ZYN002 - Cannabidiol Transdermal Gel
Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
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This trial will evaluate the efficacy and safety of ZYN002, a clear cannabidiol gel that can be applied to the skin (called transdermal application) twice a day for the treatment of behavioral symptoms of Fragile X Syndrome (FXS). Eligible participants will then participate in up to a 14 week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to \< 18 years, will be eligible to participate.
Locally preserved from ClinicalTrials.govOpen ClinicalTrials.govlast source update 2022-07-06
What this record can show
Results are reported by the registry submitter and preserved exactly. They are not independent verification or a treatment recommendation.
Registry facts
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of ZYN002, a pharmaceutically manufactured cannabidiol, formulated as a transdermal gel, for the treatment of children and adolescent with FXS. Approximately 204 male and female patients, ages 3 to \< 18 years, will undergo a screening process. Eligible participants will be randomized 1:1 to either trial drug or placebo and will undergo a 14-week treatment period. Randomization will be stratified by gender, weight category and geographic region. All participants may receive placebo during the trial. Participants who are taking anti-seizure drugs may undergo an additional 1-2 weeks of blinded treatment to taper off study drug treatment. The assignment will be done by a computer generated system and neither the trial doctor or the participant or their caregivers will know which treatment is being given to them. The dose of the treatment will depend on the weight of the participants. If the participants weigh less than or equal to 35 kg, they will receive 2 sachets of the gel twice a day (1 sachet approximately every 12 hours) and if they weigh more than 35 kg, they will receive 4 sachets of gel per day (2 sachets approximately every 12 hours). Parents/ caregivers will be instructed on proper application of the gel. The gel will be applied to clean, dry, intact skin of the upper arms/ shoulders. Blood samples will be collected for safety analysis of ZYN002. An independent analytical laboratory will also perform CGG repeat and methylation status analyses. Additionally, the parents/caregivers will be asked to complete some questionnaires. There will be other questionnaires and scales that will be completed at the site by the trial doctor. After the final dose, patients will be followed weekly for 4 weeks by telephone, prior to discharge from the study.
Linked local records
Local links use governed condition terms or exact source-reported intervention names. They are navigation candidates, not efficacy claims.
Interventions
Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
Placebo formulated as a clear gel (transdermal delivery)
Placebo Comparator · Matching PlaceboSource-reported results
Results are reported by the registry submitter and preserved exactly. They are not independent verification or a treatment recommendation.
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 to 12, and a higher value indicates a worse outcome.
Population: The prospective comparison of the full analytical set between ZYN002 and placebo.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 109 | -2.68 | 0.310 |
| Placebo | 101 | -2.29 | 0.318 |
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is between 0 and 54, and the higher score means a worse outcome.
Eligibility
primary outcomes
Time frame: Change from Baseline to end of treatment (Week 12)
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 to 12, and a higher value indicates a worse outcome.
Time frame: Change from baseline to end of treatment (Week 12)
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 and 12, and the higher score means a worse outcome.
secondary outcomes
Time frame: Change from baseline to end of treatment (Week 12)
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is between 0 and 54, and the higher score means a worse outcome.
Time frame: Change from baseline to end of treatment (Week 12)
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 and 39, and a higher mean indicates a worse outcome.
Time frame: Change in CGI-I at end of treatment (Week 12)
The Clinical Global Impressions- Improvement global improvement item is a 7-point Likert scale designed to measure behavioral symptomatic change at a specific time compared to baseline. CGI-I is a standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities. The score ranges form 1-very much improved to 7-very much worse. The percentage of patients with any improvement (minimally, much, very much improved) was assessed.
Time frame: Change from baseline in ABC-C to end of treatment (Week 12)
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is between 0 and 54, and the higher score means a worse outcome.
Publications
Population: The prospective comparison of the full analytical set between ZYN002 and placebo.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 109 | -5.88 | 0.960 |
| Placebo Transdermal Gel | 101 | -4.14 | 0.984 |
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 and 39, and a higher mean indicates a worse outcome.
