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A Study With RO4917523 in Patients With Fragile X Syndrome

This study has been completed.
Sponsor:
Information provided by:
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT01015430
First received: November 17, 2009
Last updated: December 5, 2011
Last verified: December 2011
History of Changes
  Purpose

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.


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, Double-blind, Placebo-controlled, Pharmacokinetic, Safety and Tolerability, and Exploratory Efficacy and Pharmacodynamic Effects Study of RO4917523 in Adult Patients With Fragile X Syndrome.

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters [ Time Frame: AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Efficacy: Behavior and cognition assessments [ Time Frame: Every 2 weeks throughout study ] [ Designated as safety issue: No ]

Enrollment: 40
Study Start Date: November 2009
Study Completion Date: September 2011
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: RO4917523
RO4917523 ascending doses daily po
Placebo Comparator: 2 Drug: Placebo
Placebo daily po
Experimental: 3 Drug: RO4917523
RO4917523 fixed dose daily po
Placebo Comparator: 4 Drug: Placebo
Placebo daily po

  Eligibility

Ages Eligible for Study:   18 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01015430

Locations
United States, California
Sacramento, California, United States, 95817
United States, Georgia
Decatur, Georgia, United States, 30033
United States, Illinois
Chicago, Illinois, United States, 60612
United States, Indiana
Indianapolis, Indiana, United States, 46202
United States, Tennessee
Nashville, Tennessee, United States, 37203
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

No publications provided

Responsible Party: Disclosures Group, Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01015430     History of Changes
Other Study ID Numbers: NP22578
Study First Received: November 17, 2009
Last Updated: December 5, 2011
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 May 16, 2013