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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01750957
Other study ID # NP28571
Secondary ID 2011-004349-42
Status Completed
Phase Phase 2
First received December 11, 2012
Last updated August 9, 2016
Start date February 2013
Est. completion date April 2014

Study information

Verified date August 2016
Source Hoffmann-La Roche
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Completed
Enrollment 47
Est. completion date April 2014
Est. primary completion date April 2014
Accepts healthy volunteers No
Gender Both
Age group 5 Years to 13 Years
Eligibility 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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
Placebo
orally daily, 12 weeks
RO4917523
Dose A orally daily, 12 weeks
RO4917523
Dose B orally daily, 12 weeks

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Hoffmann-La Roche

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety: Incidence of adverse events 15 weeks No
Secondary Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior) 15 weeks No
Secondary Pharmacokinetics: Clearance (CL/F) up to Week 12 No
Secondary Pharmacokinetics: Volume of distribution at steady-state (Vss/F) up to Week 12 No
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