National Registry for Egyptian Pediatric Neuromuscular Diseases
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|ClinicalTrials.gov Identifier: NCT02124616|
Recruitment Status : Recruiting
First Posted : April 28, 2014
Last Update Posted : April 28, 2014
|Condition or disease|
|Spinal Muscular Atrophy Muscular Dystrophy Muscle Diseases Myasthenic Syndromes Polyneuropathies|
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).
Participants: Eligible infants and children with inherited and acquired neuromuscular diseases.
DESIGN: This study is a prospective cohort study.
Outcome measures: Motor development assessment, respiratory and cardiac examination.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||200 participants|
|Target Follow-Up Duration:||5 Years|
|Official Title:||National Registry for Egyptian Pediatric Neuromuscular Diseases|
|Study Start Date :||April 2014|
|Estimated Primary Completion Date :||April 2020|
|Estimated Study Completion Date :||December 2020|
Prospective cohort of children with inherited or acquired neuromuscular diseases.
- Functional motor ability [ Time Frame: 1 year ]
Motor power in acquired acute neuromuscular diseases will be assessed at admission and morbidity and mortality at discharge from hospital.
Functional motor ability will be performed every 3 months for children with inherited neuromuscular diseases.
- Cardio-pulmonary function [ Time Frame: 12 months ]Pulmonary and cardiac function impairment.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02124616
|Contact: Sahar MA Hassanein, MD, PhDemail@example.com|
|Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University||Recruiting|
|Cairo, Abassia, Egypt, 11381|
|Contact: Mohsen S Elalfy, MD, PhD firstname.lastname@example.org|
|Principal Investigator: Sahar MA Hassanein, MD, PhD|
|Sub-Investigator: Nafissa El Badawy El Badawy, MD, PhD|
|Sub-Investigator: Abdelfattah Souad F Souad, MD, PhD|
|Study Chair:||Sahar MA Hassanein, MD, PhD||Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University|