Ignite Creation Date:
2025-12-25 @ 2:09 AM
Ignite Modification Date:
2026-01-03 @ 3:21 PM
Study NCT ID:
NCT04246060
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2021-11-29
First Post:
2020-01-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003554', 'term': 'Cystinosis'}], 'ancestors': [{'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003543', 'term': 'Cysteamine'}], 'ancestors': [{'id': 'D008624', 'term': 'Mercaptoethylamines'}, {'id': 'D005021', 'term': 'Ethylamines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D013438', 'term': 'Sulfhydryl Compounds'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 31}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2020-07-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-11', 'completionDateStruct': {'date': '2026-07-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-11-25', 'studyFirstSubmitDate': '2020-01-27', 'studyFirstSubmitQcDate': '2020-01-27', 'lastUpdatePostDateStruct': {'date': '2021-11-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-01-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-07-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'to descriptively assess the change from baseline and percent change from baseline in the quality of life overall summary sores at 12 months in switch cohort', 'timeFrame': '12 months'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Nephropathic Cystinosis']}, 'descriptionModule': {'briefSummary': 'Most of the real world evidence data related to efficacy of cysteamine therapy is retrospective. This study is a ambispective study to investigate the impact of cystine depletion therapy on the quality of life of patients and their parents.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'All Belgian nephropathic cystinosis patients.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria:\n\n* Confirmed diagnosis of nephropathic cystinosis\n* Undergoing cystine depletion therapy with oral cysteamine\n* Signature of informed concent.'}, 'identificationModule': {'nctId': 'NCT04246060', 'acronym': 'PROREAL', 'briefTitle': 'Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients', 'organization': {'class': 'OTHER', 'fullName': 'Chiesi SA/NV'}, 'officialTitle': 'Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis', 'orgStudyIdInfo': {'id': 'CHIESI-NIS-003'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Cohort 1', 'description': 'Patients on extended release cysteamine treatment at study enrollment', 'interventionNames': ['Drug: Cysteamine Bitartrate']}, {'label': 'Cohort 2', 'description': 'Patients switching from immediate release cysteamine to extended release cysteamine during the study', 'interventionNames': ['Drug: Cysteamine Bitartrate']}, {'label': 'Cohort 3', 'description': 'Patients remaining on immediate release cysteamine treatment', 'interventionNames': ['Drug: Cysteamine Bitartrate']}], 'interventions': [{'name': 'Cysteamine Bitartrate', 'type': 'DRUG', 'description': 'ERT', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 3']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Antwerp', 'country': 'Belgium', 'facility': 'UZA', 'geoPoint': {'lat': 51.22047, 'lon': 4.40026}}, {'city': 'Brussels', 'country': 'Belgium', 'facility': 'HUDERF', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'city': 'Brussels', 'country': 'Belgium', 'facility': 'UCL Saint-Luc', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'city': 'Ghent', 'country': 'Belgium', 'facility': 'UZ Gent', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'city': 'Leuven', 'country': 'Belgium', 'facility': 'UZ Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'city': 'Liège', 'country': 'Belgium', 'facility': 'CHC', 'geoPoint': {'lat': 50.63373, 'lon': 5.56749}}, {'city': 'Liège', 'country': 'Belgium', 'facility': 'CHU', 'geoPoint': {'lat': 50.63373, 'lon': 5.56749}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Chiesi SA/NV', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}