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A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

Primary Purpose

Fragile X Syndrome

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Placebo
RO4917523
RO4917523
Sponsored by
Hoffmann-La Roche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fragile X Syndrome

Eligibility Criteria

5 Years - 13 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Children and adolescents, 5 to 13 years of age
  • Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S

Exclusion Criteria:

  • Previous treatment with another mGlu5 receptor antagonist within the prior 3 months
  • Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study
  • Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
  • History of suicidal behavior
  • Other protocol defined inclusion/exclusion criteria may apply

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Experimental

Experimental

Arm Label

Placebo

RO4917523 Dose A

RO4917523 Dose B

Arm Description

Outcomes

Primary Outcome Measures

Safety: Incidence of adverse events

Secondary Outcome Measures

Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior)
Pharmacokinetics: Clearance (CL/F)
Pharmacokinetics: Volume of distribution at steady-state (Vss/F)

Full Information

First Posted
December 11, 2012
Last Updated
August 9, 2016
Sponsor
Hoffmann-La Roche
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1. Study Identification

Unique Protocol Identification Number
NCT01750957
Brief Title
A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
Official Title
A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
August 2016
Overall Recruitment Status
Completed
Study Start Date
February 2013 (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 randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 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
47 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Title
RO4917523 Dose A
Arm Type
Experimental
Arm Title
RO4917523 Dose B
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
orally daily, 12 weeks
Intervention Type
Drug
Intervention Name(s)
RO4917523
Intervention Description
Dose A orally daily, 12 weeks
Intervention Type
Drug
Intervention Name(s)
RO4917523
Intervention Description
Dose B orally daily, 12 weeks
Primary Outcome Measure Information:
Title
Safety: Incidence of adverse events
Time Frame
15 weeks
Secondary Outcome Measure Information:
Title
Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior)
Time Frame
15 weeks
Title
Pharmacokinetics: Clearance (CL/F)
Time Frame
up to Week 12
Title
Pharmacokinetics: Volume of distribution at steady-state (Vss/F)
Time Frame
up to Week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Children and adolescents, 5 to 13 years of age Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S Exclusion Criteria: Previous treatment with another mGlu5 receptor antagonist within the prior 3 months Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome History of suicidal behavior Other protocol defined inclusion/exclusion criteria may apply
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
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
San Diego
State/Province
California
ZIP/Postal Code
92108
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
20020
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
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
01605
Country
United States
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198-4160
Country
United States
City
New York
State/Province
New York
ZIP/Postal Code
10029
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
Greenwood
State/Province
South Carolina
ZIP/Postal Code
29646
Country
United States
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
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

12. IPD Sharing Statement

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A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

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