NCT00713453

Brief Summary

This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy

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
16

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2007

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

January 1, 2007

Completed
1.5 years 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
11.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2019

Completed
Last Updated

March 24, 2015

Status Verified

March 1, 2015

Enrollment Period

12.9 years

First QC Date

July 9, 2008

Last Update Submit

March 21, 2015

Conditions

Pompe Disease

Keywords

Pompe diseasemuscleenzyme replacement therapy

Outcome Measures

Primary Outcomes (1)

  • all morphology changes improvement

    6 months

Eligibility Criteria

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

patients with Pompe disease, prepare or on enzyme replacement therapy

You may qualify if:

  • patients with Pompe disease, prepare or on enzyme replacement therapy
  • signed informed consent

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

  • Yin-Hsiu Chien, MD

    National Taiwan University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yin-Hsiu Chien, MD

CONTACT

886-2-23123456chienyh@ntu.edu.tw

Study Design

Study Type
observational
Observational Model
CASE ONLY
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Yin-Hsiu Chien, M.D., PhD.

Study Record Dates

First Submitted

July 9, 2008

First Posted

July 11, 2008

Study Start

January 1, 2007

Primary Completion

December 1, 2019

Study Completion

December 1, 2019

Last Updated

March 24, 2015

Record last verified: 2015-03

Locations

TW(1)