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 NB-001 in Children and Adolescents With 22q11 Deletion Syndrome (NCT05290493), a randomized phase 2 study. The primary outcome is Safety and Tolerability of NB-001.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05290493 |
| Phase | Phase 2 |
| Condition | 22q11 Deletion Syndrome |
| Allocation | Randomized |
| Design model | Crossover |
| Masking | Quadruple |
| Enrollment | 37 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Nobias Therapeutics, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Children and adolescents with 22q11 deletion syndrome, described in the trial summary as having commonly associated neuropsychiatric symptoms. The registry reports an enrollment of 37. The safety analysis set is defined as participants who received at least one capsule of investigational product; the posted safety denominators are 33 for NB-001 and 34 for placebo.
Intervention
NB-001 was administered orally as two 100 mg capsules twice daily, for a total daily dose of 400 mg, with liquids. If a participant could not swallow a capsule whole, its contents could be sprinkled on applesauce.
Comparator
Matching inactive placebo capsules were administered orally twice daily. The placebo description also allowed the capsule contents to be sprinkled on applesauce if a participant could not swallow a capsule whole.
Primary question
The primary endpoint asks about the safety and tolerability of NB-001 during treatment. It covers treatment-emergent adverse events, serious adverse events, and clinically significant changes in laboratory tests, electrocardiograms, vital signs, and physical examination findings. The registry also lists clinical rating scales as secondary outcomes, but numerical results for those outcomes are not reported in the registry record.
The design is a randomized, placebo-controlled crossover trial. In general, a crossover design exposes participants to more than one treatment in different periods, so a treatment comparison may use information from the same participant under both conditions. The registry record identifies the crossover model but does not report the treatment sequence, period-level results, or washout details. Those details matter when interpreting which observations contribute to a comparison and whether results can be considered separately by period.
3. Trial Design
This was a Phase 2, randomized, placebo-controlled crossover trial of NB-001 in children and adolescents with 22q11 deletion syndrome. The registry lists quadruple masking. That term describes the recorded masking level, but the registry record does not specify which groups of people were masked or how masking was maintained.
The intervention was NB-001 and the comparator was matching inactive placebo. NB-001 was given as two 100 mg capsules orally twice daily, with a total daily dose of 400 mg. Placebo was given as two matching capsules twice daily. The administration instructions allowed capsule contents to be sprinkled on applesauce for participants unable to swallow a capsule whole.
The registry reports enrollment of 37 and a completed status. The recorded study start was 2022-02-10, and primary completion was 2023-06-01. The primary safety timeframe was 6 weeks (Day 42/ET). The registry record does not report the allocation sequence, the number or length of treatment periods, the washout procedure, or whether every participant completed both treatment periods.
The primary outcome concerns safety and tolerability. Listed secondary outcomes include the Clinical Global Impression Improvement and Severity scales, the Pediatric Anxiety Rating Scale, the inattention and hyperactivity scores of the ADHD-RS-5, and the Social Responsiveness Scale, Second Edition. The record lists these measures but does not report numerical efficacy findings for them. Nor does it report a detailed statistical analysis plan or a justification 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 | Placebo (6 Weeks) Then Washout (1 Week) Then NB-001 (6 Weeks) | NB-001 (6 Weeks) Then Washout (1 Week) Then Placebo (6 Weeks) |
|---|---|---|
| STARTED | 18 | 19 |
| COMPLETED | 15 | 17 |
| NOT COMPLETED | 3 | 2 |
Reasons for not completing this period:
| Reason | Placebo (6 Weeks) Then Washout (1 Week) Then NB-001 (6 Weeks) | NB-001 (6 Weeks) Then Washout (1 Week) Then Placebo (6 Weeks) |
|---|---|---|
| Withdrawal by Subject | 2 | 2 |
| Adverse Event | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety Analysis Set: Includes all subjects who receive at least one capsule (100 mg) of IP.
