Ignite Creation Date:
2025-12-24 @ 2:00 PM
Ignite Modification Date:
2026-01-15 @ 3:00 PM
Study NCT ID:
NCT02904395
Status:
COMPLETED
Last Update Posted:
2016-09-19
First Post:
2016-09-06
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006009', 'term': 'Glycogen Storage Disease Type II'}], 'ancestors': [{'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': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D006008', 'term': 'Glycogen Storage Disease'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': "filter paper with blood to look for enzyme activity and if enzyme activity low, PCR looking for the signature mutations for infantile Pompe's Disease"}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3413}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-09', 'completionDateStruct': {'date': '2015-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-09-13', 'studyFirstSubmitDate': '2016-09-06', 'studyFirstSubmitQcDate': '2016-09-13', 'lastUpdatePostDateStruct': {'date': '2016-09-19', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2016-09-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'acid maltase activity', 'timeFrame': 'At birth', 'description': 'The acid maltase enzyme activity is evaluated just after birth. If it is abnormal then PCR allows to identify signature mutations.'}, {'measure': "Signature mutation for pompe's disease", 'timeFrame': 'At birth', 'description': 'The acid maltase enzyme activity is evaluated just after birth. If it is abnormal then PCR allows to identify signature mutations.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ["Pompe's Disease"]}, 'descriptionModule': {'briefSummary': "Given the 100 fold increase of the incidence of Pompe's disease in Western French Guiana, the objective of the present study is to implement systematic screening in newborns in French Guiana in order to start treatment before the muscular and cardiac symptoms appear."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '2 Days', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'newborns', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* newborn\n\nExclusion Criteria:\n\n* parent refusal'}, 'identificationModule': {'nctId': 'NCT02904395', 'acronym': 'DEPIPOMP1', 'briefTitle': "Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth", 'organization': {'class': 'OTHER', 'fullName': 'Centre Hospitalier de Cayenne'}, 'officialTitle': "Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth", 'orgStudyIdInfo': {'id': 'DEPIPOMP1'}}, 'armsInterventionsModule': {'interventions': [{'name': 'observational (no intervention)', 'type': 'OTHER'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Centre Hospitalier de Cayenne', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}