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 CM-101 in PSC Patients -The SPRING Study (NCT04595825), a randomized phase 2 study. The primary outcome is Number of Participants With Treatment-emergent Adverse Events (TEAEs) (Double-blind).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04595825 |
| Phase | Phase 2 |
| Condition | Primary Sclerosing Cholangitis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 77 |
| Arms in the primary outcome | n/a |
| Lead sponsor | ChemomAb Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
Adults with primary sclerosing cholangitis (PSC) were eligible for the study. The registry record lists 77 enrolled participants and describes 76 patients in the safety population, with one patient discontinuing before dosing. Further eligibility details and the characteristics of the analyzed participants are not reported in the registry record.
Intervention
The study evaluated CM-101, an anti-human CCL24 monoclonal antibody, given as an intravenous infusion over 60 minutes, with a permitted variation of ±5 minutes. During the double-blind period, the recorded dose groups were 10 mg/kg and 20 mg/kg.
Comparator
The comparator was matching placebo, administered by intravenous infusion. Randomization assigned participants to CM-101 or placebo; the record describes a parallel-group design and double masking for the initial treatment period.
Primary question
The primary endpoint counted participants with treatment-emergent adverse events during the 15 week double-blind treatment period. The endpoint description includes any, related, serious, and severe events and identifies it as safety-related. The registry record does not report a single comparative efficacy result or an inferential analysis for this endpoint.
3. Trial Design
The SPRING Study is a randomized, double-blind, placebo-controlled phase 2a clinical trial in people with primary sclerosing cholangitis. Its parallel-group design assigned participants to CM-101 or matching placebo. The CM-101 treatment groups used doses of 10 mg/kg or 20 mg/kg, administered by intravenous infusion; placebo was also administered intravenously. The registry describes the CM-101 infusion as lasting 60 minutes, with a permitted variation of ±5 minutes.
The registry record lists 77 enrolled participants. Its primary endpoint description states that 76 patients were analyzed in the safety population because one patient discontinued before dosing. At least 68 participants and approximately 50 sites were anticipated in the brief study summary; those statements describe the study plan, rather than the final enrollment or number of participating sites. The final number of sites is not reported in the registry record.
The study began on 2020-10-01, reached primary completion on 2025-01-10, and is marked completed. The primary safety endpoint covers the 15 week double-blind treatment period. The registry also lists open-label safety outcomes and serum ALP outcomes by baseline Fibroscan categories. The outcome list includes 17 measures, but the record does not give a complete account of the timing and analysis population for every measure.
Details such as the allocation ratio, randomization procedure, planned follow-up beyond the stated endpoint timeframe, and operational methods for maintaining masking are not reported in the registry record. Nor does the record provide a full protocol or explain how participants entered the open-label period. These omissions limit reconstruction of the trial conduct from the registry entry alone.
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: A total of 134 patients were screened in the study across 52 sites globally. Of these, 57 patients failed to meet inclusion and/or exclusion criteria at screening. Inclusion/Exclusion criteria that excluded \>15% included 1. Serum ALP greater than 1.5 x ULN (31.0%) and 2. Subjects with evidence of cirrhosis (15.5%).
Double-blind
| Stage | Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Placebo (Double-blind) | Placebo (Double-blind) to CM-101 - 10mg/kg (Open-label) | Placebo (Double-blind) to CM-101 - 20mg/kg (Open-label) |
|---|---|---|---|---|---|
| STARTED | 25 | 31 | 21 | 0 | 0 |
| COMPLETED | 22 | 29 | 19 | 0 | 0 |
| NOT COMPLETED | 3 | 2 | 2 | 0 | 0 |
Open-label
| Stage | Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Placebo (Double-blind) | Placebo (Double-blind) to CM-101 - 10mg/kg (Open-label) | Placebo (Double-blind) to CM-101 - 20mg/kg (Open-label) |
|---|---|---|---|---|---|
| STARTED | 11 | 27 | 0 | 1 | 11 |
| COMPLETED | 6 | 25 | 0 | 0 | 11 |
| NOT COMPLETED | 5 | 2 | 0 | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: 76 patients analyzed in Safety Population as 1 patient discontinued prior to dosing.
