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Pompe Disease Registry Protocol

NCT00231400 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 2000

Last updated 2026-04-28

No results posted yet for this study

Summary

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes.

The objectives of the Registry are:

* To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention.
* To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.
* To characterize the Pompe disease population.
* To evaluate the long-term effectiveness of alglucosidase alfa.

Conditions

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Study Director · Genzyme, a Sanofi Company

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-09-15
Primary Completion
2034-01-31
Completion
2034-01-31

Countries

  • United States
  • Argentina
  • Australia
  • Belgium
  • Brazil
  • Bulgaria
  • Canada
  • Chile
  • China
  • Colombia
  • Croatia
  • Czechia
  • Denmark
  • France
  • Germany
  • Greece
  • Hong Kong
  • Hungary
  • India
  • Indonesia
  • Israel
  • Italy
  • Japan
  • Jordan
  • Kuwait
  • Lebanon
  • Malaysia
  • Netherlands
  • Pakistan
  • Philippines
  • Poland
  • Portugal
  • Romania
  • Russia
  • Saudi Arabia
  • Serbia
  • Singapore
  • Slovakia
  • South Korea
  • Taiwan
  • Thailand
  • United Arab Emirates
  • United Kingdom
  • Vietnam

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00231400 on ClinicalTrials.gov