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Also known as ZYN002 - Cannabidiol Transdermal Gel, ZYN002- Cannabidiol, Cannabidiol Transdermal Gel
ZYN002 · 4 trials · 3 indications
The ABC-C FXS is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials
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.
Safety assessment will include collection of any treatment emergent adverse events
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.
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.
| Arm | Type | Description |
|---|---|---|
| ZYN002 | EXPERIMENTAL | Pharmaceutically manufactured cannabidiol, which is formulated as a clear gel for transdermal delivery. |
| Placebo | PLACEBO_COMPARATOR | Placebo is formulated as a clear gel for transdermal delivery. |
| Open-label | EXPERIMENTAL | Open-label |
| ZYN002 - Cannabidiol transdermal gel | EXPERIMENTAL | ZYN002 supplied as a transdermal gel. Patients weighing less than or equal to 35 kg will be randomized to receive either 125 mg cannabidiol Q12H or placebo. Patients weighing greater than 35 kg will be randomized to receive 250 mg cannabidiol Q12H or placebo. |
| Placebo transdermal gel | PLACEBO_COMPARATOR | Matching ZYN002 placebo supplied as a transdermal gel. |
| Name | Type | Description |
|---|---|---|
| ZYN002 | DRUG | Pharmaceutically manufactured cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery) |
| Placebo | DRUG | Placebo formulated as a clear gel that can be applied to the skin (transdermal delivery) Other Names: Placebo Comparator Matching Placebo |
| ZYN002 - Cannabidiol Transdermal Gel | DRUG | Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery) |
| Placebo Transdermal Gel | OTHER | Placebo formulated as a clear gel (transdermal delivery) |
Inclusion Criteria: * Male or female children and adolescents aged 3 to \< 30 years, at the time of Screening. * Patient resides with caregiver who will continue to provide consistent care throughout the study. * Judged by the Investigator to be in generally good health at Screening based upon the ...
ZYN002 is an investigational small molecule cannabidiol transdermal gel developed by Harmony Biosciences for rare disease indications. It is being studied in 22Q11.2 Deletion Syndrome and Fragile X Syndrome. ZYN002 is applied as a gel rather than taken orally, and it remains in clinical development rather than being an approved medicine.
ZYN002 targets cannabinoid receptors. Its active ingredient is cannabidiol, delivered through the skin as a transdermal gel. By acting on cannabinoid receptors, the drug is designed to engage the endocannabinoid system, which is the biological pathway under study in the Fragile X Syndrome and 22Q11.2 Deletion Syndrome programs.
ZYN002 is developed by Harmony Biosciences Holdings, Inc., which trades on Nasdaq under the ticker HRMY. Harmony Biosciences is the sponsor of the clinical program studying the cannabidiol transdermal gel in Fragile X Syndrome and 22Q11.2 Deletion Syndrome.
ZYN002 is in Phase 2 development overall, though its Fragile X Syndrome program has included a completed Phase 3 study. The 22Q11.2 Deletion Syndrome work is at Phase 2. ZYN002 is investigational and is not an approved therapy; it continues in clinical development for these rare disease indications.
ZYN002 has been studied across several trials. These include the Phase 2 INSPIRE study in 22Q11.2 Deletion Syndrome (NCT05149898), the Phase 3 Fragile X Syndrome study (NCT04977986), the CONNECT-FX Phase 2 trial in Fragile X Syndrome (NCT03614663), and an active long-term open-label extension (NCT03802799).
Yes. ZYN002 is also known as ZYN002 - Cannabidiol Transdermal Gel and ZYN002 - Cannabidiol. These names refer to the same investigational product: a cannabidiol formulated as a gel applied to the skin. Readers searching for cannabidiol transdermal gel in Fragile X Syndrome or 22Q11.2 Deletion Syndrome are looking at the same Harmony Biosciences asset.