NCT01015430

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
40

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Nov 2009

Shorter than P25 for phase_2

Geographic Reach
1 country

5 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

Study Start

First participant enrolled

November 1, 2009

Completed
16 days until next milestone

First Submitted

Initial submission to the registry

November 17, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 18, 2009

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2011

Completed
Last Updated

November 2, 2016

Status Verified

November 1, 2016

Enrollment Period

1.2 years

First QC Date

November 17, 2009

Last Update Submit

November 1, 2016

Conditions

Outcome Measures

Primary Outcomes (1)

  • Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters

    AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43

Secondary Outcomes (1)

  • Efficacy: Behavior and cognition assessments

    Every 2 weeks throughout study

Study Arms (4)

Placebo (for RO4917523 ascending doses)

PLACEBO COMPARATOR
Drug: Placebo (for RO4917523 ascending doses)Drug: Placebo (for RO4917523 fixed dose)

Placebo (for RO4917523 fixed dose)

PLACEBO COMPARATOR
Drug: Placebo (for RO4917523 ascending doses)Drug: Placebo (for RO4917523 fixed dose)

RO4917523 ascending doses

EXPERIMENTAL
Drug: RO4917523

RO4917523 fixed dose

EXPERIMENTAL
Drug: RO4917523

Interventions

Placebo daily po

Placebo (for RO4917523 ascending doses)Placebo (for RO4917523 fixed dose)

Placebo daily po

Placebo (for RO4917523 ascending doses)Placebo (for RO4917523 fixed dose)

RO4917523 ascending doses daily po

RO4917523 ascending doses

Eligibility Criteria

Age18 Years - 50 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Adult patients, 18 to 50 years of age
  • Fragile X Syndrome
  • IQ less than 75
  • Reliable caregiver

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Unknown Facility

Sacramento, California, 95817, United States

Location

Unknown Facility

Decatur, Georgia, 30033, United States

Location

Unknown Facility

Chicago, Illinois, 60612, United States

Location

Unknown Facility

Indianapolis, Indiana, 46202, United States

Location

Unknown Facility

Nashville, Tennessee, 37203, 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

November 17, 2009

First Posted

November 18, 2009

Study Start

November 1, 2009

Primary Completion

January 1, 2011

Study Completion

January 1, 2011

Last Updated

November 2, 2016

Record last verified: 2016-11

Locations