Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)

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.
 
ClinicalTrials.gov Identifier: NCT01325220
Recruitment Status : Completed
First Posted : March 29, 2011
Last Update Posted : July 31, 2013
Sponsor:
Information provided by (Responsible Party):
Seaside Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE March 28, 2011
First Posted Date  ICMJE March 29, 2011
Last Update Posted Date July 31, 2013
Study Start Date  ICMJE June 2011
Actual Primary Completion Date June 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 28, 2011)
Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale [ Time Frame: at 8 weeks of treatment ]
This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01325220 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 Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Children With Fragile X Syndrome
Brief Summary There will be four study periods: Screening (up to 14 days in length), the Treatment Period (8 weeks), the Withdrawal Period (22 days), and Follow-up Period (up to 31 days). Subjects will be randomized to receive either STX209 (5 mg twice daily [BID], 10 mg BID or 10 mg three times daily [TID]) or placebo. Efficacy,safety and tolerability assessments will be performed periodically
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Fragile X Syndrome
Intervention  ICMJE
  • Drug: arbaclofen
    5 mg bid
    Other Name: R-baclofen, STX209
  • Drug: arbaclofen
    10 mg bid
    Other Name: R-baclofen, STX209
  • Drug: arbaclofen
    10 mg tid
    Other Name: R-baclofen, STX209
  • Drug: Placebo
    tid
Study Arms  ICMJE
  • Active Comparator: STX209 5 mg BID
    Intervention: Drug: arbaclofen
  • Active Comparator: STX209 10 mg BID
    Intervention: Drug: arbaclofen
  • Active Comparator: STX209 10 mg TID
    Intervention: Drug: arbaclofen
  • Placebo Comparator: Placebo
    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: July 30, 2013)
172
Original Estimated Enrollment  ICMJE
 (submitted: March 28, 2011)
200
Study Completion Date  ICMJE Not Provided
Actual Primary Completion Date June 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Molecular documentation of the full FMR1 mutation
  • Current pharmacological treatment regimen has been stable for at least 4 weeks prior to Screening.
  • Subjects with a history of seizure disorder must currently be receiving treatment with antiepileptics and must have been seizure free for 6 months, or must be seizure free for 3 years if not currently receiving antiepileptics.
  • If the subject is already receiving stable non-pharmacologic educational, behavioral, and/or dietary interventions, participation in these programs must have been continuous during the 2 months prior to Screening

Exclusion Criteria:

  • 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.
  • Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
  • Subjects who have taken another investigational drug within the last 30 days.
  • Subjects who are not able to take oral medications
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 11 Years   (Child)
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 NCT01325220
Other Study ID Numbers  ICMJE 209FX302
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
Study Director: Paul Wang, M.D. Seaside Therapeutics, Inc.
PRS Account Seaside Therapeutics, Inc.
Verification Date July 2013

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