Ignite Creation Date:
2025-12-25 @ 2:16 AM
Ignite Modification Date:
2026-02-24 @ 3:14 PM
Study NCT ID:
NCT02563860
Status:
COMPLETED
Last Update Posted:
2019-07-12
First Post:
2015-07-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Pharmacological Treatment of Rett Syndrome With Statins
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': 'D008148', 'term': 'Lovastatin'}], 'ancestors': [{'id': 'D009281', 'term': 'Naphthalenes'}, {'id': 'D011084', 'term': 'Polycyclic Aromatic Hydrocarbons'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'adjukic@montefiore.org', 'phone': '7189204378', 'title': 'Aleksandra Djukic', 'organization': 'Montefiore MC'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Open Label', 'description': 'Treatment with Lovastatin, dose escalating trial according to the following schedule:\n\n10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.\n\nLovastatin: dose escallating', 'otherNumAtRisk': 20, 'deathsNumAtRisk': 20, 'otherNumAffected': 1, 'seriousNumAtRisk': 20, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'mild', 'notes': 'Anxiety, upon dose escalation from 20 to 40 mg of Lovastatin.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Gait Velocity as Measured by GAITRite System', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Treatment with Lovastatin, dose escalating trial according to the following schedule:\n\n10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.\n\nLovastatin: dose escallating'}], 'classes': [{'categories': [{'measurements': [{'value': '58.6', 'groupId': 'OG000', 'lowerLimit': '43.8', 'upperLimit': '71.1'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'During final week of treatment, week 32', 'description': 'To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)', 'unitOfMeasure': 'cm/sec', 'dispersionType': 'Inter-Quartile Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Treatment with Lovastatin, dose escalating trial according to the following schedule:\n\n10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.\n\nLovastatin: dose escallating'}], 'classes': [{'title': 'Pre-treatment', 'categories': [{'measurements': [{'value': '44.85', 'groupId': 'OG000', 'lowerLimit': '25.85', 'upperLimit': '68.10'}]}]}, {'title': 'Post treatment', 'categories': [{'measurements': [{'value': '58.93', 'groupId': 'OG000', 'lowerLimit': '47.55', 'upperLimit': '70.25'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'final week of treatment, Week 32', 'description': 'Novelty score is considered a composite measure of visual memory.Test is a standardized neuropsychological test( "Rose test") adapted for eye tracking system.\n\nThe present study made use of a well-established battery of "visual paired comparison" problems .There were nine problems: five using achromatic photos of faces and four using multicolored abstract patterns paired stimuli , separated by 23°. For each problem, two identical stimuli are briefly presented side by side for familiarization, then the familiar and a new one are paired on test\n\nRecognition(memory) is indexed by a novelty score (percentage of looking to the novel target on test).\n\nHigher novelty scores were related to better recognition (index of better memory) Scores range from 0.0-1.0 (0-100%) and indicate 1) more overall looking at novel target and (2) more fixations to novel targets.\n\nScore of .5 (50%) indicates looking "by change" only with no recognition.', 'unitOfMeasure': 'percent', 'dispersionType': 'Inter-Quartile Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Calculation done on intent-to-treat population'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Open Label', 'description': 'Treatment with Lovastatin, dose escalating trial according to the following schedule:\n\nAll participants were assigned to the specified sequence of interventions and all participants received all the three dosing regimen please make this explicit in the Arm/Group Description. In either case, separate Period 1-10 mg daily for 8 week Period 2-20 mg daily for 8 weeks Period 3-40 mg daily for 16 weeks.\n\nLovastatin: dose escallating'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '19'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Open Label', 'description': 'Female patients with genetically confirmed RTT, treatment arm only.\n\nTreatment with Lovastatin, dose escalating trial according to the following schedule:\n\n10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.\n\nLovastatin: dose escallating'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '20', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '20', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '20', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 20}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-07'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-07', 'completionDateStruct': {'date': '2016-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-07-11', 'studyFirstSubmitDate': '2015-07-20', 'resultsFirstSubmitDate': '2018-05-19', 'studyFirstSubmitQcDate': '2015-09-28', 'lastUpdatePostDateStruct': {'date': '2019-07-12', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-04-15', 'studyFirstPostDateStruct': {'date': '2015-09-30', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-05-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Gait Velocity as Measured by GAITRite System', 'timeFrame': 'During final week of treatment, week 32', 'description': 'To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)'}], 'secondaryOutcomes': [{'measure': 'Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System', 'timeFrame': 'final week of treatment, Week 32', 'description': 'Novelty score is considered a composite measure of visual memory.Test is a standardized neuropsychological test( "Rose test") adapted for eye tracking system.\n\nThe present study made use of a well-established battery of "visual paired comparison" problems .There were nine problems: five using achromatic photos of faces and four using multicolored abstract patterns paired stimuli , separated by 23°. For each problem, two identical stimuli are briefly presented side by side for familiarization, then the familiar and a new one are paired on test\n\nRecognition(memory) is indexed by a novelty score (percentage of looking to the novel target on test).\n\nHigher novelty scores were related to better recognition (index of better memory) Scores range from 0.0-1.0 (0-100%) and indicate 1) more overall looking at novel target and (2) more fixations to novel targets.\n\nScore of .5 (50%) indicates looking "by change" only with no recognition.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Treatment trial', 'Lovastatin'], 'conditions': ['Rett Syndrome']}, 'descriptionModule': {'briefSummary': 'This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.', 'detailedDescription': 'Excess neuronal cholesterol plays a role in the pathophysiology of Rett syndrome(RTT) and the investigators hypothesise that inhibition of cholesterol synthesis in the CNS will reduce neuronal cholesterol and lead to improvement in Rett syndrome related symptoms.\n\nGoal: To determine the optimal dosing regiment of Lovastatin for patients with RTT and to identify the most appropriate primary outcome measure for the subsequent Phase 3 study.\n\nPhase 2, dose escalation study.\n\nPrimary outcome: Gait speed Secondary outcomes: respiratory function, cognition, EEG (encephalopathy), Rett syndrome severity scale, neurodevelopmental assessment (QOL)\n\n20 ambulatory female patients with genetically confirmed.'}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Females patients,\n* Genetically confirmed RTT,\n* Ambulatory.\n\nExclusion Criteria:\n\n* Presence of co morbid non-Rett related disease,\n* History of adverse reaction/hypersensitivity to statins,\n* Levels of aspartate-amino trans-ferase (AST), alanine-amino transferase (ALT) or creatine kinase (CK) triple the normal values,\n* Active liver disease,\n* Concomitant use of strong CYP3A4 inhibitors,\n* Condition predis-posing to renal failure secondary to rhabdomyolysis (scoliosis surgery within 6 months),\n* Oral contraceptives use.'}, 'identificationModule': {'nctId': 'NCT02563860', 'acronym': 'Statins', 'briefTitle': 'Pharmacological Treatment of Rett Syndrome With Statins', 'organization': {'class': 'OTHER', 'fullName': 'Montefiore Medical Center'}, 'officialTitle': 'Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)', 'orgStudyIdInfo': {'id': '2014-4253'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open label', 'description': 'Treatment with Lovastatin, dose escalating trial according to the following schedule:\n\n10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.', 'interventionNames': ['Drug: Lovastatin']}], 'interventions': [{'name': 'Lovastatin', 'type': 'DRUG', 'otherNames': ['Mevacor'], 'description': 'dose escallating', 'armGroupLabels': ['Open label']}]}, 'contactsLocationsModule': {'locations': [{'zip': '10467', 'city': 'The Bronx', 'state': 'New York', 'country': 'United States', 'facility': 'Montefiore Medical center', 'geoPoint': {'lat': 40.84985, 'lon': -73.86641}}], 'overallOfficials': [{'name': 'Aleksandra Djukic, MD PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Montefiore Medical Center'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Montefiore Medical Center', 'class': 'OTHER'}, 'collaborators': [{'name': 'Rett Syndrome Research Trust', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor of Clinical Neurology, Director, Tri State Rett Syndrome Center', 'investigatorFullName': 'Aleksandra Djukic', 'investigatorAffiliation': 'Montefiore Medical Center'}}}}