Ignite Creation Date:
2025-12-24 @ 7:00 PM
Ignite Modification Date:
2026-04-15 @ 7:38 PM
Study NCT ID:
NCT01750957
Status:
COMPLETED
Last Update Posted:
2016-08-11
First Post:
2012-12-11
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study of RO4917523 in Pediatric Patients 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': 'C000596770', 'term': '2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 47}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-08', 'dispFirstSubmitDate': '2016-08-09', 'completionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-08-09', 'studyFirstSubmitDate': '2012-12-11', 'dispFirstSubmitQcDate': '2016-08-09', 'studyFirstSubmitQcDate': '2012-12-13', 'dispFirstPostDateStruct': {'date': '2016-08-11', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2016-08-11', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2012-12-17', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety: Incidence of adverse events', 'timeFrame': '15 weeks'}], 'secondaryOutcomes': [{'measure': 'Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior)', 'timeFrame': '15 weeks'}, {'measure': 'Pharmacokinetics: Clearance (CL/F)', 'timeFrame': 'up to Week 12'}, {'measure': 'Pharmacokinetics: Volume of distribution at steady-state (Vss/F)', 'timeFrame': 'up to Week 12'}]}, 'conditionsModule': {'conditions': ['Fragile X Syndrome']}, 'descriptionModule': {'briefSummary': 'This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '13 Years', 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Children and adolescents, 5 to 13 years of age\n* Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S\n\nExclusion Criteria:\n\n* Previous treatment with another mGlu5 receptor antagonist within the prior 3 months\n* Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study\n* Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome\n* History of suicidal behavior\n* Other protocol defined inclusion/exclusion criteria may apply'}, 'identificationModule': {'nctId': 'NCT01750957', 'briefTitle': 'A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Hoffmann-La Roche'}, 'officialTitle': 'A Randomized, Parallel Group, Double-Blind, Placebo-Controlled, Safety and Exploratory Efficacy and Pharmacokinetic, Study of RO4917523 in Pediatric Patients With Fragile X Syndrome', 'orgStudyIdInfo': {'id': 'NP28571'}, 'secondaryIdInfos': [{'id': '2011-004349-42', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'RO4917523 Dose A', 'interventionNames': ['Drug: RO4917523']}, {'type': 'EXPERIMENTAL', 'label': 'RO4917523 Dose B', 'interventionNames': ['Drug: RO4917523']}], 'interventions': [{'name': 'Placebo', 'type': 'DRUG', 'description': 'orally daily, 12 weeks', 'armGroupLabels': ['Placebo']}, {'name': 'RO4917523', 'type': 'DRUG', 'description': 'Dose A orally daily, 12 weeks', 'armGroupLabels': ['RO4917523 Dose A']}, {'name': 'RO4917523', 'type': 'DRUG', 'description': 'Dose B orally daily, 12 weeks', 'armGroupLabels': ['RO4917523 Dose B']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90806', 'city': 'Long Beach', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 33.76696, 'lon': -118.18923}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '92108', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '20020', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '40202', 'city': 'Louisville', 'state': 'Kentucky', 'country': 'United States', 'geoPoint': {'lat': 38.25424, 'lon': -85.75941}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '01605', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'geoPoint': {'lat': 42.26259, 'lon': -71.80229}}, {'zip': '68198-4160', 'city': 'Omaha', 'state': 'Nebraska', 'country': 'United States', 'geoPoint': {'lat': 41.25626, 'lon': -95.94043}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '19063', 'city': 'Media', 'state': 'Pennsylvania', 'country': 'United States', 'geoPoint': {'lat': 39.91678, 'lon': -75.38769}}, {'zip': '29646', 'city': 'Greenwood', 'state': 'South Carolina', 'country': 'United States', 'geoPoint': {'lat': 34.1954, 'lon': -82.16179}}, {'zip': '37212', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '78258', 'city': 'San Antonio', 'state': 'Texas', 'country': 'United States', 'geoPoint': {'lat': 29.42412, 'lon': -98.49363}}, {'zip': '98145', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}], 'overallOfficials': [{'name': 'Clinical Trials', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Hoffmann-La Roche'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hoffmann-La Roche', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}