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

May 2, 2016 updated by: Taipei Veterans General Hospital, Taiwan

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

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

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Anticipated)

15

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

  • Taiwan
      • Taipei, Taiwan, 11217
        • Recruiting
        • Taipei Veteran General Hospital : Taipei City, Taiwan 11217, R.O.C.
        • Contact:

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The investigators included all patients with infantile onset Pompe disease (IOPD) who were diagnosed and follow-up at Taipei Veteran General Hospital (TVGH) and excluded patients whose guardian did not provide the inform consent.

Description

Inclusion Criteria:

  • All patients with infantile onset Pompe disease (IOPD) who were diagnosed and follow-up at Taipei Veteran General Hospital (TVGH).

Exclusion Criteria:

  • Patients whose guardian did not provide the inform consent.

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

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
patients with IOPD
observation all patients with IOPD
Other: observation study
observation study

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition
Time Frame: 1 year
1 year
Development Quotient of PDMS-II
Time Frame: 1 year
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
Raw Score of Pediatric Evaluation of Disability Inventory Total score of GMFM
Time Frame: 1 year
1 year
Total Score of GMFM
Time Frame: 1 year
1 year

Other Outcome Measures

Outcome Measure
Time Frame
Percentile of active denervation in electrodiagnostic study
Time Frame: 1 year
1 year

Collaborators and Investigators

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

Sponsor

Taipei Veterans General Hospital, Taiwan

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

January 1, 2016

Primary Completion (Anticipated)

December 1, 2016

Study Completion (Anticipated)

December 1, 2016

Study Registration Dates

First Submitted

April 26, 2016

First Submitted That Met QC Criteria

May 2, 2016

First Posted (Estimate)

May 4, 2016

Study Record Updates

Last Update Posted (Estimate)

May 4, 2016

Last Update Submitted That Met QC Criteria

May 2, 2016

Last Verified

December 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2015-12-014C

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

IPD Plan Description

publication in journal

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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