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NCT02761421 Clinical Trial

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

Glycogen Storage Disease Type II Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT02761421
Other study ID # 2015-12-014C
Secondary ID
Status Recruiting
Phase N/A
First received April 26, 2016
Last updated May 2, 2016
Start date January 2016
Est. completion date December 2016

Study information
Verified date December 2015
Source Taipei Veterans General Hospital, Taiwan
Contact chihjou lai
Phone 886-28712121
Email irbopinion@vghtpe.gov.tw
Is FDA regulated No
Health authority Taiwan: Institutional Review Board
Study type Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility
Status Recruiting
Enrollment 15
Est. completion date December 2016
Est. primary completion date December 2016
Accepts healthy volunteers No
Gender Both
Age group N/A and older
Eligibility 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 Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

Intervention

Other:
• observation study
observation study

Locations
Country Name City State
Taiwan Taipei Veteran General Hospital : Taipei City, Taiwan 11217, R.O.C. Taipei

Sponsors (1)
Lead Sponsor Collaborator
Taipei Veterans General Hospital, Taiwan

Country where clinical trial is conducted

Taiwan, 

Outcome
Type Measure Description Time frame Safety issue
Other Percentile of active denervation in electrodiagnostic study 1 year No
Primary Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition 1 year No
Primary Development Quotient of PDMS-II 1 year No
Secondary Raw Score of Pediatric Evaluation of Disability Inventory Total score of GMFM 1 year No
Secondary Total Score of GMFM 1 year No
See also
  Status Clinical Trial Phase
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Recruiting NCT04910776 - Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa Phase 3
Not yet recruiting NCT05017402 - Higher Dose of Alglucosidase Alpha for Pompe Disease
Withdrawn NCT01656590 - High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease Phase 2
Completed NCT00701129 - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease Phase 4
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT02363153 - Diet and Exercise in Pompe Disease N/A
Completed NCT00074932 - Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease N/A
Completed NCT00025896 - Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease Phase 2
Completed NCT00001331 - Genetic and Family Studies of Inherited Muscle Diseases N/A
Completed NCT00077662 - A Prospective, Observational Study in Patients With Late-Onset Pompe Disease N/A
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT00125879 - Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 Phase 2/Phase 3
Recruiting NCT04848779 - A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Active, not recruiting NCT05164055 - Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) Phase 4
Active, not recruiting NCT02635269 - Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy N/A
Completed NCT00051935 - A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II Phase 2
Completed NCT00053573 - rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) Phase 1/Phase 2
Completed NCT02801539 - Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) N/A
Completed NCT03687333 - Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment Phase 4