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Completed

NCT Number: NCT01750957

A 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.

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Key information

Conditions

Fragile X Syndrome Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Sex Chromosome Disorders X-Linked Intellectual Disability

Age range

5 year–13 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Long Beach, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Placebo

Drug

orally daily, 12 weeks

RO4917523

Drug

Dose A orally daily, 12 weeks

Primary outcomes

  1. Safety: Incidence of adverse events

    Time frame: 15 weeks

Secondary outcomes

  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

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2014
Study completion
2014
First posted
Dec 17, 2012
Registry last updated
Aug 11, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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