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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-004349-42 | EudraCT Number |
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This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator |
| |
| RO4917523 Dose A | Experimental |
| |
| RO4917523 Dose B | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | orally daily, 12 weeks |
| |
| RO4917523 |
| Measure | Description | Time Frame |
|---|---|---|
| Safety: Incidence of adverse events | 15 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior) | 15 weeks | |
| Pharmacokinetics: Clearance (CL/F) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Long Beach | California | 90806 | United States | |||
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| Drug |
Dose A orally daily, 12 weeks |
|
| RO4917523 | Drug | Dose B orally daily, 12 weeks |
|
| up to Week 12 |
| Pharmacokinetics: Volume of distribution at steady-state (Vss/F) | up to Week 12 |
| Sacramento |
| California |
| 95817 |
| United States |
| San Diego | California | 92108 | United States |
| Aurora | Colorado | 80045 | United States |
| Washington D.C. | District of Columbia | 20020 | United States |
| Decatur | Georgia | 30033 | United States |
| Chicago | Illinois | 60612 | United States |
| Iowa City | Iowa | 52242 | United States |
| Louisville | Kentucky | 40202 | United States |
| Baltimore | Maryland | 21205 | United States |
| Worcester | Massachusetts | 01605 | United States |
| Omaha | Nebraska | 68198-4160 | United States |
| New York | New York | 10029 | United States |
| Cincinnati | Ohio | 45229 | United States |
| Media | Pennsylvania | 19063 | United States |
| Greenwood | South Carolina | 29646 | United States |
| Nashville | Tennessee | 37212 | United States |
| Houston | Texas | 77030 | United States |
| San Antonio | Texas | 78258 | United States |
| Seattle | Washington | 98145 | United States |
| ID | Term |
|---|---|
| D005600 | Fragile X Syndrome |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| 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 |
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| ID | Term |
|---|---|
| C000596770 | 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine |
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