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

• ClinicalTrials.gov Identifier: NCT00788073
• Recruitment Status: Completed
• First Posted: November 10, 2008
• Results First Posted: May 6, 2013
• Last Update Posted: May 6, 2013
• Sponsor: Seaside Therapeutics, Inc.
• Information provided by (Responsible Party): Seaside Therapeutics, Inc.

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Fragile X Syndrome	Drug: STX209; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 63 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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 Start Date: November 2008
• Actual Primary Completion Date: March 2010
• Actual Study Completion Date: May 2010

Arms and Interventions

Arm	Intervention/treatment
Active Comparator: STX209; STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks	Drug: STX209; Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks; Other Name: arbaclofen
Placebo Comparator: Placebo; variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks	Drug: Placebo; variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks

Outcome Measures

Primary Outcome Measures :

1. Aberrant Behavior Checklist Irritability Subscore [ Time Frame: After 4 weeks of treatment ]
   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).

Eligibility Criteria

• Ages Eligible for Study: 6 Years to 40 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

United States, Arizona

Southwest Autism Research & Resource Center

Phoenix, Arizona, United States, 85006

United States, California

University of California-Los Angeles Neuropsychiatric Institute

Los Angeles, California, United States, 90024

M.I.N.D. Institute

Sacramento, California, United States, 95817

United States, Illinois

Rush University Medical Center

Chicago, Illinois, United States, 60612

United States, Indiana

Riley Hospital for Children

Indianapolis, Indiana, United States, 46202

United States, Massachusetts

Children's Hospital Boston

Boston, Massachusetts, United States, 02115

United States, New York

NYS Institute for Basic Research in Developmental Disabilities

Staten Island, New York, United States, 10314

United States, North Carolina

University of North Carolina Neurosciences Hospital

Chapel Hill, North Carolina, United States, 27514

United States, Pennsylvania

Suburban Research Associates

Media, Pennsylvania, United States, 19063

United States, Tennessee

Vanderbilt Kennedy Center

Nashville, Tennessee, United States, 37203

United States, Texas

Red Oaks Psychiatry Associates, P.A.

Houston, Texas, United States, 77090

United States, Washington

Seattle Children's Hospital

Seattle, Washington, United States, 98101

Sponsors and Collaborators

Seaside Therapeutics, Inc.

Investigators

• Principal Investigator: Elizabeth Berry-Kravis, MD, PhD; Rush University Medical Center
• Principal Investigator: Randi Hagerman, MD; M.I.N.D. Institute
• Principal Investigator: Craig Erikson, MD; Riley Hospital for Children
• Principal Investigator: Bryan King, MD, PhD; Seattle Children's Hospital
• Principal Investigator: James McCracken, MD; University of California, Los Angeles
• Principal Investigator: Jonathan Picker, MBChB, PhD; Boston Children's Hospital
• Principal Investigator: Linmarie Sikich, MD; University of North Carolina Neurosciences Hospital
• Principal Investigator: Jeremy Veenstra-VanderWeele, MD; Vanderbilt Kennedy Center
• Principal Investigator: Ted Brown, MD, PhD; NYS institute for Basic Research in Developmental Disabilities
• Principal Investigator: Lawrence Ginsberg, MD; Red Oaks Psychiatry Associates, PA
• Principal Investigator: Shivkumar Hatti, MD; Suburban Research Associates
• Principal Investigator: Raun Melmed, MD; Southwest Autism Research & Resource Center

More Information

• Responsible Party: Seaside Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT00788073
• Other Study ID Numbers: 22001
• First Posted: November 10, 2008
• Results First Posted: May 6, 2013
• Last Update Posted: May 6, 2013
• Last Verified: March 2013

Keywords provided by Seaside Therapeutics, Inc.:

fragile X syndrome; irritability; behavior problems

Additional relevant MeSH terms:

Fragile X Syndrome; Syndrome; Disease; Pathologic Processes; Mental Retardation, X-Linked; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System