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

NCT00788073 | Completed Phase 2 Results DMC

• 1 other identifier: 22001
• Study Type: interventional
• Target: 63
• Locations: 1 country
• Sites: 12

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.

Conditions

Fragile X Syndrome

Keywords

fragile X syndrome; irritability; behavior problems

Outcome Measures

Primary Outcomes (1)

• 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).

  After 4 weeks of treatment

Study Arms (2)

STX209

ACTIVE COMPARATOR

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

Drug: STX209

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

STX209 DRUG

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

Also known as: arbaclofen

STX209

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 6 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Seaside Therapeutics, Inc.: lead

Study Sites (12)

Southwest Autism Research & Resource Center

Phoenix, Arizona, 85006, United States

Location

University of California-Los Angeles Neuropsychiatric Institute

Los Angeles, California, 90024, United States

Location

M.I.N.D. Institute

Sacramento, California, 95817, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

NYS Institute for Basic Research in Developmental Disabilities

Staten Island, New York, 10314, United States

Location

University of North Carolina Neurosciences Hospital

Chapel Hill, North Carolina, 27514, United States

Location

Suburban Research Associates

Media, Pennsylvania, 19063, United States

Location

Vanderbilt Kennedy Center

Nashville, Tennessee, 37203, United States

Location

Red Oaks Psychiatry Associates, P.A.

Houston, Texas, 77090, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98101, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome; Mental Disorders

Interventions

arbaclofen placarbil

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Results Point of Contact

• Title: Dr. Paul Wang, Vice President of Clinical and Medical Affairs
• Organization: Seaside Therapeutics

pwang@seasidetherapeutics.com

617-374-9009

Study Officials

• 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

• James McCracken, MD

  University of California, Los Angeles

  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

• Shivkumar Hatti, MD

  Suburban Research Associates

  PRINCIPAL INVESTIGATOR

• Raun Melmed, MD

  Southwest Autism Research & Resource Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2008
• First Posted: November 10, 2008
• Study Start: November 1, 2008
• Primary Completion: March 1, 2010
• Study Completion: May 1, 2010
• Last Updated: May 6, 2013
• Results First Posted: May 6, 2013

Record last verified: 2013-03

Locations

US (12)