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 Analysis Set (SAS) includes all subjects who received at least one capsule of investigational product (IP). The numbers are different than the numbers of participants in the Participant Flow as the SAS excludes subjects who withdrew their participation prior to taking their first dose of IP. Thus, 1 subject in the Placebo/NB-001 group, and 2 subjects in the NB-001/Placebo group, were excluded from the SAS since they discontinued trial participation prior to taking any dose of IP.
| Characteristic | Unit | Placebo (6 Weeks) Then Washout (1 Week) Then NB-001 (6 Weeks) | NB-001 (6 Weeks) Then Washout (1 Week) Then Placebo (6 Weeks) | Total |
|---|---|---|---|---|
| Participants analysed | 17 | 17 | 34 | |
| Age, Continuous | Years | 12.1 (SD 3.12) | 11.9 (SD 3.30) | 12.0 (SD 3.16) |
| Sex: Female, Male: Female | Participants | 6 | 6 | 12 |
| Sex: Female, Male: Male | Participants | 11 | 11 | 22 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 | 1 | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 15 | 15 | 30 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| 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 | 2 | 0 | 2 |
| Race (NIH/OMB): White | Participants | 13 | 15 | 28 |
| Race (NIH/OMB): More than one race | Participants | 1 | 1 | 2 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 1 | 2 |
| Region of Enrollment: Canada | participants | 3 | 3 | 6 |
| Region of Enrollment: United States | participants | 14 | 14 | 28 |
| Reproductive Potential: Yes | Participants | 8 | 9 | 17 |
| Reproductive Potential: No | Participants | 9 | 8 | 17 |
6. Primary Endpoint
The registry lists the primary outcome as Safety and Tolerability of NB-001, measured over 6 weeks (Day 42/ET).
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled Safety and Tolerability of NB-001 and uses participants as its unit. It covers the type, frequency, severity, and causality of treatment-emergent adverse events and treatment-emergent serious adverse events. It also covers clinically significant changes from baseline in laboratory tests, electrocardiograms, vital signs, and physical examination findings during NB-001 treatment. The stated timeframe is 6 weeks (Day 42/ET).
Safety endpoints are used to describe unwanted events and clinically important changes observed during treatment. Considering event type, frequency, severity, and causality gives a broader description than counting events alone: the number of participants affected does not convey how serious an event was or whether investigators considered it related to treatment. Laboratory, electrocardiogram, vital-sign, and examination findings provide additional kinds of safety information beyond reported symptoms or diagnoses.
The registry defines the Safety Analysis Set as participants who received at least one capsule of investigational product. The posted adverse-event counts provide participant counts for serious events, other adverse events, and deaths. They do not provide the full endpoint detail on event type, severity, causality, or the listed clinical assessments. Those endpoint details are not reported in the registry record, so the available counts cannot be treated as a complete account of every component of the primary safety measure.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted adverse-event summary reports no serious adverse events among 33 participants at risk in the NB-001 group and none among 34 participants at risk in the placebo group. It reports no deaths in either group. For other adverse events, the counts are 18 of 33 for NB-001 and 24 of 34 for placebo. These are participant counts, not event counts, rates, or estimates of a treatment effect.
Those figures describe what was recorded for the listed categories; by themselves they do not establish that the treatments differ in safety. In particular, a zero count does not show that an event is impossible or that risk is absent. General statistical principle: when events are uncommon, a study can observe no events even when the underlying event probability is not zero. Precision depends on the amount and structure of the available information, and a point count alone does not express that uncertainty.
The comparison also needs to be read in light of the crossover design. In a crossover trial, one participant may contribute observations under more than one treatment. The group denominators in the adverse-event summary therefore should not automatically be treated as two independent sets of people. The registry record does not report participant-level paired results, treatment sequence, period-specific event counts, or an analysis accounting for the crossover structure. Without those details, the posted counts do not support a reconstruction of a paired treatment comparison.
The primary outcome covers more than the adverse-event counts. It includes event type, severity, and causality, as well as clinically significant changes in laboratory tests, electrocardiograms, vital signs, and physical examinations. Numerical results for those components and a primary-endpoint statistical comparison are not reported in the registry record. The registry record also lists several clinical rating scales as secondary outcomes, but numerical results for those measures are not reported there.
