Trial · NCT05290493
NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
NCT05290493, Phase 2, Completed, Nobias Therapeutics, Inc.; results first posted 2025-02-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. NB-001 in Children and Adolescents With 22q11 Deletion Syndrome. Trials, record NCT05290493. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05290493
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
- NCT number
- NCT05290493
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 37 participants, actual
- Lead sponsor
- Nobias Therapeutics, Inc.
- Conditions
- 22q11 Deletion Syndrome
- Interventions
- NB-001, Placebo
- First posted
- 2022-03-22
- Last updated
- 2025-02-10
- Results first posted
- 2025-02-10
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety and Tolerability of NB-001 | 6 weeks (Day 42/ET) | Type, frequency, severity, and causality of treatment-emergent adverse events (TEAEs),treatment-emergent serious adverse events (TESAEs), clinically significant changes from baseline in laboratory tests, electrocardiograms (ECGs), vital signs, and physical examination findings during treatment with NB-001. |
| Secondary | Treatment Effect of NB-001 on the Clinical Global Impression Improvement (CGI-I) Scale | 6 weeks (Day 42/ET) | The CGI-I scale is a 7-point scale that measures how much a patient's condition has improved or worsened over time. The CGI-I is scored on a scale of 1-7, with a score of 1 indicating "Very much improved" and 7 indicating "Very much worse". The least squares mean of the score on the CGI-I scale at the end of the 6-week treatment period is reported here. |
| Secondary | Treatment Effect of NB-001 on the Clinical Global Impression Severity (CGI-S) Scale | 6 weeks (Day 42/ET) | CGI-S: 7-point scale that measures a participant's overall disease severity. A 1-point improvement in the CGI-S scale is an appropriate meaningful change threshold. Possible scores are: 1 = Normal, not at all impaired; 2 = Borderline impaired; 3 = Mildly impaired; 4 = Moderately impaired; 5 = Markedly impaired; 6 = Severely impaired; 7 = Among the most extremely impaired patients. |
| Secondary | Treatment Effect of NB-001 on the Pediatric Anxiety Rating Scale (PARS) | 6 weeks (Day 42/ET) | The PARS is a clinician-rated instrument for assessing severity of anxiety symptoms associated with common anxiety disorders. The first section is a 50-item symptom checklist and the second section is comprised of 7 severity/impairment items reflecting the severity/impairment of all symptoms noted in the first section. The PARS total severity score is calculated as the sum of items 2, 3, 5, 6, and 7 from the second section of the instrument. Each item is rated on a 6-point Likert scale from 0-5 with the higher scores indicating more severe anxiety. The total severity score can range from a minimum value of 0 to a maximum value of 25. Higher scores indicate more severe anxiety. Additionally, per protocol, at baseline, a score of \>12 on the PARS 5-item total severity score (items 2+3+5+6+7) is indicative of psychiatric symptoms in the clinical range for anxiety disorder, and a score of 10 or 11 is indicative of psychiatric symptoms in the subclinical range for anxiety disorder. |
| Secondary | Treatment Effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5) - Inattention Score | 6 weeks (Day 42/ET) | The ADHD-RS-5 is a parent/guardian reported scale to measure behaviors of children and adolescents with ADHD. It consists of 18 items grouped into two subscales: inattention (items 1-9) and hyperactivity (items 10-18). Each item is scored on a scale ranging from 0 (reflecting no symptoms) to 3 (reflecting severe symptoms). The total inattention score (reported here) can range from a minimum value of 0 to a maximum value of 27. Higher total inattention scores reflect more severe symptoms. |
| Secondary | Treatment Effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5) - Hyperactivity Score | 6 weeks (Day 42/ET) | The ADHD-RS-5 is a parent/guardian reported scale to measure behaviors of children and adolescents with ADHD. It consists of 18 items grouped into two subscales: inattention (items 1-9) and hyperactivity (items 10-18). Each item is scored on a scale ranging from 0 (reflecting no symptoms) to 3 (reflecting severe symptoms). The total hyperactivity score (reported here) can range from a minimum value of 0 to a maximum value of 27. Higher total hyperactivity scores reflect more severe symptoms. |
| Secondary | Treatment Effect of NB-001 on the Social Responsiveness Scale, Second Edition (SRS-2) | 6 weeks (Day 42/ET) | The SRS-2 identifies the presence and severity of social impairment within the autism spectrum. It is a 65-item, parent-completed questionnaire which incorporates 5 content areas of social deficits. The sum of all items is calculated to provide a maximum total score of 195. However, a total derived T-score is calculated centrally and reported for this trial. A higher T-score indicates more severe impairment. The population mean T-score is 50, with a standard deviation of 10. Thus, a T-score considered within the normal range is 59 or below (i.e., minimum T-score value), and a T-score indicating a severe range is 76 or above (i.e., maximum T-score value). Scores between 60-75 fall into the mild to moderate range. |
