Ignite Creation Date:
2025-12-24 @ 12:47 PM
Ignite Modification Date:
2026-01-31 @ 10:42 PM
Study NCT ID:
NCT04184661
Status:
COMPLETED
Last Update Posted:
2022-10-06
First Post:
2019-11-28
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D063730', 'term': 'Rickets, Hypophosphatemic'}], 'ancestors': [{'id': 'D012279', 'term': 'Rickets'}, {'id': 'D001851', 'term': 'Bone Diseases, Metabolic'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D002128', 'term': 'Calcium Metabolism Disorders'}, {'id': 'D017674', 'term': 'Hypophosphatemia'}, {'id': 'D010760', 'term': 'Phosphorus Metabolism Disorders'}, {'id': 'D014808', 'term': 'Vitamin D Deficiency'}, {'id': 'D001361', 'term': 'Avitaminosis'}, {'id': 'D003677', 'term': 'Deficiency Diseases'}, {'id': 'D044342', 'term': 'Malnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001800', 'term': 'Blood Specimen Collection'}], 'ancestors': [{'id': 'D013048', 'term': 'Specimen Handling'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D011677', 'term': 'Punctures'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': '25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_CONTROL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 52}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-01-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-10', 'completionDateStruct': {'date': '2021-10-27', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-10-05', 'studyFirstSubmitDate': '2019-11-28', 'studyFirstSubmitQcDate': '2019-11-28', 'lastUpdatePostDateStruct': {'date': '2022-10-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-12-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-10-27', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'number of osteoclastic cells obtained after at the end of differentiation', 'timeFrame': '1 day', 'description': 'The analysis of osteoclastic differentiation will be obtained from the bone cells from patients with burosumab and/or 1-25 (OH) vitamin D'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hypophosphatemic Rickets']}, 'descriptionModule': {'briefSummary': 'Fibroblast growth factor 23 (FGF23) is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a Phosphating agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of Parathyroid hormone (PTH) in most tissues.\n\nThe specific role of FGF23 on bone has yet to be demonstrated. In some diseases such as hypophosphatemic rickets (HR), the direct role of FGF23 on bone has not yet been studied to our knowledge, whereas these genetic hypophosphatemias are secondary to overexpression of FGF23, whether an activating mutation of FGF23 or inhibitory mutations of its inhibitors (Dentin matrix acidic phosphoprotein 1 (DMP1) and Phosphate-regulating neutral endopeptidase, X-linked (PHEX)). However, patients with X-linked hypophosphatemic rickets (XLH) have higher circulating FGF23 levels than healthy controls and these levels are higher in treated patients.\n\nManagement of XLH consists primarily of correcting the native vitamin D defect by prescribing active vitamin D analogs as well as phosphate supplementation to improve bone mineralization and decrease dental complications, growth, and bone deformities. Recently, a new therapeutic option has been developed for XLH, burosumab, a human monoclonal antibody that binds and inhibits FGF23 activity. The use of burosumab is currently authorized in France in some pediatric patients with severe forms of XLH.\n\nIndependently of the indirect bone effects of phosphate correction and vitamin D levels, the direct role of burosumab on bone cells has never been studied. The objective of this project is to study the osteoclastic biology of patients with HR compared to control patients, and to evaluate the direct impact of the treatments used in this pathology on human osteoclasts.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'patients with hypophosphatemic rickets and controls patients without hypophosphatemic rickets', 'healthyVolunteers': False, 'eligibilityCriteria': 'hypophosphatemic rickets patients:\n\nInclusion Criteria:\n\n* children from 2 yars-old to 18 years old and adults\n* patients with HR followed in the center of calcium and phosphorus metabolism rare diseases in Lyon-\n* Patients and parent / holder of parental authority who have been informed of the study and do not object to participate\n\nExclusion Criteria:\n\n* Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery.\n* Patients under tutorship or curatorship\n* Pregnant and / or breastfeeding woman\n* Patient deprived of liberty\n\nControls patients:\n\nInclusion Criteria:\n\n* children from 2 years-old to 18 years old and adults\n* patients with normal renal function (Schwartz glomerular filtration rate (GFR) \\>90 ml/min/1.73m²)\n* Patients and parent / holder of parental authority who have been informed of the study and do not object to participate\n\nExclusion Criteria:\n\n* Patient being treated with oral corticosteroid or having received more than 3 months of corticosteroid treatment before surgery.\n* Patients under tutorship or curatorship\n* Pregnant and / or breastfeeding woman\n* Patient deprived of liberty\n* Patient treated with immunosuppressive drugs\n* Patient with inflammatory disease'}, 'identificationModule': {'nctId': 'NCT04184661', 'acronym': 'HYPO-CLASTE', 'briefTitle': 'Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts', 'organization': {'class': 'OTHER', 'fullName': 'Hospices Civils de Lyon'}, 'officialTitle': 'Effect of Burosumab and 1-25 (OH) Vitamin D on Human Osteoclasts From Patients With Hypophosphatemic Rickets (HR)', 'orgStudyIdInfo': {'id': '69HCL19_0725'}, 'secondaryIdInfos': [{'id': '2019-A02914-53', 'type': 'OTHER', 'domain': 'ID-RCB'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'hypophosphatemic rickets patients', 'description': '30 hypophosphatemic rickets patients older than 2 years will be included in this study', 'interventionNames': ['Other: blood sample']}, {'label': 'controls patients', 'description': '10 controls patients from pediatric nephrology unit without hypophosphatemic rickets, older than 2 years will be included in this study', 'interventionNames': ['Other: blood sample']}], 'interventions': [{'name': 'blood sample', 'type': 'OTHER', 'description': '25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.', 'armGroupLabels': ['controls patients', 'hypophosphatemic rickets patients']}]}, 'contactsLocationsModule': {'locations': [{'zip': '69677', 'city': 'Bron', 'country': 'France', 'facility': 'Hôpital Femme mère enfant', 'geoPoint': {'lat': 45.73865, 'lon': 4.91303}}, {'city': 'Lyon', 'country': 'France', 'facility': 'Hôpital Edouard Herriot', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Paris', 'country': 'France', 'facility': 'Hôpital Bicêtre Paris Saclay', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hospices Civils de Lyon', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}