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 Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function (NCT05001269), a na phase 2 study. The primary outcome is Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05001269 |
| Phase | Phase 2 |
| Condition | Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2 |
| Allocation | Not applicable (single group) |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 27 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company |
| Results posted | Yes |
2. Clinical Question
Population
Children from birth to 11 years of age with primary hyperoxaluria and relatively intact renal function. The primary endpoint description specifies genetically confirmed PH1, PH2, or PH3 subgroups. The defined MITT population includes participants who received at least one dose and had at least one post-baseline spot urinary oxalate-to-creatinine ratio.
Intervention
Nedosiran, identified in the intervention description as DCR-PHXC, given as an open-label monthly subcutaneous injection based on age and weight. Dose amounts and details of dose adjustment are not reported in the registry record.
Comparator
No separate control group is identified. The registry records no allocation and no masking, so this is not a randomized, masked comparison against another treatment or control condition.
Primary question
What was the mean percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in the PH1, PH2, and PH3 participant subgroups? The registry record gives the endpoint definition but does not report the numerical subgroup results or their uncertainty.
This is a descriptive question about change during treatment, not a randomized estimate of the difference between nedosiran and a comparator. With no control group, a change from baseline cannot by itself separate change occurring during treatment from changes that might have occurred over time for other reasons. The record describes a pediatric population with relatively intact renal function; it does not provide a numerical threshold for that description. It also does not report the number analyzed within each hyperoxaluria subtype for the primary endpoint. Thus, the clinical question is clear in its outcome, timeframe, and subgroups, while the available record does not supply the results needed to assess the magnitude or precision of change.
3. Trial Design
The registry identifies this as a Phase 2, open-label, multicenter study of nedosiran in pediatric patients from birth to 11 years of age with primary hyperoxaluria and relatively intact renal function. The listed conditions include primary hyperoxaluria and types 1, 2, and 3. The planned enrollment is 27, and the study status is completed. The recorded start date is 2022-02-22 and the primary completion date is 2025-02-05.
The intervention is described as a monthly subcutaneous injection of DCR-PHXC, based on age and weight. The trial title and intervention listing use the name nedosiran. The registry records no allocation and no masking. There is one open-label intervention description and no separate comparator arm, so the design does not provide a like-for-like control comparison. Participants and investigators were not masked according to the registry description.
The design model is listed as sequential. Further details about what that means for participant assignment, treatment sequence, or timing are not reported in the registry record, so the label should not be taken to imply a particular sequence of doses or groups. The record does not report a protocol-level sample-size justification, an interim-analysis plan, or a detailed statistical analysis plan.
The primary outcome is a mean percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio, described for PH1, PH2, and PH3 subgroups. The MITT definition includes participants who received at least one dose and had at least one post-baseline measurement of the ratio. The record also lists safety outcomes and changes in ECG measures. It describes 60 outcome measures, but numerical primary endpoint estimates are not reported in the registry record. The record lists zero analysis records; that registry entry alone does not establish whether analyses were conducted outside the 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.
Before assignment: This is Phase 2, open-label, single-arm uncontrolled study in pediatric participants with genetically confirmed primary hyperoxaluria (PH), with relatively intact renal function based upon estimated glomerular filtration rate (eGFR) and serum creatinine.
Overall Study
| Stage | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| STARTED | 5 | 13 | 9 |
| Modified Intent-To-Treat (MITT) | 5 | 13 | 9 |
| Safety | 5 | 13 | 9 |
| COMPLETED | 5 | 13 | 9 |
| NOT COMPLETED | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The MITT Population included all participants who received at least 1 dose of study intervention and have at least 1 post-baseline spot urinary oxalate to creatinine ratio. Here 'Number of participant analyzed' signified 'Overall number of participants analyzed' and 'Number Analyzed' signifies number of participants with available data for particular timepoint, for the respective arms.
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 population included all participants who received at least 1 dose of study intervention.
| Characteristic | Unit | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Total |
|---|---|---|---|---|---|
| Participants analysed | 5 | 13 | 9 | 27 | |
| Age, Continuous | years | 1.30 (SD 0.529) | 3.54 (SD 1.198) | 7.78 (SD 1.093) | 4.54 (SD 2.687) |
| Sex: Female, Male: Female | Participants | 1 | 6 | 4 | 11 |
| Sex: Female, Male: Male | Participants | 4 | 7 | 5 | 16 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 0 | 1 | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 4 | 12 | 8 | 24 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Asian | Participants | 1 | 5 | 0 | 6 |
| Race/Ethnicity, Customized: White | Participants | 3 | 2 | 4 | 9 |
| Race/Ethnicity, Customized: Multiple | Participants | 1 | 5 | 5 | 11 |
| Race/Ethnicity, Customized: Unknown | Participants | 0 | 1 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups, measured over Baseline (Week 0), Month 6.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Percent change |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio. The ratio is measured in a spot urine sample, and the endpoint is defined separately for participants with PH1, PH2, or PH3. The registry describes the target population as pediatric participants from birth to 11 years of age with genetically confirmed disease in one of those subgroups.
In general, a ratio to urinary creatinine can help account for differences in urine concentration between spot samples. Urinary oxalate is the measure of interest in this endpoint, while creatinine is used as the denominator in the ratio. Expressing change as a percentage frames the follow-up value relative to baseline, allowing the direction and relative size of within-participant change to be described. This is a general explanation of the measure, not a statement about the numerical findings in this study.
The endpoint is summarized as a mean, with standard deviation specified as the dispersion measure. A mean describes the average change in the analyzed group; a standard deviation describes how spread out individual changes are around that average. Neither quantity is reported numerically in the registry record for the primary endpoint. Nor are subgroup estimates, subgroup analysis counts, or confidence intervals reported. As a result, the endpoint definition identifies what was intended to be measured and how it was to be summarized, but does not establish the observed size, direction, or precision of change.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the intended primary outcome and its timeframe: mean percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio, assessed for PH1, PH2, and PH3 subgroups. It does not report numerical results for that outcome. In particular, the record does not provide the subgroup means, standard deviations, numbers analyzed, or confidence intervals. The direction and magnitude of the primary endpoint therefore cannot be described from the registry record.
This absence of numeric estimates also prevents a statistical assessment of how precisely the mean change was estimated. A confidence interval, when reported, gives a range of values compatible with the data under the analysis assumptions; its width helps communicate precision. No such interval for the primary endpoint is reported in the registry record. A standard deviation is specified as the endpoint's dispersion measure, but its value is not reported. The record also does not provide a p-value for this endpoint. It is therefore not possible to infer statistical evidence for a change, or to distinguish a small estimate from a large but uncertain one.
The study is open-label and has no separate comparator group. A within-group change from baseline, even if a value were reported, would be a descriptive result during the study rather than a randomized estimate of the effect relative to an untreated or differently treated group. Other influences on measurements over time cannot be separated from treatment-associated change by this design alone. This is a design limitation, not evidence for or against a particular result.
The endpoint is specified across three disease subgroups, but the number analyzed in each subgroup is not reported. Subgroup estimates may have different precision depending on their sizes and variability; without those details, the registry record does not support comparisons of apparent change between PH1, PH2, and PH3. Nor does it provide enough information to judge whether any subgroup differences are more than sampling variation.
The record lists 60 outcome measures, including safety outcomes and ECG changes, but it does not describe a multiplicity strategy for interpreting multiple outcomes. It lists zero analysis records. That absence means the registry record does not furnish analysis-level results; it does not prove that analyses were not performed elsewhere. The proper statistical reading is therefore limited: the outcome and intended summary are defined, while numerical efficacy estimates and their uncertainty are not reported in the registry record. No conclusion about the size or precision of the primary endpoint follows from its definition alone.
9. Statistical Methods Explained
The registry describes the primary endpoint as a continuous measure summarized by a mean and standard deviation. These are descriptive statistics: the mean conveys the average observed percent change, while the standard deviation describes variation among individual measurements. They answer different questions. A mean alone does not show whether individual participants had similar or widely varying changes, and a standard deviation alone does not tell the direction of the average change. The record does not report numerical values for either statistic for the primary endpoint.
What does a percent change describe?
In general, percent change expresses a follow-up measurement relative to a baseline measurement, rather than reporting only the absolute difference between two measurements. It can make relative changes easier to describe when starting values differ. It can also be influenced by the baseline value: the same absolute movement can correspond to different relative changes at different starting levels. This is a general property of percent-change measures, not a finding from this study. The registry specifies baseline at Week 0 and follow-up at Month 6, but does not provide the underlying participant-level values or the resulting subgroup summaries.
Why report a standard deviation?
A group mean compresses the individual observations into one central summary. The standard deviation adds information about their spread. When data are highly variable, the mean may be a less complete description of the group, so readers benefit from seeing both the central estimate and variability. The standard deviation is not the same as a confidence interval: it describes variation among observations, whereas a confidence interval describes uncertainty in an estimated quantity under a statistical model. No confidence interval for the primary endpoint is reported in the registry record.
