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Trial record 24 of 74 for:    "fragile x syndrome"

A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

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. Identifier: NCT01750957
Recruitment Status : Completed
First Posted : December 17, 2012
Last Update Posted : August 11, 2016
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief 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.

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: Placebo Drug: RO4917523 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 47 participants
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
Study Start Date : February 2013
Actual Primary Completion Date : April 2014
Actual Study Completion Date : April 2014

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
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

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

Secondary Outcome Measures :
  1. 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 ]
  2. Pharmacokinetics: Clearance (CL/F) [ Time Frame: up to Week 12 ]
  3. Pharmacokinetics: Volume of distribution at steady-state (Vss/F) [ Time Frame: up to Week 12 ]

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

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

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 identifier (NCT number): NCT01750957

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Sponsors and Collaborators
Hoffmann-La Roche
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Study Director: Clinical Trials Hoffmann-La Roche

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

Additional relevant MeSH terms:
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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