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

Primary Purpose

Fragile X Syndrome

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

STX209

Placebo

About this trial

This is an interventional treatment trial for Fragile X Syndrome focused on measuring fragile X syndrome, irritability, behavior problems

Eligibility Criteria

6 Years - 40 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Southwest Autism Research & Resource Center
University of California-Los Angeles Neuropsychiatric Institute
M.I.N.D. Institute
Rush University Medical Center
Riley Hospital for Children
Children's Hospital Boston
NYS Institute for Basic Research in Developmental Disabilities
University of North Carolina Neurosciences Hospital
Suburban Research Associates
Vanderbilt Kennedy Center
Red Oaks Psychiatry Associates, P.A.
Seattle Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

STX209

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

Aberrant Behavior Checklist Irritability Subscore

The Aberrant Behavior Checklist-Community Edition (ABC-C) is a 58-item questionnaire composed of five different independent subscales. The questionnaire is completed by the parent/caregiver and lists aberrant behaviors and asks about the severity of the problem. ABC-Irritability is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. A decreased score indicates few aberrant behaviors and clinical improvement. The entire ABC-C assessment is administered at baseline and then at the end of each Intervention Period (4 weeks after Baseline).

Secondary Outcome Measures

Full Information

NCT ID

NCT00788073

First Posted

November 7, 2008

Last Updated

March 22, 2013

Sponsor

Seaside Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT00788073

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2013

Overall Recruitment Status

Completed

Study Start Date

November 2008 (undefined)

Primary Completion Date

March 2010 (Actual)

Study Completion Date

May 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Seaside Therapeutics, Inc.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Fragile X Syndrome

Keywords

fragile X syndrome, irritability, behavior problems

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

63 (Actual)

8. Arms, Groups, and Interventions

Arm Title

STX209

Arm Type

Active Comparator

Arm Description

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

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

STX209

Other Intervention Name(s)

arbaclofen

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

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

Primary Outcome Measure Information:

Title

Aberrant Behavior Checklist Irritability Subscore

Description

Time Frame

After 4 weeks of treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

40 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials: