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ZYN002- Cannabidiol

Phase 2

Fragile X Syndrome | Small molecule | Rare Disease |Harmony Biosciences Holdings, Inc.|Last Updated: May 27, 2026

Target and mechanism

ModalitySmall molecule

Also known as ZYN002 - Cannabidiol Transdermal Gel

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment688

FDA Designations

No designations recorded

Clinical trial landscape

ZYN002- Cannabidiol · 2 trials · 1 indication

Phase 2 2
NCT03802799Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXSFragile X Syndrome
ACTIVE NOT_RECRUITING476 Analytics
NCT03614663Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)Fragile X Syndrome
COMPLETED212 Analytics
PHASE2ACTIVE NOT_RECRUITING
Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
Fragile X SyndromeUnlock trial analytics
PHASE2COMPLETED
Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)
Fragile X SyndromeUnlock trial analytics

Study Endpoints

Primary Endpoints

To evaluate the long-term safety and tolerability of ZYN002 administered as a transdermal gel formulation.
Up to 96 month.

Safety assessment will include collection of any treatment emergent adverse events

Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Social Avoidance Subscale - Full Analysis Set
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.

Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Social Avoidance Subscale - Ad Hoc Analysis
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 Endpoints

To evaluate the long-term efficacy of ZYN002 in the treatment of symptoms of FXS.
Change from baseline to end of treatment
Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Irritability Subscale - Full Analysis Set
Change from baseline to end of treatment (Week 12)
Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Socially Unresponsive/Lethargic Subscale - Full Analysis Set
Change from baseline to end of treatment (Week 12)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ZYN002EXPERIMENTALZYN002 - cannabidiol Transdermal Gel
ZYN002 - Cannabidiol transdermal gelEXPERIMENTALZYN002 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 gelPLACEBO_COMPARATORMatching ZYN002 placebo supplied as a transdermal gel.

Interventions

NameTypeDescription
ZYN002 - Cannabidiol Transdermal GelDRUGPharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
Placebo Transdermal GelOTHERPlacebo formulated as a clear gel (transdermal delivery)
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Eligibility Criteria

Age Range3 Years to 29 Years
SexALL
Healthy VolunteersNo
Study Sites26

Inclusion Criteria: * Participated in study ZYN2-CL-016 or Study ZYN2-CL-033. * Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures. * Patients and parents/caregivers must be adequately informed of the nature, risks of the study, and give wr...

Countries:United StatesAustraliaNew ZealandUnited Kingdom
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Frequently asked questions about ZYN002- Cannabidiol

What is ZYN002 used for in Fragile X Syndrome?

ZYN002 is a transdermal gel formulation of cannabidiol being developed for the treatment of Fragile X Syndrome, a rare genetic condition. It is currently in Phase 2 clinical development and is intended for use in children and adolescents aged 3 years and older.

What does ZYN002 target?

ZYN002 contains cannabidiol, a small molecule that interacts with the endocannabinoid system. The drug is administered as a transdermal gel, allowing absorption through the skin. Its mechanism of action in Fragile X Syndrome is being studied in clinical trials.

Who is developing ZYN002?

ZYN002 is being developed by Harmony Biosciences Holdings, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol HRMY. The company is conducting clinical trials to evaluate the safety and efficacy of ZYN002 in patients with Fragile X Syndrome.

What phase is ZYN002 in?

ZYN002 is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Two Phase 2 trials have been conducted or are ongoing, including a completed placebo-controlled study and an active open-label extension study.

What clinical trials is ZYN002 in?

ZYN002 has been studied in two clinical trials. The first, NCT03614663, is a completed Phase 2 study called CONNECT-FX, which evaluated the drug in 212 children and adolescents with Fragile X Syndrome. The second, NCT03802799, is an active open-label extension study assessing long-term safety in 476 participants.

Is ZYN002 the same as ZYN002 Cannabidiol Transdermal Gel?

Yes, ZYN002 is also known as ZYN002 Cannabidiol Transdermal Gel. The drug is a transdermal gel formulation of cannabidiol, and both names refer to the same investigational product being developed for Fragile X Syndrome.