Population: The prospective comparison of the full analytical set between ZYN002 and placebo.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 109 | -3.50 | 0.565 |
| Placebo Transdermal Gel | 101 | -3.14 | 0.577 |
The Clinical Global Impressions- Improvement global improvement item is a 7-point Likert scale designed to measure behavioral symptomatic change at a specific time compared to baseline. CGI-I is a standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities. The score ranges form 1-very much improved to 7-very much worse. The percentage of patients with any improvement (minimally, much, very much improved) was assessed.
Population: The prospective comparison of the full analytical set between ZYN002 and placebo.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 109 | 46 | - |
| Placebo Transdermal Gel | 101 | 37 | - |
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 and 12, and the higher score means a worse outcome.
Population: A pre-specified, ad hoc analysis of the data collected in patients with ≥90% methylation of the FMR1 gene to evaluate the effect of ZYN002 versus placebo was developed prior to unblinding the trial.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 91 | -2.99 | 0.323 |
| Placebo | 76 | -1.99 | 0.345 |
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is between 0 and 54, and the higher score means a worse outcome.
Population: A pre-specified, ad hoc analysis of the data collected in patients with ≥90% methylation of the FMR1 gene to evaluate the effect of ZYN002 versus placebo was developed prior to unblinding the trial.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 91 | -6.43 | 1.020 |
| Placebo Transdermal Gel | 76 | -4.13 | 1.088 |
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 to 39 , and the higher score means a worse outcome.
Population: A pre-specified, ad hoc analysis of the data collected in patients with ≥90% methylation of the FMR1 gene to evaluate the effect of ZYN002 versus placebo was developed prior to unblinding the trial.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 91 | -3.91 | 0.592 |
| Placebo Transdermal Gel | 76 | -2.74 | 0.631 |
The Clinical Global Impressions- Improvement global improvement item is a 7-point Likert scale designed to measure behavioral symptomatic change at a specific time compared to baseline. CGI-I is a standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities. The score ranges form 1-very much improved to 7-very much worse. The percentage of patients with any improvement (minimally, much, very much improved) was assessed.
Population: A pre-specified, ad hoc analysis of the data collected in patients with ≥90% methylation of the FMR1 gene to evaluate the effect of ZYN002 versus placebo was developed prior to unblinding the trial.
| Group | N | Value | Spread / interval |
|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel | 91 | 43 | - |
| Placebo Transdermal Gel | 76 | 25 | - |
| Milestone | ZYN002 - Cannabidiol Transdermal Gel | Placebo Transdermal Gel |
|---|---|---|
| STARTED | 110 | 102 |
| COMPLETED | 106 | 99 |
| NOT COMPLETED | 4 | 3 |
| Event | System | Groups: affected / at risk |
|---|---|---|
| vomiting | Gastrointestinal disorders | ZYN002 - Cannabidiol Transdermal Gel: 8 / 109 · Placebo Transdermal Gel: 6 / 102 |
| pyrexia | General disorders | ZYN002 - Cannabidiol Transdermal Gel: 5 / 109 · Placebo Transdermal Gel: 7 / 102 |
| application site pain | General disorders | ZYN002 - Cannabidiol Transdermal Gel: 7 / 109 · Placebo Transdermal Gel: 1 / 102 |
| upper respiratory tract infection | Infections and infestations | ZYN002 - Cannabidiol Transdermal Gel: 15 / 109 · Placebo Transdermal Gel: 7 / 102 |
| nasopharyngitis | Infections and infestations | ZYN002 - Cannabidiol Transdermal Gel: 10 / 109 · Placebo Transdermal Gel: 9 / 102 |
Time frame: Change from baseline in ABC-C to end of treatment (Week 12)
The Aberrant Behavior Checklist-Community is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. The range for score for the subscale is from 0 to 39 , and the higher score means a worse outcome.
Time frame: Change in CGI-I at end of treatment (Week 12)
The Clinical Global Impressions- Improvement global improvement item is a 7-point Likert scale designed to measure behavioral symptomatic change at a specific time compared to baseline. CGI-I is a standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities. The score ranges form 1-very much improved to 7-very much worse. The percentage of patients with any improvement (minimally, much, very much improved) was assessed.