Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001

Biomarker Testing and DNA Collection in Subjects Participating in 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

The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001 will be offered participation in 22001A which will evaluate secreted protein before and after treatment with STX209 and placebo to determine if they correlate with effectiveness of treatment or susceptibility to treatment with STX209. These same subjects will also be asked to contribute a blood sample for DNA (deoxyribonucleic acid) collection. The investigators will study the DNA to determine if STX209 works better in people with specific gene variations, or to find new gene variations that predict how well STX209 works.

Study Overview

• Status: Completed
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: STX209

• Study Type: Observational

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Southwest Autism Research & Resource Center
  • 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
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children
  • 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

• Sampling Method: Non-Probability Sample

Study Population

Fragile x syndrome subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001

Description

Inclusion Criteria:

• Subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001

Exclusion Criteria:

• Subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Arbaclofen followed by Placebo	Drug: STX209
Placebo followed by Arbaclofen	Drug: STX209

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
change in plasma proteins with treatment	After 4 weeks of treatment

Collaborators and Investigators

• Sponsor: Seaside Therapeutics, Inc.
• Investigators: Principal Investigator: James McCracken, MD, University of California, Los Angeles; 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: 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: Raun Melmed, MD, Southwest Autism Research & Resource Center

Study record dates

Study Major Dates

• Study Start: January 1, 2009
• Primary Completion (Actual): March 1, 2010

Study Registration Dates

• First Submitted: January 13, 2009
• First Submitted That Met QC Criteria: January 14, 2009
• First Posted (Estimate): January 15, 2009

Study Record Updates

• Last Update Posted (Estimate): July 31, 2013
• Last Update Submitted That Met QC Criteria: July 30, 2013
• Last Verified: July 1, 2013

More Information

Terms related to this study

• Keywords: biomarkers; 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
• Other Study ID Numbers: 22001A