Ignite Creation Date:
2025-12-25 @ 4:09 AM
Ignite Modification Date:
2025-12-26 @ 3:07 AM
Study NCT ID:
NCT01325220
Status:
COMPLETED
Last Update Posted:
2013-07-31
First Post:
2011-03-28
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005600', 'term': 'Fragile X Syndrome'}], 'ancestors': [{'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D025064', 'term': 'Sex Chromosome Disorders'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C543531', 'term': 'arbaclofen placarbil'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 172}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-07', 'lastUpdateSubmitDate': '2013-07-30', 'studyFirstSubmitDate': '2011-03-28', 'studyFirstSubmitQcDate': '2011-03-28', 'lastUpdatePostDateStruct': {'date': '2013-07-31', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-03-29', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale', 'timeFrame': 'at 8 weeks of treatment', 'description': 'This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Fragile X Syndrome']}, 'descriptionModule': {'briefSummary': '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'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Molecular documentation of the full FMR1 mutation\n* Current pharmacological treatment regimen has been stable for at least 4 weeks prior to Screening.\n* 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.\n* 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\n\nExclusion Criteria:\n\n* 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.\n* Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.\n* Subjects who have taken another investigational drug within the last 30 days.\n* Subjects who are not able to take oral medications'}, 'identificationModule': {'nctId': 'NCT01325220', 'acronym': 'Harbor-C', 'briefTitle': 'Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Seaside Therapeutics, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': '209FX302'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'STX209 5 mg BID', 'interventionNames': ['Drug: arbaclofen']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'STX209 10 mg BID', 'interventionNames': ['Drug: arbaclofen']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'STX209 10 mg TID', 'interventionNames': ['Drug: arbaclofen']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'arbaclofen', 'type': 'DRUG', 'otherNames': ['R-baclofen, STX209'], 'description': '5 mg bid', 'armGroupLabels': ['STX209 5 mg BID']}, {'name': 'arbaclofen', 'type': 'DRUG', 'otherNames': ['R-baclofen, STX209'], 'description': '10 mg bid', 'armGroupLabels': ['STX209 10 mg BID']}, {'name': 'arbaclofen', 'type': 'DRUG', 'otherNames': ['R-baclofen, STX209'], 'description': '10 mg tid', 'armGroupLabels': ['STX209 10 mg TID']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'tid', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85006', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Southwest Autism Research & Resource Center', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '90806', 'city': 'Long Beach', 'state': 'California', 'country': 'United States', 'facility': "Miller Children's Hospital", 'geoPoint': {'lat': 33.76696, 'lon': -118.18923}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'University of California-Davis, M.I.N.D. Institute', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '92108', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': 'Psychiatric Centers at San Diego', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "University of Colorado Denver, Children's Hospital", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami, Mailman Center for Child Development', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '32763', 'city': 'Orange City', 'state': 'Florida', 'country': 'United States', 'facility': 'Lake Mary Pediatrics', 'geoPoint': {'lat': 28.94888, 'lon': -81.29867}}, {'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University School of Medicine', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Rush University Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '66205', 'city': 'Fairway', 'state': 'Kansas', 'country': 'United States', 'facility': 'Kansas University Clinical Research Center', 'geoPoint': {'lat': 39.02223, 'lon': -94.6319}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '01605', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'University of Massachusetts', 'geoPoint': {'lat': 42.26259, 'lon': -71.80229}}, {'zip': '48201', 'city': 'Detriot', 'state': 'Michigan', 'country': 'United States', 'facility': "Children's Hospital of Michigan"}, {'zip': '65211', 'city': 'Columbia', 'state': 'Missouri', 'country': 'United States', 'facility': 'University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders', 'geoPoint': {'lat': 38.95171, 'lon': -92.33407}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Seaver Autism Center, Mount Sinai Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10314', 'city': 'Staten Island', 'state': 'New York', 'country': 'United States', 'facility': 'New York State Institute for Basic Research in Developmental Disabilities', 'geoPoint': {'lat': 40.56233, 'lon': -74.13986}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke Clinical Research Unit', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44308', 'city': 'Akron', 'state': 'Ohio', 'country': 'United States', 'facility': "Akron Children's Hospital", 'geoPoint': {'lat': 41.08144, 'lon': -81.51901}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '73117', 'city': 'Oklahoma City', 'state': 'Oklahoma', 'country': 'United States', 'facility': "University of Oklahoma, Physician's Child Study Center", 'geoPoint': {'lat': 35.46756, 'lon': -97.51643}}, {'zip': '19063', 'city': 'Media', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Suburban Research Associates/Elwyn Genetics', 'geoPoint': {'lat': 39.91678, 'lon': -75.38769}}, {'zip': '29646', 'city': 'Greenwood', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Greenwood Genetics Center', 'geoPoint': {'lat': 34.1954, 'lon': -82.16179}}, {'zip': '37203', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt Kennedy Center', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '75235', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Childrens Medical Center Dallas', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '77090', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Red Oaks Psychiatry Associates, P.A.', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '78258', 'city': 'San Antonio', 'state': 'Texas', 'country': 'United States', 'facility': 'Road Runner Research', 'geoPoint': {'lat': 29.42412, 'lon': -98.49363}}, {'zip': '98101', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}], 'overallOfficials': [{'name': 'Paul Wang, M.D.', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Seaside Therapeutics, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Seaside Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}