National Registry for Egyptian Pediatric Neuromuscular Diseases

Status

UNKNOWN

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT02124616

Enrollment

200

Registered

2014-04-28

Start date

2014-04-30

Completion date

2020-12-31

Last updated

2014-04-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Muscle Diseases, Muscular Dystrophy, Myasthenic Syndromes, Polyneuropathies, Spinal Muscular Atrophy

Keywords

spinal muscular atrophy, Muscular dystrophy, Muscle diseases, myasthenic syndromes, Polyneuropathies, Myopathies

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

1 Months to 18 Years

Healthy volunteers

Inclusion criteria

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

Exclusion criteria

* Chromosomal diseases. * Malformations and deformations.

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
Cardio-pulmonary function	12 months	Pulmonary and cardiac function impairment.

Countries

Egypt

Contacts

Primary ContactSahar MA Hassanein, MD, PhD

saharhassanein@med.asu.edu.eg201223183943

Outcome results