comparisons.
Primary question
The primary endpoint describes the median average daily percentage of time within a glucose target range of 70-180 mg/dL (3.9-10 mmol/L), measured by continuous glucose monitoring at baseline and end of treatment. The stated objective was to evaluate safety, tolerability, and glucose-raising effects. Numerical primary endpoint results are not reported in the registry record.
In practical terms, the endpoint asks how much of a participant’s monitored day was spent inside the specified glucose range, and how this measure was characterized at baseline and at the end of treatment. The registered outcome is a median, not a mean, and the population named for it is the Per Protocol Population. The record specifies an 8 weeks timeframe. It does not give the cohort-specific treatment details, participant-level observations, or numerical estimates needed to describe the direction or size of any change. The secondary outcomes focus on hypoglycemia measured by self-monitoring of blood glucose and continuous glucose monitoring, as well as hypoglycemia during a fasting challenge. Because the study was non-randomized and did not have a like-for-like control comparison, the clinical question is descriptive: what glucose and safety observations were recorded during this sequential RZ358 study?
3. Trial Design
This was an open-label, non-randomized Phase 2 study of RZ358 in patients with congenital hyperinsulinism. The study used a sequential model and included four RZ358 cohorts. The registry record reports an enrollment of 23. It lists 4 participants at risk for adverse events in Cohort 1, 8 in Cohort 2, 8 in Cohort 3, and 3 in Cohort 4. It does not report a randomized allocation process or a masked assessment, and no concurrent control arm is identified.
The trial status is completed. The recorded start date is 2020-02-24, and the primary completion date is 2022-04-05. The sponsor is Rezolute. The objective described in the record was to evaluate the safety, tolerability, and glucose-raising effects of RZ358. The primary outcome was the median of average daily percentage time in the glucose target range, assessed by continuous glucose monitoring at baseline and end of treatment. The specified timeframe is 8 weeks, and the named analysis population is the Per Protocol Population.
Sequential cohorts can organize treatment experience over time, but the registry record does not describe the dose levels, escalation rules, or other protocol details for these cohorts. Those details are not reported in the registry record. With no randomized comparison and no stated control group, differences among cohorts would not by themselves isolate a treatment effect: cohort membership may coincide with other differences, and the record does not give details that would allow those differences to be assessed. The arm descriptions are blank in the record.
The registry lists 14 outcome measures and an analysis count of 0. It does not provide numerical results for the primary glucose-range endpoint in the information summarized for this trial. The outcome descriptions therefore establish what the study intended to measure, but do not by themselves show the observed primary endpoint values or the precision of a between-group treatment contrast.
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: NA; open-label study
Overall Study
| Stage | RZ358 Cohort 1 | RZ358 Cohort 2 | RZ358 Cohort 3 | RZ358 Cohort 4 |
|---|---|---|---|---|
| STARTED | 4 | 8 | 8 | 3 |
| COMPLETED | 4 | 8 | 8 | 3 |
| NOT COMPLETED | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Per Protocol 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.
| Characteristic | Unit | RZ358 Cohort 1 | RZ358 Cohort 2 | RZ358 Cohort 3 | RZ358 Cohort 4 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 4 | 8 | 8 | 3 | 23 | |
| Age, Categorical: <=18 years | Participants | 4 | 7 | 8 | 3 | 22 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 1 | 0 | 0 | 1 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous | years | 5.8 (SD 4.35) | 9.3 (SD 6.88) | 5.8 (SD 5.12) | 4.0 (SD 2.00) | 6.7 (SD 5.50) |
| Sex: Female, Male: Female | Participants | 4 | 5 | 3 | 1 | 13 |
| Sex: Female, Male: Male | Participants | 0 | 3 | 5 | 2 | 10 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 4 | 6 | 8 | 3 | 21 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 2 | 0 | 0 | 2 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 4 | 6 | 7 | 3 | 20 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 1 | 0 | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 2 | 0 | 0 | 2 |
| Region of Enrollment: Turkey | participants | 0 | 3 | 4 | 0 | 7 |
| Region of Enrollment: United States | participants | 0 | 1 | 0 | 0 | 1 |
| Region of Enrollment: Denmark | participants | 0 | 1 | 0 | 0 | 1 |
| Region of Enrollment: United Kingdom | participants | 0 | 1 | 1 | 0 | 2 |
| Region of Enrollment: Georgia | participants | 1 | 0 | 0 | 0 | 1 |
| Region of Enrollment: Israel | participants | 0 | 1 | 0 | 0 | 1 |
| Region of Enrollment: Bulgaria | participants | 3 | 0 | 0 | 0 | 3 |
| Region of Enrollment: Germany | participants | 0 | 0 | 1 | 0 | 1 |
| Region of Enrollment: Russia | participants | 0 | 0 | 2 | 3 | 5 |
| Region of Enrollment: Spain | participants | 0 | 1 | 0 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT), measured over 8 weeks.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | Daily Percent Time |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the median of average daily percent time within a glucose target range of 70-180 mg/dL (3.9-10 mmol/L), measured by continuous glucose monitoring at baseline and end of treatment. It is reported in daily percent time, with a 95% confidence interval specified as the measure of dispersion. The stated timeframe is 8 weeks, and the listed population is the Per Protocol Population.
As a general statistical explanation, time in a prespecified range condenses continuous glucose monitoring readings into a measure that reflects how much of the monitored day falls inside that range. It describes time within the target rather than the number or duration of individual hypoglycemia episodes. A median summarizes the middle of the participant-level distribution and can be less influenced by unusually high or low observations than a mean. It does not show how widely individual participants’ values vary; the confidence interval is intended to express uncertainty around the reported median under the analysis method used.
The registry description reports that the median is assessed at baseline and end of treatment, but numerical medians and confidence interval limits are not reported in the registry record. Consequently, the endpoint definition alone cannot establish the direction, size, or precision of any change. Nor does a before-and-after measure in this non-randomized study, on its own, separate treatment-associated change from other influences over time.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The primary outcome is defined clearly enough to identify its measurement: median average daily percent time in the 70-180 mg/dL (3.9-10 mmol/L) glucose range by continuous glucose monitoring, at baseline and end of treatment. However, numerical results for this outcome are not reported in the registry record. There is therefore no posted median or confidence interval to interpret, and no basis in the record for describing the endpoint as having increased, decreased, or remained similar. The absence of numerical endpoint values also prevents an assessment of the magnitude and precision of any observed change.
The registry describes 14 outcome measures and reports an analysis count of 0. It includes definitions of the primary and secondary outcomes, but outcome definitions are not outcome estimates. The listed secondary measures include hypoglycemia events assessed by self-monitoring of blood glucose, time and duration in hypoglycemia assessed by continuous glucose monitoring, hypoglycemia during a fasting challenge, and hypoglycemia events at specified glucose thresholds. Numerical results for those outcomes are not reported in the registry record. Their inclusion indicates the breadth of planned measurement, not whether an outcome changed or how consistently it changed among participants.
The adverse-event record gives cohort-level counts. In Cohort 1, 0 of 4 participants had a serious adverse event and 2 of 4 had other adverse events. In Cohort 2, 2 of 8 had serious adverse events and 7 of 8 had other adverse events. In Cohort 3, 1 of 8 had serious adverse events and 5 of 8 had other adverse events. In Cohort 4, 0 of 3 had serious adverse events and 2 of 3 had other adverse events. No deaths are recorded in any cohort. These are descriptive counts from the recorded risk groups; they do not establish comparative risks between cohorts or against an untreated group.
The listed serious adverse event terms are Hypoglycemia with LOC, Tonic-Clonic Seizure, and Eosinophilic Leukocytosis, each reported as 12.5%. The record does not give further detail in the summarized information about timing, recurrence, participant-level overlap, or attribution. These entries should be read as safety reporting, not as evidence that RZ358 caused an event or that an event was unrelated to treatment.
Overall, the posted record supports description of study design and some safety counts, but it does not provide the primary numerical efficacy estimate needed to judge the observed glucose-range endpoint. Since the design was open-label and non-randomized, even a numerical before-and-after result would be descriptive rather than a randomized estimate of the treatment effect. The cohort structure and the lack of a like-for-like control comparison place limits on causal interpretation.
9. Statistical Methods Explained
The registry record identifies a median-based primary endpoint and a 95% confidence interval, but it does not report numerical endpoint estimates or a detailed statistical analysis plan. The explanations below describe general statistical concepts rather than asserting methods that are not documented for this trial.
What does a median describe?
