NCT02761421

Brief Summary

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

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
15

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2016

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2016

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

April 26, 2016

Completed
8 days until next milestone

First Posted

Study publicly available on registry

May 4, 2016

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2016

Completed
Last Updated

May 4, 2016

Status Verified

December 1, 2015

Enrollment Period

11 months

First QC Date

April 26, 2016

Last Update Submit

May 2, 2016

Conditions

Glycogen Storage Disease Type II

Keywords

IOPD

Outcome Measures

Primary Outcomes (2)

  • Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition

    1 year

  • Development Quotient of PDMS-II

    1 year

Secondary Outcomes (2)

  • Raw Score of Pediatric Evaluation of Disability Inventory Total score of GMFM

    1 year

  • Total Score of GMFM

    1 year

Other Outcomes (1)

  • Percentile of active denervation in electrodiagnostic study

    1 year

Study Arms (1)

patients with IOPD

observation all patients with IOPD

Other: observation study

Interventions

observation studyOTHER

observation study

patients with IOPD

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

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

You may not qualify if:

  • Patients whose guardian did not provide the inform consent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

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

Taipei, 11217, Taiwan

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

Observational Studies as Topic

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Clinical Studies as TopicEpidemiologic Study CharacteristicsEpidemiologic MethodsInvestigative TechniquesHealth Care Evaluation MechanismsQuality of Health CareHealth Care Quality, Access, and EvaluationPublic HealthEnvironment and Public Health

Central Study Contacts

chihjou lai

CONTACT

886-28712121irbopinion@vghtpe.gov.tw

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 26, 2016

First Posted

May 4, 2016

Study Start

January 1, 2016

Primary Completion

December 1, 2016

Study Completion

December 1, 2016

Last Updated

May 4, 2016

Record last verified: 2015-12

Data Sharing

IPD Sharing
Will share

publication in journal

Locations

TW(1)