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 | Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Placebo (Double-blind) to CM-101 - 10mg/kg or 20mg/kg (Open-label) | Total |
|---|---|---|---|---|---|
| Participants analysed | 25 | 31 | 21 | 77 | |
| Age, Continuous | years | 48.6 (SD 15.19) | 45.6 (SD 10.27) | 40.5 (SD 12.84) | 45.3 (SD 12.94) |
| Sex: Female, Male: Female | Participants | 9 | 14 | 7 | 30 |
| Sex: Female, Male: Male | Participants | 16 | 17 | 14 | 47 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 0 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 2 | 1 | 4 |
| Race (NIH/OMB): White | Participants | 19 | 28 | 20 | 67 |
| Race (NIH/OMB): More than one race | Participants | 2 | 0 | 0 | 2 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 3 | 0 | 0 | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 3 | 3 | 1 | 7 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 19 | 27 | 17 | 63 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 3 | 1 | 3 | 7 |
| Time (years) since diagnosis of large duct PSC | years | 6.4336 (SD 6.8762) | 8.284 (SD 6.9798) | 6.606 (SD 6.1843) | 7.235 (SD 6.7141) |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment-emergent Adverse Events (TEAEs) (Double-blind), measured over 15 week double-blind (DB) treatment period.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants who experienced treatment-emergent adverse events during the 15 week double-blind treatment period. The registry description identifies events as any, related, serious, or severe, and classifies this as a safety-related endpoint. It is a count of participants with events, not a measure of the number of event episodes or the duration of each event.
Safety endpoints are used to describe unwanted events recorded during treatment and to compare their occurrence across randomized groups. Counting participants with at least one event gives a direct summary of how many people were affected, but it does not show how often events recurred in the same person, their timing, or their clinical course. The registry record does not provide definitions for treatment-emergent, related, serious, or severe events, nor the rules used to attribute an event to treatment.
The posted primary outcome is not presented as one comparative estimate. The record lists group-level safety counts, but reports no effect estimate, confidence interval, or statistical test for the primary endpoint. Therefore, the counts can be read descriptively, while the precision of any between-group difference cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports primary-period safety counts for three double-blind groups. In the CM-101 10 mg/kg group, 18 of 25 participants were recorded with other adverse events and 2 of 25 with serious adverse events. In the CM-101 20 mg/kg group, the corresponding counts were 28 of 31 and 0 of 31. In the placebo group, they were 15 of 20 and 1 of 20. The record also reports zero deaths in each of these groups. These are descriptive group summaries, not estimates adjusted for participant characteristics or exposure time.
The “other adverse events” counts should not be interpreted as a mutually exclusive category alongside serious events unless the registry’s definitions establish that distinction. The record does not specify whether participants with serious events are also included in the other-event count. It also does not supply event-level detail, recurrence counts, exposure-adjusted rates, or inferential comparisons. The displayed counts therefore do not establish whether any observed group difference is compatible with random variation or reflects a treatment effect.
Open-label-period summaries are reported separately. They include participants continuing CM-101 and participants who received placebo during the double-blind period and then entered an open-label CM-101 group. For example, serious adverse events are listed for 2 of 11 in the open-label 10 mg/kg group and 0 of 27 in the open-label 20 mg/kg group; the placebo-to-open-label groups are listed as 1 of 1 and 0 of 11. These groups should not be treated as a randomized placebo comparison: the treatment periods and prior treatment histories differ, and the registry record does not provide an analysis accounting for those differences.
Although the registry marks the trial completed and reports outcome counts, it does not report a statistical analysis for the primary endpoint. The record lists 17 outcome measures and no analyses. It also does not provide a confidence interval or p-value for a between-group contrast. Consequently, the available information supports a descriptive account of recorded events, but not a claim that one randomized group had a statistically established increase or decrease in risk.
The study summary describes an assessment of safety, tolerability, and activity. However, no single efficacy outcome or comparative efficacy result is identified in the posted primary endpoint information. Secondary outcomes include serum ALP percentage changes in baseline Fibroscan categories, but the registry record does not give their numerical results or analysis details in the registry record for this trial. Clinical or efficacy conclusions therefore cannot be drawn from the safety counts alone.
9. Statistical Methods Explained
What kind of outcome is counted?
The primary endpoint is a binary participant-level outcome: each person is counted as having or not having a treatment-emergent adverse event under the registry’s endpoint definition. A group count is useful for describing how many participants were affected. It does not capture how many separate events each person experienced, how long events lasted, or the time from treatment initiation to an event. Those distinctions matter when event burden or timing is important.
As a general statistical principle, a comparison of binary outcomes may be expressed as a difference in proportions, a ratio of proportions, or another effect measure. Such measures answer different questions. An effect estimate is more informative when accompanied by a confidence interval, which describes the range of values reasonably compatible with the data and the statistical method used. The registry record does not report a comparative estimate or confidence interval for the primary endpoint, so none can be interpreted for this trial.
Why does randomization matter?
In general, random assignment aims to make treatment groups comparable, on average, with respect to factors that could affect outcomes. This supports interpretation of between-group contrasts, provided the trial is conducted and analyzed as planned. Randomization does not guarantee that all characteristics will be balanced in a particular trial, especially when groups are modest in size. The registry record does not report baseline characteristics or a prespecified adjusted analysis, so the balance of prognostic factors cannot be assessed from this information.
Blinding can reduce the influence of treatment knowledge on participant reporting, clinical assessment, and event attribution. The study is described as double-blind and placebo-controlled for the double-blind period. The registry record does not provide details about who was masked, how masking was maintained, or whether its success was assessed. Open-label outcomes are a separate context because treatment assignment is no longer concealed in the same way.
How should the counts be compared?
As a general principle, raw counts should be read with their group denominators. The posted double-blind counts include both participants with events and the number at risk in each group. A count alone can be misleading when group sizes differ; a proportion places the count in relation to the group denominator, while an effect estimate and its uncertainty are needed for a statistical comparison. No new comparison is calculated. The registry record does not report the endpoint’s inferential method, a p-value, or an interval estimate.
For safety data, event definitions and overlaps are important. A participant may experience more than one event, and may fall into more than one seriousness or severity category. The registry record does not specify whether the “other” and serious-event summaries overlap, how relatedness was judged, or whether events were coded using a particular classification system. It is therefore important not to treat the categories as independent or mutually exclusive without further documentation.
What do the planning illustrations mean?
General sample-size calculations depend on an assumed effect, a significance threshold, allocation, and a statistical model. The planning assumptions stated for the trial’s illustrations use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects defined as fractions of the observed effect. Such calculations are planning illustrations, not observed power and not evidence that a result is statistically significant. The registry record does not report the trial’s own sample-size justification, and the posted information does not provide a single primary comparison from which to assess those illustrations as a definitive design rationale.
For general interpretation, a planning calculation asks how much information a future study might need under chosen assumptions. It does not show the probability that the completed trial has detected a real effect, nor does it repair the absence of a reported comparison. The record does not state whether its assumptions match the original protocol or whether the trial had different design targets.
Why can over-dispersion matter?
As a general concept, over-dispersion occurs when event-count variability is greater than a model assumes. It can make conventional uncertainty estimates too narrow if ignored. The primary endpoint counts participants with an event rather than modeling recurrent event totals, so over-dispersion is not directly assessed by the posted summary. If repeated events were analyzed elsewhere, the relevant model and handling of extra variability are not reported in the registry record.
More broadly, the record provides outcome counts but no statistical analysis details for the primary endpoint. The analysis population is identified as the safety population, while analysis rules for other outcomes are not described. Missing-data methods, covariate adjustment, treatment of intercurrent events, and sensitivity analyses are not reported in the registry record. These omissions mean that the posted summaries can be described, but the full inferential approach cannot be reconstructed.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 17 outcome measures, including safety outcomes during double-blind and open-label periods and serum ALP percentage changes in baseline Fibroscan categories. When a study examines multiple outcomes, time points, or subgroups, the chance of finding at least one apparently unusual result can increase. Statistical plans sometimes address this multiplicity through a prespecified testing sequence or an adjustment. The registry record does not report whether multiplicity was addressed, which outcomes were formally tested, or whether any hierarchy of outcomes was prespecified.