Accordingly, the evidence available in the registry record is descriptive and incomplete for assessing the full primary endpoint or the secondary outcomes. The counts offer a limited account of the participants recorded as having adverse events. They do not, without additional analysis and uncertainty estimates, show the size or precision of a treatment contrast. A p-value, confidence interval, or adjusted estimate for the primary endpoint is not reported in the registry record.
9. Statistical Methods Explained
The registry identifies a randomized, quadruple-masked crossover design and a safety endpoint expressed as participants. The posted adverse-event summary gives participant counts by treatment category. Statistical interpretation depends not only on those counts but also on how participants were assigned, how crossover periods were handled, and what analysis was prespecified. Detailed information on those analytical choices is not reported in the registry record.
What randomization contributes
General statistical principle: randomization is intended to reduce systematic differences between treatment assignments at the start of a trial. It does not guarantee that groups will be identical, especially in a small study, and it cannot by itself resolve issues that arise after assignment, such as missing observations or treatment-period effects. The record says the trial was randomized, but does not report the allocation sequence or the method used to generate it.
What masking contributes
General statistical principle: masking is intended to limit the influence of treatment knowledge on participant reports, clinical care, outcome assessment, and analysis. The registry records quadruple masking, but does not identify which parties were masked or describe procedures for maintaining the mask. The term alone does not show whether masking was successful.
Why crossover structure matters
General statistical principle: crossover designs can compare treatments within the same participant, which can account for stable differences among participants. The observations may therefore be related rather than independent. Period effects, sequence effects, and carryover from an earlier treatment can also matter. The registry identifies a crossover model but does not report treatment sequences, period-specific outcomes, washout details, or a model for handling these issues. It is consequently not possible to tell from the record how the primary analysis incorporated the crossover structure.
Reading participant counts
The posted figures count participants with an event in each listed category. A participant count answers how many people were recorded with an event, not how many separate events occurred. If one participant had more than one event, that would not be conveyed by a count of participants. The summary lists serious events, other adverse events, and deaths, but detailed event types and severity distributions are not reported in the registry record.
General statistical principle: an estimate based on counts has uncertainty, and the amount of uncertainty depends on the underlying design and information. A confidence interval expresses a range of values compatible with the data under a specified method and assumptions. It does not guarantee that the true value lies in that range, and it does not correct for an inappropriate analysis. The registry record does not report confidence intervals or a statistical comparison for the displayed safety counts.
Understanding the planning illustration
General statistical principle: sample-size planning asks how much information a future study might need to detect a specified effect under chosen assumptions. The planning illustration associated with these results is described as using a two-sided significance level of 0.05, equal allocation between arms, a normal approximation, and effects expressed as fractions of the observed effect. These are planning assumptions, not evidence that the trial used those assumptions in its design.
Planning figures based on an observed effect are hypothetical illustrations. They are not observed power and do not establish the probability that the completed trial detected an effect. When an observed effect is uncertain, calculations based on it inherit that uncertainty. The registry record does not report a sample-size justification for the trial, and the planning assumptions should not be mistaken for its protocol or prespecified analysis plan.
Multiplicity and outcome interpretation
General statistical principle: evaluating multiple outcomes or comparisons can increase the chance of finding an apparently notable result by chance if no adjustment or hierarchy is used. The record lists multiple secondary clinical scales and indicates eight outcome measures and six analyses, but does not report a multiplicity strategy or the detailed mapping between analyses and outcomes. Without that information, the intended control of false-positive findings cannot be determined.
For the primary safety endpoint, the record describes several kinds of events and clinical assessments rather than reporting one numerical treatment contrast. The registry record does not report how those components were combined, tested, or prioritized. Conclusions should therefore remain tied to the posted descriptive counts and not imply a broader formal comparison that is absent from the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple secondary outcomes: the Clinical Global Impression Improvement and Severity scales, the Pediatric Anxiety Rating Scale, the inattention and hyperactivity scores of the ADHD-RS-5, and the Social Responsiveness Scale, Second Edition. It also records eight outcome measures and six analyses. Numerical results for the listed secondary measures and a plan for handling multiple comparisons are not reported in the registry record.
