Ignite Creation Date:
2025-12-24 @ 4:12 PM
Ignite Modification Date:
2026-01-06 @ 5:34 AM
Study NCT ID:
NCT05162066
Status:
TERMINATED
Last Update Posted:
2024-05-02
First Post:
2021-11-26
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015432', 'term': 'Glomerulonephritis, Membranoproliferative'}, {'id': 'D005922', 'term': 'Glomerulonephritis, IGA'}, {'id': 'D015433', 'term': 'Glomerulonephritis, Membranous'}], 'ancestors': [{'id': 'D005921', 'term': 'Glomerulonephritis'}, {'id': 'D009393', 'term': 'Nephritis'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@biocryst.com', 'phone': '+1 919-859-1302', 'title': 'Study Director', 'organization': 'BioCryst Pharmaceuticals Inc'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'The sponsor decided to prematurely terminate the study due to changes in the competitive landscape. Due to low enrolment in the study (N=2) and early termination of both participants, very limited data were available for analyses. Therefore, only key efficacy endpoints (24-hour uPCR, 24-hour urine protein excretion, and eGFR) and safety were analyzed and reported.'}}, 'adverseEventsModule': {'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'Safety population', 'eventGroups': [{'id': 'EG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).', 'otherNumAtRisk': 2, 'deathsNumAtRisk': 2, 'otherNumAffected': 2, 'seriousNumAtRisk': 2, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Eyelid oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Hypertransaminasaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 5, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Asymptomatic bacteriuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percent Change From Baseline in 24-hour uPCR at Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'categories': [{'measurements': [{'value': '-33.2', 'spread': 'NA', 'comment': 'Standard deviation was not calculated as only one participant was analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 12', 'description': 'Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population with available data was analyzed'}, {'type': 'PRIMARY', 'title': 'Percent Change From Baseline in 24-hour uPCR at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'categories': [{'measurements': [{'value': '-8.8', 'spread': 'NA', 'comment': 'Standard deviation was not calculated as only one participant was analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population with available data was analyzed'}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in 24-hour Urinary Protein Excretion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'title': 'Percent Change at Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-40.9', 'spread': 'NA', 'comment': 'Standard deviation was not calculated as only one participant was analyzed', 'groupId': 'OG000'}]}]}, {'title': 'Percent Change at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-25.8', 'spread': 'NA', 'comment': 'Standard deviation was not calculated as only one participant was analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 12, 24,', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population with available data was analyzed'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Estimated Glomerular Filtration Rate', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'title': 'Change at Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.0', 'spread': 'NA', 'comment': 'Standard deviation was not calculated as only one participant was analyzed', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.0', 'spread': 'NA', 'comment': 'Standard deviation was not calculated as only one participant was analyzed', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 12, 24', 'description': 'eGFR was calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for participants ≥ 18 years old and by the bedside Schwartz formula for participants ≤ 18 years old. eGFR was reported in milliliter per minute per 1.73 per square meter (mL/min/1.73m\\^2).', 'unitOfMeasure': 'mL/min/1.73m^2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population with available data was analyzed'}, {'type': 'SECONDARY', 'title': 'Number of Participants With a Treatment-emergent Adverse Event (TEAE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'An Adverse event (AE) was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Discontinued Due to a TEAE', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': "An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.\n\nA serious adverse event (SAE) was an adverse event/reaction that results in any of the following outcomes:\n\n* Death\n* Is life-threatening (participant was at immediate risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe)\n* Requires participant hospitalization (formal admission to a hospital for medical reasons) or prolongation of existing hospitalization\n* Results in persistent or significant disability/incapacity (ie, there was a substantial disruption of a person's ability to carry out normal life functions)\n* Is a congenital anomaly/birth defect", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'An AE was any untoward medical occurrence has no causal relationship with the study drug or with the clinical study itself. It was an unfavorable \\& unintended sign, symptom, syndrome, or illness that developed or worsened during clinical study.\n\nTEAEs: AEs that started on or after first dose of treatment through 30 days after the last dose of study drug. All AEs were graded using the CTCAE Grades 1 through 5.\n\nGrade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated\n\nGrade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL)\n\nGrade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL\n\nGrade 4: Life-threatening consequences; urgent intervention indicated\n\nGrade 5: Death\n\nParticipants with Grades 3 or 4 AEs were reported', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'BCX9930', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'Treatment-emergent Laboratory Abnormality were defined as an event that started on or after the first dose of study treatment through 30 days after the last dose of study drug.