| Other | Exit Interview | End of Treatment; approximately 13 weeks of trial participation | Optional qualitative exit interviews were conducted with clinicians and parent/guardian(s) of subjects who completed the protocol. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety and Tolerability of NB-001 | 6 weeks (Day 42/ET) | Safety Analysis Set: Includes all subjects who receive at least one capsule (100 mg) of IP. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Treatment Effect of NB-001 on the Clinical Global Impression Improvement (CGI-I) Scale | 6 weeks (Day 42/ET) | Full Analysis Set (FAS): Includes all subjects in the Enrolled Analysis Set (those who meet all eligibility criteria and consent to participate in study) who have at least one valid post-baseline efficacy evaluation within each treatment period. Subjects are analyzed based on the treatment to which they were randomized in each treatment period. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Treatment Effect of NB-001 on the Clinical Global Impression Severity (CGI-S) Scale | 6 weeks (Day 42/ET) | Full Analysis Set: Includes all subjects in the Enrolled Analysis Set (those who meet all eligibility criteria and consent to participate in study) who have at least one valid post-baseline efficacy evaluation within each treatment period. Subjects are analyzed based on the treatment to which they were randomized in each treatment period. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Treatment Effect of NB-001 on the Pediatric Anxiety Rating Scale (PARS) | 6 weeks (Day 42/ET) | Full Analysis Set: Includes all subjects in the Enrolled Analysis Set (those who meet all eligibility criteria and consent to participate in study) who have at least one valid post-baseline efficacy evaluation within each treatment period. Subjects are analyzed based on the treatment to which they were randomized in each treatment period. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Treatment Effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5) - Inattention Score | 6 weeks (Day 42/ET) | Full Analysis Set: Includes all subjects in the Enrolled Analysis Set (those who meet all eligibility criteria and consent to participate in study) who have at least one valid post-baseline efficacy evaluation within each treatment period. Subjects are analyzed based on the treatment to which they were randomized in each treatment period. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Treatment Effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5) - Hyperactivity Score | 6 weeks (Day 42/ET) | Full Analysis Set: Includes all subjects in the Enrolled Analysis Set (those who meet all eligibility criteria and consent to participate in study) who have at least one valid post-baseline efficacy evaluation within each treatment period. Subjects are analyzed based on the treatment to which they were randomized in each treatment period. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Treatment Effect of NB-001 on the Social Responsiveness Scale, Second Edition (SRS-2) | 6 weeks (Day 42/ET) | Includes all subjects in the Enrolled Analysis Set (those who meet all eligibility criteria and consent to participate in study) who have at least one valid post-baseline efficacy evaluation within each treatment period. Subjects are analyzed based on the treatment to which they were randomized in each treatment period. | T-score | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Exit Interview | End of Treatment; approximately 13 weeks of trial participation |
Source ClinicalTrials.gov
Results by group42
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety and Tolerability of NB-001 | NB-001 | Subjects Reporting at Least One TEAE | 18 | Participants | |
| Safety and Tolerability of NB-001 | Placebo | Subjects Reporting at Least One TEAE | 24 | Participants | |
| Safety and Tolerability of NB-001 | NB-001 | CTCAE Grade 1: Mild | 14 | Participants | |
| Safety and Tolerability of NB-001 | Placebo | CTCAE Grade 1: Mild | 20 | Participants | |
| Safety and Tolerability of NB-001 | NB-001 | CTCAE Grade 2: Moderate | 4 | Participants | |
| Safety and Tolerability of NB-001 | Placebo | CTCAE Grade 2: Moderate | 4 | Participants | |
| Safety and Tolerability of NB-001 | NB-001 | CTCAE Grade 3: Severe | 0 | Participants | |
| Safety and Tolerability of NB-001 | Placebo | CTCAE Grade 3: Severe | 0 | Participants | |
| Safety and Tolerability of NB-001 | NB-001 | CTCAE Grade 4: Life-threatening | 0 | Participants | |
| Safety and Tolerability of NB-001 | Placebo | CTCAE Grade 4: Life-threatening | 0 | Participants |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo (6 Weeks) Then Washout (1 Week) Then NB-001 (6 Weeks) | 18 | |
| Overall Study | STARTED | NB-001 (6 Weeks) Then Washout (1 Week) Then Placebo (6 Weeks) | 19 | |
| Overall Study | COMPLETED | Placebo (6 Weeks) Then Washout (1 Week) Then NB-001 (6 Weeks) | 15 | |
| Overall Study | COMPLETED | NB-001 (6 Weeks) Then Washout (1 Week) Then Placebo (6 Weeks) | 17 | |
| Overall Study | NOT COMPLETED | Placebo (6 Weeks) Then Washout (1 Week) Then NB-001 (6 Weeks) | 3 | |
| Overall Study | NOT COMPLETED | NB-001 (6 Weeks) Then Washout (1 Week) Then Placebo (6 Weeks) | 2 |
Source ClinicalTrials.gov
Adverse events28
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nasopharyngitis | Infections and infestations | NB-001 | 3 | 33 | |
| other | Nasopharyngitis | Infections and infestations | Placebo | 4 | 34 | |
| other | Upper respiratory tract infection | Infections and infestations | NB-001 | 1 | 33 | |
| other | Upper respiratory tract infection | Infections and infestations | Placebo | 3 | 34 | |
| other | Insomnia | Psychiatric disorders | NB-001 | 1 | 33 | |
| other | Insomnia | Psychiatric disorders | Placebo | 6 | 34 | |
| other | Irritability | Psychiatric disorders | NB-001 | 1 | 33 | |
| other | Irritability | Psychiatric disorders | Placebo | 3 | 34 | |
| other | Anxiety | Psychiatric disorders | NB-001 | 1 | 33 | |
| other | Anxiety | Psychiatric disorders | Placebo | 2 | 34 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | NB-001 | 0 | 33 |
| Total, all-cause mortality | Placebo | 0 | 34 |
| Total, other adverse events | NB-001 | 18 | 33 |
| Total, other adverse events | Placebo | 24 | 34 |
| Total, serious adverse events | NB-001 | 0 | 33 |
| Total, serious adverse events | Placebo | 0 | 34 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| NB-001 vs Placebo: deaths from any cause | Trial comparisons |
| NB-001 vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05290493
GET https://conductscience.org/api/trials/v1/NCT05290493/results?page=2