Pompe Disease Registry Protocol

Recruiting Observational Study
Glycogen Storage Disease Type II Pompe Disease
No Study Drug Researchers observe your health over time — no experimental treatment is given.
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Review the details below, then apply to join this clinical trial.

At a Glance
Sex
Any
Study type
Observational
Participants needed
2,000 (estimated)
Sponsor
Genzyme, a Sanofi Company · Industry
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About This Trial
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 …
Trial Locations
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Eligibility Criteria
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…
Contacts

Trial Transparency email recommended (Toll free number for US & Canada)

800-633-1610

contact-us@sanofi.com

CONTACT

Pompe Registry HelpLine

617-591-5500

CONTACT