Ignite Creation Date:
2025-12-24 @ 7:02 PM
Ignite Modification Date:
2026-01-02 @ 1:19 PM
Study NCT ID:
NCT00593957
Status:
TERMINATED
Last Update Posted:
2014-04-23
First Post:
2008-01-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Trial of Dextromethorphan in Rett Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015518', 'term': 'Rett 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': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003915', 'term': 'Dextromethorphan'}], 'ancestors': [{'id': 'D009019', 'term': 'Morphinans'}, {'id': 'D053610', 'term': 'Opiate Alkaloids'}, {'id': 'D000470', 'term': 'Alkaloids'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D006572', 'term': 'Heterocyclic Compounds, Bridged-Ring'}, {'id': 'D006576', 'term': 'Heterocyclic Compounds, 4 or More Rings'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D010616', 'term': 'Phenanthrenes'}, {'id': 'D011084', 'term': 'Polycyclic Aromatic Hydrocarbons'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'naidu@kennedykrieger.org', 'phone': '443-923-2778', 'title': 'Dr Sakkubai Naidu Professor of Neurology and Pediatrics', 'organization': 'Kennedy Krieger Institute'}, 'certainAgreement': {'piSponsorEmployee': True, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'The trial was terminated due to the FDA requiring a placebo controlled trial instead of the ongoing open label trial. As recruitment was delayed,the total number of participants was also less than anticipated.'}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'DM1( 0.25 mg/kg /Day)', 'description': 'Dextromethorphan 0.25 mg/kg per day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.', 'otherNumAtRisk': 13, 'otherNumAffected': 0, 'seriousNumAtRisk': 13, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'DM2 (2.5 mg/kg/Day)', 'description': 'Dextromethorphan 2.5 mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.', 'otherNumAtRisk': 12, 'otherNumAffected': 0, 'seriousNumAtRisk': 12, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'DM3 (5mg/kg/Day)', 'description': 'Dextromethorphan 5mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.', 'otherNumAtRisk': 10, 'otherNumAffected': 0, 'seriousNumAtRisk': 10, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Difference in EEG Spike Counts at Six Months Compared to Baseline for Each Treatment Arm.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '13', 'groupId': 'OG003'}, {'value': '12', 'groupId': 'OG004'}, {'value': '8', 'groupId': 'OG005'}]}], 'groups': [{'id': 'OG000', 'title': 'Dextromethorphan (DM)1 EEG Spike Counts at Baseline', 'description': 'Dextromethorphan(DM)I group received Dextromethorphan 0.25 mg/kg per day. The drug is given in two divided doses 12 hours apart for 6 months. EEG spike count for DM1 group measured at baseline.'}, {'id': 'OG001', 'title': 'DM2 EEG Spike Counts at Baseline', 'description': 'DM2 group participants received Dextromethorphan 2.5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months. DM2 EEG spike counts at baseline.'}, {'id': 'OG002', 'title': 'DM3 EEG Spike Counts at Baseline', 'description': 'DM3 group received Dextromethorphan 5mg/kg/day.\n\nThe drug is given in two divided doses 12 hours apart for 6 months. EEG spike counts at baseline.'}, {'id': 'OG003', 'title': 'DM1 EEG Spike Count at 6 Months', 'description': 'DM1 group received Dextromethorphan 0.25 mg/kg per day. The drug is given in two divided doses 12 hours apart for 6 months. EEG spike count for DM1 group measured at 6 months.'}, {'id': 'OG004', 'title': 'DM2 EEG Spike Counts at 6 Months', 'description': 'DM2 group participants received Dextromethorphan 2.5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months. DM2 EEG spike counts at 6 months.'}, {'id': 'OG005', 'title': 'DM3 EEG Spike Counts at 6 Months', 'description': 'DM3 group received Dextromethorphan 5mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months. EEG spike counts at 6 months.'