Trial record 2 of 12 for:    RO4917523

A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT01750957
First received: December 11, 2012
Last updated: April 7, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
Fragile X Syndrome
Drug: RO4917523
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: A RANDOMIZED, PARALLEL GROUP, DOUBLE-BLIND, PLACEBO-CONTROLLED, SAFETY AND EXPLORATORY EFFICACY AND PHARMACOKINETIC, STUDY OF RO4917523 IN PEDIATRIC PATIENTS WITH FRAGILE X SYNDROME

Resource links provided by NLM:


Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Safety: Incidence of adverse events [ Time Frame: 15 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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) [ Time Frame: 15 weeks ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Clearance (CL/F) [ Time Frame: up to Week 12 ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Volume of distribution at steady-state (Vss/F) [ Time Frame: up to Week 12 ] [ Designated as safety issue: No ]

Enrollment: 47
Study Start Date: February 2013
Estimated Study Completion Date: April 2014
Estimated Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RO4917523 Dose A Drug: RO4917523
Dose A orally daily, 12 weeks
Experimental: RO4917523 Dose B Drug: RO4917523
Dose B orally daily, 12 weeks
Placebo Comparator: Placebo Drug: Placebo
orally daily, 12 weeks

  Eligibility

Ages Eligible for Study:   5 Years to 13 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Children and adolescents, 5 to 13 years of age
  • Diagnosis of fragile X syndrome based on prior DNA testing confirming 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01750957

  Show 20 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

No publications provided

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01750957     History of Changes
Other Study ID Numbers: NP28571, 2011-004349-42
Study First Received: December 11, 2012
Last Updated: April 7, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Fragile X Syndrome
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System

ClinicalTrials.gov processed this record on April 21, 2014