Back to results

A Study of RO4917523 in Patients With Fragile X Syndrome

Primary Purpose

Fragile X Syndrome

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Placebo

RO4917523 0.5 mg

RO4917523 1.5 mg

About this trial

This is an interventional treatment trial for Fragile X Syndrome

Eligibility Criteria

14 Years - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Arm Label

RO4917523 0.5 mg

RO4917523 1.5 mg

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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

Safety (incidence of adverse events)

Secondary Outcome Measures

Change in Social Responsiveness Scale (SRS)

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

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

Change in Aberrant Behavior Checklist total score

Change in Aberrant Behavior Checklist factor scores

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

Full Information

NCT ID

NCT01517698

First Posted

January 20, 2012

Last Updated

July 6, 2016

Sponsor

Hoffmann-La Roche

1. Study Identification

Unique Protocol Identification Number

NCT01517698

Brief Title

A Study of RO4917523 in Patients With Fragile X Syndrome

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 Type

Interventional

2. Study Status

Record Verification Date

July 2016

Overall Recruitment Status

Completed

Study Start Date

May 2012 (undefined)

Primary Completion Date

April 2014 (Actual)

Study Completion Date

April 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Hoffmann-La Roche

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Fragile X Syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

185 (Actual)

8. Arms, Groups, and Interventions

Arm Title

RO4917523 0.5 mg

Arm Type

Experimental

Arm Title

RO4917523 1.5 mg

Arm Type

Experimental

Arm Title

Placebo

Arm Type

Placebo Comparator

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

placebo to RO4917523 orally once a day for 12 weeks

Intervention Type

Drug

Intervention Name(s)

RO4917523 0.5 mg

Intervention Description

0.5 mg orally once a day for 12 weeks

Intervention Type

Drug

Intervention Name(s)

RO4917523 1.5 mg

Intervention Description

1.5 mg orally once a day for 12 weeks

Primary Outcome Measure Information:

Title

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

Time Frame

12 weeks

Title

Safety (incidence of adverse events)

Time Frame

12 weeks

Secondary Outcome Measure Information:

Title

Change in Social Responsiveness Scale (SRS)

Time Frame

12 weeks

Title

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

Time Frame

12 weeks

Title

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

Time Frame

12 weeks

Title

Change in Aberrant Behavior Checklist total score

Time Frame

12 weeks

Title

Change in Aberrant Behavior Checklist factor scores

Time Frame

12 weeks

Title

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

Time Frame

12 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

14 Years

Maximum Age & Unit of Time

50 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Trials

Organizational Affiliation

Hoffmann-La Roche

Official's Role

Study Director

Facility Information:

City

Phoenix

State/Province

Arizona

ZIP/Postal Code

85006

Country

United States

City

Long Beach

State/Province

California

ZIP/Postal Code

90806

Country

United States

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

City

Washington

State/Province

District of Columbia

ZIP/Postal Code

20010

Country

United States

City

Decatur

State/Province

Georgia

ZIP/Postal Code

30033

Country

United States

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60612

Country

United States

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242-1083

Country

United States

City

Louisville

State/Province

Kentucky

ZIP/Postal Code

40202

Country

United States

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01655

Country

United States

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

City

Staten Island

State/Province

New York

ZIP/Postal Code

10314

Country

United States

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27710

Country

United States

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

City

Media

State/Province

Pennsylvania

ZIP/Postal Code

19063

Country

United States

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37203

Country

United States

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

City

San Antonio

State/Province

Texas

ZIP/Postal Code

78258

Country

United States

City

Seattle

State/Province

Washington

ZIP/Postal Code

98145

Country

United States

City

Bahia Blanca

ZIP/Postal Code

8000

Country

Argentina

City

Caba

ZIP/Postal Code

1425

Country

Argentina

City

Caba

ZIP/Postal Code

C1126AAB

Country

Argentina

City

Brampton

State/Province

Ontario

ZIP/Postal Code

L6Y 1M5

Country

Canada

City

Sherbrooke

State/Province

Quebec

ZIP/Postal Code

J1H 5N4

Country

Canada

City

Santiago

Country

Chile

City

Valparaiso

ZIP/Postal Code

2340000

Country

Chile

City

Bordeaux

ZIP/Postal Code

33076

Country

France

City

Bron

ZIP/Postal Code

69003

Country

France

City

Dijon

ZIP/Postal Code

21079

Country

France

City

Marseille

ZIP/Postal Code

13385

Country

France

City

Montpellier

ZIP/Postal Code

34295

Country

France

City

Paris

ZIP/Postal Code

75013

Country

France

City

Tours

ZIP/Postal Code

37044

Country

France

City

Aguascalientes

ZIP/Postal Code

20030

Country

Mexico

City

Mexico

ZIP/Postal Code

04530

Country

Mexico

City

Lima

ZIP/Postal Code

Lima 33

Country

Peru

City

Lima

Country

Peru

City

San Cugat del Valles

State/Province

Barcelona

ZIP/Postal Code

08195

Country

Spain

City

San Sebastian

State/Province

Guipuzcoa

ZIP/Postal Code

20009

Country

Spain

City

Torremolinos

State/Province

Malaga

ZIP/Postal Code

29620

Country

Spain

City

El Palmar. Murcia

State/Province

Murcia

ZIP/Postal Code

30120

Country

Spain

City

Madrid

ZIP/Postal Code

28040

Country

Spain

City

Madrid

ZIP/Postal Code

28046

Country

Spain

City

Gothenburg

ZIP/Postal Code

411 19

Country

Sweden

City

Belfast

ZIP/Postal Code

BT12 6AB

Country

United Kingdom

City

Dundee

ZIP/Postal Code

DD1 9SY

Country

United Kingdom

City

Edinburgh

ZIP/Postal Code

EH10 5HF

Country

United Kingdom

City

Glasgow

ZIP/Postal Code

GS 8SJ

Country

United Kingdom

City

London

ZIP/Postal Code

W1W 7EJ

Country

United Kingdom

City

Manchester

ZIP/Postal Code

M13 9WL

Country

United Kingdom

12. IPD Sharing Statement

Learn more about this trial

A Study of RO4917523 in Patients With Fragile X Syndrome

We'll reach out to this number within 24 hrs