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

Fragile X Syndrome Clinical Trial

Official title:

An Open Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects With Fragile X Syndrome

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01013480
• Other study ID #: 22002
• Status: Terminated
• Phase: Phase 2
• First received: November 12, 2009
• Last updated: December 19, 2012
• Start date: November 2009
• Est. completion date: December 2012

Study information

• Verified date: March 2011
• Source: Seaside Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 45
• Est. completion date: December 2012
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 40 Years

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• Arbaclofen
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

Locations

Country	Name	City	State
United States	University of North Carolina Neurosciences Hospital	Chapel Hill	North Carolina
United States	Rush University Medical Center	Chicago	Illinois
United States	Red Oaks Psychiatry Associates, PA	Houston	Texas
United States	University of California-Los Angeles Neuropsychiatric Institute	Los Angeles	California
United States	Suburban Research Associates	Media	Pennsylvania
United States	Vanderbilt Kennedy Center	Nashville	Tennessee
United States	M.I.N.D. Institute	Sacramento	California
United States	Seattle Children's Hospital	Seattle	Washington
United States	NYS Institute for Basic Research in Developmental Disabilities	Staten Island	New York

Sponsors (1)

Lead Sponsor	Collaborator
Seaside Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Irritability subscale of the Aberrant Behavior Checklist		1 year	No