The record identifies 76 patients in the safety population and notes that one patient discontinued before dosing. It does not state how missing outcome data were handled, whether any participants had incomplete follow-up, or whether analyses used imputation, censoring, or another approach. Those methods are not reported in the registry record. Because missingness can affect both group counts and uncertainty, the absence of this information limits interpretation.
The primary endpoint population is described as the safety population, but the registry does not report a complete analysis-population plan for all outcomes. It also does not describe an intention-to-treat analysis, protocol deviations, or exclusions after randomization beyond the patient who discontinued before dosing. Interim analyses and stopping rules are not reported in the registry record. These gaps do not invalidate the posted descriptive counts, but they prevent a full assessment of how analysis choices may have affected comparisons.
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 |
|---|---|---|---|
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Double-blind Period | 2 | 25 | 8.0% |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Double-blind Period | 0 | 31 | 0.0% |
| Placebo | Double-blind Period | 1 | 20 | 5.0% |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Open-label Period | 2 | 11 | 18.2% |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Open-label Period | 0 | 27 | 0.0% |
| Placebo (Double-blind) Then CM-101 - 10mg/kg | Open-label Period | 1 | 1 | 100.0% |
| Placebo (Double-blind) Then CM-101 - 20mg/kg | Open-label Period | 0 | 11 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Double-blind Period | 0 of 25 |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Double-blind Period | 0 of 31 |
| Placebo | Double-blind Period | 0 of 20 |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Open-label Period | 0 of 11 |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Open-label Period | 0 of 27 |
| Placebo (Double-blind) Then CM-101 - 10mg/kg | Open-label Period | 0 of 1 |
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 |
|---|---|
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Double-blind Period | 18 of 25 |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Double-blind Period | 28 of 31 |
| Placebo | Double-blind Period | 15 of 20 |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Open-label Period | 9 of 11 |
| Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Open-label Period | 25 of 27 |
| Placebo (Double-blind) Then CM-101 - 10mg/kg | Open-label Period | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Double-blind Period | Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Double-blind Period | Placebo | Double-blind Period | Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Open-label Period | Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Open-label Period | Placebo (Double-blind) Then CM-101 - 10mg/kg | Open-label Period |
|---|---|---|---|---|---|---|---|
| Obstructive Pancreatitis | Gastrointestinal disorders | 1 of 25 | 0 of 31 | 0 of 20 | 1 of 11 | 0 of 27 | 0 of 1 |
| Cholangitis | Hepatobiliary disorders | 0 of 25 | 0 of 31 | 0 of 20 | 0 of 11 | 0 of 27 | 1 of 1 |
| Wound Infection | Infections and infestations | 0 of 25 | 0 of 31 | 0 of 20 | 1 of 11 | 0 of 27 | 0 of 1 |
| Subdural Haematoma | Injury, poisoning and procedural complications | 0 of 25 | 0 of 31 | 0 of 20 | 1 of 11 | 0 of 27 | 0 of 1 |
| Colitis Ulcerative | Gastrointestinal disorders | 0 of 25 | 0 of 31 | 0 of 20 | 1 of 11 | 0 of 27 | 0 of 1 |
| Crohn's Disease | Gastrointestinal disorders | 0 of 25 | 0 of 31 | 1 of 20 | 0 of 11 | 0 of 27 | 0 of 1 |
| Cholecystectomy | Surgical and medical procedures | 1 of 25 | 0 of 31 | 0 of 20 | 0 of 11 | 0 of 27 | 0 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Double-blind Period | Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Double-blind Period | Placebo | Double-blind Period | Anti-human CCL24 Monoclonal Antibody (CM-101) - 10mg/kg | Open-label Period | Anti-human CCL24 Monoclonal Antibody (CM-101) - 20mg/kg | Open-label Period | Placebo (Double-blind) Then CM-101 - 10mg/kg | Open-label Period |