}], 'classes': [{'categories': [{'measurements': [{'value': '44.22', 'spread': '23.77', 'groupId': 'OG000'}, {'value': '25.44', 'spread': '18.6', 'groupId': 'OG001'}, {'value': '40.67', 'spread': '27.83', 'groupId': 'OG002'}, {'value': '38.29', 'spread': '30.24', 'groupId': 'OG003'}, {'value': '27.58', 'spread': '19.52', 'groupId': 'OG004'}, {'value': '36.01', 'spread': '23.29', 'groupId': 'OG005'}]}]}], 'analyses': [{'pValue': '<0.40', 'groupIds': ['OG000', 'OG003'], 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '<0.62', 'groupIds': ['OG001', 'OG004'], 'ciPctValue': '95', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '<0.38', 'groupIds': ['OG002', 'OG005'], 'ciPctValue': '95', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': 'Initial and 6-month post-treatment', 'description': 'Difference in EEG spike count means pre and 6 months post-treatment in each of three treatment groups.', 'unitOfMeasure': 'EEG spike counts per minute', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': '33/35 participants who completed the protocol with two epochs of 5 mins of non-Rapid eye movement (REM)sleep during which spikes could be counted pre and post DM intake.'}, {'type': 'SECONDARY', 'title': 'Improvement in Receptive Language as Measured by the Mullen Scale.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'DM1( 0.25 mg/kg /Day)', 'description': 'Dextromethorphan 0.25 mg/kg per day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}, {'id': 'OG001', 'title': 'DM2 (2.5 mg/kg/Day)', 'description': 'Dextromethorphan 2.5 mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}, {'id': 'OG002', 'title': 'DM3 (5mg/kg/Day)', 'description': 'Dextromethorphan 5mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'spread': '2.94', 'groupId': 'OG000'}, {'value': '-0.44', 'spread': '2.96', 'groupId': 'OG001'}, {'value': '03', 'spread': '2.45', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Change in mean between Initial and 6-month follow-up', 'description': 'The Mullen Receptive language scale pre and 6 months post DM, measured as a change in the mean score of language, by age in months.', 'unitOfMeasure': 'age in months', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': '25/35 enrolled participants completed the receptive language scale of the Mullen and underwent the analysis.'}, {'type': 'SECONDARY', 'title': 'Difference in SSI Mean Score at Six Months Compared to Baseline for Each Treatment Arm.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}, {'value': '4', 'groupId': 'OG004'}, {'value': '7', 'groupId': 'OG005'}]}], 'groups': [{'id': 'OG000', 'title': 'DM1( 0.25 mg/kg /Day) SSI Baseline', 'description': 'Screen for Social Interaction (SSI) mean score at baseline for Dextromethorphan 0.25 mg/kg per day treatment arm.'}, {'id': 'OG001', 'title': 'DM2 (2.5 mg/kg/Day)SSI Baseline', 'description': 'Screen for Social Interaction (SSI) mean score at baseline for Dextromethorphan 2.5 mg/kg/day treatment arm.'}, {'id': 'OG002', 'title': 'DM3 (5mg/kg/Day) SSI Baseline', 'description': 'Screen for Social Interaction (SSI) mean score at baseline for Dextromethorphan 5mg/kg/day treatment arm.'}, {'id': 'OG003', 'title': 'DM1( 0.25 mg/kg /Day) SSI 6 Months', 'description': 'DM1( 0.25 mg/kg /day)treatment arm Screen for Social Interaction (SSI) mean score at 6 months post-treatment.'}, {'id': 'OG004', 'title': 'DM2 (2.5 mg/kg/Day) SSI 6 Months', 'description': 'DM2( 2.5 mg/kg /day)treatment arm Screen for Social Interaction (SSI) mean score at 6 months post-treatment.'}, {'id': 'OG005', 'title': 'DM3 (5.0 mg/kg/Day) SSI 6 Months', 'description': 'DM3(5.0) mg/kg /day)treatment arm Screen for Social Interaction (SSI) mean score at 6 months post-treatment.'}], 'classes': [{'categories': [{'measurements': [{'value': '40.25', 'spread': '18.59', 'groupId': 'OG000'}, {'value': '70.00', 'spread': '19.10', 'groupId': 'OG001'}, {'value': '84.29', 'spread': '17.51', 'groupId': 'OG002'}, {'value': '48.63', 'spread': '18.58', 'groupId': 'OG003'}, {'value': '74.50', 'spread': '22.78', 'groupId': 'OG004'}, {'value': '88.29', 'spread': '18.88', 'groupId': 'OG005'}]}]}], 'analyses': [{'pValue': '<0.10', 'groupIds': ['OG000', 'OG003'], 'paramType': 'Mean Difference (Final Values)', 'paramValue': '8.38', 'groupDescription': 'Null hypothesis is that there would be no significant difference