National Registry for Egyptian Pediatric Neuromuscular Diseases

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Ain Shams University
Sponsor:
Information provided by (Responsible Party):
Sahar M.A. Hassanein, MD, Ain Shams University
ClinicalTrials.gov Identifier:
NCT02124616
First received: April 25, 2014
Last updated: NA
Last verified: April 2014
History: No changes posted
  Purpose

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
Spinal Muscular Atrophy
Muscular Dystrophy
Muscle Diseases
Myasthenic Syndromes
Polyneuropathies

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 Ain Shams University:

Primary Outcome Measures:
  • Functional motor ability [ Time Frame: 1 year ] [ Designated as safety issue: No ]

    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 ] [ Designated as safety issue: No ]
    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)
Groups/Cohorts
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.

  Eligibility

Ages Eligible for Study:   1 Month to 18 Years
Genders Eligible for Study:   Both
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.

Criteria

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 ClinicalTrials.gov identifier: NCT02124616

Contacts
Contact: Sahar MA Hassanein, MD, PhD 201223183943 saharhassanein@med.asu.edu.eg

Locations
Egypt
Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University Recruiting
Cairo, Abassia, Egypt, 11381
Contact: Mohsen S Elalfy, MD, PhD       elalfym@hotmail.com   
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
Investigators
Study Chair: Sahar MA Hassanein, MD, PhD Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
  More Information

Additional Information:
No publications provided

Responsible Party: Sahar M.A. Hassanein, MD, Professor of Pediatrics, Ain Shams University
ClinicalTrials.gov Identifier: NCT02124616     History of Changes
Other Study ID Numbers: EGYPT PED-NMD 2014
Study First Received: April 25, 2014
Last Updated: April 25, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Ain Shams University:
spinal muscular atrophy
Muscular dystrophy
Muscle diseases
myasthenic syndromes
Polyneuropathies
Myopathies

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

ClinicalTrials.gov processed this record on October 20, 2014