Fragile X SyndromePhase 2NCT00788073

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

A Phase 2 study testing STX209 in people with Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 12 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 6 to 40 years
Conditions the study is for: Fragile X Syndrome
What is being tested?: STX209
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 63 participants
Where: 12 sitesUnited States
Study dates: Nov 2008to May 2010
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 6 to 40 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

12 sites in 1 country.

City, postcode or hospital

12 sites in 1 country

United States12 sites

Children's Hospital Boston
Boston, Massachusetts, 02115
University of North Carolina Neurosciences Hospital
Chapel Hill, North Carolina, 27514
Rush University Medical Center
Chicago, Illinois, 60612
Red Oaks Psychiatry Associates, P.A.
Houston, Texas, 77090
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of California-Los Angeles Neuropsychiatric Institute
Los Angeles, California, 90024
Suburban Research Associates
Media, Pennsylvania, 19063
Vanderbilt Kennedy Center
Nashville, Tennessee, 37203
Southwest Autism Research & Resource Center
Phoenix, Arizona, 85006
M.I.N.D. Institute
Sacramento, California, 95817
Seattle Children's Hospital
Seattle, Washington, 98101
NYS Institute for Basic Research in Developmental Disabilities
Staten Island, New York, 10314

Other trials in United States

About this study

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

Conditions: Fragile X Syndrome
Being tested: STX209, Placebo
Keywords: fragile X syndrome, irritability, behavior problems
Medical subject headings: Fragile X Syndrome, Mental Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

STX209Drug
Also called arbaclofen
Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks
PlaceboDrug
variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks

Study groups in NCT00788073
Study group	Role	What they receive
STX209	Active comparator	STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks Receives: Drug: STX209
Placebo	Placebo comparator	variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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).
Measured: After 4 weeks of treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Nov 2008May 2010

First posted10 Nov 2008 (estimated)
Study startNov 2008
Primary completionMar 2010
Study completionMay 2010

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 63 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00788073
Sponsor's protocol ID: 22001
Status last verified: Mar 2013
First submitted: 7 Nov 2008
Last updated: 6 May 2013
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruitingPhase 2
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
Aggressive Behavior in Fragile X Syndome
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Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
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RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Not yet recruitingPhase 2/3
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)

All Fragile X Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 6 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00788073
Acronym / sponsor ID: 22001
Conditions: Fragile X Syndrome
Keywords: fragile X syndrome, irritability, behavior problems
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 63 participants (actual)
Lead sponsor: Seaside Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: Nov 2008
Primary completion: Mar 2010 (actual)
Study completion: May 2010 (actual)
First posted: 10 Nov 2008 (estimated)
Results: Posted 6 May 2013

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
STX209	Active comparator	Drug: STX209STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks
Placebo	Placebo comparator	Drug: Placebovariable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks

Who may qualify

Ages: 6 to 40 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (6 listed)

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.

Key exclusion criteria (6 listed)

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.

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
STX209Also: arbaclofenVariable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks	Drug	STX209
Placebovariable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks	Drug	Placebo

Primary outcome

Aberrant Behavior Checklist Irritability Subscore
Time frame: After 4 weeks of treatment
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).

Results and safety

Results posted to the registry on 6 May 2013, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
STX209:Placebo	30	29
Placebo:STX209	33	30

Primary outcome: Aberrant Behavior Checklist Irritability Subscore

Time frame: After 4 weeks of treatment. Least squares mean (Standard Error). Unit: Points on a scale

Measure	STX209:Placebo n = 63	Placebo:STX209 n = 63
Value	-5.1 (0.95)	-5.3 (0.95)

STX209:Placebo vs Placebo:STX209: Mean Difference (Final Values): -0.2, p = 0.05, ANCOVA

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
STX209	1 of 63 (1.6%)	42 of 63 (66.7%)	Not reported
Placebo	0 of 63 (0%)	31 of 63 (49.2%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2013.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Fragile X Syndrome

Not yet recruitingPhase 2
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
Aggressive Behavior in Fragile X Syndome
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruiting
Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD)
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Not yet recruitingPhase 2/3
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)

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.

Elizabeth Berry-Kravis, MD, PhD principal investigator

Rush University Medical Center

Randi Hagerman, MD principal investigator

M.I.N.D. Institute

Craig Erikson, MD principal investigator

Riley Hospital for Children

12 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing STX209 in people with Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 12 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 6 inclusion and 6 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 6 May 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 May 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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 Irritability Subscore (time frame: After 4 weeks of treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00788073.

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 6 May 2013.