A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

Roche

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome

Treatments: Drug: RO4917523; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01750957

NP28571

2011-004349-42 (EudraCT Number)

Details and patient eligibility

About

This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks.

Enrollment: 47 patients

Sex: All

Ages: 5 to 13 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Children and adolescents, 5 to 13 years of age
• Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S

Exclusion criteria

• Previous treatment with another mGlu5 receptor antagonist within the prior 3 months
• Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study
• Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
• History of suicidal behavior
• Other protocol defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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47 participants in 3 patient groups, including a placebo group

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo

RO4917523 Dose A

Experimental group

Treatment:

Drug: RO4917523

Drug: RO4917523

RO4917523 Dose B

Experimental group

Treatment:

Drug: RO4917523

Drug: RO4917523