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National Registry for Egyptian Pediatric Neuromuscular Diseases

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Sahar M.A. Hassanein, MD, Ain Shams University
Information provided by (Responsible Party):
Sahar M.A. Hassanein, MD, Ain Shams University Identifier:
First received: April 25, 2014
Last updated: NA
Last verified: April 2014
History: No changes posted
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.

Spinal Muscular Atrophy
Muscular Dystrophy
Muscle Diseases
Myasthenic Syndromes

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: National Registry for Egyptian Pediatric Neuromuscular Diseases

Resource links provided by NLM:

Further study details as provided by Sahar M.A. Hassanein, MD, Ain Shams University:

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

Estimated Enrollment: 200
Study Start Date: April 2014
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: April 2020 (Final data collection date for primary outcome measure)
Neuromuscular diseases
Prospective cohort of children with inherited or acquired neuromuscular diseases.

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.


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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02124616

Contact: Sahar MA Hassanein, MD, PhD 201223183943

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
Study Chair: Sahar MA Hassanein, MD, PhD Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
  More Information

Additional Information:
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
Study First Received: April 25, 2014
Last Updated: April 25, 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:
Lambert-Eaton Myasthenic Syndrome
Paraneoplastic Syndromes, Nervous System
Paraneoplastic Syndromes
Muscular Dystrophies
Muscular Atrophy
Muscular Atrophy, Spinal
Neuromuscular Diseases
Muscular Diseases
Pathological Conditions, Anatomical
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Nervous System Neoplasms
Neoplasms by Site
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Autoimmune Diseases
Immune System Diseases processed this record on May 25, 2017