Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)
Condition: Fragile X Syndrome
MeSH terms: Fragile X Syndrome; Syndrome
Study type: Interventional
Phase: Phase 2/Phase 3

Summary: 
      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.
    

Detailed description: 
      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.
    

Intervention name(s): ZYN002 - Cannabidiol Transdermal Gel; Placebo Transdermal Gel
Intervention type(s): Drug; Other
Intervention description(s): Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery); Placebo formulated as a clear gel (transdermal delivery)
Other intervention name(s): Placebo Comparator; Matching Placebo
Intervention MeSH terms: Cannabidiol
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment