Clinical trial NCT01517698: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2012-01-25
Enrollment: 185
Sex: ALL
Minimum age (years): 14
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Change in Anxiety Depression and Mood Scale (ADAMS) total score
timeFrame: 12 weeks
measure: Safety (incidence of adverse events)
timeFrame: 12 weeks
Secondary outcomes:
measure: Change in Social Responsiveness Scale (SRS)
timeFrame: 12 weeks
measure: Change in Clinical Global Impressions Scale - Improvement (CGI-I)
timeFrame: 12 weeks
measure: Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S)
timeFrame: 12 weeks
measure: Change in Aberrant Behavior Checklist total score
timeFrame: 12 weeks
measure: Change in Aberrant Behavior Checklist factor scores
timeFrame: 12 weeks
measure: Change in Anxiety Depression and Mood Scale (ADAMS) factor scores
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
state: Arizona
city: Long Beach
country: United States
state: California
city: Sacramento
country: United States
state: California
city: Aurora
country: United States
state: Colorado
city: Washington
country: United States
state: District of Columbia
city: Decatur
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Iowa City
country: United States
state: Iowa
city: Louisville
country: United States
state: Kentucky
city: Baltimore
country: United States
state: Maryland
city: Worcester
country: United States
state: Massachusetts
city: New York
country: United States
state: New York
city: Staten Island
country: United States
state: New York
city: Durham
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Media
country: United States
state: Pennsylvania
city: Nashville
country: United States
state: Tennessee
city: Houston
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
city: Bahia Blanca
country: Argentina
city: Caba
country: Argentina
city: Caba
country: Argentina
city: Brampton
country: Canada
state: Ontario
city: Sherbrooke
country: Canada
state: Quebec
city: Santiago
country: Chile
city: Valparaiso
country: Chile
city: Bordeaux
country: France
city: Bron
country: France
city: Dijon
country: France
city: Marseille
country: France
city: Montpellier
country: France
city: Paris
country: France
city: Tours
country: France
city: Aguascalientes
country: Mexico
city: Mexico
country: Mexico
city: Lima
country: Peru
city: Lima
country: Peru
city: San Cugat del Valles
country: Spain
state: Barcelona
city: San Sebastian
country: Spain
state: Guipuzcoa
city: Torremolinos
country: Spain
state: Malaga
city: El Palmar. Murcia
country: Spain
state: Murcia
city: Madrid
country: Spain
city: Madrid
country: Spain
city: Gothenburg
country: Sweden
city: Belfast
country: United Kingdom
city: Dundee
country: United Kingdom
city: Edinburgh
country: United Kingdom
city: Glasgow
country: United Kingdom
city: London
country: United Kingdom
city: Manchester
country: United Kingdom
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: