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 Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis (NCT02613182), a na phase 2 study. The primary outcome is Long-term Safety and Tolerability of NEOD001.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02613182 |
| Phase | Phase 2 |
| Condition | AL Amyloidosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 34 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Prothena Biosciences Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
People with light chain (AL) amyloidosis who completed Study NEOD001-001 were the intended population described in the study rationale. The registry record reports enrollment of 34 participants. Further eligibility details and characteristics of the enrolled participants are not reported in the registry record.
Intervention
Participants received NEOD001 in an open-label study. The brief summary says that participants in the current NEOD001 trials were being dosed at 24 mg/kg and that this dose would continue in this study. The listed study group is NEOD001 24 mg/kg.
Comparator
There was no concurrent comparator group. The registry lists a single-group model, no masking, and allocation as not applicable. This design can describe events recorded among participants receiving NEOD001, but it does not provide a like-for-like control comparison.
Primary question
The primary outcome asks how many participants experienced the long-term safety and tolerability events covered by the registry endpoint. The endpoint describes adverse events and serious adverse events from initiation of study drug through the last study visit, or up to 30 days after the last dose, whichever came first, assessed up to 24 months. The record lists participant counts rather than a comparative treatment effect. It does not report an analysis comparing NEOD001 with another treatment or with no treatment.
3. Trial Design
This was a Phase 2, open-label extension study of NEOD001 for people with AL amyloidosis. Its stated rationale was to offer additional treatment to subjects completing Study NEOD001-001 while continuing evaluation of long-term safety and tolerability. The registry lists one study group, called Open Label, and identifies the study drug as NEOD001. The brief summary states that the 24 mg/kg dose used in the current NEOD001 trials would continue in this study.
The design was single-group, with no masking and allocation listed as not applicable. Consequently, there was no randomized control arm and no blinded comparison. The results describe participants in the NEOD001 group; differences from an untreated or differently treated group cannot be estimated from this study design. The registry lists enrollment of 34 participants.
The study started in 2016-02, and primary completion was in 2018-05. Its status is terminated. The reason for termination, the date of termination, and whether the study ended as planned are not reported in the registry record. Those details matter when interpreting how long participants were observed and whether follow-up was complete, but they cannot be inferred from the status alone.
The primary outcome is a safety and tolerability measure expressed as a count of participants. Its timeframe begins at study-drug initiation and ends at the last study visit or up to 30 days after the last dose, whichever came first, with assessment described as up to 24 months. The registry does not report detailed eligibility criteria, the actual follow-up duration for each participant, or a protocol-based rationale for the enrollment target.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | NEOD001 24 mg/kg |
|---|---|
| STARTED | 34 |
| Number of Patients Dosed | 34 |
| COMPLETED | 0 |
| NOT COMPLETED | 34 |
Reasons for not completing this period:
| Reason | NEOD001 24 mg/kg |
|---|---|
| Death | 6 |
| Physician Decision | 1 |
| Study Terminated by Sponsor | 21 |
| Withdrawal by Subject | 6 |
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
| Characteristic | Unit | NEOD001 24 mg/kg |
|---|---|---|
| Participants analysed | 34 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 17 |
| Age, Categorical: >=65 years | Participants | 17 |
| Age, Continuous | years | 65.1 (SD 8.13) |
| Sex: Female, Male: Female | Participants | 14 |
| Sex: Female, Male: Male | Participants | 20 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 34 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 1 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 32 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Long-term Safety and Tolerability of NEOD001, measured over From initiation of study drug through the last study visit or up to 30 days after date of last dose, whichever came first, assessed up to 24 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled Long-term Safety and Tolerability of NEOD001 and is measured in participants. It concerns adverse events and serious adverse events occurring from initiation of study drug through the last study visit or up to 30 days after the last dose, whichever came first, assessed up to 24 months. The registry defines an adverse event broadly: an unfavorable and unintended diagnosis, symptom, sign, syndrome, or disease that is new after baseline or worsens if already present. Serious events include occurrences meeting specified consequences, such as death, a life-threatening event, hospitalization or prolonged hospitalization, persistent or significant disability, congenital anomaly or birth defect, or another condition judged by investigators to be a significant hazard.
A participant count is useful for describing how many people experienced events during a safety follow-up period. It is not a measure of how often events occurred, how long participants remained at risk, or whether the drug caused an event. A count also does not by itself distinguish a participant with one event from a participant with repeated events. The registry does not report the detailed counting rules for this outcome, such as how recurrent events were handled or whether the primary measure combines event categories.
