Ignite Creation Date:
2025-12-25 @ 12:40 AM
Ignite Modification Date:
2025-12-26 @ 1:50 PM
Study NCT ID:
NCT00750867
Status:
COMPLETED
Last Update Posted:
2017-02-23
First Post:
2008-09-09
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019578', 'term': 'Multiple System Atrophy'}, {'id': 'C563206', 'term': 'Thyroid Dyshormonogenesis 2A'}], 'ancestors': [{'id': 'D054969', 'term': 'Primary Dysautonomias'}, {'id': 'D001342', 'term': 'Autonomic Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D001480', 'term': 'Basal Ganglia Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009069', 'term': 'Movement Disorders'}, {'id': 'D000080874', 'term': 'Synucleinopathies'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D016756', 'term': 'Immunoglobulins, Intravenous'}], 'ancestors': [{'id': 'D007074', 'term': 'Immunoglobulin G'}, {'id': 'D007132', 'term': 'Immunoglobulin Isotypes'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'novakp@ummhc.org', 'phone': '508-334-4973', 'title': 'Dr.Peter Novak', 'organization': 'University of Massachusetts Medical School'}, 'certainAgreement': {'piSponsorEmployee': True, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'The main study limitation is its small size and open label character.'}}, 'adverseEventsModule': {'timeFrame': '1 year', 'eventGroups': [{'id': 'EG000', 'title': 'Open Label Interventional Arm', 'description': 'intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.', 'otherNumAtRisk': 9, 'otherNumAffected': 9, 'seriousNumAtRisk': 9, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'elevated blood pressure', 'notes': 'transient, responding to adjusting the infusion rate', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 32, 'numAffected': 9}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'urinary tract infection', 'notes': 'treated with antibiotics', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Accidental injury', 'notes': 'nose fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'pruritic skin rash', 'notes': '2 subjects withdrew from the study.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'increased temperature or skin flushing', 'notes': 'treated with Diphenhydramine, Acetaminophen, and slowing the infusion rate', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 5, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'restless leg syndrome', 'notes': 'treated with iron supplements', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'decreased GFR', 'notes': 'resolved', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'ankle edema', 'notes': 'treated with diuretics', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'elevated BUN', 'notes': 'resolved', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'worsening of allergies', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'nodular lung abnormality', 'notes': 'later determined to be a tangle of vein most likely inborn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'low potassium', 'notes': 'treated with potassium supplements', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'viral infection of gastrointenstinal tract', 'notes': 'resolved', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'elevated PSA', 'notes': 'resolved', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'wrist strain', 'notes': 'resolved', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'worsening of sleep apnea', 'notes': 'treated with continuous positive airway pressure (CPAP)', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Adverse Events up to Six Months Post-treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label Interventional Arm', 'description': 'intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.'}], 'classes': [{'title': 'All Adverse Events', 'categories': [{'measurements': [{'value': '43', 'groupId': 'OG000'}]}]}, {'title': 'Serious Adverse Events', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Monthly, up to 8 months (including the screening visit and the final visit)', 'description': 'The primary outcome measure was to evaluate the safety and tolerability of the IVIG infusions in patients with multiple system atrophy. The primary endpoint was defined as the frequency of adverse events (AE). AEs including their severity and relationship to the IVIG were assessed throughout the study and at least 60 days after the last infusion. The AEs were considered to be related to the IVIG infusion (infusional AE) if they occurred during an infusion or within 72 hours afterwards. Non-infusional AEs were further classified as possible related to IVIG or likely not related to IVIG. Serious AEs assessed include medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization. Any AE was defined as occurrence of any symptom regardless of intensity grade.', 'unitOfMeasure': 'Adverse events', 'reportingStatus': 'POSTED', 'populationDescription': 'Two participants dropped out from the study.'}, {'type': 'SECONDARY', 'title': 'Preliminary Efficacy of IVIg for Treatment of MSA.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label Interventional Arm', 'description': 'intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.'}], 'classes': [{'title': 'UMSARS-I, baseline', 'categories': [{'measurements': [{'value': '23.8', 'spread': '6', 'groupId': 'OG000'}]}]}, {'title': 'UMSARS-II,baseline', 'categories': [{'measurements': [{'value': '26.1', 'spread': '7.4', 'groupId': 'OG000'}]}]}, {'title': 'UMSARS-I, final visit', 'categories': [{'measurements': [{'value': '19', 'spread': '5.9', 'groupId': 'OG000'}]}]}, {'title': 'UMSARS-II, final visit', 'categories': [{'measurements': [{'value': '23.3', 'spread': '7.2', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.0128', 'groupIds': ['OG000'], 'pValueComment': 'The P-Value was obtained by comparing the UMSARS-I scores at final visit and at baseline.', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '0.025', 'groupIds': ['OG000'], 'pValueComment': 'The P-Value was obtained by comparing the UMSARS-II scores at final visit and at baseline.', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': 'Monthly, up to 8 months (including the screening visit and the final visit)', 'description': 'The secondary outcome measure was to evaluate the preliminary efficacy of IVIG for the treatment of MSA. The primary efficacy endpoint was change of the Unified MSA Rating Scale (UMSARS-I and UMSAR-II) compared to baseline. UMSARS-I and UMSARS-II are validated semiquantitative rating scales for evaluation of severity of MSA. UMSARS-I comprises a historical review of disease-related impairments and UMSARS-II comprises motor examination. UMSARS-I has 12 questions, each with assigned score 0-4, where 0 is normal and \\> are abnormal responses. Total range of UMSARS-I is 0 to 48. UMSARS-II has 12 items rated by an examiner, each with assigned score 0-4, where 0 is normal and \\> are abnormal responses. Total range of UMSARS-II is 0 to 56. The scores of UMSARS-I and UMSARS-II at baseline (month 1) was compared with the scores obtained at the final visit (month 8) which was 8 months apart. The interventions occured at months 2-7, total six times.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Open Label Interventional Arm', 'description': 'intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Open Label Interventional Arm', 'description': 'intravenous immunoglobulin (IVIg): The IVIg will be infused intravenously, monthly, 6 times, the dose will be 0.4 gram/kg for each infusion.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '59', 'groupId': 'BG000', 'lowerLimit': '55', 'upperLimit': '64'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Gender', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '9', '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': 9}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2008-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-09', 'completionDateStruct': {'date': '2012-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-01-05', 'studyFirstSubmitDate': '2008-09-09', 'resultsFirstSubmitDate': '2013-12-26', 'studyFirstSubmitQcDate': '2008-09-09', 'lastUpdatePostDateStruct': {'date': '2017-02-23', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2015-09-28', 'studyFirstPostDateStruct': {'date': '2008-09-11', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-10-26', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2009-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Adverse Events up to Six Months Post-treatment', 'timeFrame': 'Monthly, up to 8 months (including the screening visit and the final visit)', 'description': 'The primary outcome measure was to evaluate the safety and tolerability of the IVIG infusions in patients with multiple system atrophy. The primary endpoint was defined as the frequency of adverse events (AE). AEs including their severity and relationship to the IVIG were assessed throughout the study and at least 60 days after the last infusion. The AEs were considered to be related to the IVIG infusion (infusional AE) if they occurred during an infusion or within 72 hours afterwards. Non-infusional AEs were further classified as possible related to IVIG or likely not related to IVIG. Serious AEs assessed include medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization. Any AE was defined as occurrence of any symptom regardless of intensity grade.'