What would a confidence interval add?
In general, a confidence interval communicates precision by giving a range of values compatible with the observed data and the method's assumptions. A narrow interval indicates a more precise estimate than a wide interval, all else being equal. It does not eliminate design limitations or establish that a result is important in clinical terms. Here, the record does not give a primary endpoint confidence interval or enough numerical detail to derive one without making assumptions. No recalculated estimate is therefore appropriate.
What can the open-label design establish?
An open-label, single-intervention design can describe outcomes observed during treatment, but it does not create the concurrent control needed for a randomized comparison. In general, a before-and-after difference may reflect treatment, time, measurement variability, or other influences. Random allocation to a comparison condition is one way to support causal interpretation by balancing, on average, other influences between groups. This study's allocation is listed as not applicable and no comparator is identified. Its design should therefore be read as non-randomized and without a like-for-like control comparison, not as an estimate of a controlled treatment contrast.
What does the MITT definition mean?
The registry defines the MITT population as participants who received at least one dose of study intervention and had at least one post-baseline spot urinary oxalate-to-creatinine ratio. This definition conditions inclusion on both treatment exposure and availability of a post-baseline measurement. It is not equivalent to including every enrolled participant regardless of measurement availability. The record does not provide the number in this analysis population or explain how missing measurements were handled. The label alone cannot show whether participants excluded by the definition differed from those included.
How should planning calculations be understood?
In general, sample-size planning illustrations based on an observed effect are conditional calculations: they describe what a future comparison might require under stated assumptions, rather than what this study observed. The assumptions listed for the planning illustration specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is expressed as a fraction of the observed effect. Such calculations do not turn this single-intervention study into a controlled comparison. They are not observed power, and any planning figures should not be interpreted as trial outcomes. No sample-size justification from the original study protocol is reported in the registry record.
More broadly, the analysis depends on choices not fully described in the record: the exact statistical model, handling of repeated or missing observations, subgroup estimation procedures, and multiplicity adjustments are not reported. The record specifies a mean and standard deviation, but these labels do not disclose all analytic decisions. Without outcome estimates and those details, the statistical methods can be described at the level of the registered endpoint and population definition, but their implementation and results cannot be independently evaluated.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 60 outcome measures. The primary measure is the Month 6 percent change in urinary oxalate-to-creatinine ratio across PH1, PH2, and PH3 subgroups; the secondary outcomes include treatment-emergent adverse events, serious adverse events, and changes in several ECG measures. When many outcomes or subgroup results are considered, the chance of observing an apparently notable result by chance can increase. Statistical plans sometimes address this with a prespecified hierarchy or multiplicity adjustment. Whether such a strategy was planned or used is not reported in the registry record.
The record does not give a detailed statistical analysis plan, interim-analysis plan, or primary endpoint p-value. It also does not report confidence intervals or numerical subgroup estimates for the primary endpoint. No analysis records are listed in the registry. This describes what is recorded, not whether analyses were conducted outside the registry.
The stated MITT population includes participants who received at least one dose and had at least one post-baseline ratio measurement. The number meeting this definition is not reported in the registry record, nor are subgroup counts for the primary endpoint. The record does not explain how missing follow-up measurements were handled, whether any imputation was used, or whether sensitivity analyses assessed the effect of missing data. These omissions matter because the analyzed participants may not represent everyone enrolled if measurement availability is related to outcomes.
The registry does not report a protocol-based sample-size justification. A planning illustration is described using assumptions, but it is not observed power and cannot substitute for a prespecified rationale or the missing outcome estimates. Taken together, the record gives a population definition and endpoint specification but does not provide enough information to assess multiplicity control, missing-data handling, or the completeness of the primary endpoint analysis.
