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Trial record 96 of 103 for:    Pompe Disease

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

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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
Sponsor:
Information provided by (Responsible Party):
Taipei Veterans General Hospital, Taiwan

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

Condition or disease Intervention/treatment
Glycogen Storage Disease Type II Other: observation study

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.


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Study Type : Observational
Estimated Enrollment : 15 participants
Observational Model: Cohort
Time Perspective: Prospective
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


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




Primary Outcome Measures :
  1. Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition [ Time Frame: 1 year ]
  2. Development Quotient of PDMS-II [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Raw Score of Pediatric Evaluation of Disability Inventory Total score of GMFM [ Time Frame: 1 year ]
  2. Total Score of GMFM [ Time Frame: 1 year ]

Other Outcome Measures:
  1. Percentile of active denervation in electrodiagnostic study [ Time Frame: 1 year ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Criteria

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.

Information from the National Library of Medicine

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


Contacts
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Contact: chihjou lai 886-28712121 ext 7384 irbopinion@vghtpe.gov.tw

Locations
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Taiwan
Taipei Veteran General Hospital : Taipei City, Taiwan 11217, R.O.C. Recruiting
Taipei, Taiwan, 11217
Contact: chihjou lai, Master    886-2-28712121 ext 7384    irbopinion@vghtpe.gov.tw   
Sponsors and Collaborators
Taipei Veterans General Hospital, Taiwan

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Responsible Party: Taipei Veterans General Hospital, Taiwan
ClinicalTrials.gov Identifier: NCT02761421     History of Changes
Other Study ID Numbers: 2015-12-014C
First Posted: May 4, 2016    Key Record Dates
Last Update Posted: May 4, 2016
Last Verified: December 2015
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: publication in journal

Keywords provided by Taipei Veterans General Hospital, Taiwan:
IOPD

Additional relevant MeSH terms:
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Glycogen Storage Disease
Glycogen Storage Disease Type II
Genetic Diseases, Inborn
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lysosomal Storage Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors