Pompe Disease Registry

This study is currently recruiting patients.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00231400

Purpose

The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient’s treating physician.

The objectives of the Registry are:

Condition

Pompe Disease Glycogen storage disease type II (GSD-II)

Study Type: Observational

Study Design: Natural History, Longitudinal, Defined Population, Retrospective/Prospective Study

Further Study Details:
Study start: September 2004

The Pompe Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

Eligibility Criteria

Ages Eligible for Study: Both

Inclusion Criteria:
Patient must have a confirmed diagnosis of Pompe disease, documented by GAA enzyme deficiency or GAA gene mutation

Exclusion Criteria:
Patient is currently enrolled in a Genzyme-sponsored clinical trial.

Location and Contact Information

Please refer to this study by ClinicalTrials.gov identifier NCT00231400

Central Contact:
Pompe Registry HelpLine 800-745-4447, Ext. 15500 help@pomperegistry.com
Pompe Registry HelpLine 617-591-5500 help@pomperegistry.com

Registry participation is worldwide and not limited by facility; facilities not yet active may enroll upon identification of a patient.

More Information

Study ID Numbers: Pompe Registry Protocol
ClinicalTrials.gov Identifier: NCT00231400
Health Authority: United States: Institutional Review Board