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An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01013480
Recruitment Status : Terminated (all active subjects rolled into 209FX303/NCT01013480)
First Posted : November 13, 2009
Last Update Posted : December 20, 2012
Information provided by (Responsible Party):
Seaside Therapeutics, Inc.

Brief Summary:
Study 22001, "A Double-Blind, Placebo-Controlled, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects with Fragile X Syndrome" currently is evaluating the efficacy of STX209 (R-baclofen) for management of typical problem behaviors, such as irritability and aggression, in subjects with FXS. This study (22002) will enter subjects who complete Study 22001 into a long-term, open-label study.The open-label extension protocol will provide necessary data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions more closely reflective of their general medical care.

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: Arbaclofen Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects With Fragile X Syndrome
Study Start Date : November 2009
Actual Primary Completion Date : December 2012
Actual Study Completion Date : December 2012

Resource links provided by the National Library of Medicine

Drug Information available for: Baclofen

Arm Intervention/treatment
Active Comparator: STX209 Drug: Arbaclofen
A flexible dose titration will be utilized during the first four weeks to define the optimal titrated dose (OTD) for each subject. Investigators will use clinical judgment to adjust doses to the OTD. The starting dose will be 1 mg BID. The dose may be increased every four to five days to 2 mg BID, 3 mg BID, 5 mg BID and then 10 mg BID
Other Names:
  • R-baclofen
  • STX209

Primary Outcome Measures :
  1. Irritability subscale of the Aberrant Behavior Checklist [ Time Frame: 1 year ]

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Have completed all scheduled visits in protocol 22001 and have shown they can adequately follow the protocol, with sufficient medical justification to continue on open-label treatment with STX209, as assessed by the principal investigator

Exclusion Criteria:

  • Subjects with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being.
  • The occurrence or continuation of any adverse event or condition during study 22001 that, in the opinion of the Investigator, should exclude the subject from participating in this open-label extension.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01013480

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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, 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, PA
Houston, Texas, United States, 77090
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98101
Sponsors and Collaborators
Seaside Therapeutics, Inc.
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Responsible Party: Seaside Therapeutics, Inc. Identifier: NCT01013480    
Other Study ID Numbers: 22002
First Posted: November 13, 2009    Key Record Dates
Last Update Posted: December 20, 2012
Last Verified: March 2011
Keywords provided by Seaside Therapeutics, Inc.:
Fragile X Syndrome
Additional relevant MeSH terms:
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Fragile X Syndrome
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
Muscle Relaxants, Central
Physiological Effects of Drugs
Neuromuscular Agents
Peripheral Nervous System Agents
GABA-B Receptor Agonists
GABA Agonists
GABA Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action