Fragile X SyndromePhase 3Harbor-CNCT01325220
Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
A Phase 3 study testing arbaclofen in people with Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 28 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 11 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- arbaclofen
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 172 participants
- Where
- 28 sitesUnited States
- Study dates
- Jun 2011to Jun 2013
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
28 sites in 1 country.
28 sites in 1 country
United States28 sites
Akron Children's Hospital
Akron, Ohio, 44308
University of Colorado Denver, Children's Hospital
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Rush University Medical Center
Chicago, Illinois, 60612
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders
Columbia, Missouri, 65211
Childrens Medical Center Dallas
Dallas, Texas, 75235
Emory University School of Medicine
Decatur, Georgia, 30033
Children's Hospital of Michigan
Detriot, Michigan, 48201
Duke Clinical Research Unit
Durham, North Carolina, 27710
Kansas University Clinical Research Center
Fairway, Kansas, 66205
Greenwood Genetics Center
Greenwood, South Carolina, 29646
Texas Children's Hospital
Houston, Texas, 77030
Red Oaks Psychiatry Associates, P.A.
Houston, Texas, 77090
Miller Children's Hospital
Long Beach, California, 90806
Suburban Research Associates/Elwyn Genetics
Media, Pennsylvania, 19063
University of Miami, Mailman Center for Child Development
Miami, Florida, 33136
Vanderbilt Kennedy Center
Nashville, Tennessee, 37203
Seaver Autism Center, Mount Sinai Medical Center
New York, New York, 10029
University of Oklahoma, Physician's Child Study Center
Oklahoma City, Oklahoma, 73117
Lake Mary Pediatrics
Orange City, Florida, 32763
Southwest Autism Research & Resource Center
Phoenix, Arizona, 85006
University of California-Davis, M.I.N.D. Institute
Sacramento, California, 95817
Road Runner Research
San Antonio, Texas, 78258
Psychiatric Centers at San Diego
San Diego, California, 92108
Seattle Children's Hospital
Seattle, Washington, 98101
New York State Institute for Basic Research in Developmental Disabilities
Staten Island, New York, 10314
University of Massachusetts
Worcester, Massachusetts, 01605
About this study
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
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
- Conditions
- Fragile X Syndrome
- Being tested
- arbaclofen, Placebo
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
arbaclofenDrug
Also called R-baclofen, STX209
5 mg bid
arbaclofenDrug
Also called R-baclofen, STX209
10 mg bid
arbaclofenDrug
Also called R-baclofen, STX209
10 mg tid
PlaceboDrug
tid
| Study group | Role | What they receive |
|---|---|---|
| STX209 5 mg BID | Active comparator | Not described Receives: Drug: arbaclofen |
| STX209 10 mg BID | Active comparator | Not described Receives: Drug: arbaclofen |
| STX209 10 mg TID | Active comparator | Not described Receives: Drug: arbaclofen |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale
This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal
Measured: at 8 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2011 (estimated)
- Study startJun 2011
- Primary completionJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 172 participants (actual)
Sponsor and investigators
- Lead sponsor
- Seaside Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paul Wang, M.D., Study director, Seaside Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01325220
- Sponsor's protocol ID
- 209FX302
- Acronym
- Harbor-C
- Status last verified
- Jul 2013
- First submitted
- 28 Mar 2011
- Last updated
- 31 Jul 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01325220
- Acronym / sponsor ID
- Harbor-C / 209FX302
- Conditions
- Fragile X Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 172 participants (actual)
- Lead sponsor
- Seaside Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2011
- Primary completion
- Jun 2013 (actual)
- First posted
- 29 Mar 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| STX209 5 mg BID | Active comparator | Drug: arbaclofen |
| STX209 10 mg BID | Active comparator | Drug: arbaclofen |
| STX209 10 mg TID | Active comparator | Drug: arbaclofen |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 5 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| arbaclofenAlso: R-baclofen, STX2095 mg bid | Drug | STX209 5 mg BID |
| arbaclofenAlso: R-baclofen, STX20910 mg bid | Drug | STX209 10 mg BID |
| arbaclofenAlso: R-baclofen, STX20910 mg tid | Drug | STX209 10 mg TID |
| Placebotid | Drug | Placebo |
Primary outcome
Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale
Time frame: at 8 weeks of treatment
This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2013.
Related trials for Fragile X Syndrome
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Aggressive Behavior in Fragile X Syndome
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Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
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Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
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A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paul Wang, M.D. study director
Seaside Therapeutics, Inc.
28 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing arbaclofen in people with Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 28 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale (time frame: at 8 weeks of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2013.