Because the endpoint is descriptive and the study has no comparator group, it can characterize events recorded in the enrolled group but cannot establish comparative safety. The registry does not report an inferential analysis or an estimate of uncertainty for the primary endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports a single NEOD001 24 mg/kg group with 34 participants at risk for both serious and other adverse events. Twelve participants were reported as having a serious adverse event, and 34 were reported as having another adverse event. Six deaths are listed. These are descriptive counts from the posted results, not estimates of a difference between treatments. The record does not report whether deaths were considered adverse events, whether they were judged related to NEOD001, or whether the same participants contributed to more than one reported category.
The listed serious-event terms include Cardiac failure at 5.9%, Infection at 2.9%, Lung Infection at 2.9%, Respiratory syncytial virus infection at 2.9%, Skin infection at 2.9%, and Urinary tract infection at 2.9%. These are registry-listed percentages for terms, not an adjusted comparison or a causal assessment. The record does not explain the denominator or the coding and grouping rules behind each term. The terms should therefore be read as descriptions of reported events, not as proof that NEOD001 caused them.
No confidence intervals, hypothesis tests, or model-based estimates are reported. A confidence interval, in general, describes the uncertainty around an estimated quantity under the assumptions of the method used. Without an interval or a stated analysis, the precision of these participant counts cannot be quantified from the registry record. The modest enrollment and single-group design also mean there is no internal control group against which to separate events associated with the underlying condition, background care, or other causes from events that might be related to study treatment.
The study status is terminated, but the reason and timing are not reported in the registry record. That information could help readers understand the completeness and duration of observation; the status alone does not establish why the study stopped. Individual follow-up duration, the number completing planned follow-up, and details of missing observations are also not reported.
Overall, the record supports a descriptive account of safety events among enrolled participants. It does not report a treatment-effect estimate, a comparative safety analysis, or enough detail to infer causation. The stated counts should be interpreted within the open-label extension design and its reported follow-up window.
9. Statistical Methods Explained
The registry describes a single-group study with no masking and no allocation to a comparator. Its primary endpoint is a participant count for long-term safety and tolerability. The posted safety figures are therefore descriptive summaries of the NEOD001 group, rather than results from a randomized treatment comparison. The record lists no statistical analyses. The analysis methods, estimands, model specifications, and uncertainty calculations are not reported in the registry record.
What does a participant count describe?
A participant count indicates how many people met an event definition during the period being assessed. For a safety endpoint, that can be an accessible way to communicate how many participants experienced an event. It is distinct from an event count: a person who experiences repeated episodes may still count once if the measure is the number of participants with an event. The endpoint specifies participants as its unit, but detailed rules for handling recurrent events and overlapping event categories are not reported in the registry record.
Counts need to be interpreted alongside the population at risk and the observation period. A participant observed for a longer period has more opportunity to experience an event than a participant observed for less time. The registry gives a timeframe of up to 24 months and describes an endpoint period ending at the last visit or up to 30 days after the last dose, whichever came first. Individual exposure and follow-up times are not reported. The counts therefore do not convey person-time event rates or adjust for differences in observation duration.
Why does the design matter?
A single-group, open-label design can document events that occur during treatment and follow-up. It cannot estimate what would have happened to similar participants under a different treatment or without study treatment. In a randomized comparison, treatment groups can support a direct estimate of a between-group contrast, subject to the trial's methods and assumptions. No such contrast is available in this study. The absence of masking may also affect event recognition or reporting, especially for subjective outcomes; whether that occurred is not reported.
General: statistical uncertainty is often communicated with a confidence interval around an estimate. The interval reflects sampling variability and depends on the method and assumptions used. The registry does not report a confidence interval for these safety counts, so their precision is not quantified in the posted results. A count alone should not be mistaken for a precise estimate of risk in a broader population.
What do the planning assumptions mean?
The stated sample-size calculations are described as planning illustrations based on the observed effect, not as the design justification for this trial. The listed assumptions are 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. Such calculations ask what sample size a hypothetical comparison might require under those assumptions. They are not the observed power of this single-group extension study and do not change its design into a randomized comparison. The registry does not report the trial's own sample-size justification. Any numerical planning outputs should be understood as hypothetical planning quantities, not as trial findings.
General: over-dispersion occurs when variability in observed data is greater than a statistical model assumes. It can make standard errors too small if a model fails to account for the extra variation. That issue is relevant to some count-data analyses, but no count model or over-dispersion assessment is reported for this endpoint. The endpoint is described as a count of participants, and no model-based analysis is listed.
What can be concluded from the posted methods?
The record identifies the endpoint definition, the event timeframe, the study group, and summary safety counts. It does not report a statistical analysis plan, a prespecified inferential method, adjustment for baseline characteristics, handling of missing data, or rules for multiplicity. The absence of these details does not show that no such procedures were used outside the registry; it means they are not documented in the registry record. The reported participant counts should be read as descriptive information, without attributing to them adjustment, testing, or precision estimates that are not documented.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary outcome measure and no analyses. It does not report a multiplicity strategy, such as a procedure for controlling the chance of false-positive findings across multiple statistical tests. The number of listed outcome measures does not establish how many analyses may have been planned or conducted, and the registry record does not describe a statistical analysis plan.
