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An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

Primary Purpose

Fragile X Syndrome

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Arbaclofen
Sponsored by
Seaside Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fragile X Syndrome focused on measuring Fragile X Syndrome

Eligibility Criteria

6 Years - 40 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Have completed all scheduled visits in protocol 22001 and have shown they can adequately follow the protocol, with sufficient medical justification to continue on open-label treatment with STX209, as assessed by the principal investigator

Exclusion Criteria:

  • Subjects with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being.
  • The occurrence or continuation of any adverse event or condition during study 22001 that, in the opinion of the Investigator, should exclude the subject from participating in this open-label extension.

Sites / Locations

  • University of California-Los Angeles Neuropsychiatric Institute
  • M.I.N.D. Institute
  • Rush University Medical Center
  • NYS Institute for Basic Research in Developmental Disabilities
  • University of North Carolina Neurosciences Hospital
  • Suburban Research Associates
  • Vanderbilt Kennedy Center
  • Red Oaks Psychiatry Associates, PA
  • Seattle Children's Hospital

Arms of the Study

Arm 1

Arm Type

Active Comparator

Arm Label

STX209

Arm Description

Outcomes

Primary Outcome Measures

Irritability subscale of the Aberrant Behavior Checklist

Secondary Outcome Measures

Full Information

First Posted
November 12, 2009
Last Updated
December 19, 2012
Sponsor
Seaside Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01013480
Brief Title
An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome
Official Title
An Open Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects With Fragile X Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
March 2011
Overall Recruitment Status
Terminated
Why Stopped
all active subjects rolled into 209FX303/NCT01013480
Study Start Date
November 2009 (undefined)
Primary Completion Date
December 2012 (Actual)
Study Completion Date
December 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Seaside Therapeutics, Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Study 22001, "A Double-Blind, Placebo-Controlled, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects with Fragile X Syndrome" currently is evaluating the efficacy of STX209 (R-baclofen) for management of typical problem behaviors, such as irritability and aggression, in subjects with FXS. This study (22002) will enter subjects who complete Study 22001 into a long-term, open-label study.The open-label extension protocol will provide necessary data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions more closely reflective of their general medical care.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome
Keywords
Fragile X Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
45 (Actual)

8. Arms, Groups, and Interventions

Arm Title
STX209
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
Arbaclofen
Other Intervention Name(s)
R-baclofen, STX209
Intervention Description
A flexible dose titration will be utilized during the first four weeks to define the optimal titrated dose (OTD) for each subject. Investigators will use clinical judgment to adjust doses to the OTD. The starting dose will be 1 mg BID. The dose may be increased every four to five days to 2 mg BID, 3 mg BID, 5 mg BID and then 10 mg BID
Primary Outcome Measure Information:
Title
Irritability subscale of the Aberrant Behavior Checklist
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Have completed all scheduled visits in protocol 22001 and have shown they can adequately follow the protocol, with sufficient medical justification to continue on open-label treatment with STX209, as assessed by the principal investigator Exclusion Criteria: Subjects with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. The occurrence or continuation of any adverse event or condition during study 22001 that, in the opinion of the Investigator, should exclude the subject from participating in this open-label extension.
Facility Information:
Facility Name
University of California-Los Angeles Neuropsychiatric Institute
City
Los Angeles
State/Province
California
ZIP/Postal Code
90024
Country
United States
Facility Name
M.I.N.D. Institute
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
NYS Institute for Basic Research in Developmental Disabilities
City
Staten Island
State/Province
New York
ZIP/Postal Code
10314
Country
United States
Facility Name
University of North Carolina Neurosciences Hospital
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Suburban Research Associates
City
Media
State/Province
Pennsylvania
ZIP/Postal Code
19063
Country
United States
Facility Name
Vanderbilt Kennedy Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Facility Name
Red Oaks Psychiatry Associates, PA
City
Houston
State/Province
Texas
ZIP/Postal Code
77090
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States

12. IPD Sharing Statement

Learn more about this trial

An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

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