Ignite Creation Date:
2025-12-26 @ 10:56 AM
Ignite Modification Date:
2026-02-22 @ 4:53 PM
Study NCT ID:
NCT02090608
Status:
COMPLETED
Last Update Posted:
2014-03-18
First Post:
2014-03-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Paricalcitol in Fabry Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}, {'id': 'D011507', 'term': 'Proteinuria'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D014555', 'term': 'Urination Disorders'}, {'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': 'D020924', 'term': 'Urological Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C084656', 'term': 'paricalcitol'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-01', 'completionDateStruct': {'date': '2013-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-03-17', 'studyFirstSubmitDate': '2014-03-14', 'studyFirstSubmitQcDate': '2014-03-17', 'lastUpdatePostDateStruct': {'date': '2014-03-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2014-03-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Effect of paricalcitol on proteinuria reduction', 'timeFrame': '6 months', 'description': 'Fourteen Fabry patients will be selected and studied in the first six months of add-on oral PCT (1 mcg/day) and, in order to verify the dependence of proteinuria reduction on PCT, three months after drug withdrawal.'}]}, 'conditionsModule': {'conditions': ['Fabry Disease', 'Proteinuria']}, 'descriptionModule': {'briefSummary': 'Proteinuria is the predominant risk factor for renal disease progression in Fabry disease (FD). When urine protein excretion is controlled to \\<0.50 g/24 hr, the rate loss of glomerular filtration rate (GFR) is not significantly different from 0. However, enzyme replacement therapy (ERT) alone does not decrease proteinuria and it has been recommended that patients receiving ERT also receive anti Renin-Angiotensin-System (RAS) therapy. Emerging evidences show that paricalcitol (PCT) reduces proteinuria in presence of intensified inhibition of RAS; however, there is no evidence in FD. The aim of this study is to evaluate the antiproteinuric effect of PCT in FD patients with proteinuria \\>0.50 g/24 hr persisting despite the ERT and anti-RAS therapy titrated to maximum tolerated dosage.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* genetically proven FD\n* stable dose of ERT for at least 12 months\n* stable dose of ACEi or ARB titrated to maximum tolerated dosage for at least 6 months\n* persistent proteinuria \\>0.50 g/24 h despite the use of ERT and ACEi/ARBs in 2 consecutive samples within 12 weeks\n\nExclusion Criteria:\n\n* steroid/immunosuppressive treatment or glomerular filtration rate change \\>30% in the past 3 months\n* PTH levels \\<20 pg/mL\n* serum phosphorus \\>5.0 mg/dL\n* serum calcium (adjusted for albumin) \\>10.0 mg/dL\n* active malignancy.'}, 'identificationModule': {'nctId': 'NCT02090608', 'briefTitle': 'Paricalcitol in Fabry Disease', 'organization': {'class': 'OTHER', 'fullName': 'Federico II University'}, 'orgStudyIdInfo': {'id': 'PCT-FD'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Paricalcitol', 'description': 'In patients identified by the inclusion criteria, data will be collected at baseline , during administration of oral Paricalcitol (PCT) (after 1, 3 and 6 months), and three months after PCT withdrawal. PCT will administered at dosage of 1 mcg/day; this dosage was chosen as it is not associated with excessive decline of parathyroid hormone (PTH) levels in most patients', 'interventionNames': ['Drug: Paricalcitol']}], 'interventions': [{'name': 'Paricalcitol', 'type': 'DRUG', 'description': 'Paricalcitol was administered at the dose of 1 mcg/die', 'armGroupLabels': ['Paricalcitol']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80129', 'city': 'Naples', 'state': 'Naples', 'country': 'Italy', 'facility': 'federico II university, department of nephrology', 'geoPoint': {'lat': 40.85216, 'lon': 14.26811}}], 'overallOfficials': [{'name': 'eleonora riccio, md', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Federico II University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Federico II University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD', 'investigatorFullName': 'Eleonora Riccio', 'investigatorAffiliation': 'Federico II University'}}}}