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A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

This study has been completed.
Information provided by (Responsible Party):
Hoffmann-La Roche Identifier:
First received: December 11, 2012
Last updated: August 9, 2016
Last verified: August 2016
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.

Condition Intervention Phase
Fragile X Syndrome Drug: Placebo Drug: RO4917523 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Safety: Incidence of adverse events [ Time Frame: 15 weeks ]

Secondary Outcome Measures:
  • 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) [ Time Frame: 15 weeks ]
  • Pharmacokinetics: Clearance (CL/F) [ Time Frame: up to Week 12 ]
  • Pharmacokinetics: Volume of distribution at steady-state (Vss/F) [ Time Frame: up to Week 12 ]

Enrollment: 47
Study Start Date: February 2013
Study Completion Date: April 2014
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo Drug: Placebo
orally daily, 12 weeks
Experimental: RO4917523 Dose A Drug: RO4917523
Dose A orally daily, 12 weeks
Experimental: RO4917523 Dose B Drug: RO4917523
Dose B orally daily, 12 weeks


Ages Eligible for Study:   5 Years to 13 Years   (Child)
Sexes Eligible for Study:   All
Accepts 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/exclusion criteria may apply
  Contacts and Locations
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Please refer to this study by its identifier: NCT01750957

  Show 20 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche Identifier: NCT01750957     History of Changes
Other Study ID Numbers: NP28571
2011-004349-42 ( EudraCT Number )
Study First Received: December 11, 2012
Last Updated: August 9, 2016

Additional relevant MeSH terms:
Fragile X Syndrome
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System processed this record on September 21, 2017