An Open Label Extension Study in Subjects With Fragile X Syndrome

NCT01555333 | Terminated Phase 3 DMC

• 1 other identifier: 209FX303
• Study Type: interventional
• Target: 357
• Locations: 1 country
• Sites: 23

Brief Summary

This study will enroll subjects who have completed Protocols 209FX301, 209FX302, or are currently participating in Protocol 2202 into a long-term study in which all subjects will receive active drug (arbaclofen).

Conditions

Fragile X Syndrome

Outcome Measures

Primary Outcomes (1)

• Safety Measures

  Adverse Events, Suicidality Assessment, Physical Examination, Vital Signs and Weight, Laboratory Tests: Complete Blood Count, Urinalysis, Chemistry Panel

  100 weeks

Secondary Outcomes (1)

• Efficacy

  100 Weeks

Study Arms (1)

Arbaclofen

EXPERIMENTAL

Open Label Study

Drug: arbaclofen

Interventions

arbaclofen DRUG

A flexible dose titration will be utilized. Orally disintegrating tablets

Also known as: STX209

Arbaclofen

Eligibility Criteria

• Age: 5 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Successfully completed all scheduled visits of the previous protocol ( 22002, 209FX301, or 209FX302).
• A parent,LAR, or caregiver must be willing and able to accompany the subject to all study visits, participate in phone calls, complete study assessments, administer study medication, and report the subject's condition and medication use to site staff members.
• Prior to the conduct of any study-specific procedures, the subject must provide written informed consent to participate in the study ( if developmentally appropriate) or verbal assent and the parent/caregiver/LAR must provide written informed consent. If the caregiver attending the clinic visits is not the parents, caregiver, or LAR, written consent must also be obtained for the caregiver's participation in the study.
• Current treatment with no more than 3 psychoactive medications, including anti-epileptics, unless the Medical Monitor is consulted.
• Subjects with a history of seizure disorder must have been seizure free for 6 months and be taking anti-epileptics, or seizure free for 3 years if not receiving anti-epileptic treatment. If currently receiving treatment with anti-epileptics, serum concentration levels must be tested and be in therapeutic range.
• Negative pregnancy test for females of childbearing potential or be using a medically acceptable form of birth control.

You may not qualify if:

• Subjects with any condition that 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 are currently engaged in illicit drug or alcohol abuse.
• Subjects who had a serious adverse event (SAE) while taking STX209 during their previous protocol (22002,209FX301,309FX302)that the Investigator considered related to STX209, unless approval from the Medical Monitor is obtained.
• The occurrence or continuation of any AE or condition during Studies 22002, 209FX301, or 209FX302 that, in the opinion of the Investigator, should exclude this subject from participating in the open-label extension.
• Subjects taking another investigational drug, other than STX209, currently or within 30 days of Visit 1. Subject must not take any investigational drugs during this study.
• Subjects who, in the Investigator's opinion, might not be suitable for the study.
• Subjects treated with vigabatrin, tiagabine, or riluzole currently or within 2 weeks of Visit 1.
• Subjects treated with racemic baclofen currently or within 1 week of Visit 1.

Sponsors & Collaborators

• Seaside Therapeutics, Inc.: lead

Study Sites (23)

Seaside Therapeutics Site #16

Phoenix, Arizona, 85006, United States

Location

Seaside Therapeutics Site #07

Long Beach, California, 90806, United States

Location

Seaside Therapeutics Site #10

Sacramento, California, 95817, United States

Location

Seaside Therapeutics Site #17

Aurora, Colorado, 80045, United States

Location

Seaside Therapeutics Site #01

Miami, Florida, 33136, United States

Location

Seaside Therapeutics Site #14

Orange City, Florida, 32763, United States

Location

Seaside Therapeutics Site #20

Decatur, Georgia, 30033, United States

Location

Seaside Therapeutics Site #02

Chicago, Illinois, 60612, United States

Location

Seaside Therapeutics Site #23

Kansas City, Kansas, 66160, United States

Location

Seaside Therapeutics Site #12

Baltimore, Maryland, 21205, United States

Location

Seaside Therapeutics Site #08

Worcester, Massachusetts, 01605, United States

Location

Seaside Therapeutics Site #03

Columbia, Missouri, 65211, United States

Location

Seaside Therapeutics Site #22

New York, New York, 10029, United States

Location

Seaside Therapeutics Site #04

Staten Island, New York, 10314, United States

Location

Seaside Therapeutics Site #24

Chapel Hill, North Carolina, 27514, United States

Location

Seaside Therapeutics Site #21

Durham, North Carolina, 27710, United States

Location

Seaside Therapeutics Site #05

Akron, Ohio, 44308, United States

Location

Seaside Therapeutics Site #15

Oklahoma City, Oklahoma, 73117, United States

Location

Seaside Therapeutics Site #11

Media, Pennsylvania, 19063, United States

Location

Seaside Therapeutics Site #19

Nashville, Tennessee, 37212, United States

Location

Seaside Therapeutics Site #25

Houston, Texas, 77090, United States

Location

Seaside Therapeutics Site #18

San Antonio, Texas, 78258, United States

Location

Seaside Therapeutics Site #13

Seattle, Washington, 98121, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

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

Study Officials

• Paul Wang, M.D.

  Seaside Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2012
• First Posted: March 15, 2012
• Study Start: November 1, 2011
• Primary Completion: July 1, 2013
• Study Completion: July 1, 2013
• Last Updated: July 31, 2013

Record last verified: 2013-07

Locations

US (23)