Fragile X SyndromePhase 3209FX303NCT01555333
An Open Label Extension Study in Subjects With Fragile X Syndrome
A Phase 3 study testing arbaclofen in people with Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 23 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 5 to 50 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- arbaclofen
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 357 participants
- Where
- 23 sitesUnited States
- Study dates
- Nov 2011to Jul 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 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
23 sites in 1 country.
23 sites in 1 country
United States23 sites
Seaside Therapeutics Site #05
Akron, Ohio, 44308
Seaside Therapeutics Site #17
Aurora, Colorado, 80045
Seaside Therapeutics Site #12
Baltimore, Maryland, 21205
Seaside Therapeutics Site #24
Chapel Hill, North Carolina, 27514
Seaside Therapeutics Site #02
Chicago, Illinois, 60612
Seaside Therapeutics Site #03
Columbia, Missouri, 65211
Seaside Therapeutics Site #20
Decatur, Georgia, 30033
Seaside Therapeutics Site #21
Durham, North Carolina, 27710
Seaside Therapeutics Site #25
Houston, Texas, 77090
Seaside Therapeutics Site #23
Kansas City, Kansas, 66160
Seaside Therapeutics Site #07
Long Beach, California, 90806
Seaside Therapeutics Site #11
Media, Pennsylvania, 19063
Seaside Therapeutics Site #01
Miami, Florida, 33136
Seaside Therapeutics Site #19
Nashville, Tennessee, 37212
Seaside Therapeutics Site #22
New York, New York, 10029
Seaside Therapeutics Site #15
Oklahoma City, Oklahoma, 73117
Seaside Therapeutics Site #14
Orange City, Florida, 32763
Seaside Therapeutics Site #16
Phoenix, Arizona, 85006
Seaside Therapeutics Site #10
Sacramento, California, 95817
Seaside Therapeutics Site #18
San Antonio, Texas, 78258
Seaside Therapeutics Site #13
Seattle, Washington, 98121
Seaside Therapeutics Site #04
Staten Island, New York, 10314
Seaside Therapeutics Site #08
Worcester, Massachusetts, 01605
About this study
Official title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics in Subject With Fragile X Syndrome
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).
Read the detailed description
Three studies sponsored by Seaside Therapeutics, Inc., are currently evaluating the efficacy of STX209 for management of typical problem behaviors in subjects with FXS. These are Study 209FX301, "A Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Adolescents and Adults with Fragile X Syndrome;" Study 209FX302, "A Randomized, Double-Blind, Placebo-Controlled, Fixed- Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Children with Fragile X Syndrome;" and Study 22002, "An Open-Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects with Fragile X Syndrome." This study will enroll subjects who have completed Protocols 209FX301, 209FX302, or are currently participating in Protocol 22002 into a long-term, open-label study. The open-label extension protocol will provide data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions reflective of their typical medical care rather than in their previously completed study.
- Conditions
- Fragile X Syndrome
- Being tested
- arbaclofen
- 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 STX209
A flexible dose titration will be utilized. Orally disintegrating tablets
| Study group | Role | What they receive |
|---|---|---|
| Arbaclofen | Experimental | Open Label Study Receives: Drug: arbaclofen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety Measures
Adverse Events, Suicidality Assessment, Physical Examination, Vital Signs and Weight, Laboratory Tests: Complete Blood Count, Urinalysis, Chemistry Panel
Measured: 100 weeks
Secondary outcomes (1)
Efficacy
Aberrant Behavior Checklist Lethargy/Social Withdrawal Sub- Scale Clinical Global Impression Vineland Adaptive Behavior Scales Stanford Binet Intelligence Scale
Measured: 100 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2012 (estimated)
- Study startNov 2011
- Primary completionJul 2013
- Study completionJul 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 357 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
- NCT01555333
- Sponsor's protocol ID
- 209FX303
- Acronym
- 209FX303
- Status last verified
- Jul 2013
- First submitted
- 1 Mar 2012
- 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
- An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics in Subject With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT01555333
- Acronym / sponsor ID
- 209FX303 / 209FX303
- Conditions
- Fragile X Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 357 participants (actual)
- Lead sponsor
- Seaside Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2011
- Primary completion
- Jul 2013 (actual)
- Study completion
- Jul 2013 (actual)
- First posted
- 15 Mar 2012 (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.
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).
Detailed description
Three studies sponsored by Seaside Therapeutics, Inc., are currently evaluating the efficacy of STX209 for management of typical problem behaviors in subjects with FXS. These are Study 209FX301, "A Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Adolescents and Adults with Fragile X Syndrome;" Study 209FX302, "A Randomized, Double-Blind, Placebo-Controlled, Fixed- Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Children with Fragile X Syndrome;" and Study 22002, "An Open-Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects with Fragile X Syndrome." This study will enroll subjects who have completed Protocols 209FX301, 209FX302, or are currently participating in Protocol 22002 into a long-term, open-label study. The open-label extension protocol will provide data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions reflective of their typical medical care rather than in their previously completed study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arbaclofen | Experimental | Drug: arbaclofenOpen Label Study |
Who may qualify
- Ages
- 5 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
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: STX209A flexible dose titration will be utilized. Orally disintegrating tablets | Drug | Arbaclofen |
Primary outcome
Safety Measures
Time frame: 100 weeks
Adverse Events, Suicidality Assessment, Physical Examination, Vital Signs and Weight, Laboratory Tests: Complete Blood Count, Urinalysis, Chemistry Panel
1 secondary outcome
Efficacy
Time frame: 100 Weeks
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2013.
Related trials for Fragile X Syndrome
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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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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.
Paul Wang, M.D. study director
Seaside Therapeutics, Inc.
23 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 23 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 8 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 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 23 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 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 was stopped early. 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?
Safety Measures (time frame: 100 weeks). 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.