Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision (NCT04974307), a non-randomized na study. The primary outcome is Participants With Adverse Events.

2. Clinical Question

The study's stated aim was to learn whether children with CLN3 or non-CLN3-related blindness could use the device safely, easily, and usefully. Its broader assessment included tasks before and after training, with and without use of the device, and a period of device use lasting 1 week or 1 month. Those activities describe the study's context; the posted primary outcome is narrower, covering adverse events during week one.

The clinical question is therefore primarily about short-term reported safety, rather than whether the device changes vision, learning, independence, or another clinical outcome. The registry record does not report a comparator-based estimate of treatment effect. Nor does it report a separate numerical result for ease of use or usefulness in the primary outcome. The results should be read as descriptive counts within the recorded groups, not as evidence from a randomized comparison.

3. Trial Design

This was a pilot study of the OrCam MyEye 2 in a pediatric cohort with CLN3-related conditions or low vision. The registry lists the phase as not applicable, a non-randomized allocation, no masking, and a parallel model. Enrollment was 11. The study status is terminated. Its recorded start date is 2021-09-30 and primary completion date is 2023-10-06.

The registry describes two participant groups: children with CLN3 disease and children with low vision without CLN3. Both groups used the same device, which is attached to eyeglass frames and converts camera-captured information to auditory output. Participants and parents or guardians received training. Because allocation was non-randomized and no control intervention is described, differences between the groups cannot be interpreted as effects of the device or of diagnosis.

The study description includes screening procedures such as medical and family history, physical and eye examinations, vision testing, psychological and neurocognitive assessments, hearing tests, and biological samples. The description also says participants completed tasks before and after training, with and without the device, and received a device to use for 1 week or 1 month with check-ins from the study team. These details outline the broader study activities, but the primary endpoint reported in the registry is the adverse-event count for week one.

The registry does not report a protocol-level explanation for termination, a detailed recruitment flow, or why the primary outcome population is smaller than the enrollment figure. For that outcome, the group counts are 9 participants with CLN3 disease and 1 participant with low vision without CLN3. The record does not explain the difference between those counts and the overall enrollment. That gap matters when judging how representative the reported safety counts are of everyone enrolled.

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: Of 11 participants consented for the study, one participant was a screen failure

Overall Study

Analysis population for the primary outcome, as defined in the registry: All participants who completed week one of the study.

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.

6. Primary Endpoint

The registry lists the primary outcome as Participants With Adverse Events, measured over 1 week.

The primary endpoint is the number of participants with adverse events related to OrCam MyEye 2 use during 1 week. The registry defines the outcome as a count of participants, not a count of every event experienced. Events were collected through parental report, and the analysis population is described as all participants who completed week one.

A participant-based count is useful for a pilot safety assessment because it records whether any participant was reported to have an event during the specified observation period. It can provide an initial description of reported tolerability and help identify events that may warrant closer tracking in later research. It does not, by itself, describe the number of events per participant, their duration, their intensity, or their timing within the observation period.

The record reports serious and other adverse-event counts by group. For serious adverse events, the recorded counts are zero in both groups; the other-event counts are 1 among children with CLN3 disease and 0 among children with low vision without CLN3. These are descriptive observations from the recorded groups. The registry does not report confidence intervals, a hypothesis test, or an adjusted estimate for the primary outcome.

The endpoint is limited to week one even though the broader study description includes device use for 1 week or 1 month. The record does not report a longer-term adverse-event result in the posted primary outcome. It also does not report a separate numerical outcome for ease or usefulness in this endpoint. Consequently, this measure addresses only reported adverse events over the stated short-term period; it cannot establish longer-term safety or characterize all aspects of device use.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The outcome is binary at the participant level: a person either had a recorded adverse event during the specified period or did not. The registry presents it as a count of participants, rather than as a continuous measurement. General statistical interpretation of such an endpoint begins with the number of people assessed and the number with an event. Here, the record describes the outcome population as those who completed week one and lists the groups separately.

What a participant count captures

A participant count gives a direct description of how many people in the recorded group had an event. It does not count repeat events as additional participants, so it cannot describe the total burden of recurrent events. Nor does it communicate the event's clinical characteristics. Those details would require event-level reporting, such as descriptions and timing, which are not reported in the registry record.

For a binary outcome, an event proportion is often used alongside a count to convey how common the event was among those assessed. The posted information gives counts and group sizes, but the registry does not report an inferential estimate or confidence interval for the primary outcome. No additional percentage is stated. A confidence interval, as a general statistical concept, represents uncertainty around an estimated quantity under the assumptions of a chosen method. It is especially relevant when the event count and group size are small, because the observed count alone can be compatible with a broad range of underlying rates.

Why the design affects interpretation

Random allocation is a method for balancing measured and unmeasured participant characteristics across comparison groups, on average. This study is explicitly non-randomized. Its two groups are organized by diagnosis, and both are described as using the device. They are not a like-for-like control comparison. Thus, a difference in recorded events between groups could reflect group composition, reporting, or other differences, rather than a device effect. The registry does not report an adjusted comparison intended to address such differences.

