Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) (CONNECT-FX)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03614663|
Recruitment Status : Active, not recruiting
First Posted : August 3, 2018
Last Update Posted : February 5, 2020
|Condition or disease||Intervention/treatment||Phase|
|Fragile X Syndrome||Drug: ZYN002 - CBD Transdermal Gel Other: Placebo Transdermal Gel||Phase 2 Phase 3|
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of ZYN002, a pharmaceutically manufactured CBD, 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-epileptic 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 study 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 study doctor.
After the final dose, patients will be followed weekly for 4 weeks by telephone, prior to discharge from the study.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||204 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With Fragile X Syndrome|
|Actual Study Start Date :||June 12, 2018|
|Estimated Primary Completion Date :||May 30, 2020|
|Estimated Study Completion Date :||June 7, 2020|
Experimental: ZYN002 - CBD transdermal gel
ZYN002 supplied as a transdermal gel. Patients weighing less than or equal to 35 kg will be randomized to receive either 125 mg CBD Q12H or placebo.
Patients weighing greater than 35 kg will be randomized to receive 250 mg CBD Q12H or placebo.
Drug: ZYN002 - CBD Transdermal Gel
Pharmaceutically manufactured. Cannabidiol (CBD) formulated as a clear gel (transdermal delivery)
Placebo Comparator: Placebo transdermal gel
Matching ZYN002 placebo supplied as a transdermal gel.
Other: Placebo Transdermal Gel
Placebo formulated as a clear gel (transdermal delivery)
- Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 [ Time Frame: Change from Baseline to end of treatment (Week 14) ]The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
- Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 [ Time Frame: Change from Baseline to end of treatment (Week 14) ]The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
- Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 3 [ Time Frame: Change from baseline to end of treatment (Week 14) ]The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
- Clinical Global Impressions- Improvement (CGI-I) [ Time Frame: Change from baseline to end of treatment (Week 14) ]The CGI-I 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03614663