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 |
|---|---|---|---|
| Children 0 to <2 Years | 1 | 5 | 20.0% |
| Children 2 to <6 Years | 3 | 13 | 23.1% |
| Children 6 to 11 Years | 1 | 9 | 11.1% |
| Overall | 5 | 27 | 18.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Children 0 to <2 Years | 0 of 5 |
| Children 2 to <6 Years | 0 of 13 |
| Children 6 to 11 Years | 0 of 9 |
| Overall | 0 of 27 |
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 |
|---|---|
| Children 0 to <2 Years | 5 of 5 |
| Children 2 to <6 Years | 11 of 13 |
| Children 6 to 11 Years | 7 of 9 |
| Overall | 23 of 27 |
Most frequent serious adverse event terms
| Event term | Organ system | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Overall |
|---|---|---|---|---|---|
| Nephrolithiasis | Renal and urinary disorders | 0 of 5 | 1 of 13 | 1 of 9 | 2 of 27 |
| Gastroenteritis | Infections and infestations | 0 of 5 | 0 of 13 | 1 of 9 | 1 of 27 |
| Pneumonia | Infections and infestations | 0 of 5 | 1 of 13 | 0 of 9 | 1 of 27 |
| Pyelocaliectasis | Renal and urinary disorders | 0 of 5 | 1 of 13 | 0 of 9 | 1 of 27 |
| Upper respiratory tract infection | Infections and infestations | 1 of 5 | 0 of 13 | 0 of 9 | 1 of 27 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Overall |
|---|---|---|---|---|---|
| Pyrexia | General disorders | 4 of 5 | 0 of 13 | 2 of 9 | 6 of 27 |
| Injection site pain | General disorders | 1 of 5 | 2 of 13 | 2 of 9 | 5 of 27 |
| Cough | Respiratory, thoracic and mediastinal disorders | 0 of 5 | 4 of 13 | 0 of 9 | 4 of 27 |
| Diarrhoea | Gastrointestinal disorders | 1 of 5 | 2 of 13 | 1 of 9 | 4 of 27 |
| Nasopharyngitis | Infections and infestations | 2 of 5 | 2 of 13 | 0 of 9 | 4 of 27 |
| Calculus urinary | Renal and urinary disorders | 0 of 5 | 1 of 13 | 2 of 9 | 3 of 27 |
| Influenza | Infections and infestations | 2 of 5 | 1 of 13 | 0 of 9 | 3 of 27 |
| Nephrolithiasis | Renal and urinary disorders | 2 of 5 | 0 of 13 | 1 of 9 | 3 of 27 |
| Renal colic | Renal and urinary disorders | 1 of 5 | 0 of 13 | 2 of 9 | 3 of 27 |
| Upper respiratory tract infection | Infections and infestations | 0 of 5 | 3 of 13 | 0 of 9 | 3 of 27 |
Serious events with the highest arm-level rate: upper respiratory tract infection (20.0%), gastroenteritis (11.1%), nephrolithiasis (11.1%), pneumonia (7.7%), pyelocaliectasis (7.7%).
The registry reports serious adverse event counts by age group and overall. Among children 0 to <2 years, 1 affected participant is listed among 5 at risk; among children 2 to <6 years, 3 are listed among 13; among children 6 to 11 years, 1 is listed among 9. Overall, 5 affected participants are listed among 27 at risk. These are descriptive counts within the study's reported groups, not comparative estimates against a control group.
For other adverse events, the corresponding entries are 5 among 5 at risk for children 0 to <2 years, 11 among 13 for children 2 to <6 years, and 7 among 9 for children 6 to 11 years. Overall, 23 are listed among 27 at risk. The registry reports zero deaths. These counts do not indicate whether events were judged related to treatment; that relationship is not reported in the registry record.
The serious-event terms listed with percentages are upper respiratory tract infection, 20.0%; gastroenteritis, 11.1%; nephrolithiasis, 11.1%; pneumonia, 7.7%; and pyelocaliectasis, 7.7%. The registry does not provide enough detail to infer comparative risk or attribute these events to the intervention. The study has no comparator group, and the counts should be interpreted as safety observations during the study rather than as evidence of a difference from another treatment.
12. What the Result Does, and Does Not, Mean
The record defines a clinically relevant measurement for this study: change in spot urinary oxalate-to-creatinine ratio from baseline to Month 6, summarized as a mean percent change in PH1, PH2, and PH3 subgroups. It also identifies the pediatric population, open-label intervention, and safety outcomes. Those details describe what the investigators set out to assess. They do not supply the numerical findings for the primary endpoint.
Because the registry record does not report subgroup means, standard deviations, analysis counts, or confidence intervals, it is not possible to state how large the observed change was, which direction it took, or how precisely it was estimated. The endpoint definition alone is not a result. No conclusion about the magnitude or reliability of change can be drawn from the registry record's description of the measure.
Even if numerical within-group changes were available, the absence of a separate comparator would limit causal interpretation. A change observed after treatment cannot, by itself, distinguish treatment-associated change from change over time or other influences. The open-label design also means the registry does not describe masked outcome assessment. These points concern what the design can establish; they do not imply a particular outcome.
