Ignite Creation Date:
2025-12-25 @ 1:01 AM
Ignite Modification Date:
2026-02-25 @ 9:44 PM
Study NCT ID:
NCT04982393
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-12-24
First Post:
2021-07-20
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C536414', 'term': 'Primary hyperoxaluria type 1'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 207}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-12-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2028-09-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-18', 'studyFirstSubmitDate': '2021-07-20', 'studyFirstSubmitQcDate': '2021-07-20', 'lastUpdatePostDateStruct': {'date': '2025-12-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-07-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-09-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Adverse Events in Lumasiran Treated Patients', 'timeFrame': 'Up to 7 years'}], 'secondaryOutcomes': [{'measure': 'Incidence of Selected Events of Interest in PH1 Patients', 'timeFrame': 'Up to 7 years', 'description': 'Selected events of interest are defined as hepatic events, kidney stones, acute kidney injury events, nephrocalcinosis, chromic kidney disease, kidney failure, and any cardiac, bone, skin, eye, hematological, or neuropathic manifestations due to oxalosis.'}, {'measure': '12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)', 'timeFrame': 'Up to 7 years', 'description': 'SF-12 V2 is a 12-question measure capturing global quality of life and overall health status and evaluates the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health.'}, {'measure': 'Change in Urinary Oxalate Excretion', 'timeFrame': 'Baseline and every 12 months for up to 7 years'}, {'measure': 'Change in Plasma Oxalate', 'timeFrame': 'Baseline and every 12 months for up to 7 years'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PH1', 'Lumasiran', 'Primary Hyperoxaluria Type 1'], 'conditions': ['Primary Hyperoxaluria Type 1']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients diagnosed with PH1 will be managed and treated per routine clinical practice.', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Documented diagnosis of PH1, per physician's determination\n\nExclusion Criteria:\n\n* Currently enrolled in a clinical trial for any investigational agent"}, 'identificationModule': {'nctId': 'NCT04982393', 'acronym': 'BONAPH1DE', 'briefTitle': 'BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alnylam Pharmaceuticals'}, 'officialTitle': 'BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)', 'orgStudyIdInfo': {'id': 'ALN-GO1-007'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Patients with PH1', 'description': 'Patients with a diagnosis of PH1 will be eligible for the study and will be managed and treated per routine clinical practice.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '85016', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'city': 'Ghent', 'country': 'Belgium', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'city': 'Liège', 'country': 'Belgium', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 50.63373, 'lon': 5.56749}}, {'city': 'Hamilton', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 43.25011, 'lon': -79.84963}}, {'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'city': 'Laurier', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Clinical Trial Site'}, {'city': 'Bordeaux', 'country': 'France', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 44.84124, 'lon': -0.58046}}, {'city': 'Lyon', 'country': 'France', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Paris', 'country': 'France', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Berlin', 'country': 'Germany', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Cologne', 'country': 'Germany', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 50.93333, 'lon': 6.95}}, {'city': 'Hamburg', 'country': 'Germany', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 53.55073, 'lon': 9.99302}}, {'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'city': 'Orbassano', 'state': 'Torino', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 45.00547, 'lon': 7.53813}}, {'city': 'Milan', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'city': 'Verona', 'country': 'Italy', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 45.43854, 'lon': 10.9938}}, {'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'city': 'Santa Cruz de Tenerife', 'state': 'Canary Islands', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 28.46824, 'lon': -16.25462}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'city': 'Las Palmas', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 28.58232, 'lon': -16.16555}}, {'city': 'Bern', 'country': 'Switzerland', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 46.94809, 'lon': 7.44744}}, {'city': 'London', 'state': 'England', 'country': 'United Kingdom', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Alnylam Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alnylam Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}