National Registry for Egyptian Pediatric Neuromuscular Diseases

• Conditions: Muscular Dystrophy; Muscle Diseases; Spinal Muscular Atrophy; Polyneuropathies; Myasthenic Syndromes

• Registration Number: NCT02124616
• Lead Sponsor: Ain Shams University

Brief Summary

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.

Detailed Description

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 Timeline

• Status Verified: 2014-04
• Study Start: 2014-04
• Study First Submitted: 2014-04-25
• Study First Submitted QC: 2014-04-25
• Last Update Submitted: 2014-04-25
• Study First Posted: 2014-04-28
• Last Update Posted: 2014-04-28
• Primary Completion: 2020-04
• Study Completion: 2020-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

• Weakness, hypotonia.
• Nerve conduction study and electromyographic confirmation of lower motor neuron affection.

Exclusion Criteria

• Chromosomal diseases.
• Malformations and deformations.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Functional motor ability	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.

Secondary Outcome Measures

Name	Time	Method
Cardio-pulmonary function	12 months	Pulmonary and cardiac function impairment.

Trial Locations

Locations (1)

Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University; 🇪🇬 Cairo, Abassia, Egypt