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

This study has been terminated.
Sponsor:
Information provided by (Responsible Party):
Seaside Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT01555333
First received: March 1, 2012
Last updated: July 30, 2013
Last verified: July 2013
  Purpose

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 Intervention Phase
Fragile X Syndrome
Drug: arbaclofen
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Seaside Therapeutics, Inc.:

Primary Outcome Measures:
  • Safety Measures [ Time Frame: 100 weeks ] [ Designated as safety issue: Yes ]
    Adverse Events, Suicidality Assessment, Physical Examination, Vital Signs and Weight, Laboratory Tests: Complete Blood Count, Urinalysis, Chemistry Panel


Secondary Outcome Measures:
  • Efficacy [ Time Frame: 100 Weeks ] [ Designated as safety issue: No ]
    Aberrant Behavior Checklist Lethargy/Social Withdrawal Sub- Scale Clinical Global Impression Vineland Adaptive Behavior Scales Stanford Binet Intelligence Scale


Enrollment: 357
Study Start Date: November 2011
Study Completion Date: July 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arbaclofen
Open Label Study
Drug: arbaclofen
A flexible dose titration will be utilized. Orally disintegrating tablets
Other Name: STX209

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.

  Eligibility

Ages Eligible for Study:   5 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  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: NCT01555333

  Hide Study Locations
Locations
United States, Arizona
Seaside Therapeutics Site #16
Phoenix, Arizona, United States, 85006
United States, California
Seaside Therapeutics Site #07
Long Beach, California, United States, 90806
Seaside Therapeutics Site #10
Sacramento, California, United States, 95817
United States, Colorado
Seaside Therapeutics Site #17
Aurora, Colorado, United States, 80045
United States, Florida
Seaside Therapeutics Site #01
Miami, Florida, United States, 33136
Seaside Therapeutics Site #14
Orange City, Florida, United States, 32763
United States, Georgia
Seaside Therapeutics Site #20
Decatur, Georgia, United States, 30033
United States, Illinois
Seaside Therapeutics Site #02
Chicago, Illinois, United States, 60612
United States, Kansas
Seaside Therapeutics Site #23
Kansas City, Kansas, United States, 66160
United States, Maryland
Seaside Therapeutics Site #12
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Seaside Therapeutics Site #08
Worcester, Massachusetts, United States, 01605
United States, Missouri
Seaside Therapeutics Site #03
Columbia, Missouri, United States, 65211
United States, New York
Seaside Therapeutics Site #22
New York, New York, United States, 10029
Seaside Therapeutics Site #04
Staten Island, New York, United States, 10314
United States, North Carolina
Seaside Therapeutics Site #24
Chapel Hill, North Carolina, United States, 27514
Seaside Therapeutics Site #21
Durham, North Carolina, United States, 27710
United States, Ohio
Seaside Therapeutics Site #05
Akron, Ohio, United States, 44308
United States, Oklahoma
Seaside Therapeutics Site #15
Oklahoma City, Oklahoma, United States, 73117
United States, Pennsylvania
Seaside Therapeutics Site #11
Media, Pennsylvania, United States, 19063
United States, Tennessee
Seaside Therapeutics Site #19
Nashville, Tennessee, United States, 37212
United States, Texas
Seaside Therapeutics Site #25
Houston, Texas, United States, 77090
Seaside Therapeutics Site #18
San Antonio, Texas, United States, 78258
United States, Washington
Seaside Therapeutics Site #13
Seattle, Washington, United States, 98121
Sponsors and Collaborators
Seaside Therapeutics, Inc.
Investigators
Study Director: Paul Wang, M.D. Seaside Therapeutics, Inc.
  More Information

No publications provided

Responsible Party: Seaside Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT01555333     History of Changes
Other Study ID Numbers: 209FX303
Study First Received: March 1, 2012
Last Updated: July 30, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Fragile X Syndrome
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 28, 2014