General statistical principle: when several outcomes are examined, the chance of at least one apparently noteworthy finding can increase unless the analysis accounts for multiplicity. A prespecified hierarchy, adjustment method, or clear distinction between confirmatory and exploratory outcomes can help readers interpret a collection of tests. The registry record does not state whether such an approach was planned, so the status of any secondary findings cannot be evaluated from the record.
Information on missing data is also limited. The registry defines the safety analysis set as participants who received at least one capsule, and reports safety denominators of 33 for NB-001 and 34 for placebo. It does not report how many participants completed each treatment period, whether outcome data were missing, why data might have been unavailable, or how missing measurements were handled. The crossover sequence and the analysis population for the clinical rating scales are not reported in the registry record.
These omissions matter because missing observations and multiple outcomes can affect the interpretation of estimates and tests. General statistical principle: different assumptions about missingness can lead to different analyses, and the analysis population should be clear about who contributes to each result. With no reported missing-data strategy or detailed analysis populations for the secondary outcomes, the record does not allow assessment of those choices.
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 |
|---|---|---|---|
| NB-001 | 0 | 33 | 0.0% |
| Placebo | 0 | 34 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| NB-001 | 0 of 33 |
| Placebo | 0 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 |
|---|---|
| NB-001 | 18 of 33 |
| Placebo | 24 of 34 |
Most frequent non-serious adverse event terms
| Event term | Organ system | NB-001 | Placebo |
|---|---|---|---|
| Nasopharyngitis | Infections and infestations | 3 of 33 | 4 of 34 |
| Insomnia | Psychiatric disorders | 1 of 33 | 6 of 34 |
| Upper respiratory tract infection | Infections and infestations | 1 of 33 | 3 of 34 |
| Irritability | Psychiatric disorders | 1 of 33 | 3 of 34 |
| Abdominal pain upper | Gastrointestinal disorders | 1 of 33 | 3 of 34 |
| Vomiting | Gastrointestinal disorders | 2 of 33 | 2 of 34 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 2 of 33 | 2 of 34 |
| Fatigue | General disorders | 3 of 33 | 1 of 34 |
| Pyrexia | General disorders | 2 of 33 | 2 of 34 |
| Anxiety | Psychiatric disorders | 1 of 33 | 2 of 34 |
For serious adverse events, the posted summary records 0 participants affected among 33 at risk in the NB-001 group and 0 among 34 at risk in the placebo group. Deaths are recorded as 0 in both groups. The summary also reports other adverse events for 18 of 33 participants receiving NB-001 and 24 of 34 receiving placebo.
These figures are participant counts within the listed categories. They do not indicate how many total events occurred, how long each participant was observed, or the type, severity, and causality of each event. Those details are not reported in the registry record. In particular, a zero count for serious events or deaths is an observation in this trial, not proof that such events cannot occur.
The trial used a crossover design, so participants may contribute experience under more than one treatment. The summary does not give paired or period-specific event results. Its treatment-category counts should therefore be read descriptively rather than as independent-group estimates of a treatment difference. No confidence interval or formal comparison for these adverse-event counts is reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record describes a randomized, placebo-controlled crossover study of NB-001 in children and adolescents with 22q11 deletion syndrome. The primary endpoint is safety and tolerability over 6 weeks (Day 42/ET), including adverse events and clinically significant changes in several clinical assessments. The posted adverse-event figures report no serious events or deaths in either treatment category, and report participant counts for other adverse events.
Those observations do not provide a complete numerical account of the primary endpoint. The record does not report the event-level details, assessments, or statistical comparison needed to interpret all endpoint components. It also does not report numerical results for the listed secondary clinical rating scales. Therefore, the registry record does not support a numerical conclusion about efficacy from those outcomes.
General statistical principle: a treatment comparison is best understood through its estimate and uncertainty, interpreted in light of the design and the analysis. Counts alone do not show how precise a difference might be, and the crossover structure means that a paired analysis may be relevant. The necessary participant-level or period-level results and corresponding uncertainty estimates are not reported in the registry record.
These results should not be interpreted as demonstrating that NB-001 is effective, ineffective, safer, or less safe than placebo. They describe the counts recorded in the registry summary. Absence of reported serious events in these groups is not evidence that the risk is zero, while the counts of other adverse events alone cannot establish a comparative safety effect.
The registry record is also not a substitute for a complete protocol or statistical analysis plan. Details such as treatment sequence, washout, missing-data handling, multiplicity, and the analysis of the crossover design are not reported. Those limitations constrain how far the posted counts can be generalized or used to infer a treatment contrast.
13. Why This Trial Matters Statistically
This trial is statistically interesting because it combines a crossover design with a primary endpoint that covers several types of safety information. Crossover designs can use within-participant comparisons, but those comparisons require analysis that respects the relationship between observations from the same participant. The registry record identifies the design without reporting the treatment sequence, period-specific results, or statistical model, leaving important aspects of interpretation unresolved.
The safety endpoint is also broader than a single count. It includes the type, frequency, severity, and causality of adverse events, serious events, and clinically significant changes in laboratory tests, electrocardiograms, vital signs, and physical examinations. The posted adverse-event counts cover only some of this information. Distinguishing a compact summary from the full endpoint helps avoid treating a few counts as if they represented every safety assessment.
The registry lists several secondary clinical measures, which raises a general issue of multiplicity. When several outcomes are examined, the analysis plan affects how findings should be interpreted. That plan is not reported in the registry record, and numerical results for those measures are also not reported there.
Finally, the trial’s listed enrollment and the safety denominators are different quantities. The record gives enrollment of 37 and safety denominators of 33 for NB-001 and 34 for placebo. In a crossover trial, treatment-category denominators need not represent separate, independent groups of participants. Clear reporting of sequence, participant flow, period-level data, and analysis choices would be needed to make the safety comparisons more fully interpretable.
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 does not report the treatment sequence, number or length of crossover periods, or washout details. These design features are relevant to understanding which participants contributed data under each treatment and whether period or carryover effects were considered. Without period-specific or participant-level results, the posted counts cannot be used to reconstruct a within-participant comparison.
The primary endpoint covers adverse-event characteristics and several clinical assessments, but the posted summary does not provide all of those details. Event types, severity, causality, and results for laboratory tests, electrocardiograms, vital signs, and physical examinations are not reported in the registry record. The available adverse-event counts therefore describe only a portion of the endpoint information.
The registry record does not report a primary-endpoint estimate, confidence interval, p-value, or detailed statistical analysis plan. It also does not describe missing-data handling, participant completion by period, or analysis populations for the secondary outcomes. The listed secondary measures have no numerical results in the record, and a multiplicity strategy is not reported.
The record reports an enrollment of 37, while the safety summary gives denominators of 33 for NB-001 and 34 for placebo. It does not explain the relationship between enrollment and those treatment-category denominators. Because the design is crossover, the denominators should not be assumed to represent mutually exclusive participant groups.
These limitations restrict interpretation to the descriptive information recorded. They do not establish that the trial had a particular statistical shortcoming; rather, the necessary protocol and analysis details are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: NB-001 in Children and Adolescents With 22q11 Deletion Syndrome — NCT05290493.
- Registry record last updated 2025-02-10; status last verified 2025-01.
- Results first posted 2025-02-10.
- 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
NCT05290493 is a completed Phase 2 randomized, quadruple-masked, placebo-controlled crossover trial of NB-001 in children and adolescents with 22q11 deletion syndrome. The registry reports enrollment of 37, a study start of 2022-02-10, and primary completion on 2023-06-01.
The primary outcome is safety and tolerability during 6 weeks (Day 42/ET). The posted summary records 0 serious adverse events among 33 participants at risk for NB-001 and 0 among 34 for placebo; deaths are recorded as 0 in both groups. Other adverse events are reported for 18 of 33 participants in the NB-001 group and 24 of 34 in the placebo group.
Numerical efficacy results, a formal statistical comparison for the primary endpoint, and detailed crossover, missing-data, and multiplicity methods are not reported in the registry record. The available counts are descriptive and do not provide a complete analysis of the primary safety endpoint.