Clinical trial: Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome (NCT00788073)

Eligibility criteria:
Inclusion Criteria:

          -  Male or female subjects 12 to 40 years of age eventually expanding to 6 years of age

          -  Molecular documentation of the fragile X mutation.

          -  Clinical Global Impression - Severity (CGI-S) rating for problem behavior of moderate
             or higher at screening and at Visit 1

          -  An Aberrant Behavior Checklist (ABC-C) Irritability Subscale score >12 and at least 3
             items on the Irritability Subscale rated at least moderate or above.

          -  Current treatment with no more than three psychoactive medications, including
             anti-epileptics.

          -  Current pharmacological treatment regimen has been stable for at least 4 weeks.

        Exclusion Criteria:

          -  Subjects with a history of seizure disorder who are not currently receiving treatment
             with antiepileptics.

          -  Subjects with any condition, including alcohol and drug abuse, which might interfere
             with the conduct of the study, confound interpretation of the study results, or
             endanger their own well-being. This includes, but is not limited to impairment of
             renal function, evidence or history of malignancy or any significant hematological,
             endocrine, cardiovascular, respiratory, hepatic, or gastrointestinal disease.

          -  Subjects who plan to initiate or change pharmacologic or non-pharmacologic
             interventions during the course of the study.

          -  Subjects who are currently receiving treatment with racemic baclofen.

          -  Subjects currently treated with vigabatrin or tiagabine.

          -  Subjects taking another investigational drug currently or within the last 30 days.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 6 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? Entailment