Clinical trial: Investigation of Sulindac in Male Fragile X Syndrome Patients Aged 13-40 (NCT04823052)

Eligibility criteria:
Inclusion Criteria:

          -  Subject and caregiver are both able to understand English clearly and caregiver can
             read and write English to complete study assessments

          -  Males aged 13-40 years (inclusive)

          -  Has FXS with molecular genetic confirmation of the full FMR1 mutation (>200 CGG
             repeats).

          -  Weight ≥45 kg

          -  Is in general good health as deemed by the Investigator, determined by physical
             examination, medical history, and laboratory tests

          -  If receiving sertraline, is on a stable, well-tolerated dose for the previous 3 months
             with no further changes anticipated

        Exclusion Criteria:

          -  Active or history of peptic or gastric ulcer or hemorrhage

          -  Any chronic major medical comorbid condition deemed by the Investigator as presenting
             added risk to the subject, including but not limited to refractory hypertension,
             kidney disease, or liver disease

          -  Diagnosed with diabetes (Type 1 or II) or receiving any anti-diabetic medication

          -  Unstable seizure disorder defined by any seizure within 6 months prior to baseline
             visit and/or a change in any anti-convulsant drug dosing in the 60 days prior to study
             entry

          -  Individual or family history of myocardial infarction, TIA, peripheral arterial
             disease, stroke or other significant cardiovascular disease

          -  Chronic use of NSAIDs or other anti-inflammatory agents

          -  Currently taking or have taken any cannabidiol (CBD) preparation within 30 days prior
             to randomization

          -  Currently taking or have taken sulindac within 30 days prior to randomization

          -  Changes in chronic psychotropic or anti-convulsant (where taken for reasons other than
             seizure control) drug treatment within 30 days prior to randomization

          -  History of adverse effects of sulindac or other NSAIDS that would prevent safe study
             completion

          -  Unable or unwilling to take oral medication (whole capsule) or history of dysphagia or
             malabsorption

          -  Has current or suspected symptoms of COVID-19 including, but not limited to fever, dry
             cough, new loss or change of smell or taste, myalgia, fatigue, or a positive COVID-19
             test.

          -  Has abnormal baseline laboratory assessments including, but not limited to alanine
             aminotransferase (ALT) or aspartate aminotransferase (AST) >1.5 × the upper limit of
             normal (ULN), total bilirubin or creatinine >1.0 × ULN, or other clinically relevant
             laboratory abnormality

          -  Has a clinically significant abnormal electrocardiogram (ECG), heart rate, or blood
             pressure at screening as judged by the Investigator

          -  Has received an investigational drug (either approved or not approved) in any prior
             clinical study within 30 days or 5 half-lives (whichever is longer) prior to screening
Male
No healthy subjects accepted to join the trial.
Subject must be at least 13 Years old.
Subject must be at most 40 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