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Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)

Primary Purpose

Fragile X Syndrome

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
arbaclofen
arbaclofen
arbaclofen
Placebo
Sponsored by
Seaside Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fragile X Syndrome

Eligibility Criteria

5 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Southwest Autism Research & Resource Center
  • Miller Children's Hospital
  • University of California-Davis, M.I.N.D. Institute
  • Psychiatric Centers at San Diego
  • University of Colorado Denver, Children's Hospital
  • University of Miami, Mailman Center for Child Development
  • Lake Mary Pediatrics
  • Emory University School of Medicine
  • Rush University Medical Center
  • Kansas University Clinical Research Center
  • Kennedy Krieger Institute
  • University of Massachusetts
  • Children's Hospital of Michigan
  • University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders
  • Seaver Autism Center, Mount Sinai Medical Center
  • New York State Institute for Basic Research in Developmental Disabilities
  • Duke Clinical Research Unit
  • Akron Children's Hospital
  • Cincinnati Children's Hospital
  • University of Oklahoma, Physician's Child Study Center
  • Suburban Research Associates/Elwyn Genetics
  • Greenwood Genetics Center
  • Vanderbilt Kennedy Center
  • Childrens Medical Center Dallas
  • Texas Children's Hospital
  • Red Oaks Psychiatry Associates, P.A.
  • Road Runner Research
  • Seattle Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Active Comparator

Active Comparator

Active Comparator

Placebo Comparator

Arm Label

STX209 5 mg BID

STX209 10 mg BID

STX209 10 mg TID

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale
This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal

Secondary Outcome Measures

Full Information

First Posted
March 28, 2011
Last Updated
July 30, 2013
Sponsor
Seaside Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01325220
Brief Title
Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
Acronym
Harbor-C
Official Title
A Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Children With Fragile X Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
July 2013
Overall Recruitment Status
Completed
Study Start Date
June 2011 (undefined)
Primary Completion Date
June 2013 (Actual)
Study Completion Date
undefined (undefined)

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
There will be four study periods: Screening (up to 14 days in length), the Treatment Period (8 weeks), the Withdrawal Period (22 days), and Follow-up Period (up to 31 days). Subjects will be randomized to receive either STX209 (5 mg twice daily [BID], 10 mg BID or 10 mg three times daily [TID]) or placebo. Efficacy,safety and tolerability assessments will be performed periodically

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
172 (Actual)

8. Arms, Groups, and Interventions

Arm Title
STX209 5 mg BID
Arm Type
Active Comparator
Arm Title
STX209 10 mg BID
Arm Type
Active Comparator
Arm Title
STX209 10 mg TID
Arm Type
Active Comparator
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
arbaclofen
Other Intervention Name(s)
R-baclofen, STX209
Intervention Description
5 mg bid
Intervention Type
Drug
Intervention Name(s)
arbaclofen
Other Intervention Name(s)
R-baclofen, STX209
Intervention Description
10 mg bid
Intervention Type
Drug
Intervention Name(s)
arbaclofen
Other Intervention Name(s)
R-baclofen, STX209
Intervention Description
10 mg tid
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
tid
Primary Outcome Measure Information:
Title
Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale
Description
This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal
Time Frame
at 8 weeks of treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paul Wang, M.D.
Organizational Affiliation
Seaside Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Southwest Autism Research & Resource Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Facility Name
Miller Children's Hospital
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
University of California-Davis, M.I.N.D. Institute
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Psychiatric Centers at San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
92108
Country
United States
Facility Name
University of Colorado Denver, Children's Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Miami, Mailman Center for Child Development
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Lake Mary Pediatrics
City
Orange City
State/Province
Florida
ZIP/Postal Code
32763
Country
United States
Facility Name
Emory University School of Medicine
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Kansas University Clinical Research Center
City
Fairway
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
University of Massachusetts
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01605
Country
United States
Facility Name
Children's Hospital of Michigan
City
Detriot
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65211
Country
United States
Facility Name
Seaver Autism Center, Mount Sinai Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
New York State Institute for Basic Research in Developmental Disabilities
City
Staten Island
State/Province
New York
ZIP/Postal Code
10314
Country
United States
Facility Name
Duke Clinical Research Unit
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Akron Children's Hospital
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
University of Oklahoma, Physician's Child Study Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73117
Country
United States
Facility Name
Suburban Research Associates/Elwyn Genetics
City
Media
State/Province
Pennsylvania
ZIP/Postal Code
19063
Country
United States
Facility Name
Greenwood Genetics Center
City
Greenwood
State/Province
South Carolina
ZIP/Postal Code
29646
Country
United States
Facility Name
Vanderbilt Kennedy Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Facility Name
Childrens Medical Center Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Red Oaks Psychiatry Associates, P.A.
City
Houston
State/Province
Texas
ZIP/Postal Code
77090
Country
United States
Facility Name
Road Runner Research
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78258
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome

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