Masking is listed as none. In general, masking can reduce the influence of knowledge about an intervention on assessment or reporting. In a device study, participants and caregivers may know whether they use the device; the record lists no masking and says the events were collected by parental report. The registry does not explain whether any independent review was used to limit the effect of expectations on event classification.

What uncertainty can and cannot be quantified

The registry record lists no analyses for the primary outcome. It therefore does not report a test statistic, p-value, confidence interval, or adjusted model. Without those results, the posted counts should not be treated as evidence of a statistically established difference between groups. Even if an inferential test were available, a non-randomized comparison would still require careful consideration of confounding and group comparability.

General statistical reasoning also emphasizes that zero observed events do not imply zero underlying risk. With limited observation and a small group, an event may simply not have been observed during the recorded period. Precision would be conveyed by an uncertainty interval, but none is reported. Likewise, an observed event count does not establish causation. Attribution requires more than temporal occurrence, and the event-level adjudication details are not reported in the registry record.

Planning calculations are not observed results

The registry describes assumptions for a sample-size planning illustration: a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. Such calculations are planning illustrations, not additional trial observations and not evidence that the study had a particular probability of detecting an effect. They should not be interpreted as observed power. The registry does not report the trial's own sample-size justification or a statistical plan explaining whether these assumptions were used in its design.

For a binary participant outcome, general planning methods depend on the assumed event frequency, the difference one wishes to detect, the chosen error rate, and the allocation scheme. Results can be highly sensitive to those assumptions when event counts are sparse. The registry does not provide an inferential analysis of the observed adverse-event counts, and no conclusion about statistical significance can be drawn from the counts alone.

Observation period and analysis population

The endpoint's timeframe is 1 week, and the population is described as participants who completed that week. This matters because people who do not complete follow-up may have different event experiences from those who do. The registry does not report the number or characteristics of participants who did not complete week one, the reasons for non-completion, or any method for handling missing outcome data. The enrollment figure and the group counts for the endpoint do not align, and the record does not explain the gap.

Overall, the methods support a descriptive reading of the recorded short-term counts, not a causal or comparative conclusion. Important statistical details—including a full analysis plan, uncertainty estimates, missing-data handling, and event-level definitions—are not reported in the registry record.

10. Exact Estimates for Each Arm

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

R code and output

arm    <- c("Children With CLN3 Disease", "Children With Low Vision Without CLN3")
n      <- c(9, 1)
events <- c(1, 0)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Children With CLN3 Disease  9  1  11.1  0.3  48.2  2.0  43.5  2.5  44.5
ARM  Children With Low Vision Without CLN3  1  0  0.0  0.0  97.5  0.0  79.3  1.3  84.2

11. Multiplicity, Missing Data and Analysis Populations

The registry lists one primary outcome measure and no analyses. It does not report a statistical analysis plan describing prespecified tests, model choices, or how multiple outcomes or comparisons would be handled. The broader study description mentions assessments of tasks, abilities, behaviors, social skills, and learning, but the registry record does not provide numerical results for those assessments in the posted primary outcome. It is therefore not possible to assess whether multiple testing was planned or whether any adjustment was made.

Multiplicity is a general statistical concern that arises when many outcomes, time points, or comparisons are examined. The more analyses conducted, the greater the chance that an apparently notable result could occur by chance. The record does not provide enough information to determine how this issue applies to the study's broader assessments. No claim about multiplicity adjustment should be inferred.

The primary outcome population is described as participants who completed week one. The registry enrollment is 11, while the adverse-event groups list 9 participants with CLN3 disease and 1 participant with low vision without CLN3. The record does not report a participant flow accounting for the difference, reasons for non-completion, or an approach to missing outcomes. It also does not state whether any imputation or sensitivity analysis was used.

These omissions limit interpretation because complete-case results can differ from results that would have been observed if all enrolled participants had outcome data. That is a general concern, not evidence that missingness biased this study. The registry record does not report enough information to judge the direction or magnitude of any such effect, nor does it describe whether the analysis population differed from the full enrolled cohort in relevant ways.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry reports serious adverse-event counts of 0 among 9 children with CLN3 disease and 0 among 1 child with low vision without CLN3. It also lists 0 deaths in each group. The other adverse-event counts are 1 in the CLN3 group and 0 in the low-vision group. These are counts for the participants represented in the registry's group listings, not estimates for all children who might use the device.

In general, a serious adverse event is a regulatory classification and is not interchangeable with the everyday meaning of “severe.” A serious event is classified by its consequences or criteria, while severity describes intensity. The registry's serious-event counts therefore should not be read as a complete description of how burdensome any non-serious event may have been. The record does not provide event descriptions, severity ratings, timing, or duration.

The absence of a recorded serious event in these groups does not establish that serious events cannot occur. The observation period for the primary endpoint is 1 week, and one group contains only 1 participant. The registry also does not describe independent event adjudication; events were collected by parental report. These data support only a cautious description of what was recorded during the stated period.

