Ignite Creation Date:
2025-12-24 @ 10:08 PM
Ignite Modification Date:
2025-12-29 @ 6:50 AM
Study NCT ID:
NCT04014335
Status:
COMPLETED
Last Update Posted:
2025-01-23
First Post:
2019-07-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005922', 'term': 'Glomerulonephritis, IGA'}], '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': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 23}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-12-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'dispFirstSubmitDate': '2025-01-17', 'completionDateStruct': {'date': '2024-04-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-01-17', 'studyFirstSubmitDate': '2019-07-08', 'studyFirstSubmitQcDate': '2019-07-08', 'dispFirstPostDateStruct': {'date': '2024-05-14', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-01-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-07-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-02-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent Reduction in 24-hour Urine Protein Excretion', 'timeFrame': 'Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured)'}], 'secondaryOutcomes': [{'measure': 'Absolute Reduction in 24-hour Urine Protein Excretion', 'timeFrame': 'Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured)'}, {'measure': 'Absolute Reduction in Albuminuria (UACr Ratio)', 'timeFrame': 'Baseline to Week 29'}, {'measure': 'Absolute Reduction in Proteinuria (UPCr Ratio)', 'timeFrame': 'Baseline to Week 29'}, {'measure': 'Percent Change from Baseline in Plasma Factor B (FB)', 'timeFrame': 'Up to Week 29'}, {'measure': 'Percent Change from Baseline in Plasma AH50', 'timeFrame': 'Up to Week 29'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Primary IgA Nephropathy']}, 'referencesModule': {'references': [{'pmid': '39331470', 'type': 'DERIVED', 'citation': 'Tekendo-Ngongang C, Gleeson JG, Mignon L. Treating the Untreatable: Antisense Oligonucleotides as an Individualized Therapy for Rare Genetic Kidney Diseases. J Am Soc Nephrol. 2024 Dec 1;35(12):1774-1777. doi: 10.1681/ASN.0000000532. Epub 2024 Sep 27. No abstract available.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma factor B (FB) levels and serum AH50, CH50 activity in participants with primary immunoglobulin A (IgA) nephropathy.', 'detailedDescription': 'This is a Phase 2, single arm open-label clinical study in up to 25 participants that will consist of a screening period, a 24-week treatment period, an optional treatment extension period of up to an additional 48 weeks, and a 12- week post-treatment follow-up evaluation period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria\n\n* Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal OR use a highly effective method of birth control\n* Biopsy-proven primary immunoglobulin A (IgA) nephropathy\n* Hematuria\n* Proteinuria\n\nExclusion Criteria\n\n* Clinically significant abnormalities in medical history (e.g., dementia, stroke, acute coronary syndrome, thrombocytopenia, or major surgery within 3 months of Screening)\n* Diagnosis of primary or secondary immunodeficiencies of B-lymphocyte function, splenectomy, or history of recurrent meningococcal disease\n* Active infection 30 days prior to study\n* Estimated glomerular filtration rate (eGFR) ≤ 40 milliliters per minute per 1.73 square meters (mL/min/1.73m\\^2) using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)\n* Presence of another renal disease including, but not limited to, diabetes and/or diabetic nephropathy, thin basement membrane disease, Alport's disease, IgA Nephritis (Henoch-Schonlein purpura), lupus nephritis, Minimal Change Disease, post-infectious glomerulonephritis or any other cause of proteinuria or secondary IgA nephropathy (including, but not limited to Celiac disease, Crohn's disease, human immunodeficiency virus (HIV), liver cirrhosis)\n* History of renal transplant or another organ transplant\n* Treatment with another investigational drug, biological agent, or device within 6 months of screening, or 5 half-lives of investigational agent, whichever is longer\n* Administration of immunosuppressive/immunomodulatory medication 12 months prior to study drug administration, except for short-term treatments.\n* Other protocol-specified inclusion/exclusion criteria may apply"}, 'identificationModule': {'nctId': 'NCT04014335', 'briefTitle': 'A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ionis Pharmaceuticals, Inc.'}, 'officialTitle': 'An Open-Label Phase 2a Clinical Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Subjects With Primary IgA Nephropathy', 'orgStudyIdInfo': {'id': 'ISIS 696844-CS4'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'IONIS-FB-LRx', 'interventionNames': ['Drug: IONIS-FB-LRx']}], 'interventions': [{'name': 'IONIS-FB-LRx', 'type': 'DRUG', 'description': 'Participants will receive IONIS-FB-LRx, by subcutaneous injection (SC) at Week 1 and every 4 weeks through Week 25. Optional 48-week Extension, with drug dosing continuing every 4 weeks.', 'armGroupLabels': ['IONIS-FB-LRx']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2170', 'city': 'Liverpool', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'IONIS Investigative Site', 'geoPoint': {'lat': -33.91938, 'lon': 150.92588}}, {'zip': '2065', 'city': 'St Leonards', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'IONIS Investigative Site', 'geoPoint': {'lat': -33.82344, 'lon': 151.19836}}, {'zip': '3050', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': 'IONIS Investigative Site', 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': 'V6Z 1Y6', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': 'IONIS Investigative Site', 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'M4G 3E8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'IONIS Investigative Site', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': '8011', 'city': 'Christchurch', 'country': 'New Zealand', 'facility': 'IONIS Investigative Site', 'geoPoint': {'lat': -43.53333, 'lon': 172.63333}}, {'zip': '168582', 'city': 'Singapore', 'country': 'Singapore', 'facility': 'IONIS Investigative Site', 'geoPoint': {'lat': 1.28967, 'lon': 103.85007}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ionis Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}