in social abilities as measured by the SSI score in this treatment group baseline to 6 months post-treatment.', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '<0.50', 'groupIds': ['OG001', 'OG004'], 'paramType': 'Mean Difference (Final Values)', 'paramValue': '4.5', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '<0.48', 'groupIds': ['OG002', 'OG005'], 'paramType': 'Mean Difference (Final Values)', 'paramValue': '4.0', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': 'Initial and 6 month followup', 'description': 'The Screen for Social Interaction (SSI) is a 54-item parent/caregiver-report screening instrument that emphasizes reciprocal social interaction including joint attention skills. The items are positive (prosocial) and are scored on a four-point frequency scale (child displays the behavior "almost never" = 0 to "almost all the time" = 3). Thus lower scores reflect a slower or delayed development, and higher scores reflect more normative development. SSI total scores range from 0-162. There are no subscales. Difference in Screen for Social Interaction (SSI) mean scores between baseline and 6 months post-treatment for each treatment arm are reported.', 'unitOfMeasure': 'scores on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Those who provided complete information only were included. Others did not provide adequate or complete information for analysis.'}, {'type': 'SECONDARY', 'title': 'Mean SSI Score for Total Subjects at Baseline and 6 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Total Sample SSI Mean Score at Baseline', 'description': 'Study Sample Screen for Social Interaction (SSI) mean core at baseline.'}, {'id': 'OG001', 'title': 'Total Sample SSI Mean Score at 6 Months', 'description': 'Study Sample Screen for Social Interaction (SSI) mean score at 6 months post-treatment.'}], 'classes': [{'categories': [{'measurements': [{'value': '62.737', 'spread': '26.729', 'groupId': 'OG000'}, {'value': '68.684', 'spread': '25.987', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.05', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'paramValue': '5.947', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': '0-6 months', 'description': 'Analysis of Difference in Mean Screen for Social Interaction (SSI) Score between 0-6 months for total sample (n=19).', 'unitOfMeasure': 'scores on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': '19 subjects (total sample) for whom complete data was available'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'DM1( 0.25 mg/kg /Day)', 'description': 'Dextromethorphan 0.25 mg/kg per day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}, {'id': 'FG001', 'title': 'DM2 (2.5 mg/kg/Day)', 'description': 'Dextromethorphan 2.5 mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}, {'id': 'FG002', 'title': 'DM3 (5mg/kg/Day)', 'description': 'Dextromethorphan 5mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '13'}, {'groupId': 'FG002', 'numSubjects': '12'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '12'}, {'groupId': 'FG002', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '2'}]}]}], 'recruitmentDetails': 'Recruitment was from 2004 to 2010. Study initiation was delayed due to the closure of Johns Hopkins Medicine Institutional Review Board(JHMIRB). Patients were recruited from our Kennedy Krieger Institute (KKI)medical clinics, physician referrals,and parent organizations. Parents were sent letters of invitation.', 'preAssignmentDetails': 'Pharmacokinetics in enrolled subjects required her to be a fast metabolizer of Dextromethorphan (DM).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}, {'value': '38', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'DM1( 0.25 mg/kg /Day)', 'description': 'Dextromethorphan 0.25 mg/kg per day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}, {'id': 'BG001', 'title': 'DM2 (2.5 mg/kg/Day)', 'description': 'Dextromethorphan 2.5 mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}, {'id': 'BG002', 'title': 'DM3 (5mg/kg/Day)', 'description': 'Dextromethorphan 5mg/kg/day\n\nDextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.