Ignite Creation Date:
2025-12-24 @ 2:16 PM
Ignite Modification Date:
2025-12-25 @ 5:04 PM
Study NCT ID:
NCT00911495
Status:
COMPLETED
Last Update Posted:
2020-05-11
First Post:
2009-05-27
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C553182', 'term': 'rivipansel'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTrials.gov_Inquiries@pfizer.com', 'phone': '18007181021', 'title': 'Pfizer ClinicalTrials.gov Call Center', 'organization': 'Pfizer Inc.'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '28 days', 'eventGroups': [{'id': 'EG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening', 'otherNumAtRisk': 15, 'otherNumAffected': 9, 'seriousNumAtRisk': 15, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 4}], 'organSystem': 'Nervous system disorders'}, {'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders'}, {'term': 'Leukocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders'}, {'term': 'sickle cell anemia with crisis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Congenital, familial and genetic disorders'}, {'term': 'vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders'}, {'term': 'infusion site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'General disorders'}, {'term': 'hypokalemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders'}, {'term': 'arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders'}, {'term': 'cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety as Measured by the Number of Participants With Adverse Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening'}], 'classes': [{'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '28 days', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled subjects were analyzed for safety.'}, {'type': 'SECONDARY', 'title': 'Total Plasma Clearance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening'}], 'classes': [{'categories': [{'measurements': [{'value': '19.6', 'spread': '6.42', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '48 hours', 'unitOfMeasure': 'mL/h/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects were analyzed for pharmacokinetics; one subject of the 15 enrolled was lost to follow-up.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Blood Flow and Biomarkers of Adhesion', 'timeFrame': '48 hours', 'description': 'As an exploratory outcome mean change in microvascular blood flow from baseline to each time point was measured. Microvascular blood flow was also measured as microFI, perfused vessel density, and RBC velocity.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Volume of the Central Compartment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening'}], 'classes': [{'categories': [{'measurements': [{'value': '120', 'spread': '30.7', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '48 hours', 'unitOfMeasure': 'mL/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Intercompartmental Clearance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening'}], 'classes': [{'categories': [{'measurements': [{'value': '31.6', 'spread': '17.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '48 hours', 'unitOfMeasure': 'mL/h/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Volume of the Peripheral Compartment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening'}], 'classes': [{'categories': [{'measurements': [{'value': '64.2', 'spread': '19.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '48 hours', 'unitOfMeasure': 'mL/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'First subject enrolled 28 May 2009; last subject completed 6 July 2010.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'GMI-1070', 'description': 'GMI-1070 administered IV at 20 mg/kg loading dose, followed by a single dose of 10 mg/kg in the evening'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '28', 'groupId': 'BG000', 'lowerLimit': '19', 'upperLimit': '50'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '15', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-05', 'completionDateStruct': {'date': '2010-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-05-07', 'studyFirstSubmitDate': '2009-05-27', 'resultsFirstSubmitDate': '2012-09-11', 'studyFirstSubmitQcDate': '2009-05-29', 'lastUpdatePostDateStruct': {'date': '2020-05-11', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2013-05-02', 'studyFirstPostDateStruct': {'date': '2009-06-02', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2013-05-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Blood Flow and Biomarkers of Adhesion', 'timeFrame': '48 hours', 'description': 'As an exploratory outcome mean change in microvascular blood flow from baseline to each time point was measured. Microvascular blood flow was also measured as microFI, perfused vessel density, and RBC velocity.'}], 'primaryOutcomes': [{'measure': 'Safety as Measured by the Number of Participants With Adverse Events', 'timeFrame': '28 days'}], 'secondaryOutcomes': [{'measure': 'Total Plasma Clearance', 'timeFrame': '48 hours'}, {'measure': 'Volume of the Central Compartment', 'timeFrame': '48 hours'}, {'measure': 'Intercompartmental Clearance', 'timeFrame': '48 hours'}, {'measure': 'Volume of the Peripheral Compartment', 'timeFrame': '48 hours'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Sickle Cell Disease'], 'conditions': ['Sickle Cell Disease']}, 'referencesModule': {'references': [{'pmid': '24988449', 'type': 'DERIVED', 'citation': 'Wun T, Styles L, DeCastro L, Telen MJ, Kuypers F, Cheung A, Kramer W, Flanner H, Rhee S, Magnani JL, Thackray H. Phase 1 study of the E-selectin inhibitor GMI 1070 in patients with sickle cell anemia. PLoS One. 2014 Jul 2;9(7):e101301. doi: 10.1371/journal.pone.0101301. eCollection 2014.'}]}, 'descriptionModule': {'briefSummary': 'This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the outpatient setting.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '45 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age 18 to 45 years\n* Established diagnosis of SCD-SS or SCD-SB0-thal\n* At medical baseline, with no evidence of worsening of disease over the last 3 months\n* Available and agree to return for follow-up visits for the full duration of the study\n* Able to cooperate with study procedures\n* Documented and observed written informed consent\n\nExclusion Criteria:\n\n* Vaso-occlusive crisis\n* Recent major surgery, hospitalization, infection, significant bleeding, cerebrovascular accident or seizure, or transfusion\n* Currently receiving, or has received within the previous 4 weeks, any other investigational agent\n* Pregnant or lactating female; or female of childbearing age unable or unwilling to comply with birth control or abstinence during the course of the study'}, 'identificationModule': {'nctId': 'NCT00911495', 'briefTitle': 'Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'GlycoMimetics Incorporated'}, 'officialTitle': 'Phase 1/Phase 2 Study of the Safety, Pharmacokinetics, and Microvascular Effect of Titrating Doses of Intravenous GMI-1070, a Pan-Selectin Inhibitor, in Adults With Sickle Cell Disease', 'orgStudyIdInfo': {'id': 'GMI-1070-103'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'GMI-1070', 'interventionNames': ['Drug: GMI-1070']}], 'interventions': [{'name': 'GMI-1070', 'type': 'DRUG', 'description': 'Intravenous GMI-1070 given as two doses over the course of one day', 'armGroupLabels': ['GMI-1070']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital & Research Center Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'University of California at Davis, CCRC', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke Comprehensive Sickle Cell Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}], 'overallOfficials': [{'name': 'Helen Thackray, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'GlycoMimetics Incorporated'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'GlycoMimetics Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}