Missing-data methods and analysis populations are not reported in the registry record. In particular, the record does not state how participants who stopped treatment, missed visits, or had incomplete follow-up were handled in the endpoint summaries. It does not report how many participants completed follow-up or whether all enrolled participants were included in each safety summary. The 34 participants at risk in the adverse-event group summaries do not, by themselves, describe completeness of every visit or observation.
These omissions matter because safety summaries depend on how long participants are observed and how events are captured. In general, excluding participants with incomplete follow-up can give a different descriptive picture from retaining all available observations; the direction of any difference cannot be assumed. A registry reader also cannot determine whether missing observations were considered related to a participant's health or study discontinuation.
The study was terminated, but the reason is not reported. The record does not state whether termination affected the planned analysis, outcome collection, or follow-up. No imputation method, censoring rule, sensitivity analysis, or population definition is described. Consequently, the posted results can be interpreted as reported counts, but the registry record does not support a detailed assessment of missingness, multiplicity, or the analysis population.
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 |
|---|---|---|---|
| NEOD001 24 mg/kg | 12 | 34 | 35.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| NEOD001 24 mg/kg | 6 of 34 |
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 |
|---|---|
| NEOD001 24 mg/kg | 34 of 34 |
Most frequent serious adverse event terms
| Event term | Organ system | NEOD001 24 mg/kg |
|---|---|---|
| Cardiac failure | Cardiac disorders | 2 of 34 |
| Infection | Infections and infestations | 1 of 34 |
| Lung Infection | Infections and infestations | 1 of 34 |
| Respiratory syncytial virus infection | Infections and infestations | 1 of 34 |
| Skin infection | Infections and infestations | 1 of 34 |
| Urinary tract infection | Infections and infestations | 1 of 34 |
| Acute myocardial infarction | Cardiac disorders | 1 of 34 |
| Fall | Injury, poisoning and procedural complications | 1 of 34 |
| Lower limb fracture | Injury, poisoning and procedural complications | 1 of 34 |
| Hypervolaemia | Metabolism and nutrition disorders | 1 of 34 |
Most frequent non-serious adverse event terms
| Event term | Organ system | NEOD001 24 mg/kg |
|---|---|---|
| Upper respiratory tract infection | Infections and infestations | 15 of 34 |
| Oedema peripheral | Gastrointestinal disorders | 9 of 34 |
| Constipation | Gastrointestinal disorders | 8 of 34 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 8 of 34 |
| Insomnia | Psychiatric disorders | 7 of 34 |
| Fatigue | General disorders | 6 of 34 |
| Diarrhoea | Gastrointestinal disorders | 6 of 34 |
| Ecchymosis | Skin and subcutaneous tissue disorders | 6 of 34 |
| Abdominal pain | Gastrointestinal disorders | 5 of 34 |
| Nausea | Gastrointestinal disorders | 5 of 34 |
Serious events with the highest arm-level rate: cardiac failure (5.9%), infection (2.9%), lung infection (2.9%), respiratory syncytial virus infection (2.9%), skin infection (2.9%).
The NEOD001 24 mg/kg group lists 34 participants at risk for serious adverse events, with 12 participants affected. For other adverse events, 34 participants were at risk and 34 were affected. Six deaths are listed. These figures describe participants in the study group; they are not comparisons with a control group and do not establish that study drug caused an event or death.
The serious-event terms listed with percentages are Cardiac failure, 5.9%; Infection, 2.9%; Lung Infection, 2.9%; Respiratory syncytial virus infection, 2.9%; Skin infection, 2.9%; and Urinary tract infection, 2.9%. The registry does not explain whether terms could overlap for a participant, how event terms were coded, or the denominator used for the displayed percentages. Do not assume the term percentages can be added or interpreted as mutually exclusive groups.
The endpoint definition includes seriousness criteria such as death, life-threatening events, hospitalization, and other significant hazards. The registry does not report whether the six deaths were considered related to treatment, their causes, timing, or their relationship to serious adverse events. It also does not report event-level timing, duration, or recurrent-event counts. These safety figures are useful as a descriptive record, but they do not by themselves establish causality or comparative risk.
12. What the Result Does, and Does Not, Mean
The result documents safety events recorded among participants receiving NEOD001 in an open-label extension study. It reports 12 participants with serious adverse events, 34 with other adverse events, and six deaths in the listed group of 34 participants at risk. The registry also provides several serious-event terms with percentages. These data describe what was recorded in the study; they do not estimate a difference from another treatment or from no treatment.
