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An Open Label Extension Study in Subjects With Fragile X Syndrome (209FX303)

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: NCT01555333
Recruitment Status : Terminated
First Posted : March 15, 2012
Last Update Posted : July 31, 2013
Information provided by (Responsible Party):
Seaside Therapeutics, Inc.

Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: arbaclofen Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 357 participants
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 in Subject With Fragile X Syndrome
Study Start Date : November 2011
Actual Primary Completion Date : July 2013
Actual Study Completion Date : July 2013

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Arbaclofen
Open Label Study
Drug: arbaclofen
A flexible dose titration will be utilized. Orally disintegrating tablets
Other Name: STX209

Primary Outcome Measures :
  1. Safety Measures [ Time Frame: 100 weeks ]
    Adverse Events, Suicidality Assessment, Physical Examination, Vital Signs and Weight, Laboratory Tests: Complete Blood Count, Urinalysis, Chemistry Panel

Secondary Outcome Measures :
  1. Efficacy [ Time Frame: 100 Weeks ]
    Aberrant Behavior Checklist Lethargy/Social Withdrawal Sub- Scale Clinical Global Impression Vineland Adaptive Behavior Scales Stanford Binet Intelligence Scale

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Successfully completed all scheduled visits of the previous protocol ( 22002, 209FX301, or 209FX302).
  2. 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.
  3. 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.
  4. Current treatment with no more than 3 psychoactive medications, including anti-epileptics, unless the Medical Monitor is consulted.
  5. 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.
  6. Negative pregnancy test for females of childbearing potential or be using a medically acceptable form of birth control.

Exclusion Criteria

  1. 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.
  2. Subjects who are currently engaged in illicit drug or alcohol abuse.
  3. 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.
  4. 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.
  5. 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.
  6. Subjects who, in the Investigator's opinion, might not be suitable for the study.
  7. Subjects treated with vigabatrin, tiagabine, or riluzole currently or within 2 weeks of Visit 1.
  8. Subjects treated with racemic baclofen currently or within 1 week of Visit 1.

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): NCT01555333

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Sponsors and Collaborators
Seaside Therapeutics, Inc.
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Study Director: Paul Wang, M.D. Seaside Therapeutics, Inc.

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Responsible Party: Seaside Therapeutics, Inc. Identifier: NCT01555333     History of Changes
Other Study ID Numbers: 209FX303
First Posted: March 15, 2012    Key Record Dates
Last Update Posted: July 31, 2013
Last Verified: July 2013
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