A Study With RO4917523 in Patients With Fragile X Syndrome

Phase 2

Completed

• Conditions: Fragile X Syndrome
• Interventions: Drug: Placebo (for RO4917523 ascending doses); Drug: RO4917523; Drug: Placebo (for RO4917523 fixed dose)

• Registration Number: NCT01015430
• Lead Sponsor: Hoffmann-La Roche

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-11
• Study First Submitted: 2009-11-17
• Study First Submitted QC: 2009-11-17
• Study First Posted: 2009-11-18
• Primary Completion: 2011-01
• Study Completion: 2011-01
• Disposition First Submitted: 2016-07-06
• Disposition First Submitted QC: 2016-07-06
• Disposition First Posted: 2016-07-11
• Status Verified: 2016-11
• Last Update Submitted: 2016-11-01
• Last Update Posted: 2016-11-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Adult patients, 18 to 50 years of age
• Fragile X Syndrome
• IQ less than 75
• Reliable caregiver

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo (for RO4917523 ascending doses)	Placebo (for RO4917523 ascending doses)	-
Placebo (for RO4917523 ascending doses)	Placebo (for RO4917523 fixed dose)	-
RO4917523 fixed dose	RO4917523	-
Placebo (for RO4917523 fixed dose)	Placebo (for RO4917523 ascending doses)	-
Placebo (for RO4917523 fixed dose)	Placebo (for RO4917523 fixed dose)	-
RO4917523 ascending doses	RO4917523	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Efficacy: Behavior and cognition assessments	Every 2 weeks throughout study