National Registry for Egyptian Pediatric Neuromuscular Diseases

Status Recruiting

Clinical Trial 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.

Clinical Trial 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 Design

Observational Model: Case-Only, Time Perspective: Prospective

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02124616
Study type	Observational [Patient Registry]
Source	Ain Shams University
Contact	Sahar MA Hassanein, MD, PhD
Phone	201223183943
Email	saharhassanein@med.asu.edu.eg
Status	Recruiting
Phase	N/A
Start date	April 2014
Completion date	December 2020

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04851873` - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)	Phase 3
Completed	`NCT03223051` - Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy	N/A
Completed	`NCT04335942` - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor	N/A
Recruiting	`NCT05794139` - Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy	Phase 2
Not yet recruiting	`NCT06300996` - Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb	N/A
Not yet recruiting	`NCT00961103` - Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)	N/A
Completed	`NCT02003937` - Aerobic Training in Patients With Spinal Muscular Atrophy Type III	N/A
Completed	`NCT00227266` - Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy	Phase 2
Completed	`NCT00374075` - Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy	Phase 1
Enrolling by invitation	`NCT05539456` - Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
Recruiting	`NCT05779956` - Personalized Medicine for SMA: a Translational Project
Recruiting	`NCT03300869` - Natural History of Types 2 and 3 SMA in Taiwan
Recruiting	`NCT03217578` - Neonatal Spinal Muscular Atrophy (SMA) Screening
Completed	`NCT01703988` - An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy	Phase 1/Phase 2
Withdrawn	`NCT02235090` - Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting	N/A
Completed	`NCT02123186` - Newborn Screening for Spinal Muscular Atrophy	N/A
Completed	`NCT00756821` - A Pilot Study of Biomarkers for Spinal Muscular Atrophy	N/A
Completed	`NCT00004771` - Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease	Phase 2
Recruiting	`NCT05366465` - Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France
Recruiting	`NCT06310421` - Spinal Muscular Atrophy Neonatal Screening Program