This study will investigate the safety, tolerability and blood pharmacodynamics of treatment with oral administration of AZD7325 at 5 mg BID, 15 mg BID, and placebo BID, in adults with Fragile X Syndrome. The study also will also investigate measures of efficacy and biomarkers during treatment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
15
15mg PO BID
5mg PO BID
Placebo will be dosed similar to AZD7325, in terms of dosage form, frequency and duration.
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Amyloid Precursor Protein (APP)
Short-term treatment of peripheral APP dysregulation by correcting elevated levels
Time frame: Through end of study, approximately 12 weeks
Change in the Social Withdrawal subscale score of the Aberrant Behavior Checklist (ABC)
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Time frame: Through end of study, approximately 12 weeks
Change in the Pediatric Anxiety Rating Scale (PARS)
The PARS is the gold standard parent/caregiver reported anxiety outcome measure for use in Fragile X Syndrome clinical trials.
Time frame: Through end of study, approximately 12 weeks
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