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Trial record 1 of 3 for:    roche AND fragile x
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A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01750957
First Posted: December 17, 2012
Last Update Posted: August 11, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Hoffmann-La Roche
  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: 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

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 13 Years   (Child)
Sexes Eligible for Study:   All
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 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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01750957


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

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01750957     History of Changes
Other Study ID Numbers: NP28571
2011-004349-42 ( EudraCT Number )
First Submitted: December 11, 2012
First Posted: December 17, 2012
Last Update Posted: August 11, 2016
Last Verified: August 2016

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