A Study With RO4917523 in Patients With Fragile X Syndrome

Roche

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome

Treatments: Drug: Placebo (for RO4917523 ascending doses); Drug: Placebo (for RO4917523 fixed dose); Drug: RO4917523

Study type: Interventional

Funder types: Industry

Identifiers

NCT01015430

NP22578

Details and patient eligibility

About

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.

Enrollment: 40 patients

Sex: All

Ages: 18 to 50 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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40 participants in 4 patient groups, including a placebo group

Placebo (for RO4917523 ascending doses)

Placebo Comparator group

Treatment:

Drug: Placebo (for RO4917523 ascending doses)

Drug: Placebo (for RO4917523 fixed dose)

Placebo (for RO4917523 fixed dose)

Placebo Comparator group

Treatment:

Drug: Placebo (for RO4917523 ascending doses)

Drug: Placebo (for RO4917523 fixed dose)

RO4917523 ascending doses

Experimental group

Treatment:

Drug: RO4917523

Drug: RO4917523

RO4917523 fixed dose

Experimental group

Treatment:

Drug: RO4917523

Drug: RO4917523