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A Study of RO4917523 in Patients With Fragile X Syndrome

This study has been completed.
Information provided by (Responsible Party):
Hoffmann-La Roche Identifier:
First received: January 20, 2012
Last updated: July 6, 2016
Last verified: July 2016
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.

Condition Intervention Phase
Fragile X Syndrome
Drug: Placebo
Drug: RO4917523 0.5 mg
Drug: RO4917523 1.5 mg
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome.

Resource links provided by NLM:

Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Change in Anxiety Depression and Mood Scale (ADAMS) total score [ Time Frame: 12 weeks ]
  • Safety (incidence of adverse events) [ Time Frame: 12 weeks ]

Secondary Outcome Measures:
  • Change in Social Responsiveness Scale (SRS) [ Time Frame: 12 weeks ]
  • Change in Clinical Global Impressions Scale - Improvement (CGI-I) [ Time Frame: 12 weeks ]
  • Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S) [ Time Frame: 12 weeks ]
  • Change in Aberrant Behavior Checklist total score [ Time Frame: 12 weeks ]
  • Change in Aberrant Behavior Checklist factor scores [ Time Frame: 12 weeks ]
  • Change in Anxiety Depression and Mood Scale (ADAMS) factor scores [ Time Frame: 12 weeks ]

Enrollment: 185
Study Start Date: May 2012
Study Completion Date: April 2014
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RO4917523 0.5 mg Drug: RO4917523 0.5 mg
0.5 mg orally once a day for 12 weeks
Experimental: RO4917523 1.5 mg Drug: RO4917523 1.5 mg
1.5 mg orally once a day for 12 weeks
Placebo Comparator: Placebo Drug: Placebo
placebo to RO4917523 orally once a day for 12 weeks


Ages Eligible for Study:   14 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01517698

  Show 51 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche Identifier: NCT01517698     History of Changes
Other Study ID Numbers: NP27936
Study First Received: January 20, 2012
Last Updated: July 6, 2016

Additional relevant MeSH terms:
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 processed this record on May 22, 2017