Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome - Full Text View

Sponsor:	Seaside Therapeutics, Inc.
Information provided by:	Seaside Therapeutics, Inc.
ClinicalTrials.gov Identifier:	NCT00788073

Purpose

The study objective is to explore the efficacy, safety and tolerability of STX209 for treatment of irritability in subjects with FSX. We hypothesize that STX209 will improve irritability and other typical problem behaviors associated with fragile X syndrome. We also hypothesize that STX209 will be safe and well tolerated.

Condition	Intervention	Phase
Fragile X Syndrome	Drug: Arbaclofen Drug: Placebo	Phase II

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	A Double-Blind, Placebo-Controlled, Crossover, Flexible-Dose Evaluation of the Efficacy, Safety and Tolerability of STX209 in the Treatment of Irritability in Subjects 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 Seaside Therapeutics, Inc.:

Primary Outcome Measures:

Adverse events [ Time Frame: during the study ] [ Designated as safety issue: Yes ]
Aberrant Behavior Checklist Irritability Subscore [ Time Frame: After 4 weeks of treatment ] [ Designated as safety issue: No ]

Estimated Enrollment:	60
Study Start Date:	November 2008
Estimated Study Completion Date:	September 2010
Estimated Primary Completion Date:	September 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: 1 STX209	Drug: Arbaclofen Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks Other Name: STX209
Placebo Comparator: 2 Placebo	Drug: Placebo Capsule, Oral,bid to tid, 4 weeks

Eligibility

Ages Eligible for Study:	6 Years to 40 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male or female subjects 12 to 40 years of age eventually expanding to 6 years of age
Molecular documentation of the fragile X mutation.
Clinical Global Impression - Severity (CGI-S) rating for problem behavior of moderate or higher at screening and at Visit 1
An Aberrant Behavior Checklist (ABC-C) Irritability Subscale score >12 and at least 3 items on the Irritability Subscale rated at least moderate or above.
Current treatment with no more than three psychoactive medications, including anti-epileptics.
Current pharmacological treatment regimen has been stable for at least 4 weeks.

Exclusion Criteria:

Subjects with a history of seizure disorder who are not currently receiving treatment with antiepileptics.
Subjects with any condition, including alcohol and drug abuse, which might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. This includes, but is not limited to impairment of renal function, evidence or history of malignancy or any significant hematological, endocrine, cardiovascular, respiratory, hepatic, or gastrointestinal disease.
Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
Subjects who are currently receiving treatment with racemic baclofen.
Subjects currently treated with vigabatrin or tiagabine.
Subjects taking another investigational drug currently or within the last 30 days.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00788073

Locations

United States, Arizona
Southwest Autism Research & Resource Center
Phoenix, Arizona, United States, 85006
United States, California
University of California-Los Angeles Neuropsychiatric Institute
Los Angeles, California, United States, 90024
M.I.N.D. Institute
Sacramento, California, United States, 95817
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, New York
NYS Institute for Basic Research in Developmental Disabilities
Staten Island, New York, United States, 10314
United States, North Carolina
University of North Carolina Neurosciences Hospital
Chapel Hill, North Carolina, United States, 27514
United States, Pennsylvania
Suburban Research Associates
Media, Pennsylvania, United States, 19063
United States, Tennessee
Vanderbilt Kennedy Center
Nashville, Tennessee, United States, 37203
United States, Texas
Red Oaks Psychiatry Associates, P.A.
Houston, Texas, United States, 77090
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98101

Sponsors and Collaborators

Seaside Therapeutics, Inc.

Investigators

Principal Investigator:	Elizabeth Berry-Kravis, MD, PhD	Rush University Medical Center
Principal Investigator:	Randi Hagerman, MD	M.I.N.D. Institute
Principal Investigator:	Craig Erikson, MD	Riley Hospital for Children
Principal Investigator:	Bryan King, MD, PhD	Seattle Children's Hospital
Principal Investigator:	James McCracken, MD	University of California, Los Angeles
Principal Investigator:	Jonathan Picker, MBChB, PhD	Children's Hospital Boston
Principal Investigator:	Linmarie Sikich, MD	University of North Carolina Neurosciences Hospital
Principal Investigator:	Jeremy Veenstra-VanderWeele, MD	Vanderbilt Kennedy Center
Principal Investigator:	Ted Brown, MD, PhD	NYS institute for Basic Research in Developmental Disabilities
Principal Investigator:	Lawrence Ginsberg, MD	Red Oaks Psychiatry Associates, PA
Principal Investigator:	Shivkumar Hatti, MD	Suburban Research Associates
Principal Investigator:	Raun Melmed, MD	Southwest Autism Research & Resource Center

More Information

No publications provided

Responsible Party:	Study Director, Seaside Therapeutics, LLC
ClinicalTrials.gov Identifier:	NCT00788073 History of Changes
Other Study ID Numbers:	22001
Study First Received:	November 7, 2008
Last Updated:	March 28, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Seaside Therapeutics, Inc.:

fragile X syndrome
irritability
behavior problems

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