Fragile X SyndromePhase 2NCT01013480
An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome
A Phase 2 study testing Arbaclofen in people with Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 9 sites in United States.
Trial summary
Study 22001, "A Double-Blind, Placebo-Controlled, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects with Fragile X Syndrome" currently is evaluating the efficacy of STX209 (R-baclofen) for management of typical problem behaviors, such as… 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?
- Arbaclofen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 9 sitesUnited States
- Study dates
- Nov 2009to Dec 2012
- Phase
- Phase 2
- 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 6 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Have completed all scheduled visits in protocol 22001 and have shown they can adequately follow the protocol, with sufficient medical justification to continue on open-label treatment with STX209, as assessed by the principal investigator
Who cannot join
- Subjects with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being.
- The occurrence or continuation of any adverse event or condition during study 22001 that, in the opinion of the Investigator, should exclude the subject from participating in this open-label extension.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
University of North Carolina Neurosciences Hospital
Chapel Hill, North Carolina, 27514
Rush University Medical Center
Chicago, Illinois, 60612
Red Oaks Psychiatry Associates, PA
Houston, Texas, 77090
University of California-Los Angeles Neuropsychiatric Institute
Los Angeles, California, 90024
Suburban Research Associates
Media, Pennsylvania, 19063
Vanderbilt Kennedy Center
Nashville, Tennessee, 37203
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
About this study
Official title: An Open Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects With Fragile X Syndrome
Study 22001, "A Double-Blind, Placebo-Controlled, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects with Fragile X Syndrome" currently is evaluating the efficacy of STX209 (R-baclofen) for management of typical problem behaviors, such as irritability and aggression, in subjects with FXS. This study (22002) will enter subjects who complete Study 22001 into a long-term, open-label study.The open-label extension protocol will provide necessary data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions more closely reflective of their general medical care.
- Conditions
- Fragile X Syndrome
- Being tested
- Arbaclofen
- Keywords
- Fragile X Syndrome
- 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
A flexible dose titration will be utilized during the first four weeks to define the optimal titrated dose (OTD) for each subject. Investigators will use clinical judgment to adjust doses to the OTD. The starting dose will be 1 mg BID. The dose may be increased every four to five days to 2 mg BID, 3 mg BID, 5 mg BID and then 10 mg BID
| Study group | Role | What they receive |
|---|---|---|
| STX209 | Active comparator | Not described Receives: Drug: Arbaclofen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Irritability subscale of the Aberrant Behavior Checklist
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2009 (estimated)
- Study startNov 2009
- Primary completionDec 2012
- Study completionDec 2012
Why it stopped: all active subjects rolled into 209FX303/NCT01013480
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Seaside Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01013480
- Sponsor's protocol ID
- 22002
- Status last verified
- Mar 2011
- First submitted
- 12 Nov 2009
- Last updated
- 20 Dec 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT01013480
- Acronym / sponsor ID
- 22002
- Conditions
- Fragile X Syndrome
- Keywords
- Fragile X Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: all active subjects rolled into 209FX303/NCT01013480
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Seaside Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2009
- Primary completion
- Dec 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 13 Nov 2009 (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.
Study 22001, "A Double-Blind, Placebo-Controlled, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects with Fragile X Syndrome" currently is evaluating the efficacy of STX209 (R-baclofen) for management of typical problem behaviors, such as irritability and aggression, in subjects with FXS. This study (22002) will enter subjects who complete Study 22001 into a long-term, open-label study.The open-label extension protocol will provide necessary data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions more closely reflective of their general medical care.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| STX209 | Active comparator | Drug: Arbaclofen |
Who may qualify
- Ages
- 6 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Have completed all scheduled visits in protocol 22001 and have shown they can adequately follow the protocol, with sufficient medical justification to continue on open-label treatment with STX209, as assessed by the principal investigator
Key exclusion criteria (2 listed)
- Subjects with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being.
- The occurrence or continuation of any adverse event or condition during study 22001 that, in the opinion of the Investigator, should exclude the subject from participating in this open-label extension.
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, STX209A flexible dose titration will be utilized during the first four weeks to define the optimal titrated dose (OTD) for each subject. Investigators will use clinical judgment to adjust doses to the OTD. The starting dose will be 1 mg BID. The dose may be increased every four to five days to 2 mg BID, 3 mg BID, 5 mg BID and then 10 mg BID | Drug | STX209 |
Primary outcome
Irritability subscale of the Aberrant Behavior Checklist
Time frame: 1 year
Results and safety
The study was stopped early (reason given: all active subjects rolled into 209FX303/NCT01013480). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Dec 2012.
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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.
The record lists no contact. Use the official record to reach the sponsor.
9 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 Arbaclofen in people with Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 9 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 1 inclusion and 2 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: stopped early, as of the sponsor's last update on 20 Dec 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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. 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?
The study was stopped early (reason given: all active subjects rolled into 209FX303/NCT01013480). Results are not available in the source record.
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?
Irritability subscale of the Aberrant Behavior Checklist (time frame: 1 year). 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 20 Dec 2012.