A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

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

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.

Study Overview

• Status: Completed
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: Placebo; Drug: RO4917523; Drug: RO4917523

• Study Type: Interventional
• Enrollment (Actual): 47
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Long Beach, California, United States, 90806
    • Sacramento, California, United States, 95817
    • San Diego, California, United States, 92108
  • Colorado
    • Aurora, Colorado, United States, 80045
  • District of Columbia
    • Washington, District of Columbia, United States, 20020
  • Georgia
    • Decatur, Georgia, United States, 30033
  • Illinois
    • Chicago, Illinois, United States, 60612
  • Iowa
    • Iowa City, Iowa, United States, 52242
  • Kentucky
    • Louisville, Kentucky, United States, 40202
  • Maryland
    • Baltimore, Maryland, United States, 21205
  • Massachusetts
    • Worcester, Massachusetts, United States, 01605
  • Nebraska
    • Omaha, Nebraska, United States, 68198-4160
  • New York
    • New York, New York, United States, 10029
  • Ohio
    • Cincinnati, Ohio, United States, 45229
  • Pennsylvania
    • Media, Pennsylvania, United States, 19063
  • South Carolina
    • Greenwood, South Carolina, United States, 29646
  • Tennessee
    • Nashville, Tennessee, United States, 37212
  • Texas
    • Houston, Texas, United States, 77030
    • San Antonio, Texas, United States, 78258
  • Washington
    • Seattle, Washington, United States, 98145

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 13 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo	Drug: Placebo; orally daily, 12 weeks
EXPERIMENTAL: RO4917523 Dose A	Drug: RO4917523; Dose A orally daily, 12 weeks; Drug: RO4917523; Dose B orally daily, 12 weeks
EXPERIMENTAL: RO4917523 Dose B	Drug: RO4917523; Dose A orally daily, 12 weeks; Drug: RO4917523; Dose B orally daily, 12 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety: Incidence of adverse events	15 weeks

Secondary Outcome Measures

Outcome 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

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start: February 1, 2013
• Primary Completion (ACTUAL): April 1, 2014
• Study Completion (ACTUAL): April 1, 2014

Study Registration Dates

• First Submitted: December 11, 2012
• First Submitted That Met QC Criteria: December 13, 2012
• First Posted (ESTIMATE): December 17, 2012

Study Record Updates

• Last Update Posted (ESTIMATE): August 11, 2016
• Last Update Submitted That Met QC Criteria: August 9, 2016
• Last Verified: August 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome
• Other Study ID Numbers: NP28571; 2011-004349-42 (EUDRACT_NUMBER)