}], 'secondaryOutcomes': [{'measure': 'Preliminary Efficacy of IVIg for Treatment of MSA.', 'timeFrame': 'Monthly, up to 8 months (including the screening visit and the final visit)', 'description': 'The secondary outcome measure was to evaluate the preliminary efficacy of IVIG for the treatment of MSA. The primary efficacy endpoint was change of the Unified MSA Rating Scale (UMSARS-I and UMSAR-II) compared to baseline. UMSARS-I and UMSARS-II are validated semiquantitative rating scales for evaluation of severity of MSA. UMSARS-I comprises a historical review of disease-related impairments and UMSARS-II comprises motor examination. UMSARS-I has 12 questions, each with assigned score 0-4, where 0 is normal and \\> are abnormal responses. Total range of UMSARS-I is 0 to 48. UMSARS-II has 12 items rated by an examiner, each with assigned score 0-4, where 0 is normal and \\> are abnormal responses. Total range of UMSARS-II is 0 to 56. The scores of UMSARS-I and UMSARS-II at baseline (month 1) was compared with the scores obtained at the final visit (month 8) which was 8 months apart. The interventions occured at months 2-7, total six times.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['multiple system atrophy, MSA,', 'intravenous immunoglobulin, IVIg'], 'conditions': ['Multiple System Atrophy']}, 'referencesModule': {'references': [{'pmid': '23116538', 'type': 'RESULT', 'citation': 'Novak P, Williams A, Ravin P, Zurkiya O, Abduljalil A, Novak V. Treatment of multiple system atrophy using intravenous immunoglobulin. BMC Neurol. 2012 Nov 1;12:131. doi: 10.1186/1471-2377-12-131.'}]}, 'descriptionModule': {'briefSummary': 'Multiple System Atrophy (MSA) is a progressive sporadic neurodegenerative disorder leading to widespread loss of brain cells that results in parkinsonian, cerebellar and autonomic dysfunction. The cause of the MSA remains unclear. Available treatment is symptomatic only and does not alter the course of disease.\n\nAlthough the cause of MSA remains unclear, there is evidence of presence of common neuroinflammatory mechanisms in the MSA brains including activation of microglia and production of toxic cytokines. This research protocol is based on hypothesis that the MSA progression can be altered by blocking the neuroinflammatory activity.\n\nThis protocol includes administration of intravenous immunoglobulin (IVIg). IVIg contains antibodies derived from human plasma which can block the inflammatory responses in the brain that can lead to loss of brain cells.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Male or female older than 17 years.\n2. Clinical diagnosis of probable multiple system atrophy\n3. Provide written informed consent to participate in the study\n4. Understand that they may withdraw their consent at any time\n\nExclusion Criteria:\n\n1. Women who are pregnant or lactating\n2. In the investigator's opinion, have any other significant systemic, hepatic, cardiac or renal illness.\n3. In the investigator's opinion, the subjects are significantly dehydrated, as determined by clinical evaluation including measurement of skin turgor, blood urea nitrogen and creatinine values."}, 'identificationModule': {'nctId': 'NCT00750867', 'briefTitle': 'Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins', 'organization': {'class': 'OTHER', 'fullName': 'University of Massachusetts, Worcester'}, 'officialTitle': 'Treatment of Multiple System Atrophy Using Intravenous Immunoglobulins', 'orgStudyIdInfo': {'id': 'H-12784'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '1', 'description': 'Interventions included monthly infusions of intravenous immunoglobulin.', 'interventionNames': ['Drug: intravenous immunoglobulin (IVIg)']}], 'interventions': [{'name': 'intravenous immunoglobulin (IVIg)', 'type': 'DRUG', 'otherNames': ['Privigen'], 'description': 'The intravenous immunoglobulin (brand Privigen) will be infused intravenously, monthly, 6 times, for 6 months the dose will be 0.4 gram/kg for each infusion.', 'armGroupLabels': ['1']}]}, 'contactsLocationsModule': {'locations': [{'zip': '01655', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'University of Massachusetts Medical School', 'geoPoint': {'lat': 42.26259, 'lon': -71.80229}}], 'overallOfficials': [{'name': 'Peter Novak, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Massachusetts, Worcester'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Massachusetts, Worcester', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Study Principle Investigator', 'investigatorFullName': 'Peter Novak', 'investigatorAffiliation': 'University of Massachusetts, Worcester'}}}}