\n\nDextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}, {'value': '38', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '6.54', 'spread': '2.70', 'groupId': 'BG000'}, {'value': '6.31', 'spread': '3.50', 'groupId': 'BG001'}, {'value': '6.08', 'spread': '3.06', 'groupId': 'BG002'}, {'value': '6.32', 'spread': '3.02', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}, {'value': '38', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}, {'value': '38', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 38}}, 'statusModule': {'whyStopped': 'Study changed to a placebo controlled trial of dextromethorphan', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2004-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-07', 'completionDateStruct': {'date': '2010-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-03-26', 'studyFirstSubmitDate': '2008-01-04', 'resultsFirstSubmitDate': '2013-03-08', 'studyFirstSubmitQcDate': '2008-01-04', 'lastUpdatePostDateStruct': {'date': '2014-04-23', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-03-26', 'studyFirstPostDateStruct': {'date': '2008-01-15', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-04-23', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Difference in EEG Spike Counts at Six Months Compared to Baseline for Each Treatment Arm.', 'timeFrame': 'Initial and 6-month post-treatment', 'description': 'Difference in EEG spike count means pre and 6 months post-treatment in each of three treatment groups.'}], 'secondaryOutcomes': [{'measure': 'Improvement in Receptive Language as Measured by the Mullen Scale.', 'timeFrame': 'Change in mean between Initial and 6-month follow-up', 'description': 'The Mullen Receptive language scale pre and 6 months post DM, measured as a change in the mean score of language, by age in months.'}, {'measure': 'Difference in SSI Mean Score at Six Months Compared to Baseline for Each Treatment Arm.', 'timeFrame': 'Initial and 6 month followup', 'description': 'The Screen for Social Interaction (SSI) is a 54-item parent/caregiver-report screening instrument that emphasizes reciprocal social interaction including joint attention skills. The items are positive (prosocial) and are scored on a four-point frequency scale (child displays the behavior "almost never" = 0 to "almost all the time" = 3). Thus lower scores reflect a slower or delayed development, and higher scores reflect more normative development. SSI total scores range from 0-162. There are no subscales. Difference in Screen for Social Interaction (SSI) mean scores between baseline and 6 months post-treatment for each treatment arm are reported.'}, {'measure': 'Mean SSI Score for Total Subjects at Baseline and 6 Months', 'timeFrame': '0-6 months', 'description': 'Analysis of Difference in Mean Screen for Social Interaction (SSI) Score between 0-6 months for total sample (n=19).'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Rett Syndrome', 'Dextromethorphan'], 'conditions': ['Rett Syndrome']}, 'descriptionModule': {'briefSummary': "Increased brain glutamate and its N-methyl-D-aspartate (NMDA) receptors found in the brain of younger Rett syndrome (RTT) patients cause toxic damage to neurons (the brain's nerve cells), and contributing to EEG spikes. Dextromethorphan (DM) acts by blocking NMDA/glutamate receptors. This study is being done to determine if DM will prevent the harmful over-stimulation of the neurons thereby reducing EEG spike activity. Treatment with DM consists of one of 3 different doses (0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day), and aims to find out which dose if any will help improve EEG abnormalities, behavior, cognition, and reduce seizures, as well as improve breathing abnormalities, motor capabilities, bone density, and GI dysfunction.