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

The result describes a small set of participant reports over a short observation period. The registry lists no serious adverse events in either recorded group, with one other adverse event in the CLN3 group and none in the low-vision-without-CLN3 group. This is useful as an initial account of what was recorded during week one, but the counts are not a precise estimate of the frequency of events in a wider population.

The results do not establish that the device is safe for every child, that adverse events cannot occur, or that a reported event was caused by the device. They also do not establish that the device improves vision-related abilities, learning, social skills, or independence. Those are distinct questions, and the primary endpoint reported in the registry is an adverse-event count. Although the broader study description includes tasks and participant or caregiver questions, corresponding numerical results are not reported in the registry record.

The study does not provide a randomized control group for estimating what would have happened without the device. The diagnosis-based groups also are not a like-for-like comparison. Consequently, the difference in their recorded counts should not be used to attribute a difference in safety to diagnosis or device use.

The enrollment figure is 11, but the outcome group listings account for a smaller set of participants. The registry does not explain this difference or report how missing week-one outcomes were handled. This leaves uncertainty about how fully the recorded counts represent all enrolled participants.

In general, pilot studies can help describe procedures and identify questions for further study, but their results are not a substitute for larger, well-characterized comparisons. In this case, the posted evidence is limited to short-term, parent-reported adverse-event counts. The registry does not report confidence intervals, inferential analyses, or detailed event information that would support a more precise safety conclusion.

14. Why This Trial Matters Statistically

This study is statistically important to read as a pilot safety description rather than as a comparative effectiveness trial. Its non-randomized, parallel design and diagnosis-based groups determine what can be learned from the posted counts. The figures offer an early record of reported adverse events during week one, but they do not estimate a causal effect of the device.

Small groups are common in pilot research, particularly when the study concerns a specific pediatric population. Statistically, however, sparse observations provide limited precision. A zero count can be informative as a description of the observed participants, while still leaving substantial uncertainty about what might occur in a broader group. The low-vision-without-CLN3 group has 1 participant, making broad generalization especially constrained.

The endpoint also illustrates the difference between counting affected participants and characterizing events. Participant counts can answer whether an event was recorded for someone during the observation period. Without event descriptions, timing, severity, and recurrence information, they cannot fully characterize the safety experience. Parental reporting is the stated collection method, and the registry does not report independent adjudication or details of event ascertainment.

Finally, the distinction between enrollment and the primary outcome population is important. The registry reports enrollment of 11 and defines the primary population by week-one completion, while the event listings cover 9 and 1 participants across the groups. A clear participant flow and missing-data account would help readers understand which participants contribute to the results. Those details are not reported in the registry record.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

Several limitations specific to this trial constrain interpretation. The enrollment was 11, and the adverse-event group listings include 9 children with CLN3 disease and 1 child with low vision without CLN3. The registry does not explain the difference between enrollment and the listed outcome groups. Without a participant flow, it is unclear why the outcome population differs from enrollment or whether the reasons for non-completion could be related to device use or adverse events.

The groups are small and markedly unequal in their listed sizes. The study was non-randomized, and group membership was based on diagnosis. Both groups used the OrCam MyEye 2, so neither serves as a no-device control. The registry record does not report adjusted analyses that might address differences in participant characteristics. Any comparison between the groups would therefore be descriptive and potentially confounded.

The primary endpoint covers 1 week, while the broader study description says participants could receive a device for 1 week or 1 month. The registry does not report a longer-term adverse-event outcome or clarify the exposure duration for each participant in the primary analysis. The event collection method was parental report, but the record does not describe standardized definitions, independent review, or event-level details such as timing, duration, and severity.

The registry lists no analyses and does not report confidence intervals, a statistical analysis plan, a sample-size justification for the trial, or missing-data methods. The record also does not explain the reason for termination. These omissions do not establish that the study procedures were inadequate; they limit what can be concluded from the public registry information. The posted counts should therefore be interpreted as a narrow description of reported events among the participants listed, rather than a precise estimate or a causal comparison.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision — NCT04974307.
• Registry record last updated 2024-10-23; status last verified 2024-02.
• Results first posted 2024-10-23.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

19. Record Summary

NCT04974307 is a terminated, non-randomized, unmasked, parallel pilot study of the OrCam MyEye 2 in children with CLN3-related disease or low vision without CLN3. The registry lists enrollment of 11, a start date of 2021-09-30, and primary completion on 2023-10-06.

The primary outcome counted participants with adverse events related to device use during 1 week, collected by parental report, among those who completed week one. The adverse-event listings report no serious adverse events or deaths in either group, and other adverse-event counts of 1 in the CLN3 group and 0 in the low-vision group. The outcome group counts are 9 and 1, and the registry does not explain how they relate to the enrollment figure.

The record lists no analyses. It does not report confidence intervals, missing-data handling, event-level descriptions, or the reason for termination. These findings are descriptive and do not provide a randomized comparison or a precise estimate of risk.