Ignite Creation Date:
2025-12-26 @ 11:49 AM
Ignite Modification Date:
2026-02-23 @ 3:02 PM
Study NCT ID:
NCT02124616
Status:
UNKNOWN
Last Update Posted:
2014-04-28
First Post:
2014-04-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
National Registry for Egyptian Pediatric Neuromuscular Diseases
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009134', 'term': 'Muscular Atrophy, Spinal'}, {'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D015624', 'term': 'Lambert-Eaton Myasthenic Syndrome'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D009135', 'term': 'Muscular Diseases'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009157', 'term': 'Myasthenia Gravis'}, {'id': 'D020361', 'term': 'Paraneoplastic Syndromes, Nervous System'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010257', 'term': 'Paraneoplastic Syndromes'}, {'id': 'D020274', 'term': 'Autoimmune Diseases of the Nervous System'}, {'id': 'D020511', 'term': 'Neuromuscular Junction Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 200}, 'targetDuration': '5 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2014-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-04', 'completionDateStruct': {'date': '2020-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2014-04-25', 'studyFirstSubmitDate': '2014-04-25', 'studyFirstSubmitQcDate': '2014-04-25', 'lastUpdatePostDateStruct': {'date': '2014-04-28', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2014-04-28', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2020-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Functional motor ability', 'timeFrame': '1 year', 'description': 'Motor power in acquired acute neuromuscular diseases will be assessed at admission and morbidity and mortality at discharge from hospital.\n\nFunctional motor ability will be performed every 3 months for children with inherited neuromuscular diseases.'}], 'secondaryOutcomes': [{'measure': 'Cardio-pulmonary function', 'timeFrame': '12 months', 'description': 'Pulmonary and cardiac function impairment.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['spinal muscular atrophy', 'Muscular dystrophy', 'Muscle diseases', 'myasthenic syndromes', 'Polyneuropathies', 'Myopathies'], 'conditions': ['Spinal Muscular Atrophy', 'Muscular Dystrophy', 'Muscle Diseases', 'Myasthenic Syndromes', 'Polyneuropathies']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://med.asu.edu.eg/', 'label': 'Faculty of Medicine, Ain Shams University website'}, {'url': 'http://www.asu.edu.eg/', 'label': 'Ain Shams University web site'}]}, 'descriptionModule': {'briefSummary': 'Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.', 'detailedDescription': 'Aims: An open-ended multi-center, national Egyptian study to collect and analyze data for children with Neuromuscular Diseases (NMD) inherited NMD (spinal muscular atrophy (SMA), Duchenne/Becker and congenital muscular dystrophies (DMD/BMD, CMD), congenital myopathies, and congenital myasthenic syndromes) and acquired NMD (neuropathies, myasthenia gravis and myositis).\n\nParticipants: Eligible infants and children with inherited and acquired neuromuscular diseases.\n\nDESIGN: This study is a prospective cohort study.\n\nOutcome measures: Motor development assessment, respiratory and cardiac examination.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '1 Month', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'All children from birth to 18 years of age with inherited or acquired neuromuscular diseases.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Weakness, hypotonia.\n* Nerve conduction study and electromyographic confirmation of lower motor neuron affection.\n\nExclusion Criteria:\n\n* Chromosomal diseases.\n* Malformations and deformations.'}, 'identificationModule': {'nctId': 'NCT02124616', 'briefTitle': 'National Registry for Egyptian Pediatric Neuromuscular Diseases', 'organization': {'class': 'OTHER', 'fullName': 'Ain Shams University'}, 'officialTitle': 'National Registry for Egyptian Pediatric Neuromuscular Diseases', 'orgStudyIdInfo': {'id': 'EGYPT PED-NMD 2014'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Neuromuscular diseases', 'description': 'Prospective cohort of children with inherited or acquired neuromuscular diseases.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '11381', 'city': 'Cairo', 'state': 'Abassia', 'status': 'RECRUITING', 'country': 'Egypt', 'contacts': [{'name': 'Mohsen S Elalfy, MD, PhD', 'role': 'CONTACT', 'email': 'elalfym@hotmail.com'}, {'name': 'Sahar MA Hassanein, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Nafissa El Badawy El Badawy, MD, PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Abdelfattah Souad F Souad, MD, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': "Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University", 'geoPoint': {'lat': 30.06263, 'lon': 31.24967}}], 'centralContacts': [{'name': 'Sahar MA Hassanein, MD, PhD', 'role': 'CONTACT', 'email': 'saharhassanein@med.asu.edu.eg', 'phone': '201223183943'}], 'overallOfficials': [{'name': 'Sahar MA Hassanein, MD, PhD', 'role': 'STUDY_CHAIR', 'affiliation': "Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ain Shams University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor of Pediatrics', 'investigatorFullName': 'Sahar M.A. Hassanein, MD', 'investigatorAffiliation': 'Ain Shams University'}}}}