NCT01750957

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
47

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Feb 2013

Shorter than P25 for phase_2

Geographic Reach
1 country

20 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 11, 2012

Completed
6 days until next milestone

First Posted

Study publicly available on registry

December 17, 2012

Completed
2 months until next milestone

Study Start

First participant enrolled

February 1, 2013

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2014

Completed
Last Updated

August 11, 2016

Status Verified

August 1, 2016

Enrollment Period

1.2 years

First QC Date

December 11, 2012

Last Update Submit

August 9, 2016

Conditions

Fragile X Syndrome

Outcome Measures

Primary Outcomes (1)

  • Safety: Incidence of adverse events

    15 weeks

Secondary Outcomes (3)

  • 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

  • Pharmacokinetics: Clearance (CL/F)

    up to Week 12

  • Pharmacokinetics: Volume of distribution at steady-state (Vss/F)

    up to Week 12

Study Arms (3)

Placebo

PLACEBO COMPARATOR
Drug: Placebo

RO4917523 Dose A

EXPERIMENTAL
Drug: RO4917523

RO4917523 Dose B

EXPERIMENTAL
Drug: RO4917523

Interventions

PlaceboDRUG

orally daily, 12 weeks

Placebo
RO4917523DRUG

Dose A orally daily, 12 weeks

RO4917523 Dose A

Eligibility Criteria

Age5 Years - 13 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Unknown Facility

Long Beach, California, 90806, United States

Location

Unknown Facility

Sacramento, California, 95817, United States

Location

Unknown Facility

San Diego, California, 92108, United States

Location

Unknown Facility

Aurora, Colorado, 80045, United States

Location

Unknown Facility

Washington D.C., District of Columbia, 20020, United States

Location

Unknown Facility

Decatur, Georgia, 30033, United States

Location

Unknown Facility

Chicago, Illinois, 60612, United States

Location

Unknown Facility

Iowa City, Iowa, 52242, United States

Location

Unknown Facility

Louisville, Kentucky, 40202, United States

Location

Unknown Facility

Baltimore, Maryland, 21205, United States

Location

Unknown Facility

Worcester, Massachusetts, 01605, United States

Location

Unknown Facility

Omaha, Nebraska, 68198-4160, United States

Location

Unknown Facility

New York, New York, 10029, United States

Location

Unknown Facility

Cincinnati, Ohio, 45229, United States

Location

Unknown Facility

Media, Pennsylvania, 19063, United States

Location

Unknown Facility

Greenwood, South Carolina, 29646, United States

Location

Unknown Facility

Nashville, Tennessee, 37212, United States

Location

Unknown Facility

Houston, Texas, 77030, United States

Location

Unknown Facility

San Antonio, Texas, 78258, United States

Location

Unknown Facility

Seattle, Washington, 98145, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine

Condition Hierarchy (Ancestors)

X-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesSex Chromosome DisordersChromosome DisordersCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, InbornGenetic Diseases, X-LinkedHeredodegenerative Disorders, Nervous System

Study Officials

  • Clinical Trials

    Hoffmann-La Roche

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 11, 2012

First Posted

December 17, 2012

Study Start

February 1, 2013

Primary Completion

April 1, 2014

Study Completion

April 1, 2014

Last Updated

August 11, 2016

Record last verified: 2016-08

Locations

US(20)