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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. We also hypothesize that STX209 will be safe and well tolerated.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| STX209 | Active Comparator | STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks |
|
| Placebo | Placebo Comparator | variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| STX209 | Drug | Variable dose from 1 mg bid to 10 mg tid, Capsule, Oral, 4 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | After 4 weeks of treatment |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Elizabeth Berry-Kravis, MD, PhD | Rush University Medical Center | Principal Investigator |
| Randi Hagerman, MD | M.I.N.D. Institute | Principal Investigator |
| Craig Erikson, MD | Riley Hospital for Children | Principal Investigator |
| Bryan King, MD, PhD | Seattle Children's Hospital | Principal Investigator |
| James McCracken, MD | University of California, Los Angeles | Principal Investigator |
| Jonathan Picker, MBChB, PhD | Boston Children's Hospital | Principal Investigator |
| Linmarie Sikich, MD | University of North Carolina Neurosciences Hospital | Principal Investigator |
| Jeremy Veenstra-VanderWeele, MD | Vanderbilt Kennedy Center | Principal Investigator |
| Ted Brown, MD, PhD | NYS institute for Basic Research in Developmental Disabilities | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Southwest Autism Research & Resource Center | Phoenix | Arizona | 85006 | United States | ||
| University of California-Los Angeles Neuropsychiatric Institute |
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| ID | Title | Description |
|---|---|---|
| FG000 | STX209:Placebo | First Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral) Second Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral) Study Design: Placebo-controlled, Crossover study. First Intervention(28 Days)-> Withdrawal(14 Days) -> Washout(7 Days)-> Withdrawal (14 Days) Participants received all interventions. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| STX209 Then Placebo |
|
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| Placebo | Drug | variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks |
|
| Lawrence Ginsberg, MD | Red Oaks Psychiatry Associates, PA | Principal Investigator |
| Shivkumar Hatti, MD | Suburban Research Associates | Principal Investigator |
| Raun Melmed, MD | Southwest Autism Research & Resource Center | Principal Investigator |
| Los Angeles |
| California |
| 90024 |
| United States |
| M.I.N.D. Institute | Sacramento | California | 95817 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | United States |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| NYS Institute for Basic Research in Developmental Disabilities | Staten Island | New York | 10314 | United States |
| University of North Carolina Neurosciences Hospital | Chapel Hill | North Carolina | 27514 | United States |
| Suburban Research Associates | Media | Pennsylvania | 19063 | United States |
| Vanderbilt Kennedy Center | Nashville | Tennessee | 37203 | United States |
| Red Oaks Psychiatry Associates, P.A. | Houston | Texas | 77090 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98101 | United States |
| FG001 | Placebo:STX209 | First Intervention=Placebo (Placebo variable dose (same flexible dose titration protocol) bid to tid, oral) Second Intervention=STX209 (STX209 variable dose from 1mg bid to 10mg tid, capsule, oral) Study Design: Placebo-controlled, Crossover study. First Intervention(28 Days)-> Withdrawal(14 Days) -> Washout(7 Days)-> Withdrawal (14 Days) Participants received all interventions. |
| COMPLETED |
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| NOT COMPLETED |
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| Placebo Then STX209 |
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| ID | Title | Description |
|---|---|---|
| BG000 | STX209 | STX209 Variable dose, 1mg bid to 10mg tid, oral capsules, 4weeks |
| BG001 | Placebo | placebo variable dose (same flexible dose titration protocol) bid to tid, oral, 4weeks |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | Posted | Least Squares Mean | Standard Error | Points on a scale | After 4 weeks of treatment |
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| Post-Hoc | ABC-FXS Social Avoidance Subscore | After completion of the study, but during data analysis, the ABC-C assessment was independently re-validated in Fragile X Syndrome subjects. The subscales were re-factored into a Fragile-X Syndrome specific ABC-C (ABC-FX). The ABC-FX contains the same 58 questions as the original ABC-C but there are six subscales. One of the subscales is Social Avoidance, which consists of 4 items. Minimum score is 0, maximum is 12. A decreased score indicates fewer social avoidant behaviors. A post-hoc analysis was performed from the study data examining the social avoidance subscale of the ABC-FX. | Posted | Least Squares Mean | Standard Error | Points on a scale | 4 week treatment period |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | STX209 | STX209 variable dose from 1mg bid to 10mg tid, capsule, oral, 4 weeks | 1 | 63 | 42 | 63 | ||
| EG001 | Placebo | variable dose (same flexible dose titration protocol), bid to tid, capsule, Oral, 4 weeks | 0 | 63 | 31 | 63 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| increased irritability | Psychiatric disorders | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| upper respiratory tract infection | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| ||
| headache | Nervous system disorders | Non-systematic Assessment |
| ||
| sedation | Nervous system disorders | Non-systematic Assessment |
| ||
| fatigue | General disorders | Non-systematic Assessment |
| ||
| irritability | Psychiatric disorders | Non-systematic Assessment |
| ||
| diarrhea | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| increased appetite | Metabolism and nutrition disorders | Non-systematic Assessment |
| ||
| vomiting | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| aggression | Psychiatric disorders | Non-systematic Assessment |
| ||
| nasopharyngitis | Infections and infestations | Non-systematic Assessment |
|
Seaside Therapeutics agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing, but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Paul Wang, Vice President of Clinical and Medical Affairs | Seaside Therapeutics | 617-374-9009 | 1015 | pwang@seasidetherapeutics.com |
| ID | Term |
|---|---|
| D005600 | Fragile X Syndrome |
| D001523 | Mental Disorders |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D025064 | Sex Chromosome Disorders |
| D025063 | Chromosome Disorders |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D040181 | Genetic Diseases, X-Linked |
| D020271 | Heredodegenerative Disorders, Nervous System |
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| ID | Term |
|---|---|
| C543531 | arbaclofen placarbil |
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| >=65 years |
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| Male |
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