\n\nCTCAE Grades for laboratory abnormalities include:\n\nGrade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated.\n\nGrade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL.\n\nGrade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL.\n\nGrade 4: Life-threatening consequences; urgent intervention indicated.\n\nGrade 5: Death\n\nParticipants with Grades 3 or 4 laboratory abnormality were reported.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'BCX9930 - C3G Cohort', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}, {'id': 'FG001', 'title': 'BCX9930 - IgAN Cohort', 'description': 'Participants with complement IgAN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).'}, {'id': 'FG002', 'title': 'BCX9930 - PMN Cohort', 'description': 'Participants with complement PMN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Perceived lack of efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Participants with either complement 3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN), or primary membranous nephropathy (PMN) were planned to be enrolled in the study.', 'preAssignmentDetails': 'At the time the study was discontinued, only participants with historical C3G were enrolled.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'BCX9930 - C3G Cohort', 'description': 'Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).'}, {'id': 'BG001', 'title': 'BCX9930 - IgAN Cohort', 'description': 'Participants with complement IgAN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).'}, {'id': 'BG002', 'title': 'BCX9930 - PMN Cohort', 'description': 'Participants with complement PMN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '35.5', 'spread': '16.26', 'groupId': 'BG000'}, {'value': '35.5', 'spread': '16.26', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'White', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': '24-hour Urine Protein-to-creatinine Ratio (uPCR)', 'classes': [{'categories': [{'measurements': [{'value': '380.5', 'spread': '137.89', 'groupId': 'BG000'}, {'value': '380.5', 'spread': '137.89', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'description': 'Urinary protein/creatinine ratio (milligram per millimole \\[mg/mmol\\]) =urine protein concentration (mg/deciliter \\[dL\\])/ urine creatinine concentration (mmol/dL).', 'unitOfMeasure': 'milligram per millimole', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Safety population included all participants who received at least one dose of the study drug.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-08-04', 'size': 4642911, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-03-07T12:35', 'hasProtocol': True}, {'date': '2023-02-14', 'size': 2789149, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-02-26T04:21', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Three parallel treatment cohorts based on diagnosis of C3G, IgAN, or PMN. All eligible participants will receive open-label BCX9930.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}}, 'statusModule': {'whyStopped': 'Sponsor Decision', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2022-02-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-04', 'completionDateStruct': {'date': '2022-09-23', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-04-08', 'studyFirstSubmitDate': '2021-11-26', 'resultsFirstSubmitDate': '2024-04-08', 'studyFirstSubmitQcDate': '2021-12-16', 'lastUpdatePostDateStruct': {'date': '2024-05-02', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-04-08', 'studyFirstPostDateStruct': {'date': '2021-12-17', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-05-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-09-23', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent Change From Baseline in 24-hour uPCR at Week 12', 'timeFrame': 'Baseline, Week 12', 'description': 'Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.'}, {'measure': 'Percent Change From Baseline in 24-hour uPCR at Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.'}], 'secondaryOutcomes': [{'measure': 'Percent Change From Baseline in 24-hour Urinary Protein Excretion', 'timeFrame': 'Baseline, Weeks 12, 24,'}, {'measure': 'Change From Baseline in Estimated Glomerular Filtration Rate', 'timeFrame': 'Baseline, Weeks 12, 24', 'description': 'eGFR was calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for participants ≥ 18 years old and by the bedside Schwartz formula for participants ≤ 18 years old. eGFR was reported in milliliter per minute per 1.73 per square meter (mL/min/1.73m\\^2).'}, {'measure': 'Number of Participants With a Treatment-emergent Adverse Event (TEAE)', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'An Adverse event (AE) was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.'}, {'measure': 'Number of Participants Who Discontinued Due to a TEAE', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.'}, {'measure': 'Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': "An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.\n\nA serious adverse event (SAE) was an adverse event/reaction that results in any of the following outcomes:\n\n* Death\n* Is life-threatening (participant was at immediate risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe)\n* Requires participant hospitalization (formal admission to a hospital for medical reasons) or prolongation of existing hospitalization\n* Results in persistent or significant disability/incapacity (ie, there was a substantial disruption of a person's ability to carry out normal life functions)\n* Is a congenital anomaly/birth defect"}, {'measure': 'Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'An AE was any untoward medical occurrence has no causal relationship with the study drug or with the clinical study itself. It was an unfavorable \\& unintended sign, symptom, syndrome, or illness that developed or worsened during clinical study.