A Study With RO4917523 in Patients With Fragile X Syndrome - Full Text View

Sponsor:	Hoffmann-La Roche
Information provided by:	Hoffmann-La Roche
ClinicalTrials.gov Identifier:	NCT01015430

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 II

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:

Genetics Home Reference related topics: 17q21.31 microdeletion syndrome fragile X syndrome MECP2 duplication syndrome PPM-X syndrome tetrasomy 18p

MedlinePlus related topics: Fragile X Syndrome

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 February 09, 2012