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

Fragile X Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00788073
• Other study ID #: 22001
• Status: Completed
• Phase: Phase 2
• First received: November 7, 2008
• Last updated: March 22, 2013
• Start date: November 2008
• Est. completion date: May 2010

Study information

• Verified date: March 2013
• 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. completion date: May 2010
• Est. primary completion date: March 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 40 Years

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• STX209
Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks
• Placebo
variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks

Locations

Country	Name	City	State
United States	Children's Hospital Boston	Boston	Massachusetts
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, P.A.	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
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	Southwest Autism Research & Resource Center	Phoenix	Arizona
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	Aberrant Behavior Checklist Irritability Subscore	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).	After 4 weeks of treatment	No