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NCT Number: NCT01517698

A Study of RO4917523 in Patients With Fragile X Syndrome

This multi-center, randomized, double-blind, placebo-controlled, parallel-group study will investigate the efficacy and safety of RO4917523 in adolescent and adult patients with fragile X syndrome. Patients will be randomized to receive oral doses of 0.5 mg or 1.5 mg of RO4917523, or matching placebo once daily. The anticipated time on study treatment is 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

14 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Bahía Blanca, Argentina

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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 and adolescent patients, 14-50 years of age
  • Diagnosis of fragile X syndrome with a confirmed fragile X mental retardation 1 (FMR1) full mutation and qualifying scores on the Aberrant Behavior Checklist (ABC) and CGI-S
  • Patients must agree to either remain completely abstinent or to use two effective contraceptive methods during and 3 weeks after the study

Exclusion criteria

  • Previous treatment with another metabotropic glutamate (mGLU) receptor antagonist within 18 months or with RO4917523
  • Participation in a clinical trial involving an investigational (unapproved) drug within 3 months or 5 times the half-life (whichever is longer) before start of this study
  • Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
  • History of suicidal behavior

Treatment and study plan

Placebo

Drug

placebo to RO4917523 orally once a day for 12 weeks

RO4917523 0.5 mg

Drug

0.5 mg orally once a day for 12 weeks

RO4917523 1.5 mg

Drug

1.5 mg orally once a day for 12 weeks

Primary outcomes

  1. Change in Anxiety Depression and Mood Scale (ADAMS) total score

    Time frame: 12 weeks

  2. Safety (incidence of adverse events)

    Time frame: 12 weeks

Secondary outcomes

  1. Change in Social Responsiveness Scale (SRS)

    Time frame: 12 weeks

  2. Change in Clinical Global Impressions Scale - Improvement (CGI-I)

    Time frame: 12 weeks

  3. Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S)

    Time frame: 12 weeks

  4. Change in Aberrant Behavior Checklist total score

    Time frame: 12 weeks

  5. Change in Aberrant Behavior Checklist factor scores

    Time frame: 12 weeks

  6. Change in Anxiety Depression and Mood Scale (ADAMS) factor scores

    Time frame: 12 weeks

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study title

A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome.

Important dates

Study start
2012
Primary completion
2014
Study completion
2014
First posted
Jan 25, 2012
Registry last updated
Jul 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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