\n\nTEAEs: AEs that started on or after first dose of treatment through 30 days after the last dose of study drug. All AEs were graded using the CTCAE Grades 1 through 5.\n\nGrade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated\n\nGrade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL)\n\nGrade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL\n\nGrade 4: Life-threatening consequences; urgent intervention indicated\n\nGrade 5: Death\n\nParticipants with Grades 3 or 4 AEs were reported'}, {'measure': 'Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality', 'timeFrame': 'From first dose up to safety follow-up period (Week 28)', 'description': 'Treatment-emergent Laboratory Abnormality were defined as an event that started on or after the first dose of study treatment through 30 days after the last dose of study drug.\n\nCTCAE Grades for laboratory abnormalities include:\n\nGrade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated.\n\nGrade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL.\n\nGrade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL.\n\nGrade 4: Life-threatening consequences; urgent intervention indicated.\n\nGrade 5: Death\n\nParticipants with Grades 3 or 4 laboratory abnormality were reported.'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['BCX9930', 'factor D inhibitor', 'oral therapy', 'Complement 3 Glomerulopathy', 'Immunoglobulin A Nephropathy', 'Primary Membranous Nephropathy'], 'conditions': ['Complement 3 Glomerulopathy', 'Immunoglobulin A Nephropathy', 'Membranous Nephropathy']}, 'referencesModule': {'references': [{'pmid': '39899371', 'type': 'DERIVED', 'citation': 'Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available.'}]}, 'descriptionModule': {'briefSummary': 'The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Body weight ≥ 40 kilograms (kg)\n* Primary diagnosis of C3G, IgAN, or PMN confirmed by central pathology review\n* An estimated glomerular filtration rate (eGFR) ≥ 50 milliter per minute per 1.73 meter square (mL/min/1.73 m\\^2) (or ≥ 30 mL/min/1.73 m\\^2 after Data Monitoring Committee \\[DMC\\] recommendation)\n* Receiving treatment with a stable, maximum recommended or maximum tolerated dose of an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) for at least 60 days prior to the Day 1 Visit\n* Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series\n\nExclusion Criteria:\n\n* Known congenital deficiency of C1s, C1r, C1q, C2, or C4\n* History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation\n* Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition\n* History of malignancy within 5 years prior to the screening visit\n* Active serious bacterial, viral, or fungal infection or any other serious infection within 14 days of screening\n* Treatment with any systemic immunosuppressive or immunomodulatory therapy within 90 days OR anti-CD20 antibody therapies (eg, rituximab) within 180 days prior to the screening visit\n* Treatment with renin inhibitors (eg, aliskiren) or sodium-glucose-cotransporter 2 (SGLT2) inhibitors within 60 days prior to Day 1'}, 'identificationModule': {'nctId': 'NCT05162066', 'acronym': 'RENEW', 'briefTitle': 'Study to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN)', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioCryst Pharmaceuticals'}, 'officialTitle': 'An Open-Label, Safety, Tolerability, and Proof-of-Concept Study of Oral BCX9930 Therapy in Subjects With Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, or Primary Membranous Nephropathy', 'orgStudyIdInfo': {'id': 'BCX9930-211'}, 'secondaryIdInfos': [{'id': '2020-005855-19', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'C3G cohort', 'description': 'Approximately 14 eligible participants with C3G will be enrolled.', 'interventionNames': ['Drug: BCX9930']}, {'type': 'EXPERIMENTAL', 'label': 'IgAN cohort', 'description': 'Approximately 14 eligible participants with IgAN will be enrolled.', 'interventionNames': ['Drug: BCX9930']}, {'type': 'EXPERIMENTAL', 'label': 'PMN cohort', 'description': 'Approximately 14 eligible participants with PMN will be enrolled.', 'interventionNames': ['Drug: BCX9930']}], 'interventions': [{'name': 'BCX9930', 'type': 'DRUG', 'description': 'Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily', 'armGroupLabels': ['C3G cohort', 'IgAN cohort', 'PMN cohort']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Poitiers', 'country': 'France', 'facility': 'Investigative Site', 'geoPoint': {'lat': 46.58261, 'lon': 0.34348}}, {'city': 'Toulouse', 'country': 'France', 'facility': 'Investigative Site', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'city': 'Bari', 'country': 'Italy', 'facility': 'Investigative Site', 'geoPoint': {'lat': 41.12066, 'lon': 16.86982}}, {'city': 'Bergamo', 'country': 'Italy', 'facility': 'Investigative Site', 'geoPoint': {'lat': 45.69601, 'lon': 9.66721}}, {'city': 'Brescia', 'country': 'Italy', 'facility': 'Investigative Site', 'geoPoint': {'lat': 45.53558, 'lon': 10.21472}}, {'city': 'Turin', 'country': 'Italy', 'facility': 'Investigative Site', 'geoPoint': {'lat': 45.07049, 'lon': 7.68682}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': 'Investigative Site #2', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'city': 'Madrid', 'country': 'Spain', 'facility': 'Investigative Site #1', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'city': 'Madrid', 'country': 'Spain', 'facility': 'Investigative Site #2', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'city': 'Oxford', 'country': 'United Kingdom', 'facility': 'Investigative Site', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}], 'overallOfficials': [{'name': 'Carla M Nester, MD, MSA, FASN', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "University of Iowa Stead Family Children's Hospital"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioCryst Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}