Fragile X Syndrome
Conditions
Brief summary
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.
Interventions
DRUGPlacebo
orally daily, 12 weeks
DRUGRO4917523
Dose A orally daily, 12 weeks
Sponsors
Hoffmann-La Roche
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
5 Years to 13 Years
Healthy volunteers
No
Inclusion criteria
* Children and adolescents, 5 to 13 years of age * Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S
Exclusion criteria
* Previous treatment with another mGlu5 receptor antagonist within the prior 3 months * Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study * Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome * History of suicidal behavior * Other protocol defined inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety: Incidence of adverse events | 15 weeks |
Secondary
| Measure | 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) | up to Week 12 |
| Pharmacokinetics: Volume of distribution at steady-state (Vss/F) | up to Week 12 |
Countries
United States
Outcome results
None listed