A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

Hoffmann-La Roche0 sites47 target enrollmentFebruary 2013

ConditionsFragile X Syndrome

InterventionsPlacebo RO4917523

DrugsPlacebo RO4917523

Overview

Phase: Phase 2
Intervention: Placebo
Conditions: Fragile X Syndrome
Sponsor: Hoffmann-La Roche
Enrollment: 47
Primary Endpoint: Safety: Incidence of adverse events
Status: Completed
Last Updated: 9 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

February 2013

End Date

April 2014

Last Updated

9 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Hoffmann-La Roche

Responsible Party

Sponsor

Eligibility Criteria

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/exclusion criteria may apply

Arms & Interventions

Placebo

Intervention: Placebo

RO4917523 Dose A

Intervention: RO4917523

RO4917523 Dose B

Intervention: RO4917523

Outcomes

Primary Outcomes

Safety: Incidence of adverse events

Time Frame: 15 weeks

Secondary Outcomes

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)