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

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

185

Phase

  • 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

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
Intervention / Treatment
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

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

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

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

May 1, 2012

Primary Completion (ACTUAL)

April 1, 2014

Study Completion (ACTUAL)

April 1, 2014

Study Registration Dates

First Submitted

January 20, 2012

First Submitted That Met QC Criteria

January 24, 2012

First Posted (ESTIMATE)

January 25, 2012

Study Record Updates

Last Update Posted (ESTIMATE)

July 11, 2016

Last Update Submitted That Met QC Criteria

July 6, 2016

Last Verified

July 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID 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.

Clinical Trials on Fragile X Syndrome

Clinical Trials on RO4917523 0.5 mg

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Poland

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe