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.
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Hoffmann-La Roche
- Enrollment
- 40
- Primary Endpoint
- Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Eligibility Criteria
- Ages
- 18 Years to 50 Years (Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Placebo (for RO4917523 ascending doses)
Intervention: Placebo (for RO4917523 ascending doses) (Drug)
Placebo (for RO4917523 ascending doses)
Intervention: Placebo (for RO4917523 fixed dose) (Drug)
Placebo (for RO4917523 fixed dose)
Intervention: Placebo (for RO4917523 ascending doses) (Drug)
Placebo (for RO4917523 fixed dose)
Intervention: Placebo (for RO4917523 fixed dose) (Drug)
RO4917523 ascending doses
Intervention: RO4917523 (Drug)
RO4917523 fixed dose
Intervention: RO4917523 (Drug)
Outcomes
Primary Outcomes
Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters
Time Frame: AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43
Secondary Outcomes
- Efficacy: Behavior and cognition assessments(Every 2 weeks throughout study)