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A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

This study is currently recruiting participants.
Verified May 2013 by Hoffmann-La Roche
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT01750957
First received: December 11, 2012
Last updated: May 7, 2013
Last verified: May 2013
History of Changes
  Purpose

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: RO4917523
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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:

U.S. FDA Resources

Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Safety: Incidence of adverse events [ Time Frame: 15 weeks ] [ Designated as safety issue: No ]

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 ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Clearance (CL/F) [ Time Frame: up to Week 12 ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Volume of distribution at steady-state (Vss/F) [ Time Frame: up to Week 12 ] [ Designated as safety issue: No ]

Estimated Enrollment: 45
Study Start Date: February 2013
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RO4917523 Dose A Drug: RO4917523
Dose A orally daily, 12 weeks
Experimental: RO4917523 Dose B Drug: RO4917523
Dose B orally daily, 12 weeks
Placebo Comparator: Placebo Drug: Placebo
orally daily, 12 weeks

  Eligibility

Ages Eligible for Study:   5 Years to 13 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Children and adolescents, 5 to 13 years of age
  • Diagnosis of fragile X syndrome based on prior DNA testing confirming 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01750957

Contacts
Contact: Please reference Study ID Number: NP28571 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. Only) genentechclinicaltrials@druginfo.com

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

No publications provided

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01750957     History of Changes
Other Study ID Numbers: NP28571, 2011-004349-42
Study First Received: December 11, 2012
Last Updated: May 7, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Fragile X Syndrome
Mental Retardation, X-Linked
Genetic Diseases, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
 Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System

ClinicalTrials.gov processed this record on May 19, 2013