Ignite Creation Date:
2025-12-25 @ 1:23 AM
Ignite Modification Date:
2025-12-27 @ 10:53 PM
Study NCT ID:
NCT06036693
Status:
RECRUITING
Last Update Posted:
2025-01-31
First Post:
2023-07-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
MPS (RaDiCo Cohort) (RaDiCo-MPS)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008059', 'term': 'Mucopolysaccharidosis I'}, {'id': 'D016532', 'term': 'Mucopolysaccharidosis II'}, {'id': 'D009084', 'term': 'Mucopolysaccharidosis III'}, {'id': 'D009085', 'term': 'Mucopolysaccharidosis IV'}, {'id': 'D009087', 'term': 'Mucopolysaccharidosis VI'}, {'id': 'D016538', 'term': 'Mucopolysaccharidosis VII'}, {'id': 'D052517', 'term': 'Multiple Sulfatase Deficiency Disease'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D052516', 'term': 'Sulfatidosis'}, {'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': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 1000}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2017-12-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2026-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-01-29', 'studyFirstSubmitDate': '2023-07-18', 'studyFirstSubmitQcDate': '2023-09-12', 'lastUpdatePostDateStruct': {'date': '2025-01-31', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-09-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluation of the clinical data of MPS like growth for each system', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the clinical data of MPS like signs for each system', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the clinical data of MPS like symptoms for each system', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the clinical data of MPS like complications for each system', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the clinical data of MPS like psychomotor milestones', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the clinical data of MPS like cognitive evolution', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the clinical data of MPS like handicap using scales adapted to multivisceral disease for all types of MPS', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the clinical data of MPS like handicap using scales adapted to cognitive and neurologic disease for the types I, II, III VII', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the radiological data of MPS like standard bone radiographs', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the radiological data of MPS like abdominal echography', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the radiological data of MPS like echocardiography', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the radiological data of MPS like cerebral and medullar tomodensitometry', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the radiological data of MPS like magnetic resonance imaging', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the electrophysiological data of MPS like EMG', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the electrophysiological data of MPS like EEG', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the electrophysiological data of MPS like ERG', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the biochemical data of MPS like urinary GAG before specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the biochemical data of MPS like urinary GAG during specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the biochemical data of MPS like enzyme activities before specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the biochemical data of MPS like enzyme activities during specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the biochemical data of MPS like specific antibodies', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Evaluation of the molecular data of MPS', 'timeFrame': 'Through study completion, an average of 5 years'}], 'secondaryOutcomes': [{'measure': 'Description of the management of MPS diseases without specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Description of the management of MPS diseases before specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Description of the management of MPS diseases under specific treatment.', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Description of the outcome of MPS diseases without specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Description of the outcome of MPS diseases before specific treatment', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Description of the outcome of MPS diseases under specific treatment.', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Identification of mutation(s) in each MPS type', 'timeFrame': 'Through study completion, an average of 5 years'}, {'measure': 'Establishment of genotype/phenotype relationships in each MPS type.', 'timeFrame': 'Through study completion, an average of 5 years'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Mucopolysaccharidosis I', 'Mucopolysaccharidosis II', 'Mucopolysaccharidosis III', 'Mucopolysaccharidosis IV', 'Mucopolysaccharidosis VI', 'Mucopolysaccharidosis VII', 'Mucopolysaccharidosis IX', 'Multiple Sulfatase Deficiency Disease']}, 'descriptionModule': {'briefSummary': 'The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collection of extensive phenotypic data from French MPS patients.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Prevalent and incident patients will be included in the cohort RaDiCo-MPS.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed diagnosis of MPS based on clinically relevant enzyme deficiency, with abnormally elevated GAG urinary excretion and/or identification of pathogenic mutations.\n* Signed informed consent or parents/guardian non-opposition for deceased patients (minor or protected major)\n\nThere are no non-inclusion criteria.'}, 'identificationModule': {'nctId': 'NCT06036693', 'briefTitle': 'MPS (RaDiCo Cohort) (RaDiCo-MPS)', 'organization': {'class': 'OTHER_GOV', 'fullName': 'Institut National de la Santé Et de la Recherche Médicale, France'}, 'officialTitle': 'Mucopolysaccharidosis Patients in France in the Era of Specific Therapeutics', 'orgStudyIdInfo': {'id': 'C16-53'}}, 'contactsLocationsModule': {'locations': [{'city': 'Angers', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Magalie BARTH', 'role': 'CONTACT'}], 'facility': "Centre Hospitalier Universitaire d'Angers", 'geoPoint': {'lat': 47.47156, 'lon': -0.55202}}, {'city': 'Bordeaux', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Didier LACOMBE', 'role': 'CONTACT'}], 'facility': 'Hôpital des Enfants - Groupe Hospitalier Pellegrin', 'geoPoint': {'lat': 44.84124, 'lon': -0.58046}}, {'city': 'Brest', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Elise SACAZE', 'role': 'CONTACT'}], 'facility': 'Hôpital Morvan', 'geoPoint': {'lat': 48.39029, 'lon': -4.48628}}, {'city': 'Clermont-Ferrand', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Marc BERGER', 'role': 'CONTACT'}], 'facility': "Hôpital d'Estaing", 'geoPoint': {'lat': 45.77969, 'lon': 3.08682}}, {'city': 'Clichy', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Nadia BELMATOUG', 'role': 'CONTACT'}], 'facility': 'Hôpital Beaujon', 'geoPoint': {'lat': 48.90018, 'lon': 2.30952}}, {'city': 'Garches', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Dominique-Paul GERMAIN', 'role': 'CONTACT'}], 'facility': 'Hôpital Raymond-Poincaré', 'geoPoint': {'lat': 48.84226, 'lon': 2.18232}}, {'city': 'Lille', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Anne-Sophie GUEMANN', 'role': 'CONTACT'}], 'facility': 'Hôpital Jeanne de Flandre', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'city': 'Marseille', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Brigitte CHABROL', 'role': 'CONTACT'}], 'facility': 'Hôpital de la Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'city': 'Montpellier', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Agathe ROUBERTIE', 'role': 'CONTACT'}], 'facility': 'Hôpital Gui de Chauliac', 'geoPoint': {'lat': 43.61093, 'lon': 3.87635}}, {'city': 'Nancy', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'François FEILLET', 'role': 'CONTACT'}], 'facility': 'Hôpital Brabois', 'geoPoint': {'lat': 48.68439, 'lon': 6.18496}}, {'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Bénédicte HERON', 'role': 'CONTACT'}], 'facility': 'Hôpital Armand Trousseau', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Paris', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Wladimir MAUHIN', 'role': 'CONTACT'}], 'facility': 'Hôpital de la Croix Saint-Simon', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Yann NADJAR', 'role': 'CONTACT'}], 'facility': 'Hôpital de la Pitié-Salpêtrière', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Anaïs BRASSIER', 'role': 'CONTACT'}], 'facility': 'Hôpital Necker-Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Samia PICHARD', 'role': 'CONTACT'}], 'facility': 'Hôpital Robert Debré', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Pau', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Pierre BEZE-BEYRIE', 'role': 'CONTACT'}], 'facility': 'Centre Hospitalier de Pau', 'geoPoint': {'lat': 43.31117, 'lon': -0.35583}}, {'city': 'Reims', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Nathalie BEDNAREK', 'role': 'CONTACT'}], 'facility': 'American Memorial Hospital', 'geoPoint': {'lat': 49.26526, 'lon': 4.02853}}, {'city': 'Rennes', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Bérengère CADOR', 'role': 'CONTACT'}], 'facility': 'Hôpital Pontchaillou', 'geoPoint': {'lat': 48.11109, 'lon': -1.67431}}, {'city': 'Rouen', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Stéphanie TORRE', 'role': 'CONTACT'}], 'facility': 'Hôpital Charles Nicolle', 'geoPoint': {'lat': 49.44313, 'lon': 1.09932}}, {'city': 'Strasbourg', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Marie-Thérèse ABI WARDE', 'role': 'CONTACT'}], 'facility': 'Hôpital de Hautepierre', 'geoPoint': {'lat': 48.58392, 'lon': 7.74553}}, {'city': 'Toulouse', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Francis GACHES', 'role': 'CONTACT'}], 'facility': 'Clinique Monié', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'city': 'Toulouse', 'status': 'NOT_YET_RECRUITING', 'country': 'France', 'contacts': [{'name': 'Guy TOUATI', 'role': 'CONTACT'}], 'facility': 'Hôpital des Enfants', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'city': 'Tours', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Marine TARDIEU', 'role': 'CONTACT'}], 'facility': 'Hôpital Clocheville', 'geoPoint': {'lat': 47.39484, 'lon': 0.70398}}], 'centralContacts': [{'name': 'Bénédicte HERON', 'role': 'CONTACT', 'email': 'benedicte.heron@aphp.fr', 'phone': '01 44 73 65 75'}], 'overallOfficials': [{'name': 'Thierry BILLETTE DE VILLEMEUR', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'INSERM UMR 1141'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Institut National de la Santé Et de la Recherche Médicale, France', 'class': 'OTHER_GOV'}, 'responsibleParty': {'type': 'SPONSOR'}}}}