Clinical trial NCT00788073: Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Keywords: fragile X syndrome
irritability
behavior problems
Lead sponsor: Seaside Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2008-11-10
Enrollment: 63
Sex: ALL
Minimum age (years): 6
Maximum age (years): 40
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Aberrant Behavior Checklist Irritability Subscore
timeFrame: After 4 weeks of treatment
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Southwest Autism Research & Resource Center
state: Arizona
city: Los Angeles
country: United States
facility: University of California-Los Angeles Neuropsychiatric Institute
state: California
city: Sacramento
country: United States
facility: M.I.N.D. Institute
state: California
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Boston
country: United States
facility: Children's Hospital Boston
state: Massachusetts
city: Staten Island
country: United States
facility: NYS Institute for Basic Research in Developmental Disabilities
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina Neurosciences Hospital
state: North Carolina
city: Media
country: United States
facility: Suburban Research Associates
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Kennedy Center
state: Tennessee
city: Houston
country: United States
facility: Red Oaks Psychiatry Associates, P.A.
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Rush University Medical Center
name: Elizabeth Berry-Kravis, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: M.I.N.D. Institute
name: Randi Hagerman, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Riley Hospital for Children
name: Craig Erikson, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Seattle Children's Hospital
name: Bryan King, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of California, Los Angeles
name: James McCracken, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston Children's Hospital
name: Jonathan Picker, MBChB, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of North Carolina Neurosciences Hospital
name: Linmarie Sikich, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Vanderbilt Kennedy Center
name: Jeremy Veenstra-VanderWeele, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: NYS institute for Basic Research in Developmental Disabilities
name: Ted Brown, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Red Oaks Psychiatry Associates, PA
name: Lawrence Ginsberg, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Suburban Research Associates
name: Shivkumar Hatti, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Southwest Autism Research & Resource Center
name: Raun Melmed, MD
role: PRINCIPAL_INVESTIGATOR
References: