- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01555333
An Open Label Extension Study in Subjects With Fragile X Syndrome (209FX303)
July 30, 2013 updated by: Seaside Therapeutics, Inc.
An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics in Subject With Fragile X Syndrome
This study will enroll subjects who have completed Protocols 209FX301, 209FX302, or are currently participating in Protocol 2202 into a long-term study in which all subjects will receive active drug (arbaclofen).
Study Overview
Detailed Description
Three studies sponsored by Seaside Therapeutics, Inc., are currently evaluating the efficacy of STX209 for management of typical problem behaviors in subjects with FXS.
These are Study 209FX301, "A Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Adolescents and Adults with Fragile X Syndrome;" Study 209FX302, "A Randomized, Double-Blind, Placebo-Controlled, Fixed- Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Children with Fragile X Syndrome;" and Study 22002, "An Open-Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects with Fragile X Syndrome."
This study will enroll subjects who have completed Protocols 209FX301, 209FX302, or are currently participating in Protocol 22002 into a long-term, open-label study.
The open-label extension protocol will provide data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions reflective of their typical medical care rather than in their previously completed study.
Study Type
Interventional
Enrollment (Actual)
357
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Arizona
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Phoenix, Arizona, United States, 85006
- Seaside Therapeutics Site #16
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California
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Long Beach, California, United States, 90806
- Seaside Therapeutics Site #07
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Sacramento, California, United States, 95817
- Seaside Therapeutics Site #10
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Colorado
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Aurora, Colorado, United States, 80045
- Seaside Therapeutics Site #17
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Florida
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Miami, Florida, United States, 33136
- Seaside Therapeutics Site #01
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Orange City, Florida, United States, 32763
- Seaside Therapeutics Site #14
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Georgia
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Decatur, Georgia, United States, 30033
- Seaside Therapeutics Site #20
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Illinois
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Chicago, Illinois, United States, 60612
- Seaside Therapeutics Site #02
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Kansas
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Kansas City, Kansas, United States, 66160
- Seaside Therapeutics Site #23
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Maryland
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Baltimore, Maryland, United States, 21205
- Seaside Therapeutics Site #12
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Massachusetts
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Worcester, Massachusetts, United States, 01605
- Seaside Therapeutics Site #08
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Missouri
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Columbia, Missouri, United States, 65211
- Seaside Therapeutics Site #03
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New York
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New York, New York, United States, 10029
- Seaside Therapeutics Site #22
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Staten Island, New York, United States, 10314
- Seaside Therapeutics Site #04
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North Carolina
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Chapel Hill, North Carolina, United States, 27514
- Seaside Therapeutics Site #24
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Durham, North Carolina, United States, 27710
- Seaside Therapeutics Site #21
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Ohio
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Akron, Ohio, United States, 44308
- Seaside Therapeutics Site #05
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Oklahoma
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Oklahoma City, Oklahoma, United States, 73117
- Seaside Therapeutics Site #15
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Pennsylvania
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Media, Pennsylvania, United States, 19063
- Seaside Therapeutics Site #11
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Tennessee
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Nashville, Tennessee, United States, 37212
- Seaside Therapeutics Site #19
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Texas
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Houston, Texas, United States, 77090
- Seaside Therapeutics Site #25
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San Antonio, Texas, United States, 78258
- Seaside Therapeutics Site #18
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Washington
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Seattle, Washington, United States, 98121
- Seaside Therapeutics Site #13
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
5 years to 50 years (ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Successfully completed all scheduled visits of the previous protocol ( 22002, 209FX301, or 209FX302).
- A parent,LAR, or caregiver must be willing and able to accompany the subject to all study visits, participate in phone calls, complete study assessments, administer study medication, and report the subject's condition and medication use to site staff members.
- Prior to the conduct of any study-specific procedures, the subject must provide written informed consent to participate in the study ( if developmentally appropriate) or verbal assent and the parent/caregiver/LAR must provide written informed consent. If the caregiver attending the clinic visits is not the parents, caregiver, or LAR, written consent must also be obtained for the caregiver's participation in the study.
- Current treatment with no more than 3 psychoactive medications, including anti-epileptics, unless the Medical Monitor is consulted.
- Subjects with a history of seizure disorder must have been seizure free for 6 months and be taking anti-epileptics, or seizure free for 3 years if not receiving anti-epileptic treatment. If currently receiving treatment with anti-epileptics, serum concentration levels must be tested and be in therapeutic range.
- Negative pregnancy test for females of childbearing potential or be using a medically acceptable form of birth control.
Exclusion Criteria
- Subjects with any condition that 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 are currently engaged in illicit drug or alcohol abuse.
- Subjects who had a serious adverse event (SAE) while taking STX209 during their previous protocol (22002,209FX301,309FX302)that the Investigator considered related to STX209, unless approval from the Medical Monitor is obtained.
- The occurrence or continuation of any AE or condition during Studies 22002, 209FX301, or 209FX302 that, in the opinion of the Investigator, should exclude this subject from participating in the open-label extension.
- Subjects taking another investigational drug, other than STX209, currently or within 30 days of Visit 1. Subject must not take any investigational drugs during this study.
- Subjects who, in the Investigator's opinion, might not be suitable for the study.
- Subjects treated with vigabatrin, tiagabine, or riluzole currently or within 2 weeks of Visit 1.
- Subjects treated with racemic baclofen currently or within 1 week of Visit 1.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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EXPERIMENTAL: Arbaclofen
Open Label Study
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A flexible dose titration will be utilized.
Orally disintegrating tablets
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Safety Measures
Time Frame: 100 weeks
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Adverse Events, Suicidality Assessment, Physical Examination, Vital Signs and Weight, Laboratory Tests: Complete Blood Count, Urinalysis, Chemistry Panel
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100 weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Efficacy
Time Frame: 100 Weeks
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Aberrant Behavior Checklist Lethargy/Social Withdrawal Sub- Scale Clinical Global Impression Vineland Adaptive Behavior Scales Stanford Binet Intelligence Scale
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100 Weeks
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
November 1, 2011
Primary Completion (ACTUAL)
July 1, 2013
Study Completion (ACTUAL)
July 1, 2013
Study Registration Dates
First Submitted
March 1, 2012
First Submitted That Met QC Criteria
March 13, 2012
First Posted (ESTIMATE)
March 15, 2012
Study Record Updates
Last Update Posted (ESTIMATE)
July 31, 2013
Last Update Submitted That Met QC Criteria
July 30, 2013
Last Verified
July 1, 2013
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Disease
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Chromosome Disorders
- Sex Chromosome Disorders
- Syndrome
- Fragile X Syndrome
Other Study ID Numbers
- 209FX303
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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