A Study With RO4917523 in Patients With Fragile X Syndrome

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Hoffmann-La Roche

ClinicalTrials.gov Identifier:

NCT01015430

First received: November 17, 2009

Last updated: November 1, 2016

Last verified: November 2016

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: Placebo (for RO4917523 ascending doses) Drug: Placebo (for RO4917523 fixed dose) Drug: RO4917523	Phase 2


Study Type:	Interventional
Study Design:	Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double (Participant, 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: fragile X syndrome

MedlinePlus related topics: Fragile X Syndrome

Genetic and Rare Diseases Information Center resources: Fragile X Syndrome X-linked Mental Retardation and Macro-orchidism

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 ]

Secondary Outcome Measures:

Efficacy: Behavior and cognition assessments [ Time Frame: Every 2 weeks throughout study ]


Enrollment:	40
Study Start Date:	November 2009
Study Completion Date:	January 2011
Primary Completion Date:	January 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Placebo Comparator: Placebo (for RO4917523 ascending doses)	Drug: Placebo (for RO4917523 ascending doses) Placebo daily po Drug: Placebo (for RO4917523 fixed dose) Placebo daily po
Placebo Comparator: Placebo (for RO4917523 fixed dose)	Drug: Placebo (for RO4917523 ascending doses) Placebo daily po Drug: Placebo (for RO4917523 fixed dose) Placebo daily po
Experimental: RO4917523 ascending doses	Drug: RO4917523 RO4917523 ascending doses daily po
Experimental: RO4917523 fixed dose	Drug: RO4917523 RO4917523 fixed dose daily po

Eligibility


Ages Eligible for Study:	18 Years to 50 Years (Adult)
Sexes Eligible for Study:	All
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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


Responsible Party:	Hoffmann-La Roche
ClinicalTrials.gov Identifier:	NCT01015430 History of Changes
Other Study ID Numbers:	NP22578
Study First Received:	November 17, 2009
Last Updated:	November 1, 2016

Additional relevant MeSH terms:


Syndrome Fragile X Syndrome Disease Pathologic Processes Mental Retardation, X-Linked Intellectual Disability 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 August 18, 2017

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