Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome (Harbor-A)

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Seaside Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT01282268

First received: January 20, 2011

Last updated: July 30, 2013

Last verified: July 2013

History of Changes

Purpose

To explore the efficacy, safety and tolerability of STX209 (arbaclofen) administered for the treatment of social withdrawal in adolescents and adults with fragile X syndrome (FXS)

Condition	Intervention	Phase
Fragile X Syndrome	Drug: arbaclofen Drug: placebo	Phase 3


Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	A Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Adolescents and Adults With Fragile X Syndrome

Resource links provided by NLM:

Genetics Home Reference related topics: CASK-related intellectual disability MECP2 duplication syndrome PPM-X syndrome Renpenning syndrome SYNGAP1-related intellectual disability fragile X syndrome

MedlinePlus related topics: Fragile X Syndrome

Genetic and Rare Diseases Information Center resources: Fragile X Syndrome X-linked Mental Retardation and Macro-orchidism

U.S. FDA Resources

Further study details as provided by Seaside Therapeutics, Inc.:

Primary Outcome Measures:

Aberrant Behavior Checklist - FXS Social Avoidance Subscale [ Time Frame: At 8 weeks of treatment ] [ Designated as safety issue: No ]


Enrollment:	125
Study Start Date:	May 2011
Primary Completion Date:	December 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: Arbaclofen	Drug: arbaclofen orally disintegrating tablet Other Name: STX209
Placebo Comparator: Placebo	Drug: placebo orally disintegrating tablet

Eligibility


Ages Eligible for Study:	12 Years to 50 Years (Child, Adult)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Molecular documentation of the full FMR1 mutation
Current pharmacological treatment regimen has been stable for at least 4 weeks prior to Screening.
Subjects with a history of seizure disorder must currently be receiving treatment with antiepileptics and must have been seizure free for 6 months, or must be seizure free for 3 years if not currently receiving antiepileptics.
If the subject is already receiving stable non-pharmacologic educational, behavioral, and/or dietary interventions, participation in these programs must have been continuous during the 2 months prior to Screening

Exclusion Criteria:

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.
Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
Subjects who have taken another investigational drug within the last 30 days.
Subjects who are not able to take oral medications.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01282268

Show 24 Study Locations

Sponsors and Collaborators

Seaside Therapeutics, Inc.

Investigators


Study Director:	Paul Wang, M.D.	Seaside Therapeutics, Inc.

More Information


Responsible Party:	Seaside Therapeutics, Inc.
ClinicalTrials.gov Identifier:	NCT01282268 History of Changes
Other Study ID Numbers:	209FX301
Study First Received:	January 20, 2011
Last Updated:	July 30, 2013
Health Authority:	United States: Food and Drug Administration

Keywords provided by Seaside Therapeutics, Inc.:


fragile X syndrome autism spectrum disorder

Additional relevant MeSH terms:


Syndrome Fragile X 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

ClinicalTrials.gov processed this record on September 23, 2016

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