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

NCT01013480 | Terminated Phase 2

• 1 other identifier: 22002
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 9

Brief 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.

Conditions

Fragile X Syndrome

Keywords

Fragile X Syndrome

Outcome Measures

Primary Outcomes (1)

• Irritability subscale of the Aberrant Behavior Checklist

  1 year

Study Arms (1)

STX209

ACTIVE COMPARATOR

Drug: Arbaclofen

Interventions

Arbaclofen DRUG

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

Also known as: R-baclofen, STX209

STX209

Eligibility Criteria

• Age: 6 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Seaside Therapeutics, Inc.: lead

Study Sites (9)

University of California-Los Angeles Neuropsychiatric Institute

Los Angeles, California, 90024, United States

Location

M.I.N.D. Institute

Sacramento, California, 95817, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

NYS Institute for Basic Research in Developmental Disabilities

Staten Island, New York, 10314, United States

Location

University of North Carolina Neurosciences Hospital

Chapel Hill, North Carolina, 27514, United States

Location

Suburban Research Associates

Media, Pennsylvania, 19063, United States

Location

Vanderbilt Kennedy Center

Nashville, Tennessee, 37203, United States

Location

Red Oaks Psychiatry Associates, PA

Houston, Texas, 77090, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98101, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

arbaclofen placarbil

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2009
• First Posted: November 13, 2009
• Study Start: November 1, 2009
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2012
• Last Updated: December 20, 2012

Record last verified: 2011-03

Locations

US (9)