Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)
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| ClinicalTrials.gov Identifier: NCT01325220 |
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Recruitment Status :
Completed
First Posted : March 29, 2011
Last Update Posted : July 31, 2013
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Sponsor:
Seaside Therapeutics, Inc.
Information provided by (Responsible Party):
Seaside Therapeutics, Inc.
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Brief Summary:
There will be four study periods: Screening (up to 14 days in length), the Treatment Period (8 weeks), the Withdrawal Period (22 days), and Follow-up Period (up to 31 days). Subjects will be randomized to receive either STX209 (5 mg twice daily [BID], 10 mg BID or 10 mg three times daily [TID]) or placebo. Efficacy,safety and tolerability assessments will be performed periodically
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Fragile X Syndrome | Drug: arbaclofen Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 172 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Children With Fragile X Syndrome |
| Study Start Date : | June 2011 |
| Actual Primary Completion Date : | June 2013 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Fragile X syndrome
MedlinePlus related topics:
Fragile X Syndrome
| Arm | Intervention/treatment |
|---|---|
| Active Comparator: STX209 5 mg BID |
Drug: arbaclofen
5 mg bid
Other Name: R-baclofen, STX209 |
| Active Comparator: STX209 10 mg BID |
Drug: arbaclofen
10 mg bid
Other Name: R-baclofen, STX209 |
| Active Comparator: STX209 10 mg TID |
Drug: arbaclofen
10 mg tid
Other Name: R-baclofen, STX209 |
| Placebo Comparator: Placebo |
Drug: Placebo
tid |
Primary Outcome Measures :
- Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale [ Time Frame: at 8 weeks of treatment ]This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal
Information from the National Library of Medicine
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| Ages Eligible for Study: | 5 Years to 11 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Molecular documentation of the full FMR1 mutation
- Current pharmacological treatment regimen has been stable for at least 4 weeks prior to Screening.
- Subjects with a history of seizure disorder must currently be receiving treatment with antiepileptics and must have been seizure free for 6 months, or must be seizure free for 3 years if not currently receiving antiepileptics.
- If the subject is already receiving stable non-pharmacologic educational, behavioral, and/or dietary interventions, participation in these programs must have been continuous during the 2 months prior to Screening
Exclusion Criteria:
- 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.
- Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
- Subjects who have taken another investigational drug within the last 30 days.
- Subjects who are not able to take oral medications
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01325220
Locations
Show 28 study locations
Sponsors and Collaborators
Seaside Therapeutics, Inc.
Investigators
| Study Director: | Paul Wang, M.D. | Seaside Therapeutics, Inc. |
| Responsible Party: | Seaside Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT01325220 |
| Other Study ID Numbers: |
209FX302 |
| First Posted: | March 29, 2011 Key Record Dates |
| Last Update Posted: | July 31, 2013 |
| Last Verified: | July 2013 |
Additional relevant MeSH terms:
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Fragile X Syndrome Syndrome Disease Pathologic Processes Mental Retardation, X-Linked Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases Sex Chromosome Disorders Chromosome Disorders Congenital Abnormalities Genetic Diseases, Inborn |
Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Baclofen Muscle Relaxants, Central Physiological Effects of Drugs Neuromuscular Agents Peripheral Nervous System Agents GABA-B Receptor Agonists GABA Agonists GABA Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action |