A Study With RO4917523 in Patients With Fragile X Syndrome

Hoffmann-La Roche40 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Fragile X Syndrome

Interventions

Placebo (for RO4917523 ascending doses)DRUG

Placebo daily po

Placebo (for RO4917523 fixed dose)DRUG

Placebo daily po

RO4917523DRUG

RO4917523 ascending doses daily po

Eligibility

Sex: ALLMin age: 18 YearsMax age: 50 Years

Medical Language ↔ Plain English

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

Locations (5)

Unnamed facility

Sacramento, California, United States

Unnamed facility

Decatur, Georgia, United States

Unnamed facility

Chicago, Illinois, United States

Unnamed facility

Indianapolis, Indiana, United States

Unnamed facility

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

Time frame: Every 2 weeks throughout study

Data from ClinicalTrials.gov

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