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National Registry for Egyptian Pediatric Neuromuscular Diseases

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02124616
Recruitment Status : Recruiting
First Posted : April 28, 2014
Last Update Posted : April 28, 2014
Information provided by (Responsible Party):
Sahar M.A. Hassanein, MD, 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.

Condition or disease
Spinal Muscular Atrophy Muscular Dystrophy Muscle Diseases Myasthenic Syndromes Polyneuropathies

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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Case-Only
Time Perspective: Prospective
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

Neuromuscular diseases
Prospective cohort of children with inherited or acquired neuromuscular diseases.

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Cardio-pulmonary function [ Time Frame: 12 months ]
    Pulmonary and cardiac function impairment.

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All children from birth to 18 years of age with inherited or acquired neuromuscular diseases.

Inclusion Criteria:

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

Exclusion Criteria:

  • Chromosomal diseases.
  • Malformations and deformations.

Information from the National Library of Medicine

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 identifier (NCT number): NCT02124616

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Contact: Sahar MA Hassanein, MD, PhD 201223183943

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Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University Recruiting
Cairo, Abassia, Egypt, 11381
Contact: Mohsen S Elalfy, MD, PhD   
Principal Investigator: Sahar MA Hassanein, MD, PhD         
Sub-Investigator: Nafissa El Badawy El Badawy, MD, PhD         
Sub-Investigator: Abdelfattah Souad F Souad, MD, PhD         
Sponsors and Collaborators
Ain Shams University
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Study Chair: Sahar MA Hassanein, MD, PhD Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University

Additional Information:
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Responsible Party: Sahar M.A. Hassanein, MD, Professor of Pediatrics, Ain Shams University Identifier: NCT02124616     History of Changes
Other Study ID Numbers: EGYPT PED-NMD 2014
First Posted: April 28, 2014    Key Record Dates
Last Update Posted: April 28, 2014
Last Verified: April 2014

Keywords provided by Sahar M.A. Hassanein, MD, Ain Shams University:
spinal muscular atrophy
Muscular dystrophy
Muscle diseases
myasthenic syndromes

Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Atrophy
Muscular Atrophy, Spinal
Neuromuscular Diseases
Lambert-Eaton Myasthenic Syndrome
Muscular Diseases
Pathological Conditions, Anatomical
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms
Peripheral Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Autoimmune Diseases
Immune System Diseases