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NCT Number: NCT01015430

A Study With RO4917523 in Patients With Fragile X Syndrome

This randomized, double-blind multiple ascending dose study will evaluate the safety and tolerability, pharmacokinetics and efficacy of RO4917523 in patients with Fragile X Syndrome. The patients will be randomized to receive either active drug or placebo. The anticipated time on study treatment is 6 weeks. The target sample size is <100 patients.

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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

18 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Sacramento, 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

  • Adult patients, 18 to 50 years of age
  • Fragile X Syndrome
  • IQ less than 75
  • Reliable caregiver

Exclusion criteria

  • Current psychosis or presumption of psychosis
  • History of suicidal behavior or considered a high suicidal risk
  • Severe self-injurious behavior
  • Any unstable medical condition other than Fragile X Syndrome (e.g. hypertension, diabetes)
  • Current seizure disorder

Treatment and study plan

Placebo (for RO4917523 ascending doses)

Drug

Placebo daily po

Placebo (for RO4917523 fixed dose)

Drug

Placebo daily po

RO4917523

Drug

RO4917523 ascending doses daily po

Primary outcomes

  1. Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters

    Time frame: AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43

Secondary outcomes

  1. Efficacy: Behavior and cognition assessments

    Time frame: Every 2 weeks throughout study

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study title

A Randomized, Double-blind, Placebo-controlled, Pharmacokinetic, Safety and Tolerability, and Exploratory Efficacy and Pharmacodynamic Effects Study of RO4917523 in Adult Patients With Fragile X Syndrome.

Important dates

Study start
2009
Primary completion
2011
Study completion
2011
First posted
Nov 18, 2009
Registry last updated
Nov 2, 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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