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

NCT ID: NCT00788073

Last Updated: 2013-05-06

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

63 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-11-30

Study Completion Date

2010-05-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Fragile X Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

STX209

STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks

Group Type ACTIVE_COMPARATOR

STX209

Intervention Type DRUG

Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks

Placebo

variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

STX209

Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks

Intervention Type DRUG

Placebo

variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

arbaclofen

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Minimum Eligible Age

6 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Seaside Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Elizabeth Berry-Kravis, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Rush University Medical Center

Randi Hagerman, MD

Role: PRINCIPAL_INVESTIGATOR

M.I.N.D. Institute

Craig Erikson, MD

Role: PRINCIPAL_INVESTIGATOR

Riley Hospital for Children

Bryan King, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Seattle Children's Hospital

James McCracken, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, Los Angeles

Jonathan Picker, MBChB, PhD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Linmarie Sikich, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina Neurosciences Hospital

Jeremy Veenstra-VanderWeele, MD

Role: PRINCIPAL_INVESTIGATOR

Vanderbilt Kennedy Center

Ted Brown, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

NYS institute for Basic Research in Developmental Disabilities

Lawrence Ginsberg, MD

Role: PRINCIPAL_INVESTIGATOR

Red Oaks Psychiatry Associates, PA

Shivkumar Hatti, MD

Role: PRINCIPAL_INVESTIGATOR

Suburban Research Associates

Raun Melmed, MD

Role: PRINCIPAL_INVESTIGATOR

Southwest Autism Research & Resource Center

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Southwest Autism Research & Resource Center

Phoenix, Arizona, United States

Site Status

University of California-Los Angeles Neuropsychiatric Institute

Los Angeles, California, United States

Site Status

M.I.N.D. Institute

Sacramento, California, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

Children's Hospital Boston

Boston, Massachusetts, United States

Site Status

NYS Institute for Basic Research in Developmental Disabilities

Staten Island, New York, United States

Site Status

University of North Carolina Neurosciences Hospital

Chapel Hill, North Carolina, United States

Site Status

Suburban Research Associates

Media, Pennsylvania, United States

Site Status

Vanderbilt Kennedy Center

Nashville, Tennessee, United States

Site Status

Red Oaks Psychiatry Associates, P.A.

Houston, Texas, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

22001

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

ERG/5-HTP in Fragile X Syndrome (FXS)
NCT05030129 COMPLETED PHASE2