Trial · NCT05149898
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
NCT05149898, Phase 2, Completed, Harmony Biosciences Management, Inc.; results first posted 2026-03-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE). Trials, record NCT05149898. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05149898
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
- NCT number
- NCT05149898
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Conditions
- 22Q11.2 Deletion Syndrome
- Interventions
- ZYN002
- First posted
- 2021-12-08
- Last updated
- 2026-03-09
- Results first posted
- 2026-03-09
- Limitations and caveats
Dosing was adjusted based on weight. Therefore, the decision was made to collect data as a single arm (ZYN002) instead of by dose or by period because the dosing is not relevant to the interpretation of the study results and based on pharmacokinetic modeling, exposure is consistent across doses. The Protocol and Statistical Analysis Plan will not be updated. Instead, the Sponsor has written a Note to File to state how data were collected.
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) | From first dose of study drug administration (Day 1) up to end of the study, approximately 306 days | An adverse event (AE) is an undesirable medical occurrence or worsening of a pre-existing medical condition that occurs at any time after signing of the informed consent form whether or not it is considered to be related to treatment. Any AE that results in one or more of the following is considered a SAE: death, life threatening, in-patient hospitalization, persistent or significant disability/incapacity, congenital abnormality or birth defect, and other medically important events. TEAEs are defined as AEs with onset dates on or after the start of study drug. Adverse event data were collected as a single arm because, based on pharmacokinetics modeling, exposure is consistent between doses. |
| Secondary | Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | The Aberrant Behavior Checklist - Community (ABC-C) instrument is a scale for rating inappropriate and maladaptive behavior of participants with developmental disabilities, including intellectual disability and autism spectrum disorder. The ABC-C was completed by the parent/caregiver with support from the site staff. The ABC-C asks responders to rate behaviors from "0= not at all a problem" to "3= the problem is severe in degree" across 58 questions. The ABC-C consists of 5 subscales: hyperactivity noncompliance subscale (scale range from 0 to 48), irritability subscale (scale range from 0 to 45), inappropriate speech subscale (scale range from 0 to 12), stereotypic behavior subscale (scale range from 0 to 21) and social withdrawal subscale (scale range from 0 to 48). Total scores range from 0 (not at all a problem) to 174 (the problem is severe in degree). Higher scores indicate higher severity. Baseline was defined as the last assessment prior to the first dose of ZYN002. |
| Secondary | Change From Baseline in Anxiety, Depression and Mood Scale (ADAMS) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days).Period 2: Baseline (Day 1 of | The Anxiety, Depression and Mood Scale (ADAMS) was completed by the parent/caregiver with support from the site staff. The ADAMS is comprised of 28 items (question 3 is counted in two subscales), which are rated on a scale of "0=not a problem" to "3=severe problem". The ADAMS consists of 5 subscales: depressed mood subscale (scale range from 0 to 21), general anxiety subscale (scale range from 0 to 21), manic/hyperactive behavior subscale (scale range from 0 to 15), obsessive/compulsive behavior subscale (scale range from 0 to 9) and social avoidance subscale (scale range from 0 to 21). Total score range is 0 to 84. Higher scores indicate higher severity/worse outcome. Baseline was defined as the last assessment prior to the first dose of ZYN002. |
| Secondary | Change From Baseline in Clinical Global Impression-Severity (CGI-S) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | The Clinical Global Impression-Severity (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment relative to the clinician's past experience with participants who have the same diagnosis. Considering total clinical experience, a participant was assessed on severity of mental illness at the time of rating as follows: 1= normal, not at all ill; 2= borderline mentally ill; 3= mildly ill; 4= moderately ill; 5= markedly ill; 6= severely ill; or 7= extremely ill. The CGI-S score range from 0 (not a problem) to 7 (severe problem). Higher scores indicate higher severity. Baseline was defined as the last assessment prior to the first dose of ZYN002. |
| Secondary | Number of Participants With Clinical Global Impression-Improvement (CGI-I) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Week 14 (Day 98 ± 3 days); Period 2: Week 38 (Day 266 ± 3 days). | The CGI-I is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and rated as: 1= very much improved; 2= much improved; 3= minimally improved; 4= no change; 5= minimally worse; 6= much worse; or 7= very much worse. Baseline was defined as the last assessment prior to the first dose of ZYN002. Higher scores indicate worse outcome. |
| Secondary | Number of Participants With Changes in Caregiver Reported Behavioral Problems at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Week 14 (Day 98 ± 3 days); Period 2: Week 38 (Day 266 ± 3 days). | The parent/caregiver was asked the following question "What are the three behavioral, emotional, or social problems that most impacted your son/daughter and his/her family in approximately the past year?". At each study visit the parent/caregiver was reminded of their responses from the screening visit in order to rate the 3 questions for improvement or worsening. |
| Secondary | Change From Baseline in Pediatric Anxiety Rating Scale-Revised (PARS-R) Total Severity Score at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | The Pediatric Anxiety Rating Scale-Revised (PARS-R) is a clinician-rated caregiver interview that covers 61 behaviors related to anxiety. The interviewer assesses 7 items: overall severity of anxiety feelings; overall number, frequency, and severity of anxiety symptoms; overall severity of anxiety (physical symptoms); overall avoidance of anxiety-provoking situations; and anxiety interference with family and peer relationships and/or performance at home or outside of the home. Each of the 7 severity items was scored on a scale of 1 to 5, with 5 being the most severe and frequent. The total score for the PARS-R is the sum of the 7 items; range from 0 (not a problem) to 35 (severe problem). Higher scores indicate higher severity. Baseline was defined as the last assessment prior to the first dose. |
