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 Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia (NCT04614974), a randomized phase 1, phase 2 study. The primary outcome is Airway Symptom Score Change From Consult (Baseline) to 3 Month Follow-up Appointment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04614974 |
| Phase | Phase 1, Phase 2 |
| Condition | Laryngomalacia, Gastro Esophageal Reflux |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 65 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Reema Padia |
| Results posted | Yes |
2. Clinical Question
Population
Infants evaluated for laryngomalacia, a condition associated in the registry description with stridor and sometimes gastroesophageal reflux symptoms. Caregivers were asked to complete symptom questionnaires at the consult and at follow-up. The registry lists an enrollment of 65.
Intervention
Speech language therapy evaluation together with famotidine, an acid suppression therapy. The registry describes a routine swallowing evaluation by a speech language pathologist for this group.
Comparator
Speech language therapy alone, also involving a routine swallowing evaluation. The registry describes caregiver questionnaires at the consult and follow-up for both groups.
Primary question
How does the change in Pittsburgh Airway Symptom Score from consult to the 3-month follow-up compare between the group receiving speech language therapy alone and the group receiving speech language therapy plus famotidine?
The clinical question is comparative: it concerns outcomes under two assigned approaches, not whether acid suppression alone changes symptoms. The record identifies the Pittsburgh Airway Symptom Score (PASS) as the primary outcome. It describes a scale from 0-10, with higher scores indicating a worse outcome, and defines the outcome as the score change from consult to follow-up. The Infant Gastroesophageal Reflux Questionnaire is also mentioned in the arm descriptions, but the primary outcome is airway symptoms measured with PASS.
The registry reports follow-up PASS completion by 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the speech language therapy and acid suppression therapy group. It does not report the groups’ PASS changes or a direct between-group estimate in the record summarized for this analysis. As a result, the stated clinical question can be described, but the direction and size of any difference cannot be determined from the reported outcome information.
3. Trial Design
The registry describes a randomized, parallel-group trial with no masking. The listed phases are Phase 1 and Phase 2. The enrollment is 65, and the record lists the trial status as terminated. The start date is 2020-11-18 and the primary completion date is 2024-05-17. The reason for termination is not reported in the registry record.
The two groups were speech language therapy alone and speech language therapy with acid suppression therapy. The latter used famotidine. Both groups were described as receiving a routine swallowing evaluation by a speech language pathologist. Caregivers were asked to complete PASS and the Infant Gastroesophageal Reflux Questionnaire around the consult and follow-up. The primary outcome is specified at the 3-month follow-up. The registry also lists secondary outcomes concerning weight, supraglottoplasty, use of acid suppression medication, and laryngomalacia type identified at flexible laryngoscopy.
Randomization supports a comparison of assigned groups in principle, but the registry record does not describe how the random sequence was generated, whether allocation was concealed, or whether assignment used strata or blocks. With no masking, caregivers and clinical staff were aware of group assignment; the record does not describe procedures to limit the possible effects of that awareness on questionnaire completion or clinical decisions.
The arm descriptions and outcome listings establish the intended comparison and follow-up measures, but they do not give a full account of adherence, treatment changes, or the timing of all assessments. Those details are not reported in the registry record. The listed enrollment and group denominators provide context for the reported follow-up questionnaire completion, but do not by themselves establish which analysis population was used.
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: Only infant patients were considered enrolled.
Overall Study
| Stage | Speech Language Therapy Alone | Speech Language Therapy and Acid Suppression Therapy |
|---|---|---|
| STARTED | 34 | 31 |
| COMPLETED | 20 | 20 |
| NOT COMPLETED | 14 | 11 |
Reasons for not completing this period:
| Reason | Speech Language Therapy Alone | Speech Language Therapy and Acid Suppression Therapy |
|---|---|---|
| Lost to Follow-up | 14 | 11 |
Analysis population for the primary outcome, as defined in the registry: Follow-up PASS was completed by 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) of the speech language therapy and acid suppression therapy group.
