Back to resultsA Study With RO4917523 in Patients With Fragile X Syndrome
Primary Purpose
Fragile X Syndrome
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Placebo (for RO4917523 ascending doses)
Placebo (for RO4917523 fixed dose)
RO4917523
RO4917523
Sponsored by
About this trial
This is an interventional treatment trial for Fragile X Syndrome
Eligibility Criteria
Inclusion Criteria:
- Adult patients, 18 to 50 years of age
- Fragile X Syndrome
- IQ less than 75
- Reliable caregiver
Exclusion Criteria:
- Current psychosis or presumption of psychosis
- History of suicidal behavior or considered a high suicidal risk
- Severe self-injurious behavior
- Any unstable medical condition other than Fragile X Syndrome (e.g. hypertension, diabetes)
- Current seizure disorder
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Placebo Comparator
Placebo Comparator
Experimental
Experimental
Arm Label
Placebo (for RO4917523 ascending doses)
Placebo (for RO4917523 fixed dose)
RO4917523 ascending doses
RO4917523 fixed dose
Arm Description
Outcomes
Primary Outcome Measures
Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters
Secondary Outcome Measures
Efficacy: Behavior and cognition assessments
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01015430
Brief Title
A Study With RO4917523 in Patients With Fragile X Syndrome
Official Title
A Randomized, Double-blind, Placebo-controlled, Pharmacokinetic, Safety and Tolerability, and Exploratory Efficacy and Pharmacodynamic Effects Study of RO4917523 in Adult Patients With Fragile X Syndrome.
Study Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
November 2009 (undefined)
Primary Completion Date
January 2011 (Actual)
Study Completion Date
January 2011 (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 multiple ascending dose study will evaluate the safety and tolerability, pharmacokinetics and efficacy of RO4917523 in patients with Fragile X Syndrome. The patients will be randomized to receive either active drug or placebo. The anticipated time on study treatment is 6 weeks. The target sample size is <100 patients.
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
40 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo (for RO4917523 ascending doses)
Arm Type
Placebo Comparator
Arm Title
Placebo (for RO4917523 fixed dose)
Arm Type
Placebo Comparator
Arm Title
RO4917523 ascending doses
Arm Type
Experimental
Arm Title
RO4917523 fixed dose
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Placebo (for RO4917523 ascending doses)
Intervention Description
Placebo daily po
Intervention Type
Drug
Intervention Name(s)
Placebo (for RO4917523 fixed dose)
Intervention Description
Placebo daily po
Intervention Type
Drug
Intervention Name(s)
RO4917523
Intervention Description
RO4917523 ascending doses daily po
Intervention Type
Drug
Intervention Name(s)
RO4917523
Intervention Description
RO4917523 fixed dose daily po
Primary Outcome Measure Information:
Title
Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters
Time Frame
AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43
Secondary Outcome Measure Information:
Title
Efficacy: Behavior and cognition assessments
Time Frame
Every 2 weeks throughout study
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adult patients, 18 to 50 years of age
Fragile X Syndrome
IQ less than 75
Reliable caregiver
Exclusion Criteria:
Current psychosis or presumption of psychosis
History of suicidal behavior or considered a high suicidal risk
Severe self-injurious behavior
Any unstable medical condition other than Fragile X Syndrome (e.g. hypertension, diabetes)
Current seizure disorder
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
Sacramento
State/Province
California
ZIP/Postal Code
95817
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
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
12. IPD Sharing Statement
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A Study With RO4917523 in Patients With Fragile X Syndrome
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