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Pompe Disease Registry Protocol
Pompe Disease Registry Protocol

NCT00231400

RecruitingN/A

Sponsor: Genzyme, a Sanofi Company

Conditions: Glycogen Storage Disease Type II, Pompe Disease

Countries: United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile

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 overview

Sex: ALL

Healthy volunteers: No

Study type: OBSERVATIONAL

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 gene mutations.

Exclusion Criteria:

There are no exclusion criteria in this Registry
Locations (272)
  • Birmingham, Alabama, United States
  • Phoenix, Arizona, United States
  • Phoenix, Arizona, United States
  • Tucson, Arizona, United States
  • Little Rock, Arkansas, United States
  • Little Rock, Arkansas, United States
  • Irvine, California, United States
  • Loma Linda, California, United States
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • Oakland, California, United States
  • Orange, California, United States
  • Sacramento, California, United States
  • San Diego, California, United States
  • San Francisco, California, United States
  • Stanford, California, United States
  • Aurora, Colorado, United States
  • New Haven, Connecticut, United States
  • Washington D.C., District of Columbia, United States
  • Coral Gables, Florida, United States
  • Gainesville, Florida, United States
  • Jacksonville, Florida, United States
  • Tampa, Florida, United States
  • Decatur, Georgia, United States
  • Aurora, Illinois, United States
  • Chicago, Illinois, United States
  • Chicago, Illinois, United States
  • Chicago, Illinois, United States
  • Indianapolis, Indiana, United States
  • Iowa City, Iowa, United States
  • + 242 more on CT.gov