Ignite Creation Date:
2025-12-24 @ 6:55 PM
Ignite Modification Date:
2025-12-29 @ 10:53 AM
Study NCT ID:
NCT01736657
Status:
COMPLETED
Last Update Posted:
2014-07-14
First Post:
2012-11-27
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'phone': '303-205-2680', 'title': 'Ray Goodrich, Phd, VP Scientifc and Clinical Affairs', 'organization': 'Terumo BCT, Inc.'}, 'certainAgreement': {'otherDetails': 'If Study Site is performing services in a multi-center Study, it agrees not to independently publish, publicly disclose, present or discuss any results of or information pertaining to the Study until a multi-center manuscript is published; provided however, that if a multi-center manuscript is not published within one year after completion of the Study at all Study sites, Study Site may publish the Study data generated by Study Site', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Red Blood Cell Exchange for Patients With Sickle Cell Disease', 'description': 'Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.', 'otherNumAtRisk': 72, 'otherNumAffected': 10, 'seriousNumAtRisk': 72, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 72, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 72, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'sourceVocabulary': 'MedDRA'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Mean Ratio Actual Fraction of Cells Remaining (FCRa; as Measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as Predicted by the Spectra Optia System FCR Algorithm Multiplied by the Pre-Procedure % HbS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Red Blood Cell Exchange for Patients With Sickle Cell Disease', 'description': 'Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.9', 'groupId': 'OG000', 'lowerLimit': '0.855', 'upperLimit': '0.941'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Length of the procedure', 'description': 'The primary endpoint evaluated the mean ratio of the Actual Fraction of Cells Remaining (FCRa: as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp: as predicted by the Spectra Optia system FCR algorithm multiplied by the Pre-Procedure % HbS), in the evaluable population (60 pts). The pre-defined range for the mean ratio of the FCRa to the FCRp was 0.75 to 1.25.', 'unitOfMeasure': 'ratio', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': '60 patients analyzed, 73 enrolled:12 patients were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 pts), but were included in the safety analysis (72 patients); 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.'}, {'type': 'SECONDARY', 'title': 'Procedural Success of the Spectra Optia System in the Evaluable Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Red Blood Cell Exchange for Patients With Sickle Cell Disease', 'description': 'Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Length of the procedure', 'description': "The procedural success of the Spectra Optia System is defined as the ability of the device to complete a red blood cell exchange (RBCx) and to obtain a satisfactory exchange by lowering the patient's hemoglobin S, as determined by the investigator in the evaluable population (60 pts).", 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': '60 patients analyzed, 73 enrolled:12 patients were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 pts), but were included in the safety analysis (72 patients); 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.'}, {'type': 'SECONDARY', 'title': "Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population", 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Red Blood Cell Exchange for Patients With Sickle Cell Disease', 'description': 'Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.03', 'groupId': 'OG000', 'lowerLimit': '1.011', 'upperLimit': '1.045'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Length of the procedure', 'description': 'Measurement of the patient post-procedure hematocrit compared to the final target hematocrit calculated by the Spectra Optia Apheresis System. Final target hematocrit was calculated by tracking the number of red cells coming into the system versus the number of red cells removed.', 'unitOfMeasure': 'ratio', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': '60 patients analyzed, 73 enrolled:12 patients were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 pts), but were included in the safety analysis (72 patients); 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.'}, {'type': 'SECONDARY', 'title': 'Device-related Serious Adverse Events (SAE) in the Full Analysis Set', 'denoms': [{'units': 'Participants', 'counts': [{'value': '72', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Red Blood Cell Exchange for Patients With Sickle Cell Disease', 'description': 'Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'upon signing consent to 24 hours post-procedure', 'description': 'Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Seventy-three enrolled: 12 were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 patients), but these patients were included in safety analysis as Full Analysis Set; 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Red Blood Cell Exchange for Patients With Sickle Cell Disease', 'description': 'Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '73'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '72'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'insufficient vascular access', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Recruitment period: Nov 2012 through May 2013', 'preAssignmentDetails': 'Evaluable population includes 60 pts. Terumo Optia Trainer was present for Optia Operator support for 12 procedures, called "lead-in" procedures: these were not included in efficacy analysis, but were included in safety analysis (full analysis set).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '72', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Red Blood Cell Exchange for Patients With Sickle Cell Disease', 'description': 'Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '26', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '46', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '23.0', 'spread': '10.32', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '29', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '43', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '72', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Seventy-two patients were in safety analysis and 60 patients (evaluable popluation) were analyzed in the efficacy analysis. The 12 patients not in the efficacy analysis were "lead-in" patients and the RBCX procedures were conducted as the last phase in Operator training on the device.'}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 73}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-01', 'completionDateStruct': {'date': '2013-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-07-10', 'studyFirstSubmitDate': '2012-11-27', 'resultsFirstSubmitDate': '2013-08-22', 'studyFirstSubmitQcDate': '2012-11-28', 'lastUpdatePostDateStruct': {'date': '2014-07-14', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-07-10', 'studyFirstPostDateStruct': {'date': '2012-11-29', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-07-14', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Mean Ratio Actual Fraction of Cells Remaining (FCRa; as Measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as Predicted by the Spectra Optia System FCR Algorithm Multiplied by the Pre-Procedure % HbS)', 'timeFrame': 'Length of the procedure', 'description': 'The primary endpoint evaluated the mean ratio of the Actual Fraction of Cells Remaining (FCRa: as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp: as predicted by the Spectra Optia system FCR algorithm multiplied by the Pre-Procedure % HbS), in the evaluable population (60 pts). The pre-defined range for the mean ratio of the FCRa to the FCRp was 0.75 to 1.25.'}], 'secondaryOutcomes': [{'measure': 'Procedural Success of the Spectra Optia System in the Evaluable Population', 'timeFrame': 'Length of the procedure', 'description': "The procedural success of the Spectra Optia System is defined as the ability of the device to complete a red blood cell exchange (RBCx) and to obtain a satisfactory exchange by lowering the patient's hemoglobin S, as determined by the investigator in the evaluable population (60 pts)."}, {'measure': "Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population", 'timeFrame': 'Length of the procedure', 'description': 'Measurement of the patient post-procedure hematocrit compared to the final target hematocrit calculated by the Spectra Optia Apheresis System. Final target hematocrit was calculated by tracking the number of red cells coming into the system versus the number of red cells removed.'}, {'measure': 'Device-related Serious Adverse Events (SAE) in the Full Analysis Set', 'timeFrame': 'upon signing consent to 24 hours post-procedure', 'description': 'Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Sickle Cell Disease']}, 'referencesModule': {'references': [{'pmid': '25330984', 'type': 'DERIVED', 'citation': 'Quirolo K, Bertolone S, Hassell K, Howard T, King KE, Rhodes DK, Bill J. The evaluation of a new apheresis device for automated red blood cell exchange procedures in patients with sickle cell disease. Transfusion. 2015 Apr;55(4):775-81. doi: 10.1111/trf.12891. Epub 2014 Oct 21.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease.', 'detailedDescription': 'Evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease. Open label design.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* At least 12 years old\n* Enrolled in a program of regular red blood cell exchange (RBCx) to prevent symptoms/complications of sickle cell disease (SCD) or Initiating a program of regular RBCx or Receiving RBCx as a pre-surgical procedure.\n* Medically stable\n* Previous documentation of diagnosis by hemoglobin electrophoresis of a type of sickle cell disorder requiring RBCx.\n* Sufficient vascular access to accommodate the RBCx procedure as determined by the apheresis technician performing the procedure or phlebotomist responsible for obtaining intravenous access.\n* Availability of sickle trait negative, leukoreduced, ABO blood group, Rhesus factor D (Rh (D)) compatible, unexpired replacement blood. See Glossary for definition of replacement blood.\n* Able to commit to the study follow-up schedule.\n* Agree to report adverse events (AEs) during the required reporting period.\n\nExclusion Criteria:\n\n* Inability to obtain informed consent/assent from patient, or permission from parent or guardian.\n* Pregnancy (negative serum pregnancy test required for females of childbearing potential).\n* Life expectancy is fewer than 30 days from time of procedure.\n* Incarcerated or a ward of the court.\n* Refusal of blood products.\n* Failure to comply with site standard requirements for cessation of medications (e.g., angiotensin converting enzyme (ACE) inhibitors) that interfere with or increase risk of RBCx procedures.\n* History of drug or alcohol abuse that, in the opinion of the investigator, could affect the ability of the patient to comply with the study requirements Inability to comply with the protocol in the opinion of the investigator.'}, 'identificationModule': {'nctId': 'NCT01736657', 'acronym': 'ESSENTIAL', 'briefTitle': 'Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Terumo BCT'}, 'officialTitle': 'Evaluation of the Spectra Optia Apheresis Red Blood Cell Exchange Protocol in Patients With Sickle Cell Disease.', 'orgStudyIdInfo': {'id': 'CTS-5001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Red cell exchange in sickle cell', 'description': 'Open arm; Red cell blood exchange for patients with sickle cell disease', 'interventionNames': ['Device: Red blood cell exchange in sickle cell']}], 'interventions': [{'name': 'Red blood cell exchange in sickle cell', 'type': 'DEVICE', 'otherNames': ['Specta Optia Apheresis System'], 'description': 'One Red Blood Cell Exchange using Spectra Optia Apheresis System per enrolled patient', 'armGroupLabels': ['Red cell exchange in sickle cell']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': "Children's of Alabama", 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital and Research Center at Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado at Denver', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '40202', 'city': 'Louisville', 'state': 'Kentucky', 'country': 'United States', 'facility': "Kosair Children's Hospital", 'geoPoint': {'lat': 38.25424, 'lon': -85.75941}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins Medical', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}], 'overallOfficials': [{'name': 'Keith Quirolo, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hospital and Research Center at Oakland"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Terumo BCT', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}