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

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

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.

Study Overview

• Status: Terminated
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: Arbaclofen

• Study Type: Interventional
• Enrollment (Actual): 45
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90024
      • University of California-Los Angeles Neuropsychiatric Institute
    • Sacramento, California, United States, 95817
      • M.I.N.D. Institute
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • New York
    • Staten Island, New York, United States, 10314
      • NYS Institute for Basic Research in Developmental Disabilities
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • University of North Carolina Neurosciences Hospital
  • Pennsylvania
    • Media, Pennsylvania, United States, 19063
      • Suburban Research Associates
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Vanderbilt Kennedy Center
  • Texas
    • Houston, Texas, United States, 77090
      • Red Oaks Psychiatry Associates, PA
  • Washington
    • Seattle, Washington, United States, 98101
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

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

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: STX209	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; Other Names: STX209; R-baclofen

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Irritability subscale of the Aberrant Behavior Checklist	1 year

Collaborators and Investigators

• Sponsor: Seaside Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start: November 1, 2009
• Primary Completion (Actual): December 1, 2012
• Study Completion (Actual): December 1, 2012

Study Registration Dates

• First Submitted: November 12, 2009
• First Submitted That Met QC Criteria: November 12, 2009
• First Posted (Estimate): November 13, 2009

Study Record Updates

• Last Update Posted (Estimate): December 20, 2012
• Last Update Submitted That Met QC Criteria: December 19, 2012
• Last Verified: March 1, 2011

More Information

Terms related to this study

• Keywords: Fragile X Syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; GABA Agents; Neuromuscular Agents; Muscle Relaxants, Central; GABA Agonists; GABA-B Receptor Agonists; Baclofen
• Other Study ID Numbers: 22002