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 | Speech Language Therapy Alone | Speech Language Therapy and Acid Suppression Therapy | Total |
|---|---|---|---|---|
| Participants analysed | 34 | 31 | 65 | |
| Age, Continuous | Months | 2.3 (0.5 to 6.8) | 2.3 (0.2 to 6.2) | 2.3 (0.2 to 6.8) |
| Sex: Female, Male: Female | Participants | 11 | 14 | 25 |
| Sex: Female, Male: Male | Participants | 23 | 17 | 40 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 2 | 0 | 2 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 | 5 | 8 |
| Race (NIH/OMB): White | Participants | 28 | 24 | 52 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 2 | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 0 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 31 | 29 | 60 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 2 | 2 | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Airway Symptom Score Change From Consult (Baseline) to 3 Month Follow-up Appointment, measured over 3 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | score on a scale |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is change in the Pittsburgh Airway Symptom Score from the consult, treated as baseline, to the 3-month follow-up appointment. PASS is described as a score on a scale from 0-10, with a higher score indicating a worse outcome. The endpoint therefore focuses on the change in caregiver-reported airway symptoms over the stated follow-up period, rather than on a single symptom or a clinical event.
The registry specifies the median and full range as the summary measures. A median describes the middle of the observed values after they are ordered. A full range describes the lowest and highest observed values. These summaries can be useful when data are skewed or when a typical value is of interest, but they do not show the distribution’s shape or provide a direct estimate of the difference between randomized groups.
The record does not report the median change, full range, or a between-group estimate for the primary endpoint. It reports that follow-up PASS was completed by 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the speech language therapy and acid suppression therapy group. Without the outcome values and a clearly stated comparison estimate, the amount and precision of any group difference cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a randomized comparison and a primary measure, but it does not report the primary outcome values. In particular, the recorded information does not give the median PASS change in either group, the full range of changes, or a direct comparison between groups. It also does not report a confidence interval or a p-value for this outcome. Therefore, no direction, magnitude, or statistical precision of the primary comparison can be established from the outcome information in the registry record.
Follow-up PASS completion is reported for 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the speech language therapy and acid suppression therapy group. These figures describe questionnaire completion, not symptom change. The record does not explain why follow-up scores were unavailable for other participants, whether missingness differed for reasons related to symptoms, or how incomplete follow-up was handled in the analysis. Completion counts alone cannot show whether the observed responses represent the experiences of everyone assigned to each group.
The use of medians and full ranges is stated, but those summaries do not replace an explicit between-group estimate. A comparison of within-group changes is not necessarily equivalent to a comparison of changes between randomized groups. A treatment-effect interpretation requires the group contrast and its uncertainty, along with clarity about the participants and measurements included. Those details are not reported in the registry record for the primary outcome.
The registry lists the study as terminated. That status does not explain why it ended, whether the planned enrollment or follow-up was completed, or whether termination affected outcome reporting. The reason for termination and any consequences for analysis are not reported in the registry record. The completion information does show that follow-up PASS data were available for fewer than all participants listed in the group denominators; it does not establish why.
Overall, the record supports describing the design, the intended primary endpoint, and the reported follow-up completion. It does not support a conclusion about whether the groups differed in airway symptoms or how precisely such a difference was estimated. This is a limitation of the results available in the registry record, not evidence for or against either assigned approach.
9. Statistical Methods Explained
What does randomization contribute?
Random assignment is intended to make the groups comparable, on average, with respect to factors that could affect outcomes. It can therefore support a causal interpretation of a properly analyzed between-group contrast. Randomization does not guarantee that the groups will be identical, and it does not remove the need to report how assignment was performed, how outcomes were measured, and who was included in the analysis.
The registry describes this trial as randomized, parallel, and unmasked. It does not report the sequence-generation method, allocation concealment, or any stratification procedure. A parallel design assigns participants to a group and compares outcomes across those groups. In an unmasked trial, participants or caregivers and clinical staff know which approach was assigned; the record does not say whether outcome assessment was otherwise masked.
How should the endpoint summary be read?
The primary outcome is a change in PASS between consult and the 3-month follow-up. A change score represents how a participant’s measured score differs between the stated assessments. PASS is described as ranging from 0-10, with higher scores indicating worse outcomes. The registry specifies the median and full range for describing the change scores. The median gives a central location, while the range gives the observed extremes. Neither summary alone gives a direct estimate of the difference between randomized groups or its uncertainty.
The registry record does not report the observed endpoint summaries or the method used for the between-group comparison. It does not say whether the analysis compared change scores directly, adjusted for baseline PASS, or used another model. Those approaches can answer related but not identical questions, and their interpretation depends on the analysis specification. The analysis method is not reported in the registry record.
Why does follow-up completion matter?
The registry reports follow-up PASS completion by 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the speech language therapy and acid suppression therapy group. When outcomes are unavailable for some randomized participants, the analyzed responses may not represent all those assigned. The impact depends in part on why measurements are missing and on the analysis used. Missingness can be more concerning when it is associated with symptoms or other factors related to outcomes; the record does not give enough information to assess that possibility.
For a transparent interpretation, results should distinguish the randomized groups from the participants with observed follow-up outcomes and explain how missing values were handled. The registry does not report an analysis population, a missing-data method, or sensitivity analyses for the primary endpoint. It is therefore not possible to determine whether the reported completion pattern was addressed in a particular way.
What can be inferred from the reported statistical detail?
The stated summary measure is the median with full range. The record does not report a confidence interval, p-value, model specification, covariate adjustment, or a formal statistical test for the primary comparison. It also does not report a statistical analysis plan. These omissions mean that the precision of the primary result and the role of any prespecified analysis choices cannot be assessed from the registry record.
In general, a confidence interval describes uncertainty around an estimate under the assumptions of the analysis. It helps readers consider which effect sizes remain compatible with the data; it does not show that every value within the interval is equally likely or establish clinical importance. A p-value, when reported, addresses compatibility with a specified null model and does not measure the size or importance of an effect. Neither measure is reported for this primary endpoint in the registry record, so neither can be interpreted for this comparison.
The registry lists nine outcome measures and 18 analyses. Having multiple outcomes or analyses can raise multiplicity concerns when several results are tested or emphasized. The registry record does not report a multiplicity adjustment or a plan identifying which analyses were confirmatory. The primary outcome is named, but the available result information does not establish how it was analyzed relative to the other listed outcomes.
The sample-size assumptions describe a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. Such a calculation is a planning aid, not observed power and not evidence that the trial had a particular probability of detecting an effect. The registry record does not report a trial-specific sample-size justification. The available information also does not provide a single primary outcome comparison from which to judge the planning illustration as an empirical result.
Finally, the status is terminated, but the record does not explain the reason or whether statistical analyses were changed as a consequence. Without a detailed analysis plan, results for the primary endpoint, and information on missing outcomes, the safest reading is descriptive: the record states the intended comparison and endpoint but does not report enough statistical output to quantify the primary contrast.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists nine outcome measures and 18 analyses. Several outcomes are described, including weight, supraglottoplasty, medication use, and laryngomalacia type, in addition to the primary PASS endpoint. When multiple outcomes or analyses are considered, the chance of finding an apparently notable result can increase if results are selected or interpreted without a prespecified strategy. The registry record does not report a multiplicity plan, adjustment, or hierarchy for interpreting secondary outcomes.
The record names PASS change as the primary outcome and specifies the summary measure, but it does not report the primary result or the statistical analysis plan. It is therefore unclear from the registry record which comparisons were prespecified for analysis, how any additional analyses were handled, or whether the listed analyses were exploratory. This uncertainty does not imply that the analyses were inappropriate; it limits what can be concluded about their confirmatory status.
For the primary outcome, follow-up PASS was completed by 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the speech language therapy and acid suppression therapy group. The registry does not report reasons for missing follow-up scores, the approach to missing data, or sensitivity analyses. It also does not state whether the main analysis included all randomized participants, only participants with observed follow-up, or another population. These details are important because an analysis based on observed outcomes can be affected if missingness relates to the outcome or group assignment.
The record therefore documents incomplete follow-up questionnaire data but does not provide enough information to judge how missingness or multiplicity was addressed. The reason for termination and any effect on the analysis are also not reported in the registry record.
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 |
|---|---|---|---|
| Speech Language Therapy Alone | 3 | 34 | 8.8% |
| Speech Language Therapy and Acid Suppression Therapy | 3 | 31 | 9.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Speech Language Therapy Alone | 0 of 34 |
| Speech Language Therapy and Acid Suppression Therapy | 0 of 31 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Speech Language Therapy Alone | 7 of 34 |
| Speech Language Therapy and Acid Suppression Therapy | 8 of 31 |
Most frequent serious adverse event terms
| Event term | Organ system | Speech Language Therapy Alone | Speech Language Therapy and Acid Suppression Therapy |
|---|---|---|---|
| Hospital admission for viral infection | Infections and infestations | 2 of 34 | 1 of 31 |
| Hospital admission for seizure | Nervous system disorders | 0 of 34 | 2 of 31 |
| Pediatric intensive care unit admission for respiratory distress following supraglottoplasty | Surgical and medical procedures | 1 of 34 | 0 of 31 |
| Inguinal hernia with incarceration requiring surgery | Reproductive system and breast disorders | 0 of 34 | 1 of 31 |
| Hospital admission for monitoring post-motor vehicle accident | General disorders | 0 of 34 | 1 of 31 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Speech Language Therapy Alone | Speech Language Therapy and Acid Suppression Therapy |
|---|---|---|---|
| Emergency department visit viral syndrome | Infections and infestations | 5 of 34 | 5 of 31 |
| Emergency department post-fall | General disorders | 0 of 34 | 2 of 31 |
| Emergency department visit broken femur | Musculoskeletal and connective tissue disorders | 1 of 34 | 0 of 31 |
| Emergency department visit eczema and skin yeast infection | Skin and subcutaneous tissue disorders | 1 of 34 | 0 of 31 |
| Emergency department visit constipation | Gastrointestinal disorders | 1 of 34 | 0 of 31 |
| Emergency department visit abscess | Skin and subcutaneous tissue disorders | 0 of 34 | 1 of 31 |
| Emergency department visit tachypnea, fussiness, spitting up | Gastrointestinal disorders | 0 of 34 | 1 of 31 |
| Emergency department visit seizures | Nervous system disorders | 0 of 34 | 1 of 31 |
Serious events with the highest arm-level rate: hospital admission for seizure (6.5%), hospital admission for viral infection (5.9%), inguinal hernia with incarceration requiring surgery (3.2%), hospital admission for monitoring post-motor vehicle accident (3.2%), pediatric intensive care unit admission for respiratory distress following supraglottoplasty (2.9%).
The registry reports serious adverse events for 3 participants in the speech language therapy alone group, with 34 at risk, and 3 participants in the speech language therapy and acid suppression therapy group, with 31 at risk. It reports other adverse events for 7 participants in the speech language therapy alone group and 8 in the group receiving speech language therapy and acid suppression therapy. Deaths are reported as 0 in each group.
These counts describe the safety information recorded for the groups; they do not by themselves establish that an event was caused by an assigned intervention. The registry’s list of top serious adverse-event terms includes hospital admission for seizure (6.5%), hospital admission for viral infection (5.9%), inguinal hernia with incarceration requiring surgery (3.2%), hospital admission for monitoring post-motor vehicle accident (3.2%), and pediatric intensive care unit admission for respiratory distress following supraglottoplasty (2.9%).
The record does not explain the denominators used for those term-specific percentages, whether an individual could contribute to more than one term, or how severity and relatedness were assessed. These figures should therefore be read as the registry’s reported safety summaries, not as a comparative estimate of treatment-attributable risk. The number of events and the follow-up available may also affect how much can be inferred from the counts; further detail is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record establishes that investigators set out to compare speech language therapy alone with speech language therapy plus famotidine in a randomized, open-label, parallel-group study. It identifies a caregiver-reported airway symptom measure, PASS change from consult to the 3-month follow-up, as the primary outcome. It also gives follow-up questionnaire completion and safety summaries.
It does not report the primary outcome values or a between-group effect estimate. Consequently, the posted information does not establish whether PASS changed differently between the groups, the size of any difference, or how precise an estimate might be. The absence of a reported result is not evidence that the groups had the same outcomes, and it is not evidence that either approach did or did not affect symptoms.
The follow-up completion figures show that PASS data were available for 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the speech language therapy and acid suppression therapy group. They do not reveal the missing participants’ outcomes or explain why their follow-up scores were unavailable. Without the missing-data approach and analysis population, the effect of incomplete follow-up on interpretation remains uncertain.
The safety summaries report serious and other adverse events, along with no deaths in either group. These descriptive counts do not show whether events were attributable to treatment, and the registry record does not provide the details needed for a causal safety comparison.
In statistical terms, the record describes the question and design more clearly than it reports the answer. The primary endpoint’s definition and the groups’ follow-up completion are useful context, but they are not substitutes for outcome estimates and uncertainty. Conclusions about comparative symptom outcomes or safety should not be drawn from the information reported in the registry record alone.
13. Why This Trial Matters Statistically
This trial is statistically relevant because it uses random assignment to compare two approaches that both include speech language therapy, with famotidine added in one group. That structure makes the between-group contrast central: the question is whether outcomes differ under the assigned strategies, not whether symptoms change over time within either group alone. The registry identifies that intended comparison and names a patient- and caregiver-reported airway symptom endpoint.
The endpoint is also useful to consider as a measurement problem. PASS is reported on a scale from 0-10, with higher scores indicating worse outcomes, and the primary outcome is change between consult and follow-up. A clear interpretation would require the observed changes, a comparison between groups, and information about uncertainty. The registry record does not report those primary outcome results, so it offers no estimate of the comparative effect.
Follow-up completion matters for the credibility of a randomized comparison. The reported questionnaire completion differs in the two groups, but the record does not explain why data were unavailable or how missing outcomes were handled. This highlights why reporting the analysis population and missing-data approach is important alongside the assigned-group design.
The trial is listed as terminated, and the reason is not reported in the registry record. Termination status, multiple listed outcomes, and the absence of a reported analysis plan make transparent reporting especially important for understanding which analyses were intended and how the available data were used. The record illustrates that design details alone cannot resolve uncertainty about results: outcome estimates, their precision, and analysis decisions are also needed for a statistical interpretation.
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 trial is that the registry record does not report the primary PASS change values or a direct comparison between groups. It specifies the median and full range, but those results are unavailable in the record. No confidence interval, p-value, or other estimate of uncertainty for the primary comparison is reported.
Follow-up PASS completion is reported as 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the speech language therapy and acid suppression therapy group. The record does not provide reasons for unavailable scores, a missing-data method, or sensitivity analyses. It also does not specify the primary analysis population. These gaps limit assessment of whether incomplete follow-up could have influenced the observed comparison.
The trial was unmasked, and the primary outcome was caregiver-reported. The registry does not describe steps to reduce possible effects of awareness of assignment on reporting or assessment. This is a design consideration, not evidence that reporting was biased. The record also does not provide enough detail about randomization procedures, allocation concealment, adherence, or treatment changes to assess those aspects of conduct.
The record lists nine outcome measures and 18 analyses but does not report a multiplicity strategy or a statistical analysis plan. It lists the trial as terminated without reporting the reason. The enrollment, completion, and safety figures provide descriptive context, but the absent outcome and analysis details prevent a full evaluation of the trial’s comparative findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia — NCT04614974.
- Registry record last updated 2025-07-20; status last verified 2025-07.
- Results first posted 2025-07-20.
- 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 registry describes a randomized, open-label, parallel-group trial of speech language therapy alone versus speech language therapy with famotidine for laryngomalacia. The listed phases are Phase 1 and Phase 2, enrollment is 65, and status is terminated. The start date is 2020-11-18 and the primary completion date is 2024-05-17.
The primary outcome is change in Pittsburgh Airway Symptom Score from consult to the 3-month follow-up. PASS is described as a scale from 0-10, with higher scores indicating a worse outcome; the registry specifies median and full range summaries. Follow-up PASS completion was 20/34 (59%) in the speech language therapy alone group and 20/31 (65%) in the group receiving speech language therapy and acid suppression therapy.
The registry record does not report the primary outcome values, a between-group estimate, or the reason for termination. It also does not report the primary analysis method or how missing follow-up outcomes were handled. The safety record lists serious adverse events for 3 participants in each group and deaths as 0 in each group.