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Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome

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ClinicalTrials.gov Identifier: NCT00788073
Recruitment Status : Completed
First Posted : November 10, 2008
Results First Posted : May 6, 2013
Last Update Posted : May 6, 2013
Sponsor:
Information provided by (Responsible Party):
Seaside Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE November 7, 2008
First Posted Date  ICMJE November 10, 2008
Results First Submitted Date  ICMJE February 7, 2013
Results First Posted Date  ICMJE May 6, 2013
Last Update Posted Date May 6, 2013
Study Start Date  ICMJE November 2008
Actual Primary Completion Date March 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 22, 2013)
Aberrant Behavior Checklist Irritability Subscore [ Time Frame: After 4 weeks of treatment ]
The Aberrant Behavior Checklist-Community Edition (ABC-C) is a 58-item questionnaire composed of five different independent subscales. The questionnaire is completed by the parent/caregiver and lists aberrant behaviors and asks about the severity of the problem. ABC-Irritability is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. A decreased score indicates few aberrant behaviors and clinical improvement. The entire ABC-C assessment is administered at baseline and then at the end of each Intervention Period (4 weeks after Baseline).
Original Primary Outcome Measures  ICMJE
 (submitted: November 7, 2008)
  • Adverse events [ Time Frame: during the study ]
  • Aberrant Behavior Checklist Irritability Subscore [ Time Frame: After 4 weeks of treatment ]
Change History Complete list of historical versions of study NCT00788073 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
Official Title  ICMJE 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
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Fragile X Syndrome
Intervention  ICMJE
  • Drug: STX209
    Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks
    Other Name: arbaclofen
  • Drug: Placebo
    variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks
Study Arms  ICMJE
  • Active Comparator: STX209
    STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks
    Intervention: Drug: STX209
  • Placebo Comparator: Placebo
    variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 28, 2012)
63
Original Estimated Enrollment  ICMJE
 (submitted: November 7, 2008)
60
Actual Study Completion Date  ICMJE May 2010
Actual Primary Completion Date March 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 40 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00788073
Other Study ID Numbers  ICMJE 22001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Seaside Therapeutics, Inc.
Study Sponsor  ICMJE Seaside Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
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 Boston Children’s Hospital
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
PRS Account Seaside Therapeutics, Inc.
Verification Date March 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP