NCT02761421

Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT

Effect of Motor Development, Motor Function and Electrodiagnostic Characteristic of IOPD Under ERT

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 15 (estimated)
• Sponsor: Taipei Veterans General Hospital, Taiwan · Other Government
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

To investigate the motor development, motor function and electrodiagnostics presentation in IOPD under ERT.

Detailed description

Background Pompe disease is an autosomal recessive disease, enzyme replacement therapy (ERT) and new born screen (NBS) had been initiated in Taiwan since 2008. However, residual muscle weakness were noted. Decrease in amplitude of nerve compound muscle action potential (CMAP) and increase spontaneous activity were reported before the imitation of ERT. However, the motor development, motor function and electrodiagnostic presentation which were important in further direction of treatment and rehabilitation program arrangement in infantile Pompe disease (IOPD) under ERT is lacking. Aim To investigate the motor development, motor function and electrodiagnostic presentation in IOPD under ERT. Method This is an observational, prospective, longitudinal, follow-up study. Motor development was assessed by Alberta Infant Motor Scale (AIMS) and Peabody Developmental Motor Scales, Second Edition (PDMS II); motor function was assessed by Pediatric Evaluation of Disability Inventory (PEDI) and Gross Motor Function Measure (GMFM). Electrodiagnosis studies include nerve conduction study (NCS) and electromyography (EMG). Expect Effects Obtain the characteristics of motor development, motor function and electrodiagnosis presentation of IOPD under ERT, and the relation between motor development, motor function and electrodiagnosis presentation.

Conditions

• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
OTHER	observation study	observation study

Timeline

• Start date: 2016-01-01
• Primary completion: 2016-12-01
• Completion: 2016-12-01
• First posted: 2016-05-04
• Last updated: 2016-05-04

Locations

1 site across 1 country: Taiwan