| Secondary | Plasma Concentrations of Cannabidiol and Δ9-tetrahydrocannabinol | Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | Blood samples were collected to determine the plasma concentrations of cannabidiol and Δ9-tetrahydrocannabinol. |
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 | Overall Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) | From first dose of study drug administration (Day 1) up to end of the study, approximately 306 days | All participants who received at least 1 dose of ZYN002. Data were collected for all participants combined regardless of ZYN002 dose because PK modeling suggested similar exposures for the weight-based dosing regimens. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | The modified intent-to-treat (mITT) analysis set included safety participants who had at least 1 post baseline efficacy assessment. Data were collected for all participants regardless of ZYN002 dose because PK modeling suggested similar exposures for the weight-based dosing regimens. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Anxiety, Depression and Mood Scale (ADAMS) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days).Period 2: Baseline (Day 1 of | The mITT analysis set included safety participants who had at least 1 post baseline efficacy assessment. Data were collected for all participants regardless of ZYN002 dose because PK modeling suggested similar exposures for the weight-based dosing regimens. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Clinical Global Impression-Severity (CGI-S) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | The mITT analysis set included safety participants who had at least 1 post baseline efficacy assessment. Data were collected for all participants regardless of ZYN002 dose because PK modeling suggested similar exposures for the weight-based dosing regimens. | score on a scale | MEAN |
| SECONDARY | Number of Participants With Clinical Global Impression-Improvement (CGI-I) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Week 14 (Day 98 ± 3 days); Period 2: Week 38 (Day 266 ± 3 days). | The mITT analysis set included safety participants who had at least 1 post baseline efficacy assessment. Data were collected for all participants regardless of ZYN002 dose because PK modeling suggested similar exposures for the weight-based dosing regimens. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Changes in Caregiver Reported Behavioral Problems at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Week 14 (Day 98 ± 3 days); Period 2: Week 38 (Day 266 ± 3 days). | The mITT analysis set included safety participants who had at least 1 post baseline efficacy assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Pediatric Anxiety Rating Scale-Revised (PARS-R) Total Severity Score at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: Baseline (Day 1) and Week 14 (Day 98 ± 3 days); Period 2: Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | The mITT analysis set included safety participants who had at least 1 post baseline efficacy assessment. Data were collected for all participants regardless of ZYN002 dose because PK modeling suggested similar exposures for the weight-based dosing regimens. | score on a scale | MEAN |
| SECONDARY | Plasma Concentrations of Cannabidiol and Δ9-tetrahydrocannabinol | Week 14 (Day 98 ± 3 days) and Week 38 (Day 266 ± 3 days). | The Pharmacokinetic population included all participants who received at least 1 application of study drug and had a plasma concentration obtained from at least 1 post baseline assessment. | nanogram per milliliter | MEAN |
Source ClinicalTrials.gov
Results by group54
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) | ZYN002 | Any TEAE | 12 | Participants | |
| Overall Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) | ZYN002 | Any treatment-emergent SAE | 3 | Participants | |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: ZYN002 | Hyperactivity noncompliance subscale | -7.6 | Standard Deviation 10.19 | score on a scale |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 2: ZYN002 | Hyperactivity noncompliance subscale | -8.4 | Standard Deviation 10.36 | score on a scale |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: ZYN002 | Irritability subscale | -8.4 | Standard Deviation 10.42 | score on a scale |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 2: ZYN002 | Irritability subscale | -10.2 | Standard Deviation 10.38 | score on a scale |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: ZYN002 | Inappropriate speech subscale | -1.8 | Standard Deviation 2.69 | score on a scale |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 2: ZYN002 | Inappropriate speech subscale | -3.0 | Standard Deviation 2.53 | score on a scale |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 1: ZYN002 | Stereotypic behavior subscale | -2.3 | Standard Deviation 3.30 | score on a scale |
| Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) at Week 14 (Period 1) and Week 38 (Period 2) | Period 2: ZYN002 | Stereotypic behavior subscale | -3.1 | Standard Deviation 3.96 | score on a scale |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1 (14 Weeks) | STARTED | ZYN002 | 20 | |
| Period 1 (14 Weeks) | COMPLETED | ZYN002 | 17 | |
| Period 1 (14 Weeks) | NOT COMPLETED | ZYN002 | 3 | |
| Period 2 (24 Weeks) | STARTED | ZYN002 | 13 | |
| Period 2 (24 Weeks) | COMPLETED | ZYN002 | 12 | |
| Period 2 (24 Weeks) | NOT COMPLETED | ZYN002 | 1 |
Source ClinicalTrials.gov
Adverse events25
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 | Middle ear effusion | Ear and labyrinth disorders | ZYN002 | 1 | 20 | 1 |
| other | Constipation | Gastrointestinal disorders | ZYN002 | 1 | 20 | 1 |
| other | Application site pain | General disorders | ZYN002 | 2 | 20 | 2 |
| other | Application site pruritus | General disorders | ZYN002 | 1 | 20 | 1 |
| other | Nasopharyngitis | Infections and infestations | ZYN002 | 1 | 20 | 1 |
| other | Oral candidiasis | Infections and infestations | ZYN002 | 1 | 20 | 1 |
| other | Staphylococcal infection | Infections and infestations | ZYN002 | 1 | 20 | 1 |
| other | Urinary tract infection | Infections and infestations | ZYN002 | 1 | 20 | 3 |
| other | Contusion | Injury, poisoning and procedural complications | ZYN002 | 1 | 20 | 1 |
| other | Radius fracture | Injury, poisoning and procedural complications | ZYN002 | 1 | 20 | 1 |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | ZYN002 | 0 | 20 |
| Total, other adverse events | ZYN002 | 12 | 20 |
| Total, serious adverse events | ZYN002 | 3 | 20 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41888667 | DERIVED |
Source PubMed
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT05149898
GET https://conductscience.org/api/trials/v1/NCT05149898/results?page=2