- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02124616
National Registry for Egyptian Pediatric Neuromuscular Diseases
Study Overview
Status
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
Enrollment (Anticipated)
Contacts and Locations
Study Contact
- Name: Sahar MA Hassanein, MD, PhD
- Phone Number: 201223183943
- Email: saharhassanein@med.asu.edu.eg
Study Locations
-
-
Abassia
-
Cairo, Abassia, Egypt, 11381
- Recruiting
- Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
-
Contact:
- Mohsen S Elalfy, MD, PhD
- Email: 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
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
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
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
Sponsor
Investigators
- Study Chair: Sahar MA Hassanein, MD, PhD, Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Central Nervous System Diseases
- Nervous System Diseases
- Immune System Diseases
- Neoplasms
- Autoimmune Diseases of the Nervous System
- Autoimmune Diseases
- Neoplasms by Site
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Neurodegenerative Diseases
- Peripheral Nervous System Diseases
- Neuromuscular Manifestations
- Pathological Conditions, Anatomical
- Spinal Cord Diseases
- Nervous System Neoplasms
- Muscular Disorders, Atrophic
- Paraneoplastic Syndromes, Nervous System
- Paraneoplastic Syndromes
- Neuromuscular Junction Diseases
- Myasthenia Gravis
- Motor Neuron Disease
- Muscular Dystrophies
- Muscular Atrophy
- Atrophy
- Polyneuropathies
- Muscular Diseases
- Muscular Atrophy, Spinal
- Neuromuscular Diseases
- Lambert-Eaton Myasthenic Syndrome
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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