Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02761421|
Recruitment Status : Unknown
Verified December 2015 by Taipei Veterans General Hospital, Taiwan.
Recruitment status was: Recruiting
First Posted : May 4, 2016
Last Update Posted : May 4, 2016
|Condition or disease||Intervention/treatment|
|Glycogen Storage Disease Type II||Other: observation study|
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.
To investigate the motor development, motor function and electrodiagnostic presentation in IOPD under ERT.
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).
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.
|Study Type :||Observational|
|Estimated Enrollment :||15 participants|
|Official Title:||Effect of Motor Development, Motor Function and Electrodiagnostic Characteristic of IOPD Under ERT|
|Study Start Date :||January 2016|
|Estimated Primary Completion Date :||December 2016|
|Estimated Study Completion Date :||December 2016|
patients with IOPD
observation all patients with IOPD
Other: observation study
- Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition [ Time Frame: 1 year ]
- Development Quotient of PDMS-II [ Time Frame: 1 year ]
- Raw Score of Pediatric Evaluation of Disability Inventory Total score of GMFM [ Time Frame: 1 year ]
- Total Score of GMFM [ Time Frame: 1 year ]
- Percentile of active denervation in electrodiagnostic study [ Time Frame: 1 year ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02761421
|Contact: chihjou lai||886-28712121 ext email@example.com|
|Taipei Veteran General Hospital : Taipei City, Taiwan 11217, R.O.C.||Recruiting|
|Taipei, Taiwan, 11217|
|Contact: chihjou lai, Master 886-2-28712121 ext 7384 firstname.lastname@example.org|