Clinical trial: Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) (NCT03614663)

Eligibility criteria:
Inclusion Criteria:

          -  Male or female children and adolescents aged 3 to less than 18 years, at the time of
             Screening.

          -  Diagnosis of FXS through molecular documentation of FMR1 full mutation.

          -  Judged to be in good health based on physical exam, 12-lead ECG and clinical
             laboratory test results.

          -  Patients must be assessed by the Investigator as being moderately to severely impacted
             due to FXS.

          -  Patients taking psychotropic medication(s) should be on a stable regimen of not more
             than two such medications for at least fours weeks preceding Screening and must
             maintain that regimen throughout the study.

          -  If patients are receiving non-pharmacological, behavioral and/or dietary
             interventions, they must be stable and have been doing so for three months prior to
             screening.

          -  Patients and parents/caregivers must be adequately informed of the nature and risks of
             the study and given written informed consent prior to Screening.

          -  In the Investigator's opinion, patients and parents/caregivers are reliable and
             willing and able to comply with all protocol requirements and procedures.

        Exclusion Criteria:

          -  Females who are pregnant, nursing or planning a pregnancy.

          -  Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin
             levels greater than or equal to 2 times the upper limit of normal or alkaline
             phosphatase levels greater than or equal to 3 times the upper limit of normal.

          -  Use of a strong inhibitor/inducer of CYP3A4 or sensitive substrate of CYP3A4.

          -  Use of minocycline for 30 days prior to screening or throughout the study.

          -  Use of any benzodiazepine at screening or throughout the study.

          -  Use of THC or CBD-containing product within three months of Screening Visit or during
             the study.

          -  Change in pharmacologic or non-pharmacologic intervention during the course of the
             study.

          -  Any skin disease or condition including eczema, psoriasis, melanoma, acne, contact
             dermatitis, scarring, imperfections, lesions, tattoos, or discoloration that may
             affect treatment application, application site assessments or absorption of the trial
             drug.

          -  Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate
             or vigabatrin.

          -  Patients has an advanced, severe or unstable disease that may interfere with the study
             outcome evaluations.

          -  Patient has acute or progressive neurological disease, psychosis, schizophrenia or any
             other psychiatric disorder or severe mental abnormalities (other than FXS) that are
             likely to require changes in drug therapy or interfere with the study objectives or
             ability to adhere to protocol requirements.

          -  Patient has suspected or confirmed cardiovascular disease, advanced arteriosclerosis,
             structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities,
             coronary artery disease, cardiac conduction problems, exercise-related cardiac events
             including syncope and pre-syncope, risk factors for Torsades de pointes (e.g. heart
             failure, hypokalemia, family history of Long QT Syndrome) or other serious cardiac
             problems.

          -  History of treatment for, or evidence of drug abuse within the past year.

          -  Patient responds "yes" to Question 4 or 5 on the C-SSRS (Children) during Screening or
             at any time on study.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 3 Years old.
Subject must be at most 17 Years

Patient description (medical language): A 15-year-old boy with mild intellectual disability is brought to the office by his parents for a routine physical examination.  The boy is going to a school for students with learning disabilities.  The patient was adopted, and his immunizations are up to date.  Review of the patient's medical records is notable for cytogenetic studies that showed a small gap near the tip of the long arm of the X chromosome, which is consistent with fragile X syndrome, an X-linked disorder. The defect is an unstable expansion of trinucleotide repeats (CGG) in the fragile X mental retardation 1 (FMR1) gene, located on the long arm of the X chromosome. He is not using any medications and vital signs are within normal levels. His blood chemistry analysis as bellow:
Blood Chemistry Value      Normal Range     Patient Value
Glucose                    90-120 mg/dl     95 mg/dl
BUN (Blood Urea Nitrogen)  7-24 mg/dl       10 mg/dl
Creatinine                 0.7-1.4 mg/dl    0.8 mg/dl
Calcium                    8.5-10.5 mg/dl   9 mg/dl
Sodium                     134-143 mEq/L    135 mEq/L
Potassium                  3.5-4.5 mEq/L    3.7 mEq/L
Chloride                   95-108 mEq/L     98 mEq/L
CO2                        20-30 mEq/L      25 mEq/L
Blood pH                   7.38-7.42        7. 39

Patient description (patient's own words): My husband and I brought our 15-year-old son to the clinic for his routine exam. My son is going to school for special needs students. We adopted him a few years ago. His vaccinations are up to date. He already passed some chromosome testing and they found that he has a fragile X syndrome. The doctor told us that it comes from repeats in the fragile X chromosome. My son is not using any medication and his blood pressure temperature and breathing were normal during the exam. He also did a blood test. The results came back and showed that his blood sugar urea creatinine calcium sodium potassium chloride CO2 and blood pH were all within the normal range.

Does the patient match the trial criteria? 1