Ignite Creation Date:
2025-12-24 @ 11:56 PM
Ignite Modification Date:
2026-02-17 @ 6:47 PM
Study NCT ID:
NCT04269551
Status:
COMPLETED
Last Update Posted:
2025-09-22
First Post:
2020-01-29
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Canada', 'Spain']}, 'conditionBrowseModule': {'meshes': [{'id': 'D000744', 'term': 'Anemia, Hemolytic, Autoimmune'}], 'ancestors': [{'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-06-15', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2022-01-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-17', 'studyFirstSubmitDate': '2020-01-29', 'studyFirstSubmitQcDate': '2020-02-12', 'lastUpdatePostDateStruct': {'date': '2025-09-22', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2020-02-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-01-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Assessment of adverse events (AEs)', 'timeFrame': 'Screening to Day 106', 'description': 'Number of participants with adverse events (AEs)'}], 'secondaryOutcomes': [{'measure': 'Mean change from baseline in bilirubin over time', 'timeFrame': 'Day 1 to Day 106', 'description': 'Assessment of total bilirubin'}, {'measure': 'Mean change from baseline in hemoglobin over time', 'timeFrame': 'Day 1 to Day 106', 'description': 'Assessment of hemoglobin'}, {'measure': 'Complement System Classical Pathway Levels as Measured by WIESLAB Assay', 'timeFrame': 'Day 1 to Day 106', 'description': 'Inhibition by BIVV020 of the complement system classical pathway measured by the WIESLAB assay'}, {'measure': 'Complement System Alternative Pathway Levels as Measured by WIESLAB Assay', 'timeFrame': 'Day 1 to Day 106', 'description': 'Effect of BIVV0020 on the complement system alternative pathway measured by the WIESLAB assay'}, {'measure': 'Total Complement (CH50) Levels', 'timeFrame': 'Day 1 to Day 106', 'description': 'Complement CH50 is a blood test that helps us determine whether protein abnormalities and deficiencies in the complement system are responsible for any increase in autoimmune activity. It will be assessed using complement assays.'}, {'measure': 'Total Complement Factor C4 Levels', 'timeFrame': 'Day 1 to Day 106', 'description': 'Total C4 Levels will be assessed in plasma using complement assays'}, {'measure': 'PK parameter: Cmax', 'timeFrame': 'Day 1 to Day 106', 'description': 'Observed maximum plasma concentration'}, {'measure': 'PK parameter: tmax', 'timeFrame': 'Day 1 to Day 106', 'description': 'Observed first time to reach Cmax'}, {'measure': 'PK parameter: AUClast', 'timeFrame': 'Day 1 to Day 106', 'description': 'Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast'}, {'measure': 'PK parameter: AUC0-∞', 'timeFrame': 'Day 1 to Day 106', 'description': 'Calculated area under the plasma concentration versus time curve extrapolated to infinity'}, {'measure': 'Number of participants with anti-BIVV antibodies', 'timeFrame': 'Day 1 to Day 106', 'description': 'Observed number of participants with BIVV020 antibodies'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Autoimmune Haemolytic Anaemia']}, 'referencesModule': {'references': [{'pmid': '38085846', 'type': 'DERIVED', 'citation': "D'Sa S, Vos JMI, Barcellini W, Wardecki M, Perrin L, Barker G, Zilberstein M, Storek M, Chow T, Roth A. Safety, tolerability, and activity of the active C1s antibody riliprubart in cold agglutinin disease: a phase 1b study. Blood. 2024 Feb 22;143(8):713-720. doi: 10.1182/blood.2023022153."}], 'seeAlsoLinks': [{'url': 'https://www.trialsummaries.com/Study/StudyDetails?id=25315&tenant=MT_SNY_9011', 'label': 'PDY16370 Plain Language Results Summary'}]}, 'descriptionModule': {'briefSummary': 'Primary Objective:\n\nTo assess the safety and tolerability in participants with cold agglutinin disease (CAD), after a single dose of intravenous (IV) BIVV020\n\nSecondary Objectives:\n\nTo assess, in participants with cold agglutinin disease, after a single dose of intravenous (IV) BIVV020:\n\n* The effect of BIVV020 on complement mediated hemolysis\n* The pharmacodynamics (PD) of BIVV020 relating to complement inhibition\n* The pharmacokinetics (PK) of BIVV020\n* The immunogenicity of BIVV020', 'detailedDescription': 'Up to 23 weeks (screening period up to 8 weeks, treatment period 15 weeks)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion criteria :\n\n* Male and/or female patients, ≥ 18 years of age with cold agglutinin disease as defined by:\n\n 1. Chronic hemolysis per Investigator's judgement,\n 2. Polyspecific direct antiglobulin test (DAT) positive,\n 3. Monospecific DAT strongly positive for C3d,\n 4. Cold agglutinin (CAg) titer ≥ 64 at 4 C; and,\n 5. IgG DAT ≤1+.\n* A hemoglobin level ≤11 mg/dL.\n* A total bilirubin level above the normal reference range that is thought to be due to hemolysis.\n* Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitidis, including serogroup B meningococcus and Streptococcus pneumoniae) within five years of screening or willing to complete protocol specified vaccinations.\n* Having given written informed consent prior to undertaking any study-related procedure.\n\nExclusion criteria:\n\n* Cold agglutinin syndrome secondary to infection, rheumatologic disease, or known high grade hematologic malignancy, or known solid organ tumor.\n* Clinically relevant infection of any kind within one month preceding screening.\n* Treatment with anti-CD20 monotherapy within three months or anti CD20 combination therapies within six months prior to screening.\n* Concurrent treatment with systemic immunosuppressive agents targeting B- or T-cell function and/or cytotoxic agents within 3 months prior to screening. Concurrent treatment with other systemic immunosuppressants within 5.5 half-lives of the drug prior to screening.\n* Any specific complement system inhibitor within three months prior to screening.\n* Concurrent treatment with systemic corticosteroids other than a stable daily dose equivalent to ≤10 mg/day prednisone within three months prior to screening.\n* If female, pregnant or lactating.\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial."}, 'identificationModule': {'nctId': 'NCT04269551', 'briefTitle': 'A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'A Multicenter, Phase 1b, Open Label, Nonrandomized, Single Dose Study Evaluating the Safety, Tolerability and Activity of BIVV020 in Adults With Cold Agglutinin Disease', 'orgStudyIdInfo': {'id': 'PDY16370'}, 'secondaryIdInfos': [{'id': '2019-001844-22', 'type': 'EUDRACT_NUMBER'}, {'id': 'U1111-1233-0831', 'type': 'OTHER', 'domain': 'UTN'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'BIVV020 IV', 'description': 'Single administration dose 1, plus two optional doses of BIVV020 administered intravenously.', 'interventionNames': ['Drug: BIVV020']}], 'interventions': [{'name': 'BIVV020', 'type': 'DRUG', 'description': 'Pharmaceutical form:solution for injection Route of administration: intravenous', 'armGroupLabels': ['BIVV020 IV']}]}, 'contactsLocationsModule': {'locations': [{'zip': '30214', 'city': 'Fayetteville', 'state': 'Georgia', 'country': 'United States', 'facility': 'Investigational Site Number :8400002', 'geoPoint': {'lat': 33.44873, 'lon': -84.45493}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Investigational Site Number :8400009', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '10467', 'city': 'The Bronx', 'state': 'New York', 'country': 'United States', 'facility': 'Investigational Site Number :8400006', 'geoPoint': {'lat': 40.84985, 'lon': -73.86641}}, {'zip': '15232', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Investigational Site Number :8400008', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '98108', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': 'Investigational Site Number :8400004', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '45147', 'city': 'Essen', 'country': 'Germany', 'facility': 'Investigational Site Number :2760001', 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'zip': '20122', 'city': 'Milan', 'state': 'Milano', 'country': 'Italy', 'facility': 'Investigational Site Number :3800001', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '1105AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Investigational Site Number :5280001', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': '5021', 'city': 'Bergen', 'country': 'Norway', 'facility': 'Investigational Site Number :5780001', 'geoPoint': {'lat': 60.39299, 'lon': 5.32415}}, {'zip': 'NW1 2PJ', 'city': 'London', 'state': 'London, City of', 'country': 'United Kingdom', 'facility': 'Investigational Site Number :8260001', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Clinical Sciences & Operations', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Sanofi'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': "Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org"}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bioverativ, a Sanofi company', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}