NCT02399748

Brief Summary

Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.

Trial Health

77
On Track

Trial Health Score

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

Enrollment
50

participants targeted

Target at P25-P50 for all trials

Timeline
5mo left

Started Apr 2007

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

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 Progress98%
Apr 2007Dec 2026

Study Start

First participant enrolled

April 1, 2007

Completed
8 years until next milestone

First Submitted

Initial submission to the registry

March 21, 2015

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 26, 2015

Completed
11.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2026

Last Updated

July 18, 2017

Status Verified

July 1, 2017

Enrollment Period

19.7 years

First QC Date

March 21, 2015

Last Update Submit

July 13, 2017

Conditions

Pompe Disease

Outcome Measures

Primary Outcomes (1)

  • all cause morbidities

    10 years

Eligibility Criteria

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

Patients of Pompe disease who under enzyme replacement therapy

You may qualify if:

  • Confirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Taiwan University Hospital

Taipei, 100, Taiwan

RECRUITING

Related Publications (1)

  • Hsu YK, Chien YH, Shinn-Forng Peng S, Hwu WL, Lee WT, Lee NC, Po-Yu Huang E, Weng WC. Evaluating brain white matter hyperintensity, IQ scores, and plasma neurofilament light chain concentration in early-treated patients with infantile-onset Pompe disease. Genet Med. 2023 Jan;25(1):27-36. doi: 10.1016/j.gim.2022.10.005. Epub 2022 Nov 18.

    PMID: 36399131DERIVED

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

    National Taiwan University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yin-Hsiu Chien

CONTACT

+886223123456chienyh@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

March 21, 2015

First Posted

March 26, 2015

Study Start

April 1, 2007

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

December 1, 2026

Last Updated

July 18, 2017

Record last verified: 2017-07

Locations

TW(1)