As a general concept, the median is the central value when observations are ordered. It is useful when participant values are skewed or when a small number of unusually high or low observations could pull the mean away from the center of the data. A median does not describe every participant’s result, and it does not communicate the full spread of the observations. In this study, the registered endpoint is a median of average daily percent time within the glucose target range. The record does not specify the calculation details used to form participant-level averages or the exact method used to estimate the median.
How should a confidence interval be read?
As a general statistical explanation, a confidence interval conveys uncertainty around an estimate. A narrower interval indicates greater precision under the assumptions of the method; a wider interval indicates less precision. A 95% confidence interval is not a range containing 95% of individual participant values. Nor does it show that a treatment effect has a 95% probability of being inside that particular interval. Interpretation depends on the estimator, sampling process, and interval method. The record identifies a 95% confidence interval for the primary endpoint’s dispersion, but numerical limits and the interval construction method are not reported in the registry record.
What is the role of baseline and end-of-treatment measures?
Baseline and end-of-treatment measurements allow an outcome to be described at two study time points. A comparison of such measurements can show how the measured quantity changed over the observed period, if the values and analysis are reported. A before-and-after comparison is not equivalent to a randomized treatment-versus-control comparison. Without randomization and a concurrent comparator, changes may reflect treatment, the course of the condition, changes in other care, measurement variation, or other influences. The registry record does not report an adjusted estimate for a baseline-to-end-of-treatment contrast.
Why does the non-randomized design matter?
Random allocation is a general design method for balancing measured and unmeasured characteristics between treatment groups, on average. This study is registered as non-randomized, open-label, and sequential. The cohorts are not described as concurrent controls, and the record specifically does not support treating the arms as a like-for-like control comparison. Differences across sequential cohorts could reflect cohort composition or other differences as well as treatment exposure. The registry record does not report a randomized comparison, masking, or a statistical adjustment that would resolve this limitation.
What do the planning illustrations mean?
As a general concept, a sample-size calculation can illustrate how many participants per arm might be required under specified assumptions to detect a particular effect with a chosen power and significance level. The planning assumptions described for the illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. These are planning illustrations computed from an observed effect, not the trial’s own design. They should not be read as observed power, and they do not change the fact that the trial was non-randomized and did not provide a like-for-like control comparison. Any sample sizes in such illustrations are hypothetical planning quantities, not the number of participants observed in the trial. The registry record does not report that these assumptions were used to design the study.
In general, observed power is not a substitute for an estimate and its confidence interval. For interpreting results, the effect estimate and its precision are more informative than a calculation of power based on an observed result. In this case, the primary numerical estimate and interval are not reported in the registry record, so no estimate-specific precision assessment can be made.
Why can over-dispersion matter?
As a general statistical explanation, over-dispersion occurs when data vary more than a model assumes. If the analysis treats outcomes as less variable than they are, uncertainty may be understated and confidence intervals may be too narrow. This issue can matter for count outcomes, such as episode counts, when participant event rates vary substantially. The registry lists hypoglycemia event outcomes, but does not report the model or distributional assumptions used to analyze them. It is therefore not possible to say whether over-dispersion was evaluated or how it was handled.
The record does not provide a detailed account of covariate adjustment, missing-data handling, subgroup analyses, interim analyses, or multiplicity control. Those methods are not reported in the registry record. General statistical principles can explain why these choices matter, but cannot establish which procedures were used for this study.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 14 outcome measures, including one primary outcome and multiple secondary hypoglycemia measures. When several outcomes are assessed, the chance of at least one apparently notable result arising by chance can increase if each is tested separately without adjustment. As a general statistical principle, multiplicity procedures can control this issue, but the choice depends on the prespecified testing strategy and the intended interpretation of the outcomes. Multiplicity handling is not reported in the registry record. The record also does not provide p-values or numerical outcome estimates with which to assess the impact of multiple testing.
The primary endpoint is identified, and the analysis population is named as the Per Protocol Population. The record does not describe how that population was defined, whether any other analysis populations were used, or how participants who did not complete treatment were handled. Details of missing monitoring data, missing baseline or end-of-treatment measurements, imputation, and sensitivity analyses are not reported in the registry record. These omissions matter because continuous glucose monitoring endpoints depend on available monitoring observations, and the amount and pattern of missing data can affect the representativeness and precision of an estimate.
The trial is non-randomized and sequential, with no like-for-like control comparison. Thus, multiplicity and missing-data questions are only part of the interpretive limits: even complete outcome data would not turn the cohort structure into a randomized comparison. The analysis plan, interim analyses, and any adjustment for differences across cohorts are not reported in the registry record. Conclusions about statistical significance or robustness cannot be drawn from the outcome definitions alone.
