NCT00713245

Brief Summary

This study is to observe the progression of disease in late-onset Pompe disease

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
70

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started May 2008

Longer than P75 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

May 1, 2008

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

July 9, 2008

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 11, 2008

Completed
10.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2018

Completed
Last Updated

December 4, 2013

Status Verified

December 1, 2013

Enrollment Period

10.6 years

First QC Date

July 9, 2008

Last Update Submit

December 3, 2013

Conditions

Pompe Disease

Keywords

Pompe diseaselate onset

Outcome Measures

Primary Outcomes (1)

  • all muscle function

    10 years

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

individuals with acid alpha glucosidase deficiency but without obvious symptoms

You may qualify if:

  • individuals with acid alpha glucosidase deficiency without obvious symptoms
  • parents signed inform consent

You may not qualify if:

  • individuals with acid alpha glucosidase deficiency presenting symptoms

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Taiwan University Hospital

Taipei, Taiwan

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease Type II

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

Study Officials

  • Whu-Liang Hwu, MD, PhD

    National Taiwan University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Whu-Liang Hwu, MD, PhD

CONTACT

hwuwlntu@ntu.edu.tw

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 9, 2008

First Posted

July 11, 2008

Study Start

May 1, 2008

Primary Completion

December 1, 2018

Study Completion

December 1, 2018

Last Updated

December 4, 2013

Record last verified: 2013-12

Locations

TW(1)