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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo - Low-Dose - High-Dose | Experimental | Placebo AZD7325 5mg BID in gelatin capsules AZD7325 15mg BID in gelatin capsules |
|
| Placebo - High-Dose - Low-Dose | Experimental | Placebo AZD7325 15mg BID in gelatin capsules AZD7325 5mg BID in gelatin capsules |
|
| Low-Dose - Placebo - High-Dose | Experimental | AZD7325 5mg BID in gelatin capsules Placebo AZD7325 15mg BID in gelatin capsules |
|
| Low-Dose - High-Dose - Placebo | Experimental | AZD7325 5mg BID in gelatin capsules AZD7325 15mg BID in gelatin capsules Placebo |
|
| High-Dose - Low-Dose - Placebo | Experimental | AZD7325 15mg BID in gelatin capsules AZD7325 5mg BID in gelatin capsules Placebo |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AZD7325 (High-Dose) | Drug | 15mg PO BID |
|
| Measure | Description | Time Frame |
|---|---|---|
| Amyloid Precursor Protein (APP) | Short-term treatment of peripheral APP dysregulation by correcting elevated levels | Through end of study, approximately 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Through end of study, approximately 12 weeks |
| Change in the Pediatric Anxiety Rating Scale (PARS) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ernest Pedapati, MD | Cincinnati Chlidren's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
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| ID | Term |
|---|---|
| D005600 | Fragile X Syndrome |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
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| ID | Term |
|---|---|
| C576237 | 4-amino-8-(2-fluoro-6-methoxy-phenyl)-N-propylcinnoline-3-carboxamide |
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|
| High-Dose - Placebo - Low-Dose | Experimental | AZD7325 15mg BID in gelatin capsules Placebo AZD7325 5mg BID in gelatin capsules |
|
| AZD7325 (Low-Dose) | Drug | 5mg PO BID |
|
| Placebo oral capsule | Drug | Placebo will be dosed similar to AZD7325, in terms of dosage form, frequency and duration. |
|
The PARS is the gold standard parent/caregiver reported anxiety outcome measure for use in Fragile X Syndrome clinical trials. |
| Through end of study, approximately 12 weeks |
| D009422 | Nervous System Diseases |
| D025064 | Sex Chromosome Disorders |
| D025063 | Chromosome Disorders |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D040181 | Genetic Diseases, X-Linked |
| D020271 | Heredodegenerative Disorders, Nervous System |