Clinical trial NCT01750957: A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
Official title: A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2012-12-17
Enrollment: 47
Sex: ALL
Minimum age (years): 5
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Safety: Incidence of adverse events
timeFrame: 15 weeks
Secondary outcomes:
measure: 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)
timeFrame: 15 weeks
measure: Pharmacokinetics: Clearance (CL/F)
timeFrame: up to Week 12
measure: Pharmacokinetics: Volume of distribution at steady-state (Vss/F)
timeFrame: up to Week 12
Other outcomes:
Locations:
city: Long Beach
country: United States
state: California
city: Sacramento
country: United States
state: California
city: San Diego
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Washington
country: United States
state: District of Columbia
city: Decatur
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Iowa City
country: United States
state: Iowa
city: Louisville
country: United States
state: Kentucky
city: Baltimore
country: United States
state: Maryland
city: Worcester
country: United States
state: Massachusetts
city: Omaha
country: United States
state: Nebraska
city: New York
country: United States
state: New York
city: Cincinnati
country: United States
state: Ohio
city: Media
country: United States
state: Pennsylvania
city: Greenwood
country: United States
state: South Carolina
city: Nashville
country: United States
state: Tennessee
city: Houston
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: