Fragile X SyndromeNCT00823368
Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001
An observational study of Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 11 sites in United States.
Trial summary
The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001 will be offered participation in 22001A which will evaluate secreted protein before and after treatment with STX209 and placebo to determine if they correlate with effectiveness of treatment or susceptibility to… 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
- How many people
- Not given
- Where
- 11 sitesUnited States
- Study dates
- Jan 2009to Mar 2010
- Phase
- Observational
- 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
Study population: Fragile x syndrome subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001
Who may be able to join
- Subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001
Who cannot join
- Subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 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
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
About this study
Official title: Biomarker Testing and DNA Collection in Subjects Participating in 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
The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001 will be offered participation in 22001A which will evaluate secreted protein before and after treatment with STX209 and placebo to determine if they correlate with effectiveness of treatment or susceptibility to treatment with STX209. These same subjects will also be asked to contribute a blood sample for DNA (deoxyribonucleic acid) collection. The investigators will study the DNA to determine if STX209 works better in people with specific gene variations, or to find new gene variations that predict how well STX209 works.
- Conditions
- Fragile X Syndrome
- Being tested
- STX209
- Keywords
- fragile X syndrome, biomarkers
- 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.
STX209Drug
| Study group | Role | What they receive |
|---|---|---|
| Arbaclofen followed by Placebo | Not described Receives: Drug: STX209 | |
| Placebo followed by Arbaclofen | Not described Receives: Drug: STX209 |
What is being measured
The primary outcome is the main question the study is designed to answer.
change in plasma proteins with treatment
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.
- First posted15 Jan 2009 (estimated)
- Study startJan 2009
- Primary completionMar 2010
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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
Sponsor and investigators
- Lead sponsor
- Seaside Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elizabeth Berry-Kravis, MD, PhD, Principal investigator, Rush University Medical CenterRandi Hagerman, MD, Principal investigator, M.I.N.D. InstituteCraig Erikson, MD, Principal investigator, Riley Hospital for ChildrenBryan King, MD, PhD, Principal investigator, Seattle Children's HospitalJames McCracken, MD, Principal investigator, University of California, Los AngelesJonathan Picker, MBChB, PhD, Principal investigator, Boston Children's HospitalLinmarie Sikich, MD, Principal investigator, University of North Carolina Neurosciences HospitalJeremy Veenstra-VanderWeele, MD, Principal investigator, Vanderbilt Kennedy Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00823368
- Sponsor's protocol ID
- 22001A
- Status last verified
- Jul 2013
- First submitted
- 13 Jan 2009
- 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
- Biomarker Testing and DNA Collection in Subjects Participating in 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
- NCT00823368
- Acronym / sponsor ID
- 22001A
- Conditions
- Fragile X Syndrome
- Keywords
- fragile X syndrome, biomarkers
- Study type
- Observational
- Recruitment status
- Completed
- Lead sponsor
- Seaside Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Jan 2009
- Primary completion
- Mar 2010 (actual)
- First posted
- 15 Jan 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.
The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001 will be offered participation in 22001A which will evaluate secreted protein before and after treatment with STX209 and placebo to determine if they correlate with effectiveness of treatment or susceptibility to treatment with STX209. These same subjects will also be asked to contribute a blood sample for DNA (deoxyribonucleic acid) collection. The investigators will study the DNA to determine if STX209 works better in people with specific gene variations, or to find new gene variations that predict how well STX209 works.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arbaclofen followed by Placebo | – | Drug: STX209 |
| Placebo followed by Arbaclofen | – | Drug: STX209 |
Who may qualify
- Ages
- 6 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Fragile x syndrome subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001
Key inclusion criteria (1 listed)
- Subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001
Key exclusion criteria (1 listed)
- Subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001
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 |
|---|---|---|
| STX209 | Drug | Arbaclofen followed by Placebo, Placebo followed by Arbaclofen |
Primary outcome
change in plasma proteins with treatment
Time frame: After 4 weeks of treatment
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
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
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fragile X Syndrome, run by Seaside Therapeutics, Inc. at 11 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 1 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 11 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.
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?
change in plasma proteins with treatment (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?
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.