|---|---|---|---|---|---|---|---|
| Fatigue | General Disorders And Administration Site Conditions | 5 of 25 | 12 of 31 | 6 of 20 | 1 of 11 | 8 of 27 | 0 of 1 |
| Headache | Nervous System Disorders | 2 of 25 | 9 of 31 | 4 of 20 | 0 of 11 | 5 of 27 | 0 of 1 |
| Pruritus | Skin And Subcutaneous Tissue Disorders | 5 of 25 | 4 of 31 | 3 of 20 | 2 of 11 | 6 of 27 | 0 of 1 |
| Diarrhoea | Gastrointestinal Disorders | 2 of 25 | 3 of 31 | 2 of 20 | 1 of 11 | 4 of 27 | 0 of 1 |
| Nasopharyngitis | Infections And Infestations | 0 of 25 | 4 of 31 | 2 of 20 | 0 of 11 | 6 of 27 | 0 of 1 |
| Infusion Site Pain | General Disorders And Administration Site Conditions | 3 of 25 | 1 of 31 | 3 of 20 | 1 of 11 | 1 of 27 | 0 of 1 |
| Dizziness | Nervous System Disorders | 0 of 25 | 3 of 31 | 3 of 20 | 0 of 11 | 2 of 27 | 0 of 1 |
| Influenza | Infections And Infestations | 0 of 25 | 3 of 31 | 2 of 20 | 0 of 11 | 2 of 27 | 0 of 1 |
| Upper Respiratory Tract Infection | Infections And Infestations | 2 of 25 | 2 of 31 | 1 of 20 | 0 of 11 | 2 of 27 | 0 of 1 |
| Urinary Tract Infection | Infections And Infestations | 3 of 25 | 1 of 31 | 2 of 20 | 0 of 11 | 1 of 27 | 0 of 1 |
Serious events with the highest arm-level rate: cholangitis (100.0%), obstructive pancreatitis (9.1%), wound infection (9.1%), subdural haematoma (9.1%), colitis ulcerative (9.1%).
For the double-blind period, the registry lists serious adverse events for 2 of 25 participants receiving CM-101 10 mg/kg, 0 of 31 receiving CM-101 20 mg/kg, and 1 of 20 receiving placebo. It reports zero deaths in each of those groups. These figures count participants, not necessarily separate serious-event episodes. The registry does not report a statistical comparison or confidence interval for these counts.
The open-label summaries are separate: serious events are listed for 2 of 11 in the CM-101 10 mg/kg group and 0 of 27 in the CM-101 20 mg/kg group. Among participants moving from placebo into open-label CM-101, the record lists 1 of 1 in the 10 mg/kg group and 0 of 11 in the 20 mg/kg group. These summaries have different treatment histories and are not equivalent to the randomized double-blind comparison.
The registry also names serious-event terms and gives percentages, including Cholangitis at 100.0% and several other terms at 9.1%. The denominator and counting basis for these term percentages are not reported in the registry record, so they should not be read as percentages of all enrolled participants or as comparative risks. Serious-event counts describe what was recorded; by themselves, they do not establish causation or a treatment difference.
12. What the Result Does, and Does Not, Mean
The posted results show that treatment-emergent adverse events were recorded in each double-blind group, with the group counts differing across the CM-101 dose groups and placebo. Serious-event counts were also recorded, and the registry reports zero deaths in the double-blind groups. These observations are descriptive summaries for the stated safety population and treatment period.
They do not establish that CM-101 caused or prevented any particular event, or that one group had a statistically reliable difference in safety. The registry record gives no comparative effect estimate, confidence interval, or p-value for the primary endpoint. It also does not provide event-level descriptions, exposure-adjusted rates, or a complete account of event definitions and overlap. The counts alone cannot resolve these uncertainties.
The trial’s stated purpose includes assessing activity, but the posted information described for the primary endpoint is safety-focused. The registry lists serum ALP outcomes as secondary measures, including categories based on baseline Fibroscan values, but numerical results and analysis details for those outcomes are not reported in the registry record. The safety summaries should not be used to infer efficacy.
