National Registry for Egyptian Pediatric Neuromuscular Diseases

April 25, 2014 updated by: Sahar M.A. Hassanein, MD, Ain Shams University
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.

Study Overview

Status

Unknown

Conditions

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.

Study Type

Observational

Enrollment (Anticipated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Egypt
    • Abassia
      • Cairo, Abassia, Egypt, 11381
        • Recruiting
        • Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
        • Contact:
        • Principal Investigator:
          • Sahar MA Hassanein, MD, PhD
        • Sub-Investigator:
          • Nafissa El Badawy El Badawy, MD, PhD
        • Sub-Investigator:
          • Abdelfattah Souad F Souad, MD, PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 month to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

All children from birth to 18 years of age with inherited or acquired neuromuscular diseases.

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • Chromosomal diseases.
  • Malformations and deformations.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cardio-pulmonary function
Time Frame: 12 months
Pulmonary and cardiac function impairment.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ain Shams University

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2014

Primary Completion (Anticipated)

April 1, 2020

Study Completion (Anticipated)

December 1, 2020

Study Registration Dates

First Submitted

April 25, 2014

First Submitted That Met QC Criteria

April 25, 2014

First Posted (Estimate)

April 28, 2014

Study Record Updates

Last Update Posted (Estimate)

April 28, 2014

Last Update Submitted That Met QC Criteria

April 25, 2014

Last Verified

April 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EGYPT PED-NMD 2014

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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