\n\nThe study will include 90 females and males with RTT, 2 years-14.99 years of age, with a mutation in the methyl CpG binding protein 2 (MECP2) gene, and spikes on EEG, with or without clinical seizures.", 'detailedDescription': 'Patients meeting eligibility criteria(mutation +ve and having EEG spikes), will be admitted to the Pediatric Clinical Research Unit at Johns Hopkins Hospital and will have pharmacokinetics of DM determined to establish that they are rapid metabolizers of the drug. The baseline studies on initial admission include neurological, neuropsychology,EEG, gastroenterology, Occupational and Physical therapy evaluations. If the subject is a rapid metabolizer they will be randomized to one of the three drug doses. They are contacted by telephone, weekly in the first month, and monthly thereafter. They will be examined by a neurologist at 2 weeks,1 month, and 3 months during the drug trial. At each of these visits they will also be monitored for changes in complete blood count (CBC), electrolytes, and EKG. At the end of the 6 month drug trial the patients will be readmitted to Johns Hopkins Hospital when all baseline studies are repeated. Cost of travel, hospitalization and interim tests are free to participants.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '15 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. those who have classic or atypical RTT with a proven mutation in the MeCP2 gene;\n2. those with documented EEG evidence of spike activity who may or may not have clinical seizures;\n3. subjects must be between 2years -14.99 years of age.\n\nExclusion Criteria:\n\n1. those without an established mutation in the MeCP2 gene;\n2. those who do not have EEG evidence of spike activity;\n3. those with mutations in the MeCP2 gene but who have had brain resection or surgical intervention; for example, tumor, hydrocephalus, severe head trauma; or, an associated severe medical illnesses such as vasculopathies, malignancies, diabetes, thyroid dysfunction, etc;\n4. those on medications that could interact with DM, e.g. monoamine oxidase (MAO) inhibitors, selective serotonin reuptake inhibitor (SSRI), sibutramine etc. to avoid a serotonin syndrome; quinidine and drugs metabolized by the Cytochrome P450 (CYP450) isoform cytochrome P450 2D6 (CYP2D6) (e.g. amiodarone, haloperidol, propafenone, thioridazine);\n5. those proven to be intermediate or slow metabolizers of DM;\n6. those with reported adverse reactions to DM;\n7. those whose pregnancy test is positive; and,\n8. those showing poor compliance with any aspect of the study;\n9. foster children'}, 'identificationModule': {'nctId': 'NCT00593957', 'briefTitle': 'Trial of Dextromethorphan in Rett Syndrome', 'organization': {'class': 'OTHER', 'fullName': 'Hugo W. Moser Research Institute at Kennedy Krieger, Inc.'}, 'officialTitle': 'Trial of Dextromethorphan in Rett Syndrome', 'orgStudyIdInfo': {'id': 'FD2408'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'DM1( 0.25 mg/kg /day)', 'description': 'Dextromethorphan 0.25 mg/kg per day', 'interventionNames': ['Drug: Dextromethorphan']}, {'type': 'EXPERIMENTAL', 'label': 'DM2 (2.5 mg/kg/day)', 'description': 'Dextromethorphan 2.5 mg/kg/day', 'interventionNames': ['Drug: Dextromethorphan']}, {'type': 'EXPERIMENTAL', 'label': 'DM3 (5mg/kg/day)', 'description': 'Dextromethorphan 5mg/kg/day', 'interventionNames': ['Drug: Dextromethorphan']}], 'interventions': [{'name': 'Dextromethorphan', 'type': 'DRUG', 'otherNames': ['Delsym'], 'description': 'Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.', 'armGroupLabels': ['DM1( 0.25 mg/kg /day)', 'DM2 (2.5 mg/kg/day)', 'DM3 (5mg/kg/day)']}, {'name': 'Dextromethorphan', 'type': 'DRUG', 'otherNames': ['Delsym'], 'description': 'Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.', 'armGroupLabels': ['DM1( 0.25 mg/kg /day)', 'DM2 (2.5 mg/kg/day)', 'DM3 (5mg/kg/day)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute/Johns Hopkins Medical Institutions', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}], 'overallOfficials': [{'name': 'SakkuBai Naidu, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hugo W. Moser Research Institute at Kennedy Krieger, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hugo W. Moser Research Institute at Kennedy Krieger, Inc.', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor of Neurology and Pediatrics', 'investigatorFullName': 'SakkuBai Naidu, M.D.', 'investigatorAffiliation': 'Hugo W. Moser Research Institute at Kennedy Krieger, Inc.'}}}}