The open-label counts have a different interpretive status from the randomized double-blind results. Participants in those groups had different treatment histories, and no placebo-controlled comparison is reported for the open-label period. As a general statistical principle, comparisons across such groups may be affected by selection, exposure duration, and other differences. The record does not report analyses that address those factors.
Overall, the registry supports a limited description of recorded safety events. It does not provide enough inferential detail to determine the precision of treatment comparisons or to support broader conclusions about benefit and harm. Additional protocol and analysis information would be needed for a fuller statistical assessment.
13. Why This Trial Matters Statistically
This trial illustrates why a registry entry can be useful for identifying the clinical question and the outcomes collected while still offering limited evidence for comparative inference. The record specifies a randomized, double-blind, placebo-controlled design and identifies the primary endpoint as a participant count of treatment-emergent adverse events. It also reports group-level safety counts, making the basic descriptive pattern visible.
Statistical interpretation depends on more than the counts themselves. Readers need to know how events were defined and classified, whether categories overlap, how follow-up and missing data were handled, and how uncertainty around group contrasts was estimated. The registry record does not report those details or a primary endpoint analysis. The distinction between descriptive results and inferential evidence is especially important when interpreting safety outcomes with small counts.
The study also has multiple listed outcomes spanning treatment periods and outcome types. Without a reported analysis plan or multiplicity strategy, it is not possible to determine which comparisons were intended to be confirmatory or how multiple testing was handled. Open-label observations may add information about events recorded during continued treatment, but they do not replace the randomized comparison.
For biostatisticians and clinical researchers, the SPRING record is a reminder to separate what a registry explicitly reports from what would require a protocol, statistical analysis plan, or full results publication. The record documents the trial design, enrollment, safety population, and selected adverse-event summaries. It does not, on its own, provide a complete basis for estimating comparative treatment effects or judging their precision.
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 registry record contains limited detail for reconstructing the statistical analysis. It identifies the primary safety endpoint and the safety population but reports no primary endpoint analysis, comparative estimate, confidence interval, or p-value. Definitions for treatment-emergent, related, serious, and severe events are not reported in the registry record. Nor does the record explain whether serious events are included in the “other” adverse-event counts.
Participant flow is only partly described. The record states that 76 patients were analyzed in the safety population because one patient discontinued before dosing, while enrollment was 77. Reasons for discontinuation, group-specific disposition, protocol deviations, and completeness of follow-up are not reported in the registry record. Missing-data handling and analysis populations for secondary outcomes are also not reported.
The open-label summaries cannot be read as the same randomized comparison as the double-blind groups. Treatment history differs, and the registry record does not report an approach for accounting for those differences. The term-specific percentages for serious adverse events are listed without a reported denominator or clear counting basis. These details constrain interpretation of both the safety summaries and any attempt to compare groups.
The registry describes 17 outcome measures but does not report analyses, multiplicity handling, interim analyses, or the statistical plan. It also does not provide numerical secondary efficacy results in the record information summarized for this trial. These are limitations of the information available in the registry record, not proof that analyses or documentation do not exist elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: CM-101 in PSC Patients -The SPRING Study — NCT04595825.
- Registry record last updated 2026-07-02; status last verified 2026-06.
- Results first posted 2026-07-02.
- 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
The SPRING Study is a completed, randomized, double-blind, placebo-controlled phase 2a trial of CM-101 in adults with primary sclerosing cholangitis. The registry reports 77 enrolled participants and identifies a safety population of 76 patients, with one patient discontinuing before dosing. The primary endpoint counts participants with treatment-emergent adverse events during the 15 week double-blind treatment period.
Double-blind safety counts are reported for CM-101 10 mg/kg, CM-101 20 mg/kg, and placebo, with separate open-label summaries also listed. The registry reports no primary endpoint analysis or comparative uncertainty estimate. Missing-data procedures, multiplicity handling, and a full statistical analysis plan are not reported in the registry record. The posted counts therefore provide descriptive safety information, but not a complete inferential comparison or a basis for efficacy conclusions.