A Study of RO4917523 in 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: NCT01517698
Recruitment Status : Completed
First Posted : January 25, 2012
Last Update Posted : July 11, 2016
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief Summary:
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 or disease Intervention/treatment Phase
Fragile X Syndrome Drug: Placebo Drug: RO4917523 0.5 mg Drug: RO4917523 1.5 mg Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 185 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (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.
Study Start Date : May 2012
Actual Primary Completion Date : April 2014
Actual Study Completion Date : April 2014

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
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

Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Information from the National Library of Medicine

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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

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): NCT01517698

  Hide Study Locations
United States, Arizona
Phoenix, Arizona, United States, 85006
United States, California
Long Beach, California, United States, 90806
Sacramento, California, United States, 95817
United States, Colorado
Aurora, Colorado, United States, 80045
United States, District of Columbia
Washington, District of Columbia, United States, 20010
United States, Georgia
Decatur, Georgia, United States, 30033
United States, Illinois
Chicago, Illinois, United States, 60612
United States, Iowa
Iowa City, Iowa, United States, 52242-1083
United States, Kentucky
Louisville, Kentucky, United States, 40202
United States, Maryland
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Worcester, Massachusetts, United States, 01655
United States, New York
New York, New York, United States, 10029
Staten Island, New York, United States, 10314
United States, North Carolina
Durham, North Carolina, United States, 27710
United States, Ohio
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Media, Pennsylvania, United States, 19063
United States, Tennessee
Nashville, Tennessee, United States, 37203
United States, Texas
Houston, Texas, United States, 77030
San Antonio, Texas, United States, 78258
United States, Washington
Seattle, Washington, United States, 98145
Bahia Blanca, Argentina, 8000
Caba, Argentina, 1425
Caba, Argentina, C1126AAB
Canada, Ontario
Brampton, Ontario, Canada, L6Y 1M5
Canada, Quebec
Sherbrooke, Quebec, Canada, J1H 5N4
Santiago, Chile
Valparaiso, Chile, 2340000
Bordeaux, France, 33076
Bron, France, 69003
Dijon, France, 21079
Marseille, France, 13385
Montpellier, France, 34295
Paris, France, 75013
Tours, France, 37044
Aguascalientes, Mexico, 20030
Mexico, Mexico, 04530
Lima, Peru, Lima 33
Lima, Peru
San Cugat del Valles, Barcelona, Spain, 08195
San Sebastian, Guipuzcoa, Spain, 20009
Torremolinos, Malaga, Spain, 29620
El Palmar. Murcia, Murcia, Spain, 30120
Madrid, Spain, 28040
Madrid, Spain, 28046
Gothenburg, Sweden, 411 19
United Kingdom
Belfast, United Kingdom, BT12 6AB
Dundee, United Kingdom, DD1 9SY
Edinburgh, United Kingdom, EH10 5HF
Glasgow, United Kingdom, GS 8SJ
London, United Kingdom, W1W 7EJ
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Hoffmann-La Roche
Study Director: Clinical Trials Hoffmann-La Roche

Responsible Party: Hoffmann-La Roche Identifier: NCT01517698     History of Changes
Other Study ID Numbers: NP27936
First Posted: January 25, 2012    Key Record Dates
Last Update Posted: July 11, 2016
Last Verified: July 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