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 |
|---|---|---|---|
| RZ358 Cohort 1 | 0 | 4 | 0.0% |
| RZ358 Cohort 2 | 2 | 8 | 25.0% |
| RZ358 Cohort 3 | 1 | 8 | 12.5% |
| RZ358 Cohort 4 | 0 | 3 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| RZ358 Cohort 1 | 0 of 4 |
| RZ358 Cohort 2 | 0 of 8 |
| RZ358 Cohort 3 | 0 of 8 |
| RZ358 Cohort 4 | 0 of 3 |
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 |
|---|---|
| RZ358 Cohort 1 | 2 of 4 |
| RZ358 Cohort 2 | 7 of 8 |
| RZ358 Cohort 3 | 5 of 8 |
| RZ358 Cohort 4 | 2 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | RZ358 Cohort 1 | RZ358 Cohort 2 | RZ358 Cohort 3 | RZ358 Cohort 4 |
|---|---|---|---|---|---|
| Hypoglycemia with LOC | Endocrine disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
| Tonic-Clonic Seizure | Nervous system disorders | 0 of 4 | 0 of 8 | 1 of 8 | 0 of 3 |
| Eosinophilic Leukocytosis | Blood and lymphatic system disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | RZ358 Cohort 1 | RZ358 Cohort 2 | RZ358 Cohort 3 | RZ358 Cohort 4 |
|---|---|---|---|---|---|
| Covid-19 | Infections and infestations | 0 of 4 | 2 of 8 | 0 of 8 | 1 of 3 |
| Phlebitis | Vascular disorders | 0 of 4 | 1 of 8 | 0 of 8 | 1 of 3 |
| Anaemia | Blood and lymphatic system disorders | 0 of 4 | 1 of 8 | 1 of 8 | 0 of 3 |
| Dermatitis Contact | Skin and subcutaneous tissue disorders | 0 of 4 | 1 of 8 | 0 of 8 | 1 of 3 |
| Extravasation | General disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
| Fatigue | General disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
| Infusion Site Reaction | General disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
| Medical Device Pain | General disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
| Cough | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
| Respiratory Disorder | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 1 of 8 | 0 of 8 | 0 of 3 |
Serious events with the highest arm-level rate: hypoglycemia with loc (12.5%), tonic-clonic seizure (12.5%), eosinophilic leukocytosis (12.5%).
The registry reports serious and other adverse events by cohort, with the number of participants at risk stated for each. Cohort 1 had 0 participants with serious adverse events among 4 at risk and 2 with other adverse events among 4. Cohort 2 had 2 with serious adverse events among 8 at risk and 7 with other adverse events among 8. Cohort 3 had 1 with serious adverse events among 8 at risk and 5 with other adverse events among 8. Cohort 4 had 0 with serious adverse events among 3 at risk and 2 with other adverse events among 3. The record reports 0 deaths in each cohort.
These counts describe recorded participants, not event rates adjusted for time at risk. The record does not provide exposure duration by participant, event timing, repeat-event details, or a comparator group. Because participants may have experienced more than one event or event type, the counts for separate categories should not be treated as mutually exclusive without further information. The record does not specify attribution in the summarized safety data, so the counts alone do not establish whether events were caused by treatment.
Three serious adverse event terms are listed at 12.5% each: Hypoglycemia with LOC, Tonic-Clonic Seizure, and Eosinophilic Leukocytosis. Further detail about event-level counts and overlap is not reported in the registry record. These figures are descriptive safety observations from a small, sequential study and are not a comparison of RZ358 with a control treatment.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase 2 study of RZ358 in patients with congenital hyperinsulinism was completed and that its primary outcome was defined as median average daily percent time in a specified glucose range, measured by continuous glucose monitoring at baseline and end of treatment. It also describes secondary outcomes focused on hypoglycemia and provides adverse-event counts by cohort. This information helps clarify what the trial set out to measure and what safety categories were reported.
The record does not report numerical results for the primary endpoint. It therefore does not support a conclusion about the observed median, the size or direction of a change, or the precision of that change. Outcome definitions and the study’s stated objective are not evidence of an outcome result. The listed secondary outcomes likewise do not establish that hypoglycemia changed, because numerical estimates are not reported in the registry record.
