A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

Fragile X Syndrome Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01750957
• Other study ID #: NP28571
• Secondary ID: 2011-004349-42
• Status: Completed
• Phase: Phase 2
• First received: December 11, 2012
• Last updated: August 9, 2016
• Start date: February 2013
• Est. completion date: April 2014

Study information

• Verified date: August 2016
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 47
• Est. completion date: April 2014
• Est. primary completion date: April 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years to 13 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• Placebo
orally daily, 12 weeks
• RO4917523
Dose A orally daily, 12 weeks
• RO4917523
Dose B orally daily, 12 weeks

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Incidence of adverse events		15 weeks	No
Secondary	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	No
Secondary	Pharmacokinetics: Clearance (CL/F)		up to Week 12	No
Secondary	Pharmacokinetics: Volume of distribution at steady-state (Vss/F)		up to Week 12	No