A Study With RO4917523 in Patients With Fragile X Syndrome

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.

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01015430

Enrollment

Registered

2009-11-18

Start date

2009-11-30

Completion date

2011-01-31

Last updated

2016-11-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fragile X Syndrome

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.

Interventions

DRUGPlacebo (for RO4917523 ascending doses)

Placebo daily po

DRUGPlacebo (for RO4917523 fixed dose)

Placebo daily po

DRUGRO4917523

RO4917523 ascending doses daily po

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

ALL

Age

18 Years to 50 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters	AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43

Secondary

Measure	Time frame
Efficacy: Behavior and cognition assessments	Every 2 weeks throughout study

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 19, 2026