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NCT02124616 Clinical Trial

National Registry for Egyptian Pediatric Neuromuscular Diseases

Spinal Muscular Atrophy Clinical Trial

Official title:
National Registry for Egyptian Pediatric Neuromuscular Diseases

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT02124616
Other study ID # EGYPT PED-NMD 2014
Secondary ID
Status Recruiting
Phase N/A
First received April 25, 2014
Last updated April 25, 2014
Start date April 2014
Est. completion date December 2020

Study information
Verified date April 2014
Source Ain Shams University
Contact Sahar MA Hassanein, MD, PhD
Phone 201223183943
Email saharhassanein@med.asu.edu.eg
Is FDA regulated No
Health authority United States: Institutional Review Board
Study type Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Recruiting
Enrollment 200
Est. completion date December 2020
Est. primary completion date April 2020
Accepts healthy volunteers No
Gender Both
Age group 1 Month to 18 Years
Eligibility Inclusion Criteria:

- Weakness, hypotonia.

- Nerve conduction study and electromyographic confirmation of lower motor neuron affection.

Exclusion Criteria:

- Chromosomal diseases.

- Malformations and deformations.

Study Design

Observational Model: Case-Only, Time Perspective: Prospective

Related Conditions & MeSH terms

Locations
Country Name City State
Egypt Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University Cairo Abassia

Sponsors (1)
Lead Sponsor Collaborator
Ain Shams University

Country where clinical trial is conducted

Egypt, 

Outcome
Type Measure Description Time frame Safety issue
Primary Functional motor ability 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.		 1 year No
Secondary Cardio-pulmonary function Pulmonary and cardiac function impairment. 12 months No
See also
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Recruiting NCT05366465 - Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France
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