The adverse-event counts document events among participants in each cohort, and the record reports no deaths. These observations are not a controlled comparison, and the record does not provide details sufficient to establish treatment attribution. Counts from a sequential, non-randomized study cannot show whether event frequencies differ because of treatment, participant characteristics, exposure time, or other factors.
More generally, an observed change from baseline to end of treatment in a single treatment group would not, on its own, show what would have happened over the same period without treatment. A control comparison and an appropriate analysis would be needed to estimate a comparative treatment effect. Here, a randomized control comparison is not reported, and the cohort arms are not like-for-like controls. Accordingly, the registered design and results support descriptive interpretation, not a causal claim about RZ358’s effects.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of how outcome definition and design shape what can be concluded. Its primary measure is a median of average daily time in a glucose range, based on continuous glucose monitoring. That is distinct from an episode count or a measure of time in hypoglycemia, and the secondary outcomes show that the registry tracked several different dimensions of glucose patterns. Keeping these outcomes distinct is important: a measure of time in a target range does not by itself describe the frequency or duration of hypoglycemic events.
The design is also central to interpretation. The study was open-label, non-randomized, and sequential, with four cohorts and no like-for-like control comparison. A sequential cohort structure may organize study experience, but it does not provide the protections against confounding that random allocation can offer. Any comparison between cohorts needs to be treated cautiously, especially because cohort-level treatment details are not reported in the registry record.
The record illustrates the difference between an outcome being registered and its result being numerically available. A precise endpoint definition, a stated confidence interval, and a detailed list of secondary outcomes do not substitute for estimates, interval limits, or a complete statistical analysis account. The primary numerical results are not reported in the registry record, so the size and precision of an endpoint change cannot be assessed.
Finally, the available safety figures are useful as descriptive counts, but they do not provide a controlled estimate of risk. Reading them alongside the design and reporting limitations avoids treating a small cohort count as a definitive comparative finding. In this study, statistical interpretation depends as much on the absence of a control comparison and numerical endpoint estimates as it does on the stated measurement plan.
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.
Several trial-specific limitations constrain interpretation. First, the study was non-randomized, open-label, and sequential. No concurrent control group or like-for-like control comparison is identified in the registry record. The cohort structure therefore cannot by itself support a causal comparison of treatment effects. The record does not describe whether cohort differences were adjusted for or provide enough detail to assess comparability.
Second, the registry record does not report numerical results for the primary glucose-range endpoint. Although it specifies a median and a 95% confidence interval, the median values and interval limits are not available in the record. The size, direction, and precision of any baseline-to-end-of-treatment change therefore cannot be evaluated. Numerical results for the listed secondary outcomes are also not reported in the registry record.
Third, treatment details for the four cohorts are limited. The arm descriptions are blank, and dose levels and rules for moving between sequential cohorts are not reported in the registry record. Without those details, the meaning of a cohort-level comparison cannot be established. The analysis count is 0, and the record does not provide a detailed statistical analysis plan.
Finally, methods for missing monitoring data, analysis-population definitions beyond the named Per Protocol Population, multiplicity control, covariate adjustment, and interim analyses are not reported in the registry record. Adverse-event counts lack the summarized detail needed to assess duration of exposure, timing, participant-level overlap, or attribution. These limitations do not negate the descriptive information that is recorded, but they restrict how far it can be interpreted.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism — NCT04538989.
- Registry record last updated 2025-05-28; status last verified 2025-05.
- Results first posted 2025-05-28.
- 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.
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18. Record Summary
This completed Phase 2 study evaluated RZ358 in patients with congenital hyperinsulinism. It was open-label, non-randomized, and sequential, with four cohorts and an enrollment of 23. The recorded start date is 2020-02-24, and the primary completion date is 2022-04-05.
The primary outcome was the median average daily percent time within 70-180 mg/dL (3.9-10 mmol/L) by continuous glucose monitoring at baseline and end of treatment, with an 8 weeks timeframe and the Per Protocol Population specified. Numerical primary endpoint results are not reported in the registry record. The registry lists 14 outcome measures and an analysis count of 0.
Safety counts are reported by cohort, including serious and other adverse events, and no deaths are recorded. The non-randomized design does not provide a like-for-like control comparison. Numerical endpoint results and additional statistical methods are not reported in the registry record, limiting assessment of treatment effects and their precision.