Pompe Disease Registry Protocol

ID#: NCT00231400

Age: Birth - 66+

Gender: All

Healthy Subjects: No

Recruitment Status: Recruiting

Start Date: September 15, 2004

End Date: January 31, 2034

Contact Information:
Trial Transparency email recommended (Toll free number for US & Canada)
800-633-1610
Pompe Registry HelpLine
617-591-5500
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.
Eligibility:

Inclusion Criteria: All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.

Exclusion Criteria: There are no exclusion criteria in this Registry
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