The safety counts describe events recorded among participants in the study, including serious events, other events, and zero reported deaths. Without a control group, those observations do not estimate how event occurrence compares with another treatment or with no treatment. The registry's adverse-event entries are not a substitute for the missing primary endpoint results, and the primary endpoint does not answer every question about safety.
Overall, the registry record supports a description of the study's design, target population, endpoint, and reported safety counts. It does not support a quantitative conclusion about the primary endpoint or a controlled treatment effect. The distinction is important: defining an outcome and specifying a statistical summary are not the same as reporting an estimate and its uncertainty.
13. Why This Trial Matters Statistically
This trial is statistically notable for studying a pediatric population from birth to 11 years of age across three primary hyperoxaluria subgroups. The registry's primary endpoint is defined in a way that focuses on change over time in a spot urinary oxalate-to-creatinine ratio, rather than on a direct randomized comparison. Describing the outcome and population clearly is a prerequisite for interpreting any eventual estimate.
The design also illustrates the limits of interpreting before-and-after results. With an open-label intervention and no separate comparator, changes over the study period cannot be cleanly separated from time-related or other influences. This distinction helps prevent a within-group observation from being mistaken for a controlled estimate of treatment effect. It is a general design consideration and does not presume the direction of the results.
Subgroup reporting is especially important when an endpoint is defined for PH1, PH2, and PH3 separately. The subgroup estimates and analysis counts are not reported in the registry record, so their precision and comparability cannot be assessed. A pooled description, if available elsewhere, would not automatically answer whether patterns differ by subtype.
The registry also lists safety and ECG outcomes, reflecting that interpretation is broader than a single efficacy measure. Yet the record does not provide a detailed analysis plan for multiple outcomes or missing measurements. These features show why statistical interpretation depends not only on choosing an endpoint, but also on reporting who was analyzed, how uncertainty was quantified, and how multiple questions were handled.
Finally, the absence of numerical primary results is itself consequential for evidence appraisal. The record makes the study question and design identifiable, but does not allow readers to judge the observed size or precision of the primary endpoint. This is a distinction between information about what a trial intended to measure and information about what its analysis found.
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 central limitation for interpreting this record is that numerical primary endpoint results are not reported in the registry record. The mean percent changes, standard deviations, confidence intervals, and numbers analyzed for PH1, PH2, and PH3 are absent. The registry therefore does not support an assessment of the direction, magnitude, or precision of the primary endpoint.
The study has no separate comparator group, and allocation is listed as not applicable. Its open-label design cannot provide a randomized estimate of a difference between treatment conditions. Any within-group change, if reported elsewhere, would need to be interpreted as change during the study rather than as an isolated causal effect of treatment. The record does not provide a detailed account of outcome assessment procedures or masking beyond the designation of no masking.
The subgroup sizes for the primary endpoint are not reported, and the record does not explain whether each subgroup had enough observations for precise estimation. Although the MITT definition is stated, the size of that analysis population is not given. The record also does not report how missing post-baseline measurements were handled, whether participants with incomplete follow-up were assessed in sensitivity analyses, or whether the analysis population differed from the enrolled population.
Additional limitations include the absence of a detailed statistical analysis plan, a protocol-level sample-size justification, an interim-analysis plan, and a reported multiplicity strategy for the 60 listed outcomes. The registry lists zero analysis records, but that fact does not show whether analyses were conducted elsewhere. Safety events are descriptive observations without a control group, and the reported counts do not establish comparative risk. These gaps limit statistical interpretation of the registry record without determining what results may have been reported in other sources.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function — NCT05001269.
- Registry record last updated 2026-04-16; status last verified 2026-03.
- Results first posted 2026-04-16.
- 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
NCT05001269 is a completed Phase 2, open-label, multicenter study of nedosiran in pediatric patients from birth to 11 years of age with primary hyperoxaluria and relatively intact renal function. The registry lists enrollment of 27, no allocation, no masking, and a sequential model. The recorded study dates are 2022-02-22 for start and 2025-02-05 for primary completion.
The primary endpoint is mean percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio for PH1, PH2, and PH3 subgroups. The MITT population is defined as participants who received at least one dose and had at least one post-baseline measurement. Numerical primary endpoint results and uncertainty estimates are not reported in the registry record.
Safety entries include serious and other adverse events by age group and overall, with zero deaths reported. The record lists 60 outcome measures and zero analysis records. It does not describe a separate comparator, a detailed statistical analysis plan, or methods for missing data and multiplicity.