A Study of RO4917523 in Patients With Fragile X Syndrome

July 6, 2016 updated by: Hoffmann-La Roche

A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety 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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
185

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Bahia Blanca, Argentina, 8000
      • Caba, Argentina, 1425
      • Caba, Argentina, C1126AAB
  • Canada
    • Ontario
      • Brampton, Ontario, Canada, L6Y 1M5
    • Quebec
      • Sherbrooke, Quebec, Canada, J1H 5N4
  • Chile
      • Santiago, Chile
      • Valparaiso, Chile, 2340000
  • France
      • Bordeaux, France, 33076
      • Bron, France, 69003
      • Dijon, France, 21079
      • Marseille, France, 13385
      • Montpellier, France, 34295
      • Paris, France, 75013
      • Tours, France, 37044
  • Mexico
      • Aguascalientes, Mexico, 20030
      • Mexico, Mexico, 04530
  • Peru
      • Lima, Peru
      • Lima, Peru, Lima 33
  • Spain
      • Madrid, Spain, 28040
      • Madrid, Spain, 28046
    • Barcelona
      • San Cugat del Valles, Barcelona, Spain, 08195
    • Guipuzcoa
      • San Sebastian, Guipuzcoa, Spain, 20009
    • Malaga
      • Torremolinos, Malaga, Spain, 29620
    • Murcia
      • El Palmar. Murcia, Murcia, Spain, 30120
  • Sweden
      • 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
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85006
    • California
      • Long Beach, California, United States, 90806
      • Sacramento, California, United States, 95817
    • Colorado
      • Aurora, Colorado, United States, 80045
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
    • Georgia
      • Decatur, Georgia, United States, 30033
    • Illinois
      • Chicago, Illinois, United States, 60612
    • Iowa
      • Iowa City, Iowa, United States, 52242-1083
    • Kentucky
      • Louisville, Kentucky, United States, 40202
    • Maryland
      • Baltimore, Maryland, United States, 21205
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
    • New York
      • New York, New York, United States, 10029
      • Staten Island, New York, United States, 10314
    • North Carolina
      • Durham, North Carolina, United States, 27710
    • Ohio
      • Cincinnati, Ohio, United States, 45229
    • Pennsylvania
      • Media, Pennsylvania, United States, 19063
    • Tennessee
      • Nashville, Tennessee, United States, 37203
    • Texas
      • Houston, Texas, United States, 77030
      • San Antonio, Texas, United States, 78258
    • Washington
      • Seattle, Washington, United States, 98145

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years to 50 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: DOUBLE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
PLACEBO_COMPARATOR: Placebo
Drug: Placebo
placebo to RO4917523 orally once a day for 12 weeks
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change in Anxiety Depression and Mood Scale (ADAMS) total score
Time Frame: 12 weeks
12 weeks
Safety (incidence of adverse events)
Time Frame: 12 weeks
12 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Change in Social Responsiveness Scale (SRS)
Time Frame: 12 weeks
12 weeks
Change in Clinical Global Impressions Scale - Improvement (CGI-I)
Time Frame: 12 weeks
12 weeks
Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S)
Time Frame: 12 weeks
12 weeks
Change in Aberrant Behavior Checklist total score
Time Frame: 12 weeks
12 weeks
Change in Aberrant Behavior Checklist factor scores
Time Frame: 12 weeks
12 weeks
Change in Anxiety Depression and Mood Scale (ADAMS) factor scores
Time Frame: 12 weeks
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hoffmann-La Roche

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2012

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 1, 2014

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 1, 2014

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 20, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 24, 2012

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 25, 2012

Study Record Updates

Last Update Posted (ESTIMATE)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 11, 2016

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 6, 2016

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NP27936

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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