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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02124616
|Contact: Sahar MA Hassanein, MD, PhDfirstname.lastname@example.org|
|Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University||Recruiting|
|Cairo, Abassia, Egypt, 11381|
|Contact: Mohsen S Elalfy, MD, PhD email@example.com|
|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|