The single-group design is central to interpretation. Without a concurrent comparator, the posted counts cannot distinguish events associated with the underlying AL amyloidosis or other circumstances from events potentially associated with study drug. The open-label design and the absence of a reported analysis plan further limit what can be concluded about event ascertainment and statistical precision. No confidence intervals, hypothesis tests, or adjusted estimates are reported.
The study's stated purpose was to provide additional NEOD001 treatment to subjects completing Study NEOD001-001 and continue evaluating long-term safety and tolerability. Its primary endpoint uses a defined observation window, but actual follow-up time for each participant and follow-up completeness are not reported. The registry also identifies the study as terminated without reporting why. These gaps limit interpretation of how comprehensively the intended observation period was completed.
Accordingly, the counts should be read as descriptive findings from this enrolled group, not as proof that NEOD001 caused the reported events or as evidence of comparative safety. The record does not support a conclusion about treatment benefit, and it does not report a statistical test of any treatment effect. General statistical principle: an event count is most informative when paired with a clear event definition, observation period, analysis population, and an appropriate comparator where a comparative question is being asked. Some of those details are documented; others are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically informative in part because it illustrates the difference between a safety description and a comparative treatment analysis. The registry records events in a single open-label group. Those counts can describe what happened among participants who received NEOD001, but they cannot estimate how the same population would have fared under a different treatment or without treatment. A reader should not interpret a count as a treatment effect when the design contains no comparator.
The outcome is expressed as participants with events, which is distinct from counting every event episode or measuring time to an event. That distinction matters for safety interpretation: participant counts do not reveal repeated events, event timing, or differences in exposure duration. The record defines a broad event timeframe, but participant-level follow-up and detailed event-counting rules are not reported.
The registry also makes visible the limits of public reporting. It gives the study status, enrollment, endpoint description, and safety summaries, while leaving the reason for termination, the statistical analysis plan, missing-data methods, and analysis populations undocumented. These omissions restrict assessment of precision and completeness. They do not justify filling gaps with assumptions.
The stated planning calculations are explicitly hypothetical, using a two-arm equal-allocation framework and an assumed effect related to the observed effect. Those planning assumptions are separate from the actual single-group extension design. Keeping planning illustrations separate from observed results is essential: a hypothetical sample-size calculation is not observed power and cannot turn descriptive data into a randomized comparison.
For biostatistical reading, the key lesson is to align the claim with the design and analysis actually reported. The registry supports a descriptive safety account, not a causal or comparative estimate. Recognizing that boundary is as important as reading the event counts themselves.
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, this was a single-group, open-label study with allocation listed as not applicable. It had no like-for-like control comparison, so background events and treatment-associated events cannot be separated using the posted results. The registry explicitly characterizes the study as not being a randomized comparison.
Second, the study enrolled 34 participants. The registry reports participant counts, but does not report confidence intervals or other measures of statistical uncertainty. The precision of the safety summaries therefore cannot be assessed from the record. The listed percentages for serious-event terms are not accompanied by an explanation of their denominators or coding rules.
Third, the study status is terminated, and the reason for termination is not reported. The record does not state how many participants completed planned follow-up, how long each participant was observed, or whether termination affected event collection. Although the endpoint timeframe is described as up to 24 months, actual follow-up durations are not reported.
Fourth, important analysis details are not reported in the registry record. These include the statistical analysis plan, analysis populations, missing-data handling, recurrent-event rules, event attribution, and any sensitivity analyses. The record also does not explain whether the reported deaths were considered treatment-related or how they overlapped with serious adverse-event reporting.
Finally, the stated sample-size approach is a planning illustration, not the study's design justification or an analysis of observed power. Its hypothetical two-arm assumptions do not match the single-group study design. These limitations do not invalidate the descriptive counts, but they restrict the conclusions that can be drawn from them.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis — NCT02613182.
- Registry record last updated 2019-05-16; status last verified 2019-05.
- Results first posted 2019-05-16.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT02613182 is a Phase 2, open-label extension study of NEOD001 in subjects with AL amyloidosis. Its stated rationale was continued treatment for subjects completing Study NEOD001-001 and further evaluation of long-term safety and tolerability. The registry lists a single group receiving NEOD001 24 mg/kg, enrollment of 34, no masking, and no randomized comparator. The study started in 2016-02, reached primary completion in 2018-05, and is listed as terminated.
The primary endpoint measures participants with long-term safety and tolerability events over the stated follow-up window, assessed up to 24 months. The posted safety results list 12 participants with serious adverse events, 34 with other adverse events, and six deaths. No statistical analyses are listed. The reason for termination, analysis plan, missing-data methods, participant-level follow-up, and treatment attribution for events are not reported in the registry record